Regulatory tracker: 5 years on, Rubraca turns accelerated prostate cancer nod into full FDA approval

Welcome to Fierce Pharma's regulatory tracker for the second half of 2025. On this page, we're recording the regulatory progress of in-market products, including expansions into key geographies and new indications. Be sure to come back regularly for the latest updates.

UPDATED: Thursday, December 18 at 2:45 p.m. ET

• More than five years after winning an accelerated nod in metastatic castration-resistant prostate cancer, PARP inhibitor Rubraca has converted that clearance into a full FDA approval. The green light covers treatment of deleterious BRCA mutation (BRCAm) (Germline and-or somatic)-associated metastatic castration-resistant prostate cancer (mCRPC) in adults who’ve previously received an androgen receptor-directed therapy, the FDA said in a Dec. 17 release.

  The drug was approved based on data from TRITON3, the phase 3 study that helped secure Rubraca its accelerated nod back in 2020. In the study, BRCAm prostate cancer patients on Rubraca charted a median radiographic progression-free survival (rPFS) of 11.2 months, compared to 6.4 months in a control group that included a physician’s choice of an androgen receptor pathway inhibitor the patients had not previously received.

  Median overall survival clocked in at 23.2 months in the Rubraca arm, versus 21.2 months for the control cohort.

  Rubraca was original developed by Clovis Oncology, which filed for bankruptcy in Dec. 2022. In looking to sell off its remaining assets, the drugmaker handed over Rubraca to Switzerland’s Pharma& Schweiz for $70 million upfront, plus potential regulatory and sales milestones. 

• Over in the United Kingdom, the National Institute for Health and Care Excellence (NICE)—which weighs new drugs’ cost effectiveness for the NHS—has given its final blessing to Johnson & Johnson’s Lazcluze (lazertinib) for the first-line treatment of advanced non-small cell lung cancer (NSCLC) in adults whose tumors have EGFR exon 19 deletions or exon 21 L858R substitution mutations.

  With the recommendation, eligible patients can now receive the J&J treatment through the U.K.’s National Health Service in England and Wales, J&J said in a release.

  NICE issued its recommendation after reviewing data from J&J’s late-stage Mariposa study, in which patients taking Lazcluze lived for a median of 23.7 months without their disease worsening, compared to 16.6 months in patients on AstraZeneca’s Tagrisso. This performance allowed the study to meet its primary endpoint, and Lazcluze further helped “significantly” improve overall survival versus Tagrisso on a key secondary endpoint, too, the company said.

  Some 35,000 people were diagnosed with lung cancer in England between 2019 and 2023, J&J estimates. NSCLC is the most common form of lung cancer, making up some 80% to 85% of cases. Further, EGFR mutations are found in around 15% of cases of NSCLC in European patients, with most of those being the types of mutations included in Lazcluze’s NICE recommendation, J&J pointed out. 

UPDATED: Monday, December 15 at 3:20 p.m. ET

• Sprout Pharmaceuticals' Addyi has picked up a label expansion in the United States.

  More than a decade after the drug's first FDA approval, Addyi is now approved to treat hypoactive sexual desire disorder (HSDD) in women older than 65, the company said in a press release.

  The approval "closes a gap in care for millions of postmenopausal women experiencing frustrating low sexual desire," the company explained.

  Back in 2015, the drug scored a controversial FDA nod after two prior rejections. Before today, its label restricted use to "premenopausal women with acquired, generalized" HSDD under specific circumstances.

• Right on the heels of an endorsement from the European Medicines Agency's (EMA's) drug review committee, GSK's depemokimab is keeping its regulatory momentum rolling with an approval in the United Kingdom.

  The U.K.'s Medicines and Healthcare products Regulatory Agency (MHRA) today endorsed the twice-yearly biologic in two indications. In asthma, the drug is approved as an add-on treatment for patients 12 and older. And in severe chronic rhinosinusitis with nasal polyps, it's approved as an add-on for adults.

  The drug is specifically approved for patients whose symptoms remain uncontrolled despite treatment with standard of care. The drug will carry the brand name Exdensur in the United Kingdom.

  Just last week, CHMP signed off on the medicine with similar stipulations in the two diseases.

UPDATED: Friday, December 12 at 8:40 a.m. ET

• GSK's vaccine for respiratory syncytial virus, Arexvy, is set to gain a wider approval in Europe with a new positive decision from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP).

  The committee recommended the shot be approved for all adults, GSK said in a Dec. 12 press release. As it stands, the vaccine is approved in Europe for people ages 50 to 59 who are at an increased risk for RSV disease, and for people 60 and older.

  The European Commission is expected to give a final decision on the wider approval in February, GSK said.

  The wider approval will put the shot on equal footing with rivals from Pfizer and Moderna, which already boast approvals in the general adult population.

• China has approved (PDF) Sino Biopharmaceutical’s culmerciclib, making the drug the world’s first CDK2/4/6 inhibitor to reach the market. It is indicated for use in combination with AstraZeneca’s Faslodex to treat patients with HR-positive, HER2-negative breast cancer who have progressed following prior endocrine therapy.

  In a phase 3 trial coded TQB3616-III-01, the culmerciclib regimen led to a 64% reduction in the risk of disease progression or death versus Faslodex alone. The combo prolonged the median progression-free survival time by 9.2 months to 16.6 months.

  Sino Biopharm has already filed a first-line application for the culmerciclib-Faslodex cocktail in China in July. Back in March, the company announced that the TQB3616-III-02 trial pitting the combo against Faslodex alone in previously untreated HR-positive, HER2-negative breast cancer has also met its PFS endpoint at an interim analysis. 

• The CHMP also signed off on Stada and Bio-Thera Solutions' biosimilar to Simponi this week. 

  The biosimilar, to be marketed as Gotenfia, secured an endorsement to treat rheumatoid arthritis, psoriatic arthritis, ankylosing spondylitis, non-radiographic axial spondyloarthritis and ulcerative colitis in adult patients, plus juvenile idiopathic arthritis in for patients aged 2 years and older.

  Like the Arexvy recommendation, the Gotenfia nod remains subject to a final sign off from the European Commission.

  Bio-Thera Solutions developed the drug and licensed it to Stada last year.

• A new formulation of BioCryst's hereditary angioedema (HAE) treatment Orladeyo has gained an FDA approval.

  Specifically, the agency approved the company's once-daily oral pellet formulation as a prophylactic therapy for pediatric patients ages 2 to 12, the company said in a press release.

  Prior to this approval, pediatric patients with HAE could receive prophylactic therapies intravenously or through subcutaneous injections, which the company notes "can be burdensome for younger patients living with HAE and their caregivers."

• China’s National Medical Products Administration has approved InnoCare’s next-generation TRK inhibitor, zurletrectinib, for treating patients 12 years of age and older with solid tumors harboring NTRK gene fusions.

  In a registrational trial, the drug showed an objective response rate of 89.1%, and two-year progression-free survival and overall survival rates of 77.4% and 90.8%, respectively. InnoCare said the results look better than first-generation TRK inhibitors.

  Zurletrectinib has been included in the NMPA’s “SPARK Program,” a pilot initiative that encourages the development of pediatric cancer drugs. InnoCare said it plans to file for approval in patients as young as 2 years of age in the near future.

  NTRK fusion genes and have been identified in over 26 solid tumors, and about 6,500 new cases of tumors with NTRK fusion genes occur annually in China, InnoCare estimates.

   

UPDATED: Monday, December 8 at 9:10 a.m. ET

• Agios Pharmaceuticals is still awaiting an FDA decision for mitapivat as a treatment for thalassemia after the company's FDA Prescription Drug User Fee Act (PDUFA) target date passed on Sunday.

  The company is seeking approval for the drug as a treatment for adults with non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia. The application is "under active review," the drugmaker said in a Dec. 8 press release.

  Agios is "collaborating closely" with the agency to finalize label documents, the company said. The FDA hasn't provided an expected timeline on the decision, according to Agios.

  Mitapivat, boasting a brand name Pyrukynd, is already approved by the FDA as a treatment for PK deficiency.

  Agios submitted its supplemental New Drug Application for the thalassemia indication early this year. The FDA already delayed its decision on the drug by three months back in September.

UPDATED: Wednesday, November 26 at 9:00 a.m. ET

• Eisai has completed its rolling FDA Biologics License Application for the Leqembi Iqlik subcutaneous starter dose, the company said in a Nov. 26 press release.

  If the application is approved, the drug would be the only anti-amyloid treatment for Alzheimer's disease with at-home dosing available from the start, Eisai explained. As it stands, the drug is approved for at-home maintenance dosing after an 18-month initiation phase with the infused dose.

  Subcutaneous dosing of the Alzheimer's disease antibody offers the potential to reduce treatment burdens for patients and ease infrastructure bottlenecks associated with infused therapies. Approval and uptake of the subcutaneous dose has been viewed by analysts and market watchers as a key inflection point in the ongoing launch.

• Sun's Unloxcyt received an FDA label update featuring new longer-term data showing that patients with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) experienced durable clinical responses.

  In the the CK-301-101 trial, at least 50% of patients achieved an objective response, while 14% of patients with metastatic CSCC and 32% of those with locally advanced CSCC achieved stable disease. As of the latest follow-up, the median duration of response was not reached in either patient group.

  The drug won its original approval nearly a year ago as a treatment for patients with metastatic or locally advanced CSCC who are not candidates for curative surgery or curative radiation.

  "As a company committed to addressing the unmet needs of the patient communities we support, these pivotal data highlight that more patients responded and maintained their responses to Unloxcyt for longer than observed in the primary analysis," Sun Pharma CEO Richard Ascroft said in a statement. "The updated label reinforces Unloxcyt as an evolution in checkpoint inhibition."

   

UPDATED: Friday, November 21 at 11:30 a.m. ET

• England's National Institute for Health and Care Excellence (NICE) has cleared Alnylam’s blockbuster Amvuttra for use by the National Health Service (NHS) as a treatment for wild-type or hereditary transthyretin amyloid cardiomyopathy (ATTR-CM). 
  
  The endorsement comes two years after the U.K. agency endorsed Amvuttra as a treatment for polyneuropathy of hereditary transthyretin-mediated amyloidosis (hATTR-PN).

  ATTR-CM is a progressive disorder in which misfolded protein deposits collect in the heart, causing stiffness and difficulty in pumping blood. Symptoms include shortness of breath, fatigue and arrhythmias, potentially leading to heart failure.

  As an RNA interference drug, Amvuttra brings a new mechanism of action to treat ATTR-CM. The FDA originally cleared Amvuttra for hATTR-PN in 2022, followed by an ATTR-CM label expansion in March of this year. 

  The company reported Amvuttra sales at $685 million for the third quarter and doubling of demand for the treatment since the second quarter.

• The FDA has expanded the label for AstraZeneca and Merck’s oral, selective MEK inhibitor Koselugo to treat adult patients with neurofibromatosis type 1 (NF1) who have symptomatic, inoperable plexiform neurofibromas (PN).

  The FDA originally signed off on Koselugo in 2020 as a treatment for pediatric patients ages2 and older with NF1. In April of this year, the U.S. regulator expanded its use in a granular formulation to children as young as age 1.

  NF1 is a rare, progressive, genetic disorder which is usually diagnosed in early childhood and causes PN tumors to grow on nerve tissues and can affect the brain, spinal cord and nerves.

  “We celebrate this FDA approval of Koselugo for adults with NF1 plexiform neurofibromas—a major step forward for NF patients everywhere,” Annette Bakker, Ph.D., the CEO of the Children’s Tumor Foundation, said in a release. “Koselugo has already changed what is possible for children with NF1, and now adults will benefit from that same progress.”

UPDATED: Thursday, November 20 at 8:40 a.m. ET

• Merck's subcutaneous form of Keytruda has picked up a wide-ranging approval in the European Union. 

  The drug will be branded as Keytruda SC in Europe and is now approved in all 33 of the original Keytruda's indications for adults in the region, Merck said in a Nov. 19 press release.

  In the U.S., the subcutaneous form of the oncology blockbuster is branded as Keytruda Qlex, having passed muster with the FDA back in September. 

  The drug can be administered in one minute every three weeks or in two minutes every six weeks, Dr. Marjorie Green, head of oncology global clinical development at Merck Research Laboratories, said in a statement.

  Availability of the drug in EU member states will "depend on multiple factors," Merck said in the release, including the completion of reimbursement talks with national authorities.

• AbbVie and Genmab's Epkinly has secured an expanded approval in the United States.

  The FDA endorsed the drug in combination with rituximab and lenalidomide as a regimen for adults with relapsed or refractory (R/R) follicular lymphoma (FL). 

  Last year, the AbbVie and Genmab drug gained an FDA approval for use as a monotherapy for patients with FL who've tried at least two prior lines of therapy. That approval set up a contest between Epkinly and Roche’s Lunsumio, which are both members of the CD20xCD3 bispecific T-cell engager class.

  In addition to its FL approvals, Epkinly scored its first FDA nod as a treatment for third-line diffuse large B-cell lymphoma. 

• After granting prior accelerated green lights to Johnson & Johnson's Darzalex Faspro and Amgen's Imdelltra, the FDA has converted the approvals into full, traditional endorsements.

  Darzalex Faspro's full approval covers its use in combination with bortezomib, cyclophosphamide, and dexamethasone (VCd) as a regimen for newly diagnosed patients with light chain (AL) amyloidosis. Previously, the drug scored an accelerated approval in that use back in 2021.

  As for Imdelltra, the drug now carries a full approval to treat adults with extensive stage small cell lung cancer who've experienced disease progression on or after platinum-based chemotherapy. Imdelltra's accelerated approval in that setting only came last May.

   

UPDATED: Tuesday, November 18 at 10:02 a.m. ET

• Eli Lilly can add patients in India to its reach for its Alzheimer’s disease med after scoring an approval from the Central Drugs Standard Control Organization (CDSCO).

  The drug regulator cleared Lilly’s donanemab, which is branded in Kisunla in its approved markets, as a treatment for those with mild cognitive impairment or mild dementia due to Alzheimer’s disease. To be eligible, patients must have confirmed amyloid pathology. 

  The amyloid-targeting treatment is delivered via an intravenous infusion every four weeks to remove excessive buildup of amyloid plaques and slow cognitive and functional decline.

  Over 8 million people in India are expected to have dementia by 2030, according to reports. Alzheimer’s disease remains the most common cause of dementia globally.

  “The approval of donanemab marks a significant milestone in our mission to address the urgent needs of people living with Alzheimer’s disease in India,” Lilly India’s president and general manager, Winselow Tucker, said in a statement. “By offering an innovative therapy that targets amyloid plaques and slows cognitive decline, we aim to give patients and their families more time and a better quality of life.

• Canadian drug regulators signed off on Esperion’s cholesterol med Nilemdo (bempedoic acid), which will be commercialized by the drugmaker’s Canadian partner HLS Therapeutics.

  Nilemdo, which goes by the moniker Nexletol in the U.S., is a non-statin oral mediation that’s used to lower low-density lipoprotein (LDL) cholesterol levels and reduce the risk of major cardiovascular events. Health Canada’s Nilemdo label specifically permits its use in those at risk of cardiovascular disease.

  HLS Therapeutics, which also partners with Esperion on combination therapy Nexlizet (bempedoic acid and ezetimibe), is now gearing up for a commercial launch in the second quarter of next year. About 1 in 12 Canadians aged 20 of older are living with diagnosed heart disease.

UPDATED: Thursday, November 13 at 10:33 a.m. ET

• Roche’s blood cancer therapy Columvi has widened its reach in England, where the drug can now be used as a second-line treatment for patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL).

  The National Institute for Health and Care Excellence (NICE) specifically signed off on the CD20xCD3 T-cell engaging bispecific antibody’s availability on England’s National Health Service (NHS) for those who are ineligible for autologous stem cell treatment. Previously, Columvi’s use was limited to patients who had already undergone two prior therapy lines of therapy, the same approval it holds in the U.S.

  In trials, 54% of those treated with Roche’s med and chemotherapy were still alive after two years, compared to 34% of patients on the standard Rituxan-based regimen.

  Around 5,500 patients are diagnosed with DLBCL in England each year, while around 300 stand to benefit from the new approval, according to the NHS. Columvi, an intravenous infusion given every three weeks, will be immediately available to eligible patients in the region through NHS’ Cancer Drug Fund, which fast-tracks access to certain cancer therapies. 

  Roche is “delighted” with the positive NICE guidance and pledges to make the treatment available across the U.K. “as quickly as possible,” the company’s U.K. hematology lead Pia Ballschmieter, Ph.D., said in a statement.

  The NICE endorsement follows that of European regulators earlier this year. The FDA, meanwhile, denied Roche’s efforts to expand Columvi into earlier treatment lines in July after an advisory panel determined that the trial results were not applicable to patients in the U.S.  

   

UPDATED: Friday, November 7 at 2:15 p.m. ET

• The FDA will take an extra three months to consider a potential label expansion for Rhythm Pharmaceuticals’ Imcivree. The delay pushes the FDA's target decision date out to March 20, 2026, and follows a request for additional sensitivity analyses that came from the agency in October. 

  Imcivree had been in the running for a potential approval in acquired hypothalamic obesity by December 20, but the FDA will now need more time for its review after determining that the new analyses Rhythm provided constitute a "major amendment" to its application. The FDA request centers on Rhythm's late-stage pivotal trial, and the regulator did not request any new data, Rhythm said on Nov. 7.

  The company has “every confidence” that the additional analyses will confirm the strength of its data, and the company plans to collaborate with the FDA while continuing launch preparations in parallel, CEO David Meeker, M.D., commented in a press release.

  Imcivree is Rhythm’s sole commercial product and is currently marketed as a weight management med for patients with obesity due to Bardet-Biedl syndrome or other rare genetic deficiencies. A nod in hypothalamic obesity would allow the drug to reach patients with obesity caused by damage or dysfunction in the brain’s hypothalamus.  

   

UPDATED: Tuesday, November 4 at 9:00 a.m. ET

• Chiesi and Protalix BioTherapeutics have requested that the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) re-review its previous negative opinion on a less frequent dosing regimen of the pair's Fabry disease therapy Elfabrio.

  While Elfabrio is currently approved in Europe to be given at 1 mg/kg of body weight every two weeks, Chiesi and Protalix are prosing a dosing regimen of 2 mg/kg every 4 weeks. 

  Last month, the CHMP concluded that the data submitted from the open-label, switch-over Bright trial and its extension study were insufficient to demonstrate similar efficacy for the four-week dosing schedule compared to the existing regimen.
   

UPDATED: Friday, October 31 at 9:00 a.m. ET

• China's National Medical Products Administration has handed out the world's first approval for an EGFR-targeted antibody-drug conjugate. The approval is for Lepu Biopharma's becotatug vedotin (MRG003) for the treatment of recurrent or metastatic nasopharyngeal cancer, the company said in a release (PDF).

  In a pivotal study conducted in Chinese patients with nasopharyngeal cancer who had failed at least two lines of therapy, including chemotherapy and PD-1/L1 inhibitor, becotatug vedotin significantly reduced the risk of progression or death by 37% versus chemo, according to results presented at the 2025 American Society of Clinical Oncology annual meeting. The drug more than doubled the median progression-free survival time from 2.8 months to 5.8 months.

  The drug also showed a trend toward improving overall survival (OS). As of the data cutoff in December 2024, the median OS was 17.1 months in the experimental arm, versus 12 months in the control group, accompanied by a favorable 27% death-risk reduction that was not statistically significant. The life-extension benefit widened to 41% after adjusting for trial crossover.

  A phase 3 study is underway to pair becotatug vedotin with Lepu's anti-PD-1 agent pucotenlimab in second-line nasopharyngeal carcinoma. The company is also testing the EGFR ADC in a phase 3 study in head and neck cancer.
   

UPDATED: Wednesday, October 29 at 3:57 p.m. ET

• Eli Lilly’s ulcerative colitis offering Omvoh just got more convenient with a new FDA approval that clears the drug as a single-injection maintenance regimen.

  The once-monthly injection replaces the previous dosing of two injections and has been confirmed bioequivalent in a phase 1 study. The new maintenance dose, which is also citrate-free, will be available in the U.S. in early 2026 as a prefilled pen or prefilled syringe.

  Omvoh treatment starts with three infusions every four weeks before patients can transition to subcutaneous self-injection every four weeks for maintenance treatment.  Providing the same outcomes in a single injection offers patients an option that “fits more seamlessly into their lives” SVP of U.S. immunology Ashley Diaz-Grandados explained in a company release. 

  The nod follows up on Omvoh's expansion earlier this year into Crohn's disease, another key immunology indication as the other form of inflammatory bowel disease. 

• BioMarin’s bid to widen the patient population of its phenylketonuria (PKU) treatment Palynziq is under FDA review, with a decision date of Feb. 28, 2026.

  The company is seeking a label expansion that would cover adolescents aged 12 to 17. Palynziq is the only approved enzyme substitution therapy that can treat adults with PKU, a genetic condition marked by deficiency of the phenylalanine hydroxylase (PAH) enzyme. Patients are largely diagnosed at birth and must adhere to a strict diet.

  In a study of the drug’s benefits in adolescents, BioMarin’s treatment demonstrated statistically significant lowering of blood phenylalanine (Phe) concentration compared to diet alone. The company is also looking to share the data with regulators in Europe, it said.

   

UPDATED: Friday, October 24 at 3:00 p.m. ET

• Nearly a year after Syndax Pharmaceuticals achieved its original FDA approval for acute myeloid leukemia (AML) drug Revuforj, the U.S. regulator has expanded the med's label to include patients with another subset of the disorder.

  The new nod covers those age 1 and older with relapsed or refractory AML with a susceptible nucleophosmin 1 (NPM1) mutation. In November of last year, the FDA approved the menin inhibitor for those with rearrangements of the lysine methyltransferase 2A (KMT2A) gene, which is another genetic form of AML.

  While Revuforj is the first treatment approved specifically for patients with the NPM1 mutation, another could be on the way as Kura Oncology and commercial partner Kyowa Kirin face a Nov. 30 FDA decision date for their menin inhibitor ziftomenib. 

  New York-based Syndax is on a roll with the FDA. In August of last year, the company scored an endorsement for Incyte-partnered Niktimvo, a treatment for graft-versus-host disease (GVHD).

  In the first half of this year, Revuforj generated $49 million in sales, while Niktimvo—which was launched in January of this year—pulled in $50 million.

• Azurity Pharmaceuticals has scored an FDA approval for its blood pressure medicine Javadin. The oral solution was developed for patients who have difficulty swallowing tablets or capsules.

  Javadin becomes the first immediate-release, ready-to-use oral clonidine formulation for the treatment of hypertension. The berry-flavored treatment can eliminate the need for tablet cutting, compounding or the use of transdermal delivery products to lower blood pressure.

  According to the Massachusetts-based company, a recent study showed that more than a third of primary care patients have difficulty swallowing oral medications, with many resorting to splitting or crushing their tablets or opening their capsules to ingest them.

  “Javadin lowers blood pressure within 30 to 60 minutes,” Jay Singh, Azurity’s Chief Scientific Officer, said in a release. “Unlike many antihypertensive agents that act peripherally, Javadin works centrally by stimulating alpha-adrenoreceptors in the brain, resulting in decreased peripheral and renal vascular resistance, lower heart rate, and reduced blood pressure.”

  It is the second FDA approval in a week for Azurity, which also won a recent nod for Ferabright, the first iron-based contrast agent indicated for magnetic resonance imaging (MRI) of the brain in adults with known or suspected malignant neoplasms.

• Bayer’s hormone-sensitive prostate cancer (HSPC) blockbuster Nubeqa has landed a thumbs up from England’s National Institute for Health and Care Excellence (NICE).

  The endorsement is for the use of Nubeqa along with androgen deprivation therapy (ADT) in patients with advanced HSPC if chemotherapy is not suitable.

  Clinical studies indicate that the combination of Nubeqa and ADT is more effective than ADT alone and matches the efficacy of other drugs approved by NICE that are used in combination with ADT including Astellas’ Xtandi and Johnson & Johnson’s Erleada.

UPDATED: Wednesday, October 22 at 2:05 p.m. ET

• A new indication for Amgen and AstraZeneca’s Tezspire broadens the treatment landscape for patients with chronic rhinosinusitis with nasal polyps (CRSwNP), adding the first biologic that targets thymic stromal lymphopoietin (TSLP).

  The FDA signed off on the drug’s use as an add-on maintenance treatment for adult and pediatric patients with inadequately controlled CRSwNP, a condition that impacts up to 320 million people worldwide. The disease is a complex epithelial-driven inflammatory disorder that causes persistent inflammation and benign polyp growths in the nasal cavity, leading to airflow obstruction, congestion and an impaired sense of smell.

  The nod is Tezspire’s second indication following its initial approval in severe asthma. In studies, the drug demonstrated a statistically significant and clinically meaningful reduction in nasal polyp severity and resulted in near-elimination of the need for surgery, plus a significant reduction in systemic corticosteroid use, compared to placebo.

• The European Commission also green-lit Tezspire’s use in CRSwNP, albeit with a slightly different label that covers adults with severe disease. In both markets, the drug is available as a single-use pre-filled syringe and auto-injector for self-administration. Amgen and AZ jointly commercialize Tezspire in the U.S., while AZ records product sales outside of the U.S. 

• Astellas and Merck’s combination therapy of Padcev and Keytruda has landed on the FDA’s desk as a potential treatment for certain patients muscle-invasive bladder cancer (MIBC). The agency plans a speedy review and has set a decision date of April 7, 2026.

  The companies are positioning the combo regimen as a perioperative therapy that can treat MIBC patients who are ineligible for cisplatin-containing chemotherapy both before and after a radical cystectomy. Astellas and Keytruda’s phase 3 study, presented at the recent European Society of Medical Oncology (ESMO) congress, showed that the combination cut patients' risk of disease recurrence, progression or death by 60%—and slashed the risk of death by 50%—compared to surgery alone.

  If approved, the regimen would be a key option for patients who can’t take cisplatin-based chemotherapy. Antibody-drug conjugate Padcev plus PD-1 inhibitor Keytruda is currently approved to treat patients with locally advanced or metastatic urothelial cancer. 

• Boehringer Ingelheim’s idiopathic pulmonary fibrosis (IPF) treatment Jascayd has already crossed the regulatory finish line in two major markets, with an approval from China’s National Medical Products Administration (NMPA) coming two weeks after its first nod from the FDA.

  The oral drug is the first new IPF therapy in over a decade, marking a welcome new option for the up to 3.6 million people worldwide that may be affected by the progressive fibrosing interstitial lung disease. Patients often succumb to the disease within five years of diagnosis and suffer symptoms such as a dry and persistent cough, shortness of breath and fatigue. 

• Sanofi and Regeneron’s Dupixent is growing its reach in chronic obstructive pulmonary disease (COPD) with a new nod in Canada.

  Health Canada approved the drug as an add-on maintenance treatment for adult patients with COPD that’s characterized by raised blood eosinophils (type 2 inflammation) and that is inadequately controlled by the combination of an inhaled corticosteroid, a long-acting beta2-agonist and a long-acting muscarinic antagonist. 

  The Canada nod is Dupixent’s sixth in the country and adds to the drug’s list of global COPD approvals in Europe, the U.S., China and Japan. COPD is the fifth-leading cause of death in Canada and the fourth-leading cause worldwide, according to the partners.

UPDATED: Tuesday, October 21 at 10:30 a.m. ET

• Right on the heels of a first-of-its-kind data readout for Summit Therapeutics and Akeso's ivonescimab at the European Society for Medical Oncology congress, Summit has revealed regulatory plans for the PD-1xVEGF bispecific in the U.S. for another indication. 

  Based on results from the HARMONi clinical trial, Summit plans to seek FDA approval for ivonescimab and chemo as a treatment for patients with previously treated EGFR-mutated nonsquamous non-small cell lung cancer (NSCLC).

  In the global HARMONi study, the combo reduced patients' risk of progression or death by 48% compared with chemotherapy alone. While the trial didn't reach statistical significance on its other dual primary endpoint, overall survival, Summit believes the comprehensive data package shows that patients in the setting "can benefit from the ivonescimab regimen," according to an Oct. 20 press release (PDF).

  Previously, the FDA has "noted that a statistically significant overall survival benefit is necessary to support marketing authorization in this setting," Summit said in its release. Nonetheless, the company is forging ahead "after careful consideration" and based on "discussions with key opinion leaders and those physicians who have administered ivonescimab to patients in a clinical study setting."

  The disclosure comes right after the drug showed it can delay tumor progression versus a PD-1 inhibitor as part of a combination with chemotherapy in patients with first-line NSCLC. Those data are from a China-only study.
   

UPDATED: Wednesday, October 15 at 1:30 p.m. ET

• China’s National Medical Products Administration (NMPA) has expanded its approval for GSK’s shingles shot Shingrix, allowing it to be used by adults age 18 and older who are at an increased risk of infection due to immunodeficiency or immunosuppression caused by known disease or therapy.

  The application was supported by six clinical trials of those aged 18 years and older who had undergone recent stem cell transplantation, kidney transplant or who have blood cancer, solid tumors or HIV. 

  In 2020, China endorsed the shot for people age 50 and older. Shingrix remains the only vaccine available in the country to protect against the infection. Roughly 6 million cases of shingles are diagnosed every year in China.

  The FDA approved Shingrix for people 50 and older in 2017 and followed with a 2021 nod which expanded its use to adults who are immunocompromised.

UPDATED: Monday, October 13 at 11:18 a.m. ET

• Teva is unlocking a major market for Uzedy with a new FDA approval for the medicine.

  The FDA cleared the drug, which is a long-acting form of the atypical antipsychotic risperidone, as a once-monthly monotherapy or add-on therapy to lithium or valproate for the maintenance treatment of bipolar I disorder (BD-1) in adults. The approval was based on existing clinical data for Uzedy and previous findings on approved risperidone formulations.

  Teva’s offering to the bipolar market "may address existing unmet needs and treatment gaps," the company's U.S. head of commercial, Chris Fox, said in a statement. It's the first marketed subcutaneous, long-acting form of risperidone that uses Teva's SteadyTeq technology to control the steady release of the drug.

  Uzedy won its original approval in 2023 as a treatment for schizophrenia. 

• A new biosimilar to Regeneron and Bayer’s blockbuster eye drug Eylea has been cleared by the FDA in Celltrion’s Eydenzelt.

  The drug, an injectable VEGF inhibitor, won its FDA approval in the same indications Eylea currently holds. In trials, Eydenzelt proved biosimilarity to Eyelea in a 52-week trial measuring corrected visual acuity and other secondary endpoints.a

• MannKind’s insulin powder Afrezza is up for FDA review in a pediatric population that would make for the first needle-free insulin option for children and adolescents in the U.S.

  The FDA expects to decide on Afrezza’s potential label expansion by May 29, 2026. MannKind’s application incudes data from its phase 3 INHALE-1 study in people between the ages of 4 to 17 who have Type 1 or Type 2 diabetes. In the study, Afrezza combined with basal insulin was compared to multiple daily injections with basal insulin and proved non-inferiority in patients' mean change in HbA1c levels over 26 weeks. 

  The drug, an inhaled insulin powder, was first approved for adults in 2014 and is recognized as a standard of care by the American Diabetes Association. 

• The FDA tweaked two indications that it had previously granted to AbbVie’s Rinvoq, allowing the drug to treat certain additional patients.

  Rinvoq’s indications for moderately to severely active ulcerative colitis (UC) and moderately to severely active Crohn's disease (CD) were previously limited to adult patients who had an inadequate response or intolerance to one or more tumor necrosis factor (TNF) blockers. Now, the FDA has broadened the scope of those indications by allowing Rinvoq to also be used in UC and CD patients who have received at least one approved systemic therapy in the event TNF blockers are “clinically inadvisable.”

  The label update allows new options to physicians treating UC and Crohn’s, which are diseases that can impact “every aspect of a patient’s life,” AbbVie’s head of immunology clinical development Kori Wallace, M.D., Ph.D. said in a statement. Rinvoq earned its UC nod in 2022, with CD added to the label in 2023. 

UPDATED: Thursday, October 9 at 10:45 a.m. ET

• The National Institute for Health and Care Excellence (NICE) has changed its tune on Pfizer’s lung cancer med Lorviqua, endorsing the therapy as a first-line treatment after initially turning it down in 2023.

  Now, patients in England who have anaplastic lymphoma kinase (ALK)-positive advanced non-small-cell lung cancer (NSCLC) and who have not previously received an ALK inhibitor can access the drug through the National Health Service (NHS). Previously, Lorviqua was only available as a second-line option in England, although NHS Scotland has offered it for first-line treatment since 2022.

  Back in 2023, NICE wasn’t sure enough of the drug’s benefits over existing treatments to ensure it would be a good use of NHS resources.

  “Since there’s currently no cure for the disease, it’s essential that patients are able to access therapies that can help them have a good quality of life and live progression-free for years,”  Yvonne Diaz, co-founder of Oncogene Cancer Research, explained in a statement.

• Johnson & Johnson’s Simponi can reach a larger patient population after it won a label extension from the FDA enabling its use in children with moderate to severe ulcerative colitis (UC).

  With the nod, Simponi is the first UC treatment that can offer a monthly maintenance dose in the pediatric population. In its phase 3 PURSUIT program, the company found that 32% of patients achieved clinical remission by week 6 and some 57% of those patients maintained the clinical remission of symptoms through week 54.

  Simponi originally hit the market in 2009 as a treatment for rheumatoid arthritis, psoriatic arthritis and ankylosing spondylitis. It later picked up a nod as a treatment for adults with UC in 2013.

  The treatment is administered through a pre-filled syringe and can be self-administered by patients ages 12 and older after proper training. About 20% of the over 1 million people with UC in the U.S. are pediatric patients, according to J&J. 

• Young children with mild to moderate atopic dermatitis have a new topical treatment option in Arcutis Biotherapeutics’ Zoryve cream.

  With a new FDA approval, the steroid-free, once-daily cream can now be used to treat children ages 2 to 5 years anywhere on the body for any duration of time.

  Atopic dermatitis is typically diagnosed during childhood. The younger patient population is particularly impacted by the itch symptoms of the disease, as their skin barrier is less developed.  Zoryve importantly does not include “ingredients known to compromise the skin barrier,” Arcutis CEO Frank Watanabe noted in a statement. 

  The nod makes for Zoryve’s sixth in just over three years, adding to its prowess as the top prescribed branded topical therapy across the three major inflammatory skin diseases.

UPDATED: Friday, October 3 at 11:00 a.m. ET

• In the 11th hour before the government shutdown this week, the FDA approved a second generic version of the abortion pill mifepristone.

  In a letter sent out (PDF) Tuesday, the FDA signed off on Evita Solution’s application for 200 mg mifepristone tablets, deeming them bioequivalent and therapeutically on par with Danco Laboratories’ original reference product Mifeprex.

  The approval was a long time coming for Evita, which filed its application for review with the FDA back in October of 2021. With its green light, the FDA has now cleared two copycat versions of mifepristone for the U.S. market. Up until now, GenBioPro was the only other company with an approved mifepristone generic in the U.S., receiving sign-off for its product in 2019.

  The green light comes as access to abortion medication is under serious fire in the U.S. Last year, the Supreme Court moved to preserve mail-order access to mifepristone in a move hailed by the biopharma industry. But as of 2025, new FDA leadership installed under the Trump administration has sought to revive scrutiny of the Risk Evaluation and Mitigation Strategies (REMS) for the drug, which has now been on the market for 25 years.

  The approval of Evita’s generic has stoked fresh condemnation from some Republican lawmakers, like Sen. Josh Hawley, R-Mo., who called the FDA’s decision “shocking” in a post on X.

  Nevertheless, certain pro-choice states and members of the industry have continued to push for maintained mifepristone access this year. 

UPDATED: Monday, September 29 at 2:30 p.m. ET

• Johnson & Johnson's Tremfya continues to expand its reach as now the FDA has approved the IL-23 inhibitor as a treatment for two pediatric patient populations.

  The agency endorsed Tremfya as a treatment for children 6 years and older who weigh at least 40 kg (88 pounds) with moderate to severe plaque psoriasis (PsO) who are candidates for systemic therapy or phototherapy.

  In addition, children 6 and older with active psoriatic arthritis are now eligible for the drug, provided they weigh at least 40 kg, J&J said in a Sept. 29 release.

  The agency considered numerous data sources in making the endorsements. In PsO, J&J ran a late-stage pediatric study called PROTOSTAR. The trial showed that Tremfya outperformed placebo when it came to helping patients achieve certain psoriasis improvement milestones.

  "The approval of the first and only pediatric indications for an IL-23 inhibitor marks an important step forward not only for children, but also for the parents and care partners who support them every day," Brandee Pappalardo, VP of medical affairs for dermatology and rheumatology at J&J innovative medicines, said in a statement.

  We remain committed to advancing research that demonstrates the long-term safety and efficacy of Tremfya and to exploring its full potential for adult and pediatric patients," Pappalardo added.

UPDATED: Wednesday, September 24 at 8:45 a.m. ET

• The combination of AstraZeneca and Daiichi Sankyo's Enhertu and Roche's Perjeta has gained an FDA priority review as a potential first-line treatment for patients with HER2-positive metastatic breast cancer.

  With the speedy review, the FDA aims to make a decision on Daiichi and AZ's application by January 23, 2026, the partners said in a Sept. 24 release.

  The companies are leveraging data from the DESTINY-Breast09 clinical trial in their FDA filing. In the study, the Enhertu and Perjeta combination reduced patients' risk of disease progression or death by 44% compared with the current standard of care.

  Enhertu is already approved across a range of cancer indications. In breast cancer, the drug is approved to treat patients with unresectable or metastatic HER2-positive cancer who have received a prior anti-HER2-based regimen.

  “Receiving priority review moves us closer to offering Enhertu to patients even earlier in the metastatic treatment pathway as a potential new first-line treatment option," Daiichi's head of R&D, Ken Takeshita, M.D., said in the release.

• In Australia, Biogen and Eisai's Leqembi has secured approval to treat mild cognitive impairment or mild dementia due to Alzheimer's disease in adult patients who are either Apolipoprotein E non-carriers or heterozygous carriers.

  Australia's Therapeutic Goods Administration (TGA) previously rejected the medicine in February 2025, but Eisai requested a review and was ultimately able to win over the regulator.

  As of last year, an estimated 425,000 people with dementia lived in Australia, Biogen said in a Sept. 24 release.

UPDATED: Thursday, September 18 at 10:17 a.m. ET

• Europe’s first therapy for postpartum depression (PPD) has arrived with the approval of Biogen’s Zurzuvae.

  The European Commission signed off on the med, a once-daily oral pill meant to be taken over a 14-day treatment course, to treat PPD in adults following childbirth. PPD is a leading cause of maternal mortality in Europe and is one of the most common medical conditions linked to pregnancy, as up to 20% of women experience symptoms, according to Biogen. However, many cases go undiagnosed and untreated due to varying clinical guidelines for depression management during and after pregnancy.

  The nod was based on Biogen’s Skylark study. The trial met its primary endpoint, with treated patients achieving significant reductions on the Hamilton Rating Scale for Depression (HAMD-17), a common measure for weighing depression severity, at day 15 compared to placebo. 

  Zurzuvae first hit the market in the U.S. in 2023 and most recently nabbed a nod in the U.K. 

• Ono Pharmaceutical’s Deciphera subsidiary can introduce a new treatment option for underserved patients with specific tumor type following the European Commission's recent approval for Romvimza (vimseltinib).

  Romvimza is indicated in Europe to treat adult patients with symptomatic tenosynovial giant cell tumors (TGCT) associated with clinically relevant physical function deterioration for those who have exhausted surgical options or who can’t pursue surgery due to unacceptable risks.

  The tumor type is a rare, locally aggressive neoplasm that is caused by a translocation in the colony-stimulating factor 1 (CSF1) gene and can grow and damage surrounding tissues or structures.

  Surgery is usually the main treatment option, but the tumors often recur. For some, surgical resection can cause severe morbidity.

  In studies, the drug showed statistically significant and clinically meaningful improvements across a range of motion, physical functioning and pain measures.

• AstraZeneca’s FDA label for rescue asthma inhaler Airsupra has been updated to include new evidence from a study further demonstrating the drug’s efficacy.

  Specifically, the agency added results from the company’s phase 3b Batura trial to the label. The trial showed that as-needed Airsupra reduced patients' risk of severe exacerbation by 46%, compared with as-needed albuterol, in those with mild asthma. 

  Albuterol is the U.S.’s most commonly used rescue medication for asthma, according to AstraZeneca. 

  With the label change, patients across “all asthma severities have a superior choice for their asthma rescue medication,” the company’s VP of U.S. respiratory and immunology, Liz Bodin, said in a statement. 

UPDATED: Wednesday, September 17 at 10:35 a.m. ET

• For patients with limited-stage small cell lung cancer (LS-SCLC) living in the United Kingdom, a new treatment option has arrived at long last, courtesy of hometown hero AstraZeneca.

  Thanks to an endorsement (PDF) from England’s National Institute for Health and Care Excellence (NICE), AZ’s immunotherapy Imfinzi, also known as durvalumab, is now recommended for use by the country’s National Health Service (NHS) in adults with LS-SCLC that has not progressed after platinum-based chemoradiotherapy.

  To date, treatment options for LS-SCLC patients in the U.K. have been limited, revolving around active monitoring through regular doctor visits and scans, NICE said in its recommendation.

  While most people with small-cell lung cancer see their disease metastasize and spread to other parts of the body, the cancer is contained to a single area that can be treated with radiotherapy in the roughly 30% of patients who develop LS-SCLC, the UK price watchdog noted. There have been limited advances in the treatment of LS-SCLC over the past few decades and previously, no maintenance therapies existed for the condition following chemotherapy and radiotherapy, NICE added.

  NICE based its guidance on results from AstraZeneca’s late-stage Adriatic trial, which showed that Imfinzi helped significantly extend patients’ lives over placebo. The organization said that it believes AZ’s drug is cost effective given the potential benefits it offers LS-SCLC patients.

  Aside from the U.K. endorsement, Imfinzi has also picked up LS-SCLC green lights in places like the United States and the European Union. 

UDPATED: Monday, September 15 at 9:35 a.m. ET

• Novo Nordisk’s oral GLP-1 Rybelsus can now be used to improve cardiovascular outcomes for patients in Europe after the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) approved a label update that reflects the drug's cardiovascular benefits.

  Rybelsus, approved in 2019, is Novo’s only approved oral version of its semaglutide and is currently indicated to treat type 2 diabetes. In the company’s phase 3b SOUL trial, the drug demonstrated a superior reduction in major adverse cardiovascular events (MACE) compared with placebo. The company plans to present additional findings from the trial later this week at the European Association for the Study of Diabetes annual meeting.

  Novo expects a decision on Rybelsus’s label update to also come in the U.S. this year. The drugmaker has additionally filed an FDA application for a pill version of its Wegovy, another semaglutide-based product that treats obesity and cardiovascular disease. 

• Krystal Biotech’s topical gene therapy Vyjuvek received a FDA label update that allows the drug's use in newborn babies and, in other age groups, at-home administration by patients and caregivers.

  Vyjuvek is a gel that treats wounds caused by rare skin disorder dystrophic epidermolysis bullosa (DEB) by delivering copies of the COL7A1 gene, acting as a crucial “glue” to hold the skin layers together. Patients with DEB have extremely fragile skin and are prone to wounds and blistering due to mutations in the COL7A1 gene.

  Along with clearing at-home administration by patients and their caregivers, the FDA label update also enables an easier wound dressing routine by permitting wound dressings to be removed during regular dressing changes as opposed to the previous rule of waiting 24 hours for re-application. The update stands to help Vyjuvek integrate further into existing wound care routines, Krystal pointed out, and was approved based on real-world data and an open-label extension study. 

   

UDPATED: Thursday, September 11 at 8:58 a.m. ET

• The FDA has decided that a previously planned advisory committee meeting to discuss Travere Therapeutics' application for Flispari in focal segmental glomerulosclerosis (FSGS) is no longer needed, the company said.

  The FDA is still reviewing the submission with a target action date of Jan. 13, 2026. 

  If approved, Filspari would be the first medication for FSGS, a rare and serious kidney disorder defined by progressive scarring of the kidney that often leads to kidney failure. The disease is estimated to affect more than 40,000 patients in the U.S., with a similar prevalence in Europe, according to Travere.

  "For Filspari, we think the cancelation bodes well for eventual approval," Jefferies analysts said in Sept. 10 note.

  Uncertainty surrounding the application stemmed from a phase 3 flop in which Filspari failed to significantly beat off-label irbesartan when looking at changes in patients' estimated glomerular filtration rate (eGFR), a measure of kidney function, after 108 weeks.

  However, a recent public-private collaboration called Proteinuria and GFR as Clinical Trial Endpoints in Focal Segmental Glomerulosclerosis (PARASOL), which the FDA is involved in, suggested a transition away from eGFR in favor of Urine Protein-to-Creatinine Ratio (UPCR) as an approvable endpoint. Filspari has demonstrated its ability to reduce proteinuria, measured by UPCR, in clinical trials.

• The Chinese National Medical Products Administration (NMPA) has approved Sanofi's Tzield (teplizumab) as the first disease-modifying therapy in autoimmune Type 1 diabetes (T1D). The drug is cleared to delay the onset of stage 3 T1D in patients ages 8 and older with stage 2 disease.

  The approval is based on the positive results from the TN-10 phase 2 study, which showed that Tzield delayed the median onset of stage 3 T1D by 48.4 months vs 24.4 months observed in the placebo group.

  “This approval represents the beginning of a new era of care for stage 2 type 1 diabetes patients in China, one focused on the potential of Tzield to prevent the natural progression of T1D by its unique beta-cell function preserving capabilities,” Olivier Charmeil, Sanofi's EVP of general medicines, said in a statement.

   

UPDATED: Tuesday, September 9 at 8:15 a.m. ET

• Takeda's Vonvendi, a recombinant factor replacement therapy for patients with von Willebrand Disease (VWD), has picked up a label expansion from the FDA.

  The drug is now approved to be given as a routine prophylaxis to reduce the frequency of bleeding episodes in adult patients, including those with Type 1 and 2 disease, Takeda said in a Sept. 8 release. 

  Further, the drug scored a green light be used as an on-demand and perioperative bleeding management therapy for pediatric patients.

  Previously, Vonvendi was only approved for on-demand and perioperative use in adults with VWD and for routine prophylactic use in adults with severe Type 3 VWD.

  With the new nod, the drug can be used in a broad range of patients with the genetic bleeding disorder.

• In Canada, partners Medison Pharma and Ipsen announced the regulatory approval of Bylvay to treat cholestatic pruritus in patients 12 and older with Alagille Syndrome.

  Two years ago, the drug picked up a Canadian approval to treat pruritus in patients 6 months or older with Progressive Familial Intrahepatic Cholestasis (PFIC).

  With the latest nod, Bylvay is the only drug approved in Canada to treat the pair of liver-related diseases.

  Medison and Ipsen are in a multiregional partnership to bring the drug to patients in Canada and Israel. 

   

UPDATED: Thursday, September 4 at 9:10 a.m. ET

• Novavax's LP.8.1-formulated COVID-19 vaccine, Nuvaxovid, has gained regulatory approval in Japan.

  The shot is approved as an initial immunization (first and second vaccination) for people age 6 and older and as a booster for people 12 and older, Novavax said in a Sept. 4 release.

  Under a 2020 licensing deal, Takeda is responsible for production and commercialization of the vaccine in Japan. Earlier this year, Novavax said it had secured better financial terms for the deal.

  The new approval triggers an undisclosed milestone payment from Takeda to Novavax, the latter company said in its release this week.

   

UPDATED: Thursday, September 4 at 8:50 a.m. ET

• The FDA is pushing back its decision target date for Agios' Pyrukynd in thalassemia by three months after the company provided additional information to support its application. 

  The agency now expects to make a decision on Agios' filing by Dec. 7. Previously, the agency had targeted Sept. 7 for its decision.
  
  Agios is seeking approval for its oral pyruvate kinase (PK) activator to treat adults with non-transfusion-dependent (NTD) and transfusion-dependent (TD) alpha- or beta-thalassemia.
  
  During the review, an FDA information request prompted Agios to submit a proposed Risk Evaluation and Mitigation Strategy to reduce patients' risk of experiencing hepatocellular injury. This constituted a major amendment to the filing, resulting in the delay, Agios said in a Sept. 4 release.
  
  “We look forward to continuing our collaborative engagement with the FDA, with the goal of bringing this disease-modifying oral medicine to adult patients with thalassemia in the U.S.," Agios CEO Brian Goff said in a statement.

• In Japan, Roche's Chugai announced it's implementing new safety measures for the Duchenne muscular dystrophy gene therapy Elevidys.

  Following a review of the therapy's available safety information, Chugai said it has agreed to add acute liver failure as a serious adverse reaction to the medicine's label in Japan. Going forward, patients will need to undergo pre- and post-administration tests for detection of liver dysfunction and liver failure, Chugai said in its Sept. 4 release.

  The drug is approved to treat ambulatory DMD patients ages 3 to 8 in Japan.

  The development comes after two non-ambulatory patients who had received the drug passed away in the U.S.

UPDATED: Wednesday, September 3 at 8:35 a.m. ET

• On the heels of securing an approval for a subcutaneous version of Leqembi for maintenance treatment of Alzheimer's disease, Eisai has started an FDA rolling submission seeking another approval for the autoinjector, dubbed Leqembi Iqlik, as a weekly starting dose.

  The company and its partner Biogen are proposing Leqembi Iqlik, administered in two 250 mg injections, as a once-weekly starting dose, versus the current 10 mg/kg intravenous dosing once every two weeks. 

  During the maintenance phase, the subcutaneous formulation comes with 360 mg of the drug in a single-dose prefilled autoinjector, which takes about 15 seconds to administer.

  The autoinjector "would expand the option for patients and care partners to receive Leqembi treatment from initiation to maintenance at home," Eisai said in a statement.

  The application is based on data from the open-label extension cohort of the phase 3 Clarity AD trial. Eisai did not mention when it plans to finish the rolling submission.

UPDATED: Friday, August 29 at 10:40 a.m. ET

• The FDA is adding an earlier safety monitoring recommendation to the prescribing information for Eisai and Biogen's Alzheimer’s disease treatment Leqembi. 

  Patients receiving Leqembi are now recommended to undergo an MRI before receiving their third infusion of the drug in order to identify those with amyloid-related imaging abnormalities with edema (ARIA-E).

  In Leqembi’s prior FDA prescribing information, MRI imaging was recommended before patients' 5th, 7th and 14th infusions. After an “in-depth analysis” of the ARIA-E safety issue, the agency has determined that earlier monitoring could help alleviate the risk. The recommendations for the subsequent scans are unchanged.

  Specifically, the FDA identified six deaths early in treatment, prompting a deeper look into serious ARIA-E outcomes prior to patients' 5th infusion of Leqembi.

  As such, the FDA will update its prescribing information to note the recommended MRI between the 2nd and 3rd Leqembi infusions. ARIA-E is characterized by brain swelling or fluid buildup and is often asymptomatic, although serious and life-threatening events can occur. 

  The prescribing information for Eli Lilly’s rivaling Alzheimer’s disease treatment Kisunla calls for MRIs before patients' 2nd, 3rd, 4th and 7th infusions. 

• Travere Therapeutics’ IgA nephropathy (IgAN) med Filspari has new safety monitoring language in its FDA prescribing information.

  The agency updated the drug’s Risk Evaluation and Mitigation Strategy (REMS) labeling to reduce the frequency of liver function monitoring to every three months after treatment starts and to remove the previous embryo-fetal toxicity (EFT) monitoring requirement. 

  Previously, patient liver function monitoring was recommended monthly.

• GSK’s uncomplicated urinary tract infection (uUTI) treatment Blujepa is cleared for use in the U.K. after scoring a nod from the Medicines and Healthcare products Regulatory Agency (MHRA).

  The pill is a new oral antibiotic that can be used in females age 12 and older. GSK’s offering represents a key new treatment option to target increasingly drug-resistant bacteria and is meant to be taken twice daily for five days.

  If untreated, uUTI’s can cause sepsis or permanent kidney damage. 

• Biogen’s Zurzuvae is poised to unlock the U.K. postnatal depression market with an approval from the MHRA.

  The drug is the first oral treatment cleared to treat moderate or severe postnatal depression in the U.K., a common condition that affects more than 1 in every 10 women within a year of giving birth. 

  Postnatal depression (or postpartum depression) can consist of persistent feelings of sadness and anxiety that can manifest in extreme fatigue, changes in appetite and disrupted sleep patterns. 

  Zurzuvae made it to the U.S. in 2023 and comes as a capsule that is meant to be taken nightly for 14 days. 

UPDATED: Wednesday, August 27 at 9:20 a.m. ET

• BeOne Medicines, the company formerly known as BeiGene, has impressed European regulators once again with its PD-1 drug Tevimbra. 

  On Wednesday, the European Commission approved Tevimbra in combination with platinum-containing chemotherapy as a neoadjuvant (pre-surgery) treatment, followed by Tevimbra monotherapy after surgery, for adults with resectable non-small cell lung cancer (NSCLC) at high risk of disease recurrence. 

  Europe's drug regulator granted the approval recommendation after reviewing data from BeOne's phase 3 RATIONALE-315 study, which demonstrated a "statistically significant and clinical meaningful" benefit to overall survival for patients on Tevimbra versus those on chemotherapy and placebo, the company said. 

  The drug also demonstrated a "significant" event-free survival benefit, BeOne noted in a press release. 

  The green light marks Tevimbra's fifth lunger cancer approval in the European Union. Meanwhile, the European Commission in July endorsed the drug in a separate indication, clearing Tevimbra plus the chemotherapy drugs gemcitabine and cisplatin for initial treatment of adults with metastatic or recurrent nasopharyngeal carcinoma (NPC).

UPDATED: Tuesday, August 26 at 11:00 a.m. ET

• The European Commission has signed off on Gilead Sciences' long-acting injection, Yeytuo, for preventing HIV infection. 
  
  In granting marketing authorization for the transformational prophylactic, which is administered every six months, Europe joins the U.S., which approved the drug under the commercial name Yeztugo two months ago. The World Health Organization also put its stamp of approval on the product last month.

  Yeytuo was reviewed in Europe under an accelerated pathway as a product of major interest for public health. The approval follows a positive opinion last month from the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP). 
  
  The nod also comes with an added year of market protection in Europe as regulators determined that Yeytuo brought significant clinical benefit compared to existing therapies.

  “With around 25,000 new HIV diagnoses in the EU and European Economic Area every year, it’s clear that current prevention options are not working for everyone who needs or wants them, especially among vulnerable populations,” Jean-Michel Molina, M.D., who is the head of the infectious diseases department at the Saint-Louis and Lariboisière Hospitals in Paris, said in Gilead’s release.
  
  Gilead has filed for review of the PrEP medicine in several other countries including Australia, Brazil, Canada, Argentina, Mexico and South Africa.

• Canada has approved Moderna’s updated version of Spikevax, its COVID-19 mRNA vaccine targeting the currently circulating SARS-CoV-2 LP.8.1 variant.

  The shot will be produced at Moderna’s drug substance site in Quebec, with Novocol Pharma performing the fill-finish duties in Ontario, the company said. It will be provided in a pre-filled syringe and is endorsed for those age 6 months and older.

  Europe signed off on the updated version of Spikevax four weeks ago. In May, the FDA endorsed Moderna’s next-generation COVID vaccine mNEXSPIKE for use in all adults 65 and older, as well as in individuals ages 12 to 64 who have one or more underlying risk factor for severe COVID.

UPDATED: Monday, August 25 at 9:40 a.m. ET

• The FDA has approved Amgen's Repatha as a treatment for adults at an increased risk for major adverse cardiovascular events (MACE) due to uncontrolled low-density lipoprotein cholesterol (LDL-C), or "bad cholesterol." 

  The label update removes a requirement that patients be diagnosed with cardiovascular disease to be eligible for the PCSK9 inhibitor, Amgen noted in a press release.

  In addition, the FDA endorsed Repatha as a monotherapy to treat patients with a rare, genetic form of high cholesterol known as homozygous familial hypercholesterolemia.

  Repatha generated $2.22 billion in sales in 2024, a 36% increase from the prior year.

• In China, regulators have approved AstraZeneca and Daiichi Sankyo's Datroway for adults with unresectable or metastatic HR-positive, HER2-negative breast cancer who have received prior endocrine therapy and at least one line of chemotherapy in the advanced setting.

  About 357,000 cases of breast cancer were diagnosed in China in 2022, according to a Monday press release from Daiichi Sankyo.

  China's National Medical Products Administration leveraged results from the TROPION-Breast01 phase 3 study to make the approval decision. In the study, Datroway reduced patients' risk of disease progression or death by 37% compared to investigator's choice of chemotherapy.

  Datroway is a TROP2-directed DXd antibody-drug conjugate discovered by Daiichi Sankyo.

UPDATED: Friday, August 22 at 8:45 a.m. ET

• England's National Institute for Health and Care Excellence (NICE) has endorsed the combination of Astellas' Padcev and Merck & Co.'s Keytruda as a treatment for unresectable or metastatic urothelial cancer in patients who are eligible for platinum‑containing chemotherapy.

  The combo represents a new first-line treatment for adults with advanced bladder cancer, offering a potential "step change" in caring for patients, NICE said in an Aug. 21 press release. 

  Evidence from clinical trials shows that the drugs can "significantly" extend progression-free survival and overall survival compared with standard chemotherapy treatments, according to the agency.

  The combination therapy is available through the National Health Service starting Aug. 21.

• In China, regulators have approved the world's first hexavalent rotavirus vaccine.

  The oral vaccine, developed by a subsidiary of Sinopharm, is approved for the prevention of acute gastroenteritis in infants and young children caused by rotavirus, according to an Aug. 22 announcement from the company.

UPDATED: Wednesday, August 20 at 8:35 a.m. ET

• After Madrigal's Rezdiffra broke ground in the United States as the first approved medication for the treatment of metabolic dysfunction-associated steatohepatitis (MASH), the drug has now done the same in Europe.

  The European Commission granted a conditional approval to the therapy as a treatment for adults with noncirrhotic MASH with moderate to advanced liver fibrosis, Madrigal said in a Tuesday press release. The drug's EU label doesn't stipulate the need for a biopsy to be eligible for treatment, Madrigal CEO Bill Sibold noted in a statement.

  The disease poses an increasing burden on healthcare systems worldwide and is a leading cause of liver-related mortality, according to the drugmaker. In Europe, the company estimates some 370,000 patients are already diagnosed with MASH with moderate to advanced fibrosis.

  In a phase 3 trial, Rezdiffra met both its fibrosis reduction and MASH resolution primary endpoints. 

  To advance the launch, Madrigal plans to engage in reimbursement discussions with individual EU countries. The timing of the launches in EU countries will vary, starting with Germany later this year, the company said.

UPDATED: Tuesday, August 19 at 11:40 a.m. ET

• Novo Nordisk’s Ozempic has stacked another use on top of its existing Canadian label, allowing it to treat a broader group of patients in the country.

  The new nod clears the medicine to reduce the risk of sustained estimated glomerular filtration rate (eGFR) decline, end-stage kidney disease, and cardiovascular death in adults with type 2 diabetes and chronic kidney disease (CKD), according to Novo's Aug. 19 press release.

  Health Canada originally approved the drug for improving glycemic control in those with type 2 diabetes mellitus. 

  About 4 million Canadians are impacted by CKD and around 40% of those with type 2 diabetes develop CKD, according to Novo. Ozempic proved itself in its FLOW trial, which began in 2019 and ended early due to overwhelming signs of efficacy. In the study, the drug showed that it can delay the progression of CKD and lower patients' risk of kidney and cardiovascular mortality.

  The FDA approved Ozempic in the same use earlier this year.

UPDATED: Monday, August 18 at 2:37 p.m. ET

• Valneva’s chikungunya vaccine Ixchiq has won clearance for use in a broader population in Canada.

  Health Canada has authorized use of the vaccine in people aged 12 years and older, expanding the shot's previous Canadian nod in adults. The label extension also includes new antibody persistence data showing that Ixchiq's immune response was sustained for 24 months in 97% of patients, with that response equally durable in younger and older adults.

  Meanwhile, in the six-month data that won Ixchiq its label expansion, a single dose of the vaccine achieved a high and sustained immune response in 99.1% of adolescents. 

  The Canadian nod comes after Valneva scored an adolescent label extension for Ixchiq in Europe in April.

  Ixchiq became the world’s first chikungunya vaccine to win approval in an endemic country earlier this year with a marketing authorization for adults in Brazil. In the U.S., the vaccine is cleared for use in adults of all ages who are at “high risk” of exposure to the virus. 

• SpringWorks Therapeutics has been cleared to launch the world's first desmoid tumor treatment Ogsiveo in Europe. 

  The company—which was recently acquired by Germany's Merck KGaA—won marketing authorization from the European Commission for its oral gamma secretase inhibitor as a monotherapy for adults with progressing desmoid tumors who require systemic treatment. 

  The rare, locally aggressive tumor type is difficult to manage due to its unpredictable nature. Desmoid tumors form in the connective tissues of the body, causing severe pain, loss of mobility, limited function and disfigurement. In Europe, about 1,300 to 2,300 new desmoid tumor cases are diagnosed yearly, according to SpringWorks. 

  In SpringWorks’ phase 3 DeFi study, Ogsiveo charted a statistically significant improvement over placebo by helping reduce patients' risk of disease progression by 71%. The drug also won out on metrics like objective response rate and patient-reported outcomes. 

  Ogsiveo was approved in the U.S. in 2023. 

UPDATED: Thursday, August 14 at 9:35 a.m. ET

• Shilpa Medicare is ushering in a landmark treatment for non-alcoholic fatty liver disease (NAFLD) with a world-first approval in India, according to an Aug. 14 press release from the company.

  India's Central Drugs Standard Control Organization signed off on Shilpa’s nor ursodeoxycholic acid (NorUDCA) tablets, marking the first-ever therapy cleared to target the liver disease, the company said. 

  NAFLD impacts 1.2 billion people across the world, or 1 in 4 people, with an estimated 188 million patients living in India. The disease often goes undiagnosed until irreversible damage occurs, according to Shilpa. 

  NorUDCA works through a dual-action mechanism that joins anti-inflammatory benefits with bile acid regulation, helping to hinder NAFLD from progressing into liver complications such as cirrhosis, liver failure and nonalcoholic steatohepatitis (NASH), which is the most severe form of NAFLD.

  The company looks to bring the treatment to other markets across the globe and aims to rapidly commercialize NorUDCA, according to the release.

UPDATED: Monday, August 11 at 8:50 a.m. ET

• GSK's gepotidacin has secured an FDA priority review as a potential treatment for uncomplicated urogenital gonorrhea, the company said in a Monday press release.

  Specifically, the FDA is reviewing GSK's filing for the drug in patients 12 and older who weigh at least 45 kilograms (99 pounds), with a decision expected by Dec. 11.

  If approved, the drug would become the first oral option in this indication, according to Sharecast News. As it stands, patients with uncomplicated urogenital gonorrhea need to undergo treatment with injectable therapies.

  In a phase 3 trial, GSK's drug demonstrated non-inferiority to intramuscular ceftriaxone and oral azithromycin, a leading treatment regimen for gonorrhea. 

  Gepotidacin, a first-in-class antibiotic, scored its original FDA approval earlier this year as a treatment for uncomplicated urinary tract infections.

• In England, the National Institute for Health and Care Excellence (NICE) has endorsed Chiesi's Raxone as a treatment for patients 12 and older with visual impairment caused by Leber's hereditary optic neuropathy (LHON). 

  With the decision, Raxone becomes the first drug endorsed by NICE in this indication, according to an Aug. 7 press release.

  The disease is caused by a genetic mutation that prevents cells in patients' eyes from functioning normally, according to NICE. It usually starts with blurred vision in one or both eyes and can quickly progress to blindness. LHON primarily affects young men and boys.

  It's estimated about 250 people in England could be eligible for the therapy, according to NICE.

UPDATED: Wednesday, August 6 at 8:55 a.m. ET

• The FDA has approved Teva's Ajovy for the preventive treatment of episodic migraine in children and adolescents ages 6 to 17 who weigh at least 45 kilograms (99 pounds).

  With the nod, Ajovy becomes the first CGRP inhibitor approved by the agency for the preventive treatment of episodic migraine in pediatric patients and migraine in adults, Teva said in an August 6 release.

  The drug is administered by way of subcutaneous injection once per month, either in a doctor's office or at a patient's home. 

  In the U.S., 1 out of 10 children and adolescents suffer from migraines, causing missed school days and trouble with schoolwork, among other disruptions to patients' daily lives.

  “With this FDA approval, Ajovy now offers younger patients a new treatment option, addressing a long-standing gap in care and offering families added support as they navigate the challenges of this condition," Chris Fox, Teva's U.S. Commercial and Innovative Franchise Lead and Head of Global Marketing Business, said in a statement.

UPDATED: Monday, August 4 at 10:00 a.m. ET

• Novartis' Leqvio has picked up an FDA label expansion to be used as a monotherapy in adults with hypercholesterolemia.

  Previously, the drug's label stated that it should be used with statin therapy in patients with primary hyperlipidemia. The new label expansion, which was "proactively requested" by the FDA, enables first-line use of the therapy, Novartis said in a July 31 press release.

  After an initial dose and another one after three months, Leqvio can be given twice yearly. The drug is approved to reduce patients' low-density lipoprotein cholesterol (LDL-C).

  "With this new indication enabling Leqvio's use as monotherapy along with diet and exercise, we now have the potential to help even more patients achieve their LDL-C lowering goals earlier in their treatment journey," Novartis U.S. president Victor Bultó said in a statement.

• Also scoring a recent FDA label expansion was Novo Nordisk's Alhemo. 

  The drug was previously approved as a once-daily prophylaxis to prevent or reduce the frequency of bleeding episodes in patients ages 12 and older who have hemophilia A or B with inhibitors. Now, the FDA approved the subcutaneous therapy for those without inhibitors.

  As it stands, many treatment options for hemophilia A or B without inhibitors are administered via intravenous infusions. The approval marks the arrival of a new subcutaneous drug for these patients, Novo Nordisk said in a July 31 press release.

  "By building on the initial indication for Alhemo for those with hemophilia with inhibitors—an especially significant development in hemophilia B where challenges still exist despite advanced treatment options—Novo Nordisk continues its 35+ year legacy in rare bleeding disorders to continue to help address the unmet needs of this community," Novo's SVP of clinical development, medical & regulatory affairs, Anna Windle, Ph.D., said in a statement.

UPDATED: Wednesday, July 30 at 4:00 p.m. ET

• AstraZeneca’s bid to move its Imfinzi into early-stage stomach cancers has moved forward with the FDA, earning priority review designation.

  The company is hoping to win an approval for resecatable, stage 2 to 3a gastric and gastroesophageal junction cancers. If granted, Imfinzi would be the first immunotherapy-based regimen that can treat patients before, during, and after surgeries in the so-called perioperative setting. 

  In AZ’s phase 3 Matterhorn study, patients treated with an Imfinzi-based perioperative regimen proved a 29% reduction in the risk of disease progression, recurrence or death compared with chemotherapy alone.

  Gastric cancer is the fifth most common cancer worldwide and the fifth leading cause of cancer-related deaths, with nearly one million new patients diagnosed in 2022, according to AZ. Disease recurrence is common for patients despite surgery and treatment. 

  With its priority review, the FDA expects to decide on the approval during the fourth quarter of 2025. 

• AbbVie is hoping to push another combination regimen of its Venclexta over the FDA finish line.

  The company submitted its FDA application for a fixed-duration, all-oral combination of Venclexta and AstraZeneca’s Calquence (acalabrutinib) in previously untreated patients with chronic lymphocytic leukemia (CLL). The combo improved progression-free survival rates for patients enrolled in the phase 3 Amplify trial compared to standard chemoimmunotherapy.

  If approved, the Venclexta-Calquence combo would offer patients the opportunity for time off treatment, due to the fixed-treatment duration approach. Venclexa, which AbbVie markets with partner Genentech, is approved to treat blood cancers in more than 80 countries. 

• PTC Therapeutics won a broad FDA label for its Sephience, making the drug eligible to cover all disease subtypes of phenylketonuria (PKU) in both adult and pediatric patients.

  With the new approval, Sephience can treat hyperphenylalaninemia and sepiapterin-responsive PKU, allowing more people with the genetic disease to benefit from the drug. PKU is caused by a gene defect and can lead to a build-up of phenylalanine in the blood and brain, which can cause severe and irreversible disabilities such as delayed development, intellectual disability and memory loss. PTC’s Sephience works by reducing blood phenylalanine levels, as proven in the company’s phase 3 Aphenity trial. 

• Ascendis Pharma’s Skytrofa can now replace endogenous growth hormone in U.S. adults with rare disease growth hormone deficiency (GHD) thanks to a new FDA approval.

  Skytrofa is a prodrug of human growth hormone somatropin and was previously approved in 2021 as a treatment for pediatric GHD. According to Ascendis, Skytrofa is already the treatment of choice for pediatric GHD and the expansion into the adult population will support the company’s goal of becoming a leading endocrinology rare disease company.

  With once-weekly administration, patients can benefit from sustained release of active somatropin as opposed to the standard daily injections. 

  Those with GHD are at risk of medical complications, metabolic syndrome, cardiovascular concerns and impaired quality of life.  

UPDATED: Thursday, July 24 at 3:45 p.m. ET

• The roller coaster ride for GSK’s Blenrep continued on Thursday with an approval from the European Union as a combo agent to treat multiple myeloma patients who have received at least one prior line of therapy.

  The nod came one day after the FDA delayed its decision on Blenrep in the same indication by three months following a thumbs down from the agency's Oncologic Drugs Advisory Committee (ODAC).

  The EU endorsement covers Blenrep in combination with Takeda’s Velcade and the chemotherapy dexamethasone, or with Bristol Myers Squibb’s Pomalyst and dexamethasone, to treat patients with relaxed or refractory multiple myeloma. 

  Paving the way for the EU approval were two phase 3 trials in which the Blenrep combos topped triplet therapies that included Johnson & Johnson’s Darzalex in measures of efficacy including overall survival.

  GSK is angling for a Blenrep comeback. The drug was previously endorsed by the FDA in 2020 as a monotherapy in the fifth-line multiple myeloma setting before the company pulled the treatment from global markets two years later after a confirmatory trial fell short.

• The European Commission also has approved a new indication for the subcutaneous formulation of Johnson & Johnson’s Darzalex, which is now cleared as a monotherapy for adults with smoldering multiple myeloma (SMM) who are at risk of developing multiple myeloma.

  It is the first time a drug has been approved to treat patients with high-risk SMM, J&J said. Darzalex has been on the market as a treatment for multiple myeloma for a decade.
  
  The EU nod is based on results from a phase 3 study, which showed that fixed-duration treatment with Darzalex reduced the risk of progression to active multiple myeloma or death by 51% compared to active monitoring.

• In yet another European Commission approval, the bloc has signed off on Ipsen’s Cabometyx for adult patients with unresectable or metastatic, well differentiated pancreatic and extra-pancreatic neuroendocrine tumors (pNETs and epNETs) who have progressed following at least one prior systemic therapy other than somatostatin analogues.

  The approval is in a tough-to-treat indication, as most forms of NETs develop slowly in different parts of the body and require multiple lines of therapy as the disease progresses. Additionally, treatment options vary and can be limited depending on the tumor site. 

  Cabometyx paved the way for its most recent nod in a phase 3 trial in which pNET patients on Ipsen's drug, at a median follow up of 13.8 months, charted a median progression-free survival (PFS) score of 13.8 months. That compared to just 4.4 months for trial participants on placebo. 

  In the epNET cohort, the PFS figures at a median follow up of 10.2 months were 8.4 for Cabometyx versus 3.9 months for placebo. 

UPDATED: Wednesday, July 23 at 9:10 a.m. ET

• Roche’s Itovebi regimen can now treat certain breast cancer patients in Europe after winning a nod from the European Commission.

  The regulator specifically cleared the use of Itovebi in combination with Pfizer’s Ibrance and fulvestrant for patients with PIK3CA-mutated, estrogen receptor (ER)-positive, HER2-negative locally advanced or metastatic breast cancer after recurrence by 12 months of completing adjuvant endocrine treatment.

  Roche’s approval is based on data from its phase 3 INAVO120 trial, which showed that the regimen can reduce the risk of disease worsening or death by 57% and can cut the risk of death by 33% compared with Ibrance and fulvestrant alone.

  The Itovebi regimen’s use in this indication has also been cleared in the U.S., China, Canada and other global markets. Up to 40% of ER-positive breast cancers have a PIK3CA mutation, according to Roche. The company is also studying the drug in other combinations across other PIK3CA-mutated breast cancer populations. 

• Canadian regulators granted marketing authorization to Vertex’s new cystic fibrosis triplet therapy Alyftrek.

  Health Canada checked off on Alyftrek’s use in cystic fibrosis patients who are six years old and older and who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

  The approval allows the drug to reach some 3,800 patients in Canada, including 60 who were not previously eligible for a CFTR modulator, according to Vertex.

  Alyftrek, made up of deutivacaftor, tezacaftor and vanzacaftor, is the first once-daily CFTR modulator. The triplet drug met primary and secondary endpoints in phase 3 studies across multiple age groups. 

  Besides Canada, the drug has picked up approvals in the U.S., the U.K., and recently the European Union.

UPDATED: Monday, July 21 at 1:00 p.m. ET

• The U.S. FDA has accepted Bristol Myers Squibb's label expansion filing for TYK2 inhibitor Sotyktu as a treatment for adults with active psoriatic arthritis.

  The agency plans to make a decision on the filing by March 6, 2026, BMS said in a July 21 press release.

  Besides the FDA, regulators in China, Japan and Europe are reviewing the drug in the proposed use, BMS said in its release.

  Sotyktu is already approved by the FDA to treat adults with moderate-to-severe plaque psoriasis who are candidates for systemic therapy or phototherapy. 

  In psoriatic arthritis, results from the POETYK PsA-1 and POETYK PsA-2 trials are supporting the company's filings. The studies found that more patients treated with Sotyktu achieved a 20% improvement in signs and symptoms of disease after 16 weeks compared with those given placebo.

• In England, the National Institute for Health and Care Excellence (NICE) has signed off on the tree pollen tablet Itulazax, the allergy-focused drugmaker ALK reported.

  In final draft guidance published July 21, NICE endorsed the clinical benefits and cost-effectiveness of Itulazax as a treatment for uncontrolled tree pollen allergy, Denmark's ALK said in a press release.

  Approximately 16 million people are affected by hay fever in the U.K., and many manage their symptoms with over-the-counter medicines, according to ALK. But about 4 million have moderate to severe tree pollen hay fever and may be looking for an alternative treatment option.

• That wasn't the only good news for ALK in recent days. The company's epinephrine nasal spray, EURneffy, secured regulatory approval in the U.K. on Friday.

  The Medicines and Healthcare products Regulatory Agency endorsed the spray for the emergency treatment of serious allergic reactions, according to a July 18 press release.

  ALK licensed global rights to the drug, excluding in the U.S., through a November 2024 deal with ARS Pharmaceuticals worth up to $465 million. ARS markets the spray as neffy in the U.S.

UPDATED: Thursday, July 17 at 10:52 a.m. ET

• GSK’s shingles vaccine Shingrix will make its debut in a new prefilled syringe after snagging FDA approval, offering a more convenient administration process.

  Shingrix was previously available in separate vials consisting of a powder antigen and a liquid adjuvant, which healthcare professionals would need to combine before administering a dose. Now, the vaccine can be more easily given to its approved population of adults 50 and older or those 18 and older who are at increased risk for shingles. An estimated 1 million people develop shingles annually in the U.S., according to GSK.

  The FDA signed off on the new prefilled syringe based on data demonstrating technical comparability between the Shingrix vaccine presentations. The prefilled syringe is currently under review with regulators in Europe. 

• The U.K.’s National Institute for Health and Care Excellence (NICE) will allow more patients to benefit from Novartis’ Kisqali after expanding its recommendation to a wider group of those with hormone receptor-positive, HER2-negative early breast cancer.

  Previously, the treatment option was only available for eligible patients whose cancer had spread to nearby lymph nodes. In its final draft guidance, NICE has removed the lymph node-related restriction, allowing thousands more at a high risk of recurrence to take the med as an adjuvant treatment in combination with an aromatase inhibitor.

  In clinical trials, Novartis showed that adding Kisqali to an aromatase inhibitor can extend the time before cancer returns compared with an aromatase inhibitor alone. Around 5,700 people in the U.K. could benefit from the decision, according to NICE. 

• Johnson & Johnson’s innovative bladder cancer therapy TAR-200 will get a speedy FDA review after the agency granted the company’s drug application priority review status.

  TAR-200 is a unique intravesical system designed to locally deliver the chemotherapy gemcitabine into the bladder for certain patients with Bacillus Calmette-Guérin-unresponsive high-risk non-muscle invasive bladder cancer (HR-NMIBC).

  The gemcitabine releasing system is the first and only that can provide sustained local delivery into the bladder and is administered via a catheter in an outpatient setting without anesthesia. TAR-200 remains in the bladder for three weeks per treatment cycle and previously received a breakthrough therapy designation from the FDA. 

• AstraZeneca and Daiichi Sankyo’s Enhertu has been awarded another breakthrough therapy designation from the FDA, this time for its proposed combination with Roche’s Perjeta (pertuzumab) as a first-line treatment for patients with unresectable or metastatic HER2 positive breast cancer.

  The antibody-drug conjugate (ADC) has received nine breakthrough therapy designations to date, including five for metastatic breast cancer. The FDA awarded the combo the latest designation based on data from the companies’ DESTINY-Breast09 study, a phase 3 trial presented at this year’s American Society of Clinical Oncology annual meeting.

UPDATED: Tuesday, July 15 at 3:10 p.m. ET

• As Vertex Pharmaceuticals moves ahead with the launch of its next cystic fibrosis offering Alyftrek, the company has inked a reimbursement deal in England.

  The company and NHS England reached a deal for all eligible patients to access the once-daily triplet therapy, Vertex said in a July 14 press release. Along with this deal, England's National Institute for Health and Care Excellence issued a positive final draft recommendation on the medicine.

  Alyftrek, Vertex's fifth CF offering, is a combination of deutivacaftor, tezacaftor and vanzacaftor. The drug is licensed for CF patients ages 6 and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

  "We’re pleased to have reached this agreement with NHS England that recognises the value that this new medicine brings to CF patients, their families and society," Vertex International SVP Ludovic Fenaux said in a statement. 

  Besides the rollout in England, the drug scored an approval from the U.S. FDA late last year and the launch is underway in several European countries.

UPDATED: Monday, July 14 at 9:35 a.m. ET

• A GSK label expansion bid for respiratory syncytial virus vaccine Arexvy is under review at the FDA.

  The company is seeking approval for Arexvy to be used in adults ages 18 to 49 who are at an increased risk for severe infection, GSK said in a July 14 press release. The FDA has now accepted the filing.

  The vaccine is currently approved for all adults ages 60 and older, and for adults ages 50 to 59 who are at a higher risk of severe infection. The CDC, meanwhile, recommends RSV vaccines for all adults ages 75 and older, and for people ages 50 to 74 who are at a higher risk of severe infection.

  Risk factors for severe RSV infection include chronic cardiovascular disease, asthma, kidney disease and diabetes, according to the CDC.

  As for GSK's new expansion bid, the company says 21 million people in the U.S. are under the age of 50 and have at least one risk factor.

  GSK expects the FDA's decision to come in the first half of 2026, according to the company's press release.

• Atara Biotherapeutics has resubmitted its biologics license application for Ebvallo as a treatment for patients 2 years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy.

  The development comes six months after the FDA rejected the off-the-shelf cell therapy over manufacturing findings identified during a plant inspection.

  Atara projects its current cash holdings can fund the company into the first quarter of next year, according to a July 14 press release. In addition, the drugmaker would be in line for a $40 million milestone payment from partner Pierre Fabre Laboratories if the drug wins FDA approval.

UPDATED: Friday, July 11 at 8:50 a.m. ET

• After a review by the safety committee of the European Medicines Agency, the regulator is removing a temporary restriction on Valneva's chikungunya vaccine.

  Back in May, the regulator recommended against the continued use of the vaccine, Ixchiq, in people 65 and older as it weighed reports of serious adverse events in elderly people.

  Now, EMA says the shot should only be given "when there is a significant risk of chikungunya infection and after a careful consideration of the benefits and risks." 

  Serious side effects have been seen "mainly in people 65 years and older and in those with several underlying medical conditions," the agency said in a July 11 announcement.

  While there is a side effect risk for some, the vaccine remains "effective at triggering the production of antibodies against the chikungunya virus which may be of particular benefit for older people who are at increased risk of severe chikungunya infection," the EMA said.

  In the U.S., authorities recommended a pause of the vaccine rollout in people 60 and older in May. At the time, U.S. officials said there had been 17 reports of serious adverse events, including 2 deaths, in Ixchiq recipients ages 62 to 89 around the world.

• Moderna's COVID-19 vaccine, Spikevax, has picked up a full U.S. approval in children aged 6 months to 11 years who are at an increased risk from the disease.

  The vaccine was previously available for these children under an Emergency Use Authorization, Moderna noted in a July 10 press release.

  With the nod, the vaccine is available to people 6 months to 64 years of age who are at a high risk for severe disease, and for everyone 65 and older.

UPDATED: Thursday, July 10 at 9:55 a.m. ET

• Three months after winning a thumbs up from the U.K.’s drug regulator in Crohn's disease, Eli Lilly’s Omvoh (mirikizumab) has scored the blessing of the nation’s drug cost watchdog.

  The National Institute for Health and Care Excellence (NICE) has signed off on Omvoh in moderately to severely active Crohn’s in adults under three potential conditions: Patients will be able to access the treatment if their disease didn’t respond well enough or stopped responding to a previous biologic therapy; if a previous biologic drug wasn’t well tolerated; or if tumor necrosis factor (TNF)-alpha inhibitors are not suitable for that patient, according to final draft guidance published on July 10.

  While “indirect comparisons of mirikizumab with other biological treatments are uncertain,” there is sufficient evidence—bolstered by clinical expert opinion—that “mirikizumab is likely to work as well as risankizumab,” NICE said of the recommendation, referring to the generic name for AbbVie’s Skyrizi.

  Now that the U.K.’s drug regulator, the Medicines and Healthcare Products Regulatory Agency (MHRA), and NICE have aligned on Omvoh, the drug will be made available as a treatment option for patients in England and Wales within 30 and 60 days, respectively, Pharmaphorum noted on Thursday.

  Aside from AbbVie’s Skyrizi, Omvoh will also be sharing the U.K. Crohn’s market with Takeda’s Entyvio (vedolizumab) and Johnson & Johnson’s Stelara (ustekinumab), the latter of which also has biosimilar versions available in the country. 

• NICE has also endorsed the BTK inhibitor Brukinsa (zanubrutinib) from BeOne Medicines—formerly known as BeiGene—in relapsed or refractory mantle cell lymphoma (MCL). The vote of confidence specifically covers the use of BeOne’s drug in adults who’ve had one prior line of treatment.

  Patients with relapsed or refractory MCL seeking additional treatment after first-line therapy typically turn to AbbVie’s Imbruvica (ibrutinib). Brukinsa “works in a similar way” and “would be offered to a similar population” under the recommendation, NICE pointed out in its guidance. NICE did caveat that data from an indirect comparison between Brukinsa and Imbruvica are “uncertain because of differences between the people in the included trials.”

  NICE has previously recommended BeOne’s drug in certain patients with Waldenstrom’s macroglobulinemia, as well as those with chronic lymphocytic leukemia (CLL) and marginal zone lymphoma (MZL).

• BeOne has also secured a green light for its PD-1 inhibitor Tevimbra (tislelizumab) in the European Union.

  The approval, finalized by the European Commission (EC), covers the use of Tevimbra alongside the chemotherapy drugs gemcitabine and cisplatin for initial treatment of adults with metastatic or recurrent nasopharyngeal carcinoma (NPC).

  The EC based its decision on BeOne’s late-stage RATIONALE-309 study, in which the Tevimbra-chemotherapy cocktail reduced the risk of disease progression or death by 48% at the time of the trial’s first interim analysis. Median progression-free survival (PFS) for patients on the Tevimbra regimen clocked in at 9.2 months, versus 7.4 months for patients on placebo plus gemcitabine and cisplatin, BeOne said in a July 10 press release.

  Those results were reinforced by data from an additional 12 months of follow-up in the trial, in which patients on the Tevimbra cocktail achieved a median overall survival (OS) of 45.3 months, compared to 31.8 months for patients in the study’s control cohort.

  Tevimbra also boasts EU approvals in gastric or gastroesophageal junction adenocarcinoma, unresectable esophageal squamous cell carcinoma, extensive-stage small cell lung cancer and three separate non-small cell lung cancer indications. 

• Meanwhile, over in China, Ascentage Pharma has landed an approval for its B-cell lymphoma 2 (Bcl-2) selective inhibitor lisaftoclax in adults with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who’ve previously tried at least one prior therapy, including BTK inhibitors.

  With the green light, lisaftoclax becomes the second Bcl-2 inhibitor approved globally behind AbbVie’s Venclexta (venetoclax) and the first approved in China for patients with CLL/SLL, Ascentage said on July 10.

  China’s National Medical Products Administration (NMPA) endorsed the drug after reviewing data from a pivotal phase 2 study looking at overall response rate (ORR) as the primary endpoint. Lisaftoclax displayed “compelling efficacy” and charted an ORR that satisfied the study’s main objective in patients previously treated with BTK inhibitors or immunochemotherapy, Ascentage noted in its release. 

UPDATED: Tuesday, July 8 at 9:20 a.m. ET

• A little over half a year after acquiring central nervous system specialist Intra-Cellular Therapies, Johnson & Johnson is advancing plans for its newly inherited schizophrenia medicine Caplyta.

  J&J has submitted a supplemental new drug application to the FDA leveraging positive data on Caplyta’s (lumateperone’s) ability to prevent relapse in schizophrenia patients. The company based its filing on a phase 3 withdrawal study, which showed that patients on Caplyta took significantly longer to experience symptom relapse than those on placebo during a 26-week treatment phase.

  Patients on Caplyta also experienced a 63% reduction in the risk of relapse versus placebo, J&J said in a July 8 press release. Further, Caplyta helped significantly delay time to all-cause discontinuation, including relapse, compared to the dummy drug.

  Relapses in schizophrenia refer to the recurrence of symptoms like psychosis, hallucinations and other disruptive behaviors. On average, adults with schizophrenia experience nine relapses in less than six years, J&J noted in its release.

  "Relapse prevention is a critical goal for the long-term care and management of this debilitating disorder," Bill Martin, Ph.D., the global therapeutic leader for neuroscience at Johnson & Johnson Innovative Medicine, said in a statement.

  Caplyta is currently approved in the U.S. for the treatment of schizophrenia in adults, and as a treatment for depressive episodes linked to bipolar 1 or 2 disorder.

• Across the pond, the U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has granted marketing authorization to ImmunityBio’s lymphocyte-stimulating agent, Anktiva, in combination with the Bacillus Calmette-Guérin (BCG) vaccine, to treat BCG-unresponsive non-muscle invasive bladder cancer with carcinoma in situ (NMIBC CIS).

  The green light marks the second in the world for Anktiva after the FDA approved the drug in a similar bladder cancer indication last April.

  Anktiva functions as an IL-15 agonist that activates and proliferates natural killer (NK) cells, plus CD4+ and CD8+ T cells. The drug is designed to restore immune competence by reversing lymphopenia, wherein cancer and conventional cancer therapies like chemotherapy, radiation and checkpoint inhibitors curb the number of a patient’s immune cells and blunt their function, ImmunityBio explained in its approval announcement.

  ImmunityBio estimates that there are around 16,400 to 18,000 patients diagnosed with NMIBC in the U.K. each year. With approvals secured in the U.S. and the U.K., ImmunityBio is next jockeying for a greenlight from the European Medicines Agency (EMA). An EMA thumbs up would allow ImmunityBio to market its therapy across all 27 European Union member states, plus Iceland, Norway and Liechtenstein.

UPDATED: Wednesday, July 2 at 10:30 a.m. ET

• A little over a year after Merck & Co. scored U.S. approval for its pulmonary arterial hypertension (PAH) newcomer Winrevair (sotatercept), the New Jersey drugmaker has locked in an expedited review timeline to potentially update the medicine’s label.

  The FDA has granted priority review to Merck’s application and set a target date of Oct. 25 to decide whether to revise Winrevair’s label with impressive morbidity and mortality data from Merck’s phase 3 ZENITH trial.

  The study, which was the first late-stage PAH outcomes trial to leverage a primary endpoint consisting of major morbidity and mortality events, recently stopped early on the advice of a third-party data monitoring committee thanks to “overwhelming efficacy,” Merck noted in a July 2 press release.

  In the trial, Winrevair led to a 76% reduction in the risk of a composite of all-cause death, lung transplantation and hospitalization in PAH patients for at least 24 hours compared to placebo.

  Winrevair was approved by the FDA last March as an add-on therapy to standard of care in a subset of PAH patients defined by the World Health Organization (WHO). Merck picked up the drug in its $11.5 billion acquisition of Acceleron. Analysts have predicted peak sales of Winrevair could land anywhere between $2 billion and $4 billion. 

• Over in Europe, Vertex Pharmaceuticals is continuing to grow its cystic fibrosis (CF) empire with a European Commission (EC) approval of its once-a-day triple therapy Alyftrek—comprising deutivacaftor, tezacaftor and vanzacaftor—in CF patients ages 6 years and older who have at least one non-class 1 mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

  The EC gave its blessing after reviewing data from two head-to-head studies that found Alyftrek was non-inferior to Vertex’s entrenched CF therapy Kaftrio in combination with ivacaftor on a standard measure of lung function and superior when it came to reducing sweat chloride and spurring greater improvements in CFTR function.

  The EC green light significantly expands the reach of Vertex’s CF therapeutics overseas, with some 31,000 CF patients in the European Union (EU) now eligible to receive the new therapy, the company pointed out.

  The drug is expected to launch in Ireland, Denmark and Germany first as Vertex continues to work out reimbursement deals with other EU member states.

  The thumbs up from the EC comes after the FDA cleared Alyftrek in a similar patient population in late December. 

• Elsewhere, London-based Hikma has won the FDA’s blessing for a new, ready-to-infuse formulation of the antibiotic vancomycin for the treatment of septicemia, infective endocarditis, skin and skin structure infections, bone infections and lower respiratory tract infections in adults and children as young as 1 month or older.

  Hikma will market the novel formulation of the well-established anti-infective under the commercial moniker Tyzavan, which the company says stands for “time-saving vancomycin.”

  The green light earns Tyzavan the distinction of being the only vancomycin product that can be kept at room temperature and doesn’t require compounding, thawing, activation or dilution before use, which ought to hasten treatment by reducing preparation steps, Hikma said in a July 2 press release.

  U.S. sales of injectable vancomycin approached $200 million in 2024, Hikma noted, citing data from the health information and research firm IQVIA.