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: 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.