Welcome to Fierce Pharma's regulatory tracker for the first half of 2026. 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: Tuesday, March 24 at 2:55 p.m. ET
Daiichi Sankyo and AstraZeneca’s Enhertu chalked up a new approval in Japan, winning clearance to treat patients with HER2-positive advanced or recurrent solid tumors that are refractory or intolerant to standard treatments.
Japan’s Ministry of Health, Labour and Welfare (MHLW) reviewed four phase 2 trials to make its decision, including an investigator-initiated Japanese trial called Herald and three other studies in which Enhertu achieved clinically meaningful responses across a “broad range” of tumors, Daiichi said in a March 23 press release. In the Herald study, the drug charted a confirmed objective response rate of 56.5% across patients with an array of tumors including cervical, pancreatic, gastric and colorectal, as well as non-small cell lung cancer and multiple other cancer types.
With the nod, the drug is the first tumor agnostic HER2-directed medicine to hit the market in Japan for previously treated HER2 metastatic solid tumors.
Enhertu continued its regulatory winning streak with a second Japanese approval Monday, this time in the form of a label expansion from the country's Pharmaceuticals and Medical Devices Agency (PDMA).
The new label covers the second-line treatment of patients with HER2-positive unresectable advanced or recurrent gastric cancer, bumping up the antibody drug-conjugate (ADC) into an earlier treatment line than its previous third-line nod. Gastric cancer is the third most common cancer in Japan.
Japanese regulators signed off on the expansion based on data from the Destiny-Gastric04 trial, in which Enhertu demonstrated a 30% reduction in the risk of death compared to Eli Lilly’s VEGFR2 inhibitor Cyramza (ramucirumab) plus the chemotherapy paclitaxel. Median overall survival for Enhertu-treated patients was 14.7 months, versus 11.4 months on the Cyramza regimen. The trial made Enhertu the first HER2-directed medicine to show a survival benefit in the second-line metastatic setting in a randomized clinical trial, Daiichi said this week.
Japan’s Ministry of Health, Labour and Welfare also blessed Chugai Pharmaceutical and Roche’s combination of Lunsumio and Polivy with a world-first approval for relapsed or refractory large B-cell lymphoma.
In its clinical trials, the Lunsumio-Polivy combo achieved an objective response rate of 69.7% compared to 44.1% in those who took a regimen of Roche’s Rituxan and the chemotherapies gemcitabine and oxaliplatin (R-GemOx). At the time of the phase 3 Sumo study’s primary analysis, Lunsumio and Polivy flexed a 59% reduction in the risk of disease progression or death over the R-GemOx group, Chugai said.
Chugai, a Roche-owned company, markets both drugs in Japan and is “committed to delivering this treatment to patients as quickly as possible,” CEO Osamu Okuda commented in a company release.
Bayer and Regeneron’s superstar eye drug Eylea is widening its global footprint with another Japanese nod for its 8mg high-dose version.
The approval is high-dose Eylea’s third in Japan and covers patients with visual impairment due to macular edema following retinal vein occlusion (RVO) including branch, central and hemiretinal vein occlusion. RVO is the second most common cause of vision loss due to retinal vascular disease and can cause abrupt vision loss if not treated and diagnosed early.
Bayer and Regeneron’s Quasar trial stacked up the 8mg Eylea dose against its original 2mg version, finding that patients treated with the high-dose iteration maintained their visual acuity and required an average of three fewer injections than those on the older 2mg version, Bayer said. Regeneron and Bayer jointly developed the Eylea franchise, but Bayer is responsible for marketing the meds outside of the U.S.
UPDATED: Friday, March 20 at 4:10 p.m. ET
Bristol Myers Squibb’s checkpoint inhibitor Opdivo (nivolumab) has bumped its way up the treatment chain in classical Hodgkin lymphoma and solidified two accelerated nods in related indications in the U.S.
On Friday, the FDA approved Opdivo alongside a triplet chemotherapy regimen of doxorubicin, vinblastine, and dacarbazine (AVD) in adults and kids ages 12 and older with previously untreated, stage 3 or 4 classical Hodgkin lymphoma (cHL).
At the same time, the FDA granted traditional approvals to Opdivo in adults with relapsed or refractory cHL after treatment with autologous hematopoietic stem cell transplantation (HSCT) and brentuximab vedotin, and in patients who’ve tried three or more lines of systemic treatment that includes autologous HSCT.
BMS’ drug won accelerated green lights in those indications in 2016 and 2017, respectively, according to the FDA announcement.
The FDA based its decision on data from a National Cancer Institute-sponsored trial that enrolled 994 stage 3 and 4 cHL patients ages 12 and older. It showed superiority for the Opdivo-AVD arm over the control cohort on the study’s key metric of progression-free survival. Patients in the control cohort received Adcetris (brentuximab vedotin) plus AVD. Pfizer gained access to Adcetris, a CD30-targeted antibody-drug conjugate, through its acquisition of Seagen.
Meanwhile, after a median follow-up of 36.7 months, the trial logged 1.8% deaths in the Opdivo-AVD arm versus a 3.4% death rate in the Adcetris-AVD control, according to the FDA.
Toward the end of 2024, almost exactly 10 years after its initial FDA approval, the FDA approved Opdivo Qvantig, a subcutaneous version of the intravenous PD-1 inhibitor in nearly all of its existing solid tumor indications. The subcutaneous formulation does not currently have the cHL indications.
UPDATED: Thursday, March 19 at 9:15 a.m. ET
After Sarepta's exon-skipping agents for Duchenne muscular dystrophy (DMD) fell short in a 9-year confirmatory trial back in November, the company stressed that it still planned to seek full approvals for the drugs.
Now, Sarepta has provided an update on its interactions with the FDA regarding this effort. On Thursday, the company said it received feedback from the FDA "confirming that we can submit our data" from the 9-year confirmatory study, called Essence, as well as real-world evidence on the drugs, as part of its supplemental new drug approvals.
The medicines, Vyondys 53 and Amondys 45, won their respective FDA accelerated approvals in 2019 and 2021. The company aims to convert those approvals into full nods with the new filings.
While the drugs didn't meet statistical significance on a key measurement of DMD patient mobility compared with placebo in Essence, Sarepta has stressed that "numerical trends favored" the treatments.
"We appreciate the FDA’s openness to our submitting the supplemental applications for Amondys 45 and Vyondys 53 and willingness to consider all available data—from the Essence confirmatory study and the real-world evidence generated over the past several years," Sarepta's president of R&D and technical operations, Louise Rodino‑Klapac, Ph.D., said in a statement.
Sarepta plants to submit its sNDAs for the drugs by the end of April.
UPDATED: Tuesday, March 17 at 2:45 p.m. ET
With a green light from Britain's drug regulator, Sun Pharma has won its second global approval for severe alopecia areata medicine Leqselvi.
The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has signed off on Sun’s JAK inhibitor, also known as deuruxolitinib, to treat the hair loss-causing autoimmune disease in adults.
With the nod, Leqselvi will now be assessed by the country’s National Institute for Health and Care Excellence (NICE) to determine its availability through the U.K.’s National Health Service (NHS).
The MHRA based its approval decision on two Leqselvi studies in which the drug topped placebo on a measure of scalp hair coverage at 24 weeks. Overall, around 30% of the participants on Leqselvi saw scalp coverage grow to 80%, while some 23% of Leqselvi patients had 90% or more scalp hair coverage at that 24-week mark, according to the MHRA release.
Sun snared approval for Leqselvi from the FDA in the summer of 2024, although the company did not launch its medicine in the U.S. until roughly a year later, following a now-resolved patent dispute with Incyte.
UPDATED: Monday, March 16 at 9:20 a.m. ET
After the successful phase 3 Matterhorn study, AstraZeneca's Imfinzi has scored a new approval in the European Union.
Regulators in the bloc approved the drug as part of a perioperative regimen with standard-of-care FLOT chemotherapy (fluorouracil, leucovorin, oxaliplatin, and docetaxel). The regimen features two cycles of Imfinzi in combination with chemotherapy before and after surgery, followed by Imfinzi monotherapy.
The new perioperative treatment approach is for adults with resectable, early-stage and locally advance gastric and gastroesophageal junction cancers.
In the drug's late-stage study, the addition of Imfinzi to the chemotherapy regimen before and after surgery lowered the risk of disease recurrence, progression or death by 29% compared with FLOT alone in these patients.
In the U.S., regulators have approved Novartis' Cosentyx as a treatment for moderate to severe hidradenitis suppurativa in patients ages 12 and older.
The approval makes Cosentyx the first IL-17A inhibitor approved for this population, as well as the first new mechanism of action in this patient group in nearly a decade, Novartis said in a March 13 press release.
The approval is particularly important because hidradenitis suppurativa often emerges around puberty and can cause irreversible scarring and disabilities. The disease is a chronic inflammatory skin condition that causes patients to develop boil-like lesions.
"Hidradenitis suppurativa affects far more than skin; it impacts confidence, emotional well-being and relationships during a formative period for many pediatric patients," Brindley Brooks, founder and CEO of the patient group HS Connect, said in a statement. "For families watching their children struggle, this FDA approval brings hope for earlier intervention."
UPDATED: Monday, March 9 at 2:10 p.m. ET
Daiichi Sankyo and AstraZeneca’s reigning antibody-drug conjugate (ADC) Enhertu is homing in on another potential approval, with the FDA this week granting the drug priority review in a subset of HER2-positive breast cancer patients. Daiichi and AZ are specifically pursuing a green light in patients with residual invasive disease after neoadjuvant treatment with a HER2-targeted drug.
The FDA priority review tag follows receipt of a breakthrough therapy designation in the indication in December and tees up Enhertu for a regulatory decision by July 7, Daiichi and AZ said in a March 9 press release.
The partners’ application leans on results from the late-stage Destiny-Breast05 study, in which Enhertu curbed the risk of invasive disease recurrence or death by 53% versus a control of trastuzumab emtansine (Roche’s Kadcyla) in the specific breast cancer patient population.
Enhertu also reduced the risk of distant disease recurrence and brain metastases by 51% and 36%, respectively, compared to the trial’s Kadcyla control.
“For patients with residual invasive disease after neoadjuvant therapy, identifying additional treatments following surgery is critical to help further reduce the risk of recurrence and help prevent progression to metastatic disease,” Ken Takeshita, M.D., Daiichi’s global head of R&D, said in a statement.
The U.S. filing comes as submissions based on Destiny-Breast05 are already under review in the European Union and Japan, AZ and Daiichi said. And ahead of the new July decision date set by the FDA, the partners are also awaiting the U.S. regulator’s verdict on their Enhertu application for neoadjuvant treatment in HER2-positive stage 2 or 3 breast cancer.
Since its initial 2019 nod in HER2-positive unresectable or metastatic breast cancer patients who’ve received two or more anti-HER2 regimens, Enhertu has gone on to pick a suite of breast cancer indications, and the drug late last year scored a coveted first-line nod in combination with Roche’s Perjeta.
In another bit of FDA news, ImmunityBio said on March 9 that it has submitted additional information in support of its application in papillary disease following “multiple meetings” with the regulator.
The Culver City, California-based drugmaker is aiming for approval of its IL-15 receptor agonist Anktiva plus Bacillus Calmette-Guérin (BCG) in BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with papillary tumors. ImmunityBio’s filing in the indication was dealt with a refusal-to-file letter last year, as the company and the FDA appeared split on whether data from a single-arm trial, rather than a randomized study, were sufficient.
ImmunityBio says it’s been in ongoing discussions with the FDA since January, over which time the agency requested more data for its Anktiva review. ImmunityBio specified that those requests did not call for any new clinical trials.
While ImmunityBio submitted that info in February, the FDA in March asked for updated efficacy data, too, prompting ImmunityBio’s latest filing.
Celebrity businessman and biotech entrepreneur Patrick Soon-Shiong’s ImmunityBio scored its first U.S. approval for Anktiva—also alongside the BCG vaccine—to treat patients with BCG-unresponsive non-muscle invasive bladder cancer back in April 2024.
More recently, ImmunityBio issued detailed results on Anktiva in papillary-only NMIBC in December.
UPDATED: Thursday, March 5 at 11:05 a.m. ET
Sweden’s Hansa Biopharma now has a date on the calendar to potentially bring its kidney transplant med imlifidase to the U.S., with the FDA this week assigning an approval decision target of Dec. 19.
The FDA is evaluating the therapy as an option for highly sensitized patients awaiting kidney transplantation, a group that makes up about 10% to 15% of people on transplant waiting lists. Those patients also carry high levels of pre-formed donor specific antibodies (DSA), primarily from previous transplants, blood transfusion or pregnancies, the company explained.
DSAs can trigger an immediate immune response against a donor organ, which can cause tissue damage and graft rejection, Hansa said. Highly sensitized patients face long or even indefinite wait times for a transplant as compatible donors are difficult to find, leaving patients dependent on long-term dialysis while awaiting a matched organ. There are currently no FDA-approved treatments available in the U.S. that address the population.
Imlifidase, or Idefirix as it’s marketed under conditional approvals in Europe, the U.K. and other countries, is a desensitization treatment meant for highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. The drug is an antibody-cleaving enzyme that can inhibit immunoglobulin G (IgG) immune response within hours after administration.
In Hansa's U.S. trial, imlifidase helped patients chart statistically significant improvements in kidney function at 12 months. The study will go on for five years, including a long-term follow-up as part of the FDA’s accelerated approval pathway. Hansa had requested a priority review from the FDA upon its drug application submission in December, but the FDA opted for the standard review cycle instead.
Still, the company considers the FDA’s review an “important milestone,” bringing it “one step closer to potentially offering imlifidase as a transformative therapy option for patients who today have very limited access to a life-changing kidney transplant,” CEO Renée Aguiar-Lucander said in a statement.
UPDATED: Monday, March 2 at 11:15 a.m. ET
The FDA has expanded the label of Novo Nordisk’s once-weekly long-acting growth hormone Sogroya as a treatment for three additional growth disorders.
Sogroya can now be used by children age 2.5 years and older with idiopathic short stature (ISS), growth failure associated with Noonan syndrome and those born small for gestational age (SGA) and with no catch-up growth by age 2.
Sogroya was originally approved in the U.S. in 2020 for growth hormone deficiency. The new approvals for the once-weekly drug provide an alternative to daily injections.
“Daily injections have defined the growth disorder treatment paradigm for more than 40 years. Our scientific leadership and focus on advancing care in rare diseases led us to the development of Sogroya,” Nicky Kelepouris, who heads up Novo’s rare endocrine disorders U.S. unit, said in a release.
In May of last year, Novo presented results from a phase 3 trial basket study which demonstrated Sogroya’s effectiveness in each of the three indications. The 52-week trial included 307 patients, with a two-year extension study ongoing to investigate its safety.
The FDA has signed off on a label expansion for enzyme substitution therapy Palynziq to include patients age 12 and older with phenylketonuria (PKU) to reduce blood phenylalanine (Phe) concentrations.
The drug was approved eight years ago for adults with the disorder.
Palynziq, which is the only enzyme substitution therapy for people with PKU, racked up sales of $433 million last year, which were up 22% year over year. The nod is backed by a phase 3 study in those ages 12-17 that showed statistically significant reductions in Phe compared to diet alone.
UPDATED: Wednesday, February 25 at 9:30 a.m. ET
The FDA has converted a 2024 accelerated approval for a Braftovi combination regimen by Pfizer into a traditional approval. The indication covers Braftovi, used in tandem with Eli Lilly's Erbitux and fluorouracil-based chemotherapy, for the treatment of adult patients with metastatic colorectal cancer (mCRC) with a BRAF V600E mutation.
The original go-ahead, supported by objective response data (ORR) from the phase 3 Breakwater trial, used mFOLFOX6 as the only chemo component. The full nod is now backed by progression-free survival and overall survival data, and includes additional data on the use of the chemo regimen FOLFOXIRI.
Compared with chemo—with or without bevacizumab—the cocktail of Braftovi, Erbitux and mFOLFOX6 reduced patients' risk of death by 51% in the Breakwater trial. Further, the ORR was 64% for Braftovi-Erbitux-FOLFOXIRI, compared to 39% for FOLFOXIRI with or without bevacizumab.
“As the only targeted combination regimen shown to deliver a significant improvement in certain outcomes for patients with BRAF V600E‑mutant metastatic colorectal cancer, BRAFTOVI is uniquely positioned to redefine first‑line treatment and establish a new standard of care,” Aamir Malik, Pfizer's chief U.S. commercial officer, said in a Feb. 24 statement.
Johnson & Johnson has filed an application with the FDA, looking to make Imaavy the first treatment for warm autoimmune hemolytic anemia (wAIHA), a rare and serious autoantibody disease that affects approximately 1 in 8,000 in the U.S.
The submission is based on the phase 2/3 Energy trial, although detailed results do not appear to have been published.
According to J&J, the study showed that more patients treated with Imaavy achieved the primary endpoint of a durable hemoglobin response compared with placebo. A durable response was defined as achieving a hemoglobin level at or above 10 g/dL and an increase of at least 2 g/dL for at least 28 days, without the need for rescue therapy.
What's more, a greater number of patients treated with Imaavy experienced rapid and sustained improvement in fatigue as assessed by FACIT-Fatigue, which J&J said is of "significant importance to people living with wAIHA."
Viatris said the FDA has accepted the company's application to expand the use of its Ryzumvi eye drop to treat presbyopia, or age-related farsightedness, which affects approximately 90% of adults in the U.S. over the age of 45, according to the company. The FDA is expected to deliver a verdict by Oct. 17, 2026.
The submission is supported by two phase 3 trials, VEGA-2 and VEGA-3, both of which met their primary and all key secondary endpoints, with no treatment-related serious adverse events, according to Viatris.
Ryzumvi is currently approved in the U.S. for the treatment of pharmacologically-induced mydriasis produced by adrenergic agonists or parasympatholytic agents.
UPDATED: Tuesday, February 24 at 9:50 a.m. ET
Sanofi and Regeneron's megablockbuster immunology drug Dupixent has gained yet another FDA approval, this time in allergic fungal rhinosinusitis (AFRS).
The U.S. regulator signed off on the drug as a treatment for adults and children ages 6 and older with AFRS based on late-stage trial data showing Dupixent reduced nasal signs and symptoms and systemic corticosteroid use or surgery compared to placebo, according to a Feb. 24 press release.
Patients with AFRS have a hypersensitivity to fungi, often requiring sino-nasal surgery, which is a minimally invasive surgical procedure to widen sinus drainage pathways.
The disease often impacts people living in warm, humid climates who frequently come across nasal spores in the environment.
“As the first medicine approved specifically for AFRS, Dupixent has been proven to lessen multiple signs and symptoms of disease, help break the cycle of recurrence, and reduce the risk for subsequent surgeries and corticosteroids by 92%,” Alyssa Johnsen, M.D., Ph.D., global therapeutic area head for immunology development at Sanofi, said in a statement. “We look forward to working with regulators in other countries to bring this innovative option to more patients in need.”
Dupixent is also approved in the sino-nasal disease chronic rhinosinusitis with nasal polyps. Last year, the drug generated 15.7 billion euros ($18.5 billion) worldwide.
UPDATED: Tuesday, February 17 at 3:55 p.m. ET
Harmony Biosciences is rounding out the U.S. patient pool eligible for its sleep disorder pill Wakix after notching a pediatric nod from the FDA that positions the drug as a treatment for cataplexy in people ages 6 and older with narcolepsy.
The new addition to Wakix’s label makes it the only non-scheduled treatment for both adult and pediatric narcolepsy patients in the U.S. with or without cataplexy. That non-scheduled classification represents an "important distinction that supports its clinical utility," Harmony's CEO, Jeffrey Dayno, M.D., commented in a press release. Cataplexy is a common symptom of narcolepsy that involves a sudden weakening of muscles, often when triggered by a strong emotion.
Wakix was first approved in 2019 for the treatment of excessive daytime sleepiness (EDS) in adults with narcolepsy, with the adult cataplexy indication tacked on in 2020. In 2024, the drug reached the pediatric population for the first time with an FDA approval for younger patients with narcolepsy and EDS. Now, the company is looking to secure pediatric exclusivity for Wakix, which would grant an extra six months of regulatory exclusivity for the “growing franchise,” CEO Dayno noted.
GSK’s new twice-yearly asthma treatment Exdensur is landing in Europe after the European Commission signed off on two indications, making the drug the first and only ultra-long-acting biologic approved to treat respiratory diseases in the bloc.
Exdensur’s label in Europe specifies its use as an add-on maintenance treatment for severe asthma with type 3 inflammation characterized by blood eosinophil count in those ages 12 and older who are inadequately controlled despite high dose inhaled corticosteroids and another asthma controller. The drug can also be used as an add-on therapy with intranasal corticosteroids in adults with severe chronic rhinosinusitis with nasal polyps (CRSwNP) for whom treatment with systemic corticosteroids or surgery does not provide adequate disease control.
The approval is based on four trials that demonstrated sustained efficacy through twice-yearly dosing with Exdensur and comes after the FDA green lit the med as an add-on maintenance treatment for severe asthma characterized by an eosinophilic phenotype in December. In Europe, about 42 million people are affected by asthma, with 5% to 10% of patients experiencing severe asthma, according to GSK.
“Exdensur may help redefine care for the millions of patients living with these persistent and burdensome conditions, supporting them in achieving their treatment goals with just two doses a year,” GSK’s head of respiratory, immunology and inflammation R&D Kaivan Khavandi said in a company press release.
With a stamp of approval from the European Commission, Novo Nordisk has officially added a 7.2 mg once-weekly maintenance dose of the obesity blockbuster Wegovy to its European arsenal.
The new injectable dose strength marks Wegovy’s sixth in Europe, allowing physicians another option for patients who need additional weight loss after treatment with the 2.4mg dose. Starting now, the injection can be prescribed as three 2.4 mg injections, to be taken in a single sitting once a week, according to the company. Novo has also applied for a 7.2 mg single-dose pen in Europe, which could become available this year if approved.
European regulators signed off on the "more effective" dose strength, as Novo calls it, based on two clinical trials. In the studies, patients taking Wegovy as instructed charted average weight loss of 21%, with about 1 in 3 people shedding 25% or more of their body weight.
The 7.2 mg dose is already available in the U.K. and is currently under review in the U.S.
UPDATED: Tuesday, February 17 at 8:30 a.m. ET
Two months after Johnson & Johnson's Rybrevant Faspro picked up its first FDA approval, the subcutaneous lung cancer drug has scored a label expansion to be given monthly.
On Tuesday, J&J touted a "simplified" monthly dosing regimen for the drug's combination with lazertinib for the first-line treatment of epidermal growth factor receptor EGFR-mutated advanced non-small cell lung cancer. Previously, the combo was approved as an every-two-week regimen.
For weeks 1 through 4, patients must still receive weekly doses of Rybrevant Faspro. Beginning week 5, the doses can shift to monthly administration.
Rybrevant Faspro picked up its initial FDA nod back in December, part of J&J's ambition to challenge AstraZeneca's leading Tagrisso in the field. With the subcutaneous dosing nod, administration of Rybrevant moved from hours to minutes, with far fewer administration-related reactions.
"Building on unmatched overall survival and regimens that support proactive side effect management, this once-monthly injection now delivers the simplest and fastest combination therapy for patients with EGFR-mutated non-small cell lung cancer," Mahadi Baig, M.D., vice president of U.S. medical affairs at J&J, said in a statement.
UPDATED: Friday, February 13 at 10:35 a.m. ET
Roughly two months after scoring a thumbs up in the same indication from the FDA, Amgen's Uplizna has been cleared for treatment of generalized myasthenia gravis (gMG) in Europe.
The European Commission has green lit Uplizna, also known as inebilizumab, as an add-on to standard therapy for adults with gMG who are AChR or MuSK antibody positive.
The approval introduces another targeted therapy into the increasingly crowded treatment field for gMG. After two initial loading doses, Uplizna is dosed just twice a year, Amgen noted in a Feb. 12 press release.
European regulators signed off on Amgen's drug after reviewing data from the company's late-stage Myasthenia Gravis Inebilizumab Trial, which also included a steroid tapering protocol.
At the trial's 26-week mark, treatment with Uplizna was associated with a 1.9-point difference on a clinical measure of gMG symptoms versus placebo, Amgen said of the data around the time of its U.S. approval last year.
Generalized myasthenia gravis is a rare autoimmune disease that can cause fluctuating muscle weakness. It is estimated to affect between 56,000 and 123,000 people in Europe.
Aside from Uplizna, Johnson & Johnson's gMG med Imaavy and argenx's FcRn blocker Vyvgart are also approved in Europe, as is the argenx drug's subcutaneous formulation Vyvgart Hytrulo.
Sticking on the EU, the European Medicines Agency's safety committee, PRAC, has recommended that drugs containing levamisole be withdrawn from the European market.
PRAC made its call after an EU-wide review of the medicines found that their benefits no longer outweigh the risks for treatment of parasitic worm infections.
Specifically, the safety board found that leukoencephalopathy can be a rare but serious side effect from taking levamisole-containing drugs. Leukoencephalopathy damages the white matter of the brain and can be debilitating and potentially fatal if left untreated, according to the EMA.
Levamisole is an anthelminthic medicine used to treat infections caused by five different types of parasitic worm. The treatment stimulates nicotinic acetylcholine receptors, which are proteins found on the surface of the worm's nerve cells, causing paralysis of the parasite's muscles so it can be expelled from the gut, according to the EMA.
The drugs implicated in the safety recommendation are known as Decaris and Levamisol Arena and are authorized in Hungary, Lithuania, Latvia and Romania, according to the European regulator.
Over in China, Eli Lilly has scored an approval for its drug mirikizumab, sold in the U.S. as Omvoh, in adults with moderate to severe active Crohn's disease and ulcerative colitis.
The thumbs up from China's National Medical Products Administration (NMPA) was informed by a pair of phase 3 programs, Lilly noted in a recent announcement on its WeChat account (Chinese).
In the phase 3 VIVID-1 study in Crohn's, the antibody drug helped 45% of patients achieve clinical remission at week 12 compared to 20% of patients receiving placebo. Meanwhile, 38% of mirikizumab patients achieved a clinical response at week 12 and endoscopic response at the one-year mark versus just 9% of patients in the study's control arm.
As for the ulcerative colitis program, dubbed LUCENT, Lilly tested its med across two phase 3 studies looking at mirikizumab as both an induction and maintenance therapy. The drug's performance allowed LUCENT to meet primary and key secondary endpoints by helping patients achieve sustained clinical remission and significantly improving some of the some of the most severe ulcerative colitis symptoms.
Mirikizumab has already picked up inflammatory bowel disease nods in multiple other countries, including the U.S. and EU. Notably for Lilly, the green light marks the company's first innovative drug approved in China in a digestive and immunology indication, which Lilly said marks "a key step forward in this treatment area."
UPDATED: Monday, February 9 at 1:00 p.m. ET
Eli Lilly has secured approvals from the FDA and the European Medicines Agency (EMA) for Alzheimer’s disease drug Kisunla, but the company is striking out with regulators in the U.K.
For a second time, Scotland has rejected the amyloid beta-directed antibody. The Scottish Medicines Consortium (SMC) has determined that Kisunla does not meet its cost-effectiveness standard.
This marks the second thumbs down issued by the SMC for Kisunla in the last seven months.
In June of last year, the U.K.’s National Institute for Health and Care Excellence (NICE) rejected Kisunla and Biogen and Eisai’s Alzheimer’s treatment Leqembi, also citing a lack of cost effectiveness. It was the third red light issued by NICE for the infused drugs over the last few years, preventing the National Health Service from providing them in England and Wales.
The FDA endorsed Leqembi on an accelerated basis in January of 2023 and converted the nod to a full approval six months later. The U.S. regulator signed off on Kisunla in July of 2024. Last year, the EMA granted marking authorization to Leqembi in April and Kisunla in September.
UPDATED: Friday, February 6 at 10:55 am. ET
The FDA removed a prior "limitations of use" restriction it had placed on Gilead Sciences’ CAR-T Yescarta, allowing it to be used in patients with relapsed or refractory (R/R) primary central nervous system lymphoma (PCNSL).
Yescarta is approved for R/R large B-cell lymphoma, but previously wasn’t permitted to treat those with the rare, fast-growing PCNSL subtype. Prognoses related to this disease, which originates in the brain, spinal cord, eye, or cerebrospinal fluid, are typically poor, with a five-year survival rate of about 30%. The cancer type has no standard-of-care treatment options and an estimated 1,500 cases are diagnosed annually in the U.S.
Dana-Farber Cancer Institute ran a phase 1 study to evaluate the safety of Yescarta in patients with PCNSL, as those with the disease had previously been excluded from the clinical trials supporting Yescarta’s initial approval, global head of development at Gilead’s Kite unit, Gallia Levy, M.D., Ph.D. explained in a company release.
With the safety study's positive outcome, Yescarta is now the only CAR T-cell therapy that has the "limitations of use" removed from its label for PCNSL.
The U.S.’ first oral film to treat erectile dysfunction (ED) is here with an FDA approval for IBSA’s Vybrique, a dissolving, single-dose sildenafil formulation indicated for men aged 18 and older.
Sildenafil in its tablet formulation is best known as Pfizer’s Viagra. Vybrique’s dissolving version was made using IBSA’s FilmTec technology. The approval introduces a new option that dissolves on the tongue without the need for food or water.
ED remains a "sensitive topic where many men value discretion,” IBSA’s USA subsidiary’s CEO Nicholas Hart explained in a company release. “The FDA approval of Vybrique provides men experiencing ED with a novel treatment option that helps meet the evolving needs of patients today.”
The drug will be offered in four dosage strengths via direct home delivery and is expected to hit the commercial market in March.
Pfizer aims to grow the reach of Hympavzi with an FDA application to treat patients with hemophilia A or B who are 6 and older with inhibitors. The company also aims to include those ages 6 to 11 years old without inhibitors in the drug's label.
The additional indications would add to the drug’s existing approval for patients 12 years of age and older who have hemophilia A without factor VIII inhibitors or hemophilia B without factor IX inhibitors. The FDA will complete its review and issue a decision in the second quarter of this year, Pfizer said in a release.
UPDATED: Tuesday, February 3 at 2:30 pm. ET
AstraZeneca and Daiichi Sankyo’s Datroway has received a regulatory tailwind in the U.S. as the partners seek to expand the antibody-drug conjugate’s (ADC’s) reach in breast cancer.
On Tuesday, AZ and Daiichi revealed that the drug won a priority review tag from the FDA for treatment of adults with unresectable or metastatic triple-negative breast cancer (TNBC) who aren’t eligible for PD-1/PD-L1 immunotherapy.
The review is being conducted as part of the FDA’s Project Orbis framework, which allows for the concurrent submission and review of potential cancer medicines. With the priority review tag, a decision on Datroway in the prospective new indication is expected to come down from the FDA sometime in the second quarter.
The partners suspect that, if approved, Datroway could become the standard of care in the TNBC population they’re pursuing. Roughly 70% of patients with metastatic TNBC aren’t candidates for immunotherapy, with chemotherapy currently the only approved 1st-line treatment for the group, according to AZ and Daiichi.
AZ and Daiichi are leveraging data from Datroway’s late-stage TROPION-Breast02 study, which pitted the ADC against chemotherapy and met its dual primary endpoints in overall survival and progression-free survival.
Datroway’s ascent has been swift. After nabbing an FDA green light to treat HR-positive, HER2-negative breast cancer in patients who’ve tried prior endocrine-based therapy and chemotherapy last January, the drug in June added a second indication in a subset of non-small cell lung cancer patients.
AbbVie also made regulatory moves this week, announcing on Feb. 3 that it submitted applications to both the FDA and the European Medicines Agency (EMA) for a potential Rinvoq label expansion in adults and adolescents with non-segmental vitiligo (NSV).
Should the JAK inhibitor win approval, it would become the first systemic medication for vitiligo, AbbVie noted in a press release.
Vitiligo is a chronic autoimmune disease most commonly marked by symmetrical and bilateral depigmented white patches on the skin, which AbbVie notes can be “prone to unpredictable progression even after long periods of stability.” The company asserts that there are three pillars in vitiligo treatment: disease stabilization, re-pigmentation and maintaining re-pigmentation.
AbbVie is using data from its Viti-Up program—which included two replicate phase 3 studies—to inform the Rinvoq applications. The drug enabled both studies to hit their primary endpoints, thanks to statistically significant score improvements over placebo on measures of de-pigmentation covering the entire body and the face.
Rinvoq is one of two AbbVie meds, alongside Skyrizi, that the company has positioned to fill Humira’s shoes. The drug already boasts approvals in a suite of immunology indications, including rheumatoid arthritis, atopic dermatitis, ulcerative colitis and Crohn’s, and ankylosing spondylitis, among others.
Meanwhile, over in China, Bayer has picked up an approval for its androgen receptor inhibitor (ARi) Nubeqa, also known as darolutamide.
China’s National Medical Products Administration (NMPA) has signed off on the medicine in combination with androgen deprivation therapy (ADT) for patients with metastatic hormone-sensitive prostate cancer (mHSPC).
With the green light, Nubeqa plus ADT is now cleared in China for the treatment of adults with mHSPC both with and without the chemotherapy docetaxel, Bayer noted in a Feb. 03 release.
China’s drug regulator approved Nubeqa after reviewing data from the company’s ARANOTE study. In the phase 3 trial, the Nubeqa-ADT combo cut the risk of radiological progression or death by 46% compared to placebo plus ADT. Bayer added that its drug was generally well tolerated and showed lower discontinuation rates from adverse events when compared to the trial’s control regimen.
Prostate cancer is the second most common cancer in men, and the burden of the disease is on the rise in China, by Bayer’s estimate. The company noted that 315,310 new cases and 81,540 prostate cancer deaths are predicted to occur in China by 2030.
UPDATED: Monday, February 2 at 4:40 p.m. ET
In Europe, Acadia Pharmaceuticals' application for trofinetide in Rett syndrome has been met with a negative trend vote from the Committee for Medicinal Products for Human Use (CHMP), the company said Monday.
Assuming the CHMP officially turns away the company's approval bid this month, Acadia plans to request a re-examination.
"While the negative trend vote is disappointing and not what we hoped for, we believe the strong data that supported the approval of trofinetide for the treatment of Rett syndrome in the United States, Canada, and Israel speak to the meaningful benefits that trofinetide can deliver," Acadia CEO Catherine Owen Adams said in a statement.
Two years ago, Acadia's drug won an FDA approval to become the first approved treatment for the neurodevelopmental disorder.
Rett syndrome affects one out of every 10,000 to 15,000 female births worldwide, according to Acadia. After an initial period of apparently normal development, patients' development starts to slow around 18 months. Patients can then see a developmental regression around this time, followed by slower development afterward.
"We look forward to working with the EMA and other stakeholders to advance trofinetide as an important potential treatment option in the EU," Owen Adams added. "Our commitment to the Rett syndrome community in the EU remains steadfast, and we are fully dedicated to making trofinetide available to individuals and families who urgently need a new therapeutic option.”
UPDATED: Thursday, January 29 at 1:42 p.m. ET
The FDA has accepted Summit Therapeutics' application seeking approval of Akeso-partnered ivonescimab in combination with chemotherapy in EGFR-mutated nonsquamous non-small cell lung cancer, the U.S. company said Thursday. The target decision date is Nov. 14, 2026.
The application is a risky bet. Summit filed the closely watched PD-1xVEGF bispecific last year based on data from the phase 3 HARMONi trial, which missed its overall survival goal, even though the FDA had told the company that a statistically significant OS would be necessary to obtain an approval here.
The trial readout was complicated by the rollover of patients from the Chinese HARMONi-A trial, which met its OS endpoint, and by a slower-than-expected enrollment of U.S. patients. Summit is arguing that ivonescimab has shown a consistent benefits in U.S. and Chinese patients.
Elevar Therapeutics has refiled its Hengrui Pharma-partnered combination of PD-1 inhibitor camrelizumab and VEGFR inhibitor rivoceranib with the FDA for the first-line treatment of liver cancer, Elevar's parent company HLB said.
The FDA had previously rejected the regimen twice because of shortfalls at a Hengrui manufacturing facility.
UPDATED: Monday, January 26 at 10:45 a.m. ET
The European Union has expanded its endorsement for GSK’s respiratory syncytial virus (RSV) vaccine Arexvy, allowing its use by those age 18 and older.
The shot, which helps prevent lower respiratory tract disease (LRTD) caused by RSV, was previously approved in Europe in June of 2023 for individuals age 60 and older. A year later, the EU expanded its endorsement to those between ages 50 and 59 who are at increased risk because of underlying conditions.
The expansion comes as the demand for vaccines is rapidly declining in the United States and companies are becoming more reliant on sales in Europe and the rest of the world. In the third quarter of last year, GSK reported (PDF) a 34% increase in sales of Arexvy, which came despite a 21% decline of its sales in the U.S.
In the quarter, GSK’s overall vaccine revenue fell by 18% overall due largely to a sales decline in the U.S. for Arexvy and blockbuster shingles shot Shingrix. With the results, the company flipped its vaccines sales projection for 2025 from an increase to a decline. The company will report 2025 sales on Februray 4.
The FDA signed off on Arexvy in May of 2023 for those age 60 and older, then expanded the nod to individuals age 50 and older who are at increased risk of severe infection. In 2024, however, the CDC shifted its recommendation for RSV vaccine use to those age 75 and older and others ages 50 to 74 who are at increased risk.
The FDA has accepted Biogen and Eisai’s application for Alzheimer’s disease drug Leqembi to be administered by way of an autoinjector during the initiation phase of treatment. The potential new option would make Leqembi the first anti-amyloid Alzheimer’s treatment available for at-home use for initiation and maintenance dosing.
The U.S. regulator has granted a priority review for the application and established a decision date of May 24 of this year. With an approval, the autoinjector could be used to administer a once-weekly starting dose, as an alternative to the current bi-weekly intravenous (IV) dosing over the initiation period which lasts for 18 months.
The FDA originally signed off on Leqembi as an infused treatment in January of 2023 and converted the conditional nod to a full endorsement six months later. In August of last year, the agency approved the subcutaneous autoinjector for once-weekly maintenance.
Eli Lilly’s anti-amyloid drug Kisunla reached the market in July of 2024. There is no at-home dosing option for the infused treatment. In the third quarter of last year, Biogen reported sales of Leqembi at $121 million, compared (PDF) to sales of $70 million for Kisunla.
UPDATED: Friday, January 23 at 9:45 a.m. ET
The National Institute for Health and Care Excellence (NICE) has changed its tune on the combination of Pfizer's once-daily PARP inhibitor Talzenna and enzalutamide (Xtandi) in patients with untreated hormone-relapsed metastatic prostate cancer.
In new final draft guidance, the U.K. agency calls for the drugs to be used in cases when chemotherapy is not clinically indicated or when abiraterone plus prednisolone are not appropriate.
Pfizer must also provide its drug under the "commercial arrangement" it offered to NICE, the agency said.
"We are continuing to focus on what matters most to people by recommending this effective treatment that can make a huge difference to the lives of people with advanced prostate cancer," Helen Knight, director of medicines evaluation at NICE, said in a statement.
In recommending against the pairing last August, the agency at the time said "available evidence does not suggest that talazoparib with enzalutamide is value for money in this population."
In China, authorities have approved GSK's Trelegy as a treatment for adults with uncontrolled asthma.
The approval from China's National Medical Products Administration (NMPA) adds to the drug's prior nod in the country in chronic obstructive pulmonary disease (COPD).
About 46 million adults in China are affected by asthma, GSK said in a Friday press release.
UPDATED: Thursday, January 22 at 10:20 a.m. ET
England’s National Institute for Health and Care Excellence (NICE) has given a thumbs up to Roche’s Gazyvaro as a treatment for lupus, following nods in the same indication from the FDA in October and the European Commission in December.
After an induction period, Gazyvaro is infused twice a year and is taken in combination with mycophenolate mofetil, a daily immunosuppression tablet. The treatment is designed to restore kidney function for those with lupus nephritis, an autoimmune disorder that causes inflammation and organ damage.
Up to one-third of people living with the disorder progress to end-stage kidney disease. It affects more than 1.7 million people worldwide and most predominantly women of color and childbearing age.
Roche's monoclonal antibody, which targets the protein CD20, originally reached the market in 2013 as a blood cancer treatment in the U.S., where the drug goes by the commercial name Gazyva.
With the lupus nod, Gazyva could now achieve sales of $1.7 billion by 2030, according to GlobalData.
AstraZeneca and Daiichi Sankyo have earned a sixth nod in China for their blockbuster Enhertu, this time as a second-line treatment for stomach cancer. The approval covers patients with HER2 metastatic cancer who have been treated with a trastuzumab-based (Herceptin) therapy. The nod marks an evolution from Enhertu's previous endorsement for third-line use, which AZ and Daiichi secured in 2024.
Last year, the companies presented data from a phase 3 trial positioning Enhertu as the first HER2-directed drug to mount a patient survival benefit in the second-line indication.
Enhertu reached the market in the U.S. in 2019 and has been cleared by the FDA for several types of breast cancer. The U.S. regulator also approved the Daiichi-developed antibody-drug conjugate as a treatment for second-line gastric cancer in 2021. Enhertu generated sales of $3.6 billion in the first nine months of 2025.
UPDATED: Wednesday, January 21 at 09:58 a.m. ET
The European Commission has approved Ionis and Otsuka's Dawnzera (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients ages 12 years and older. The EU go-ahead follows an FDA nod in August for the same indication.
Dawnzera, which comes in a self-administered subcutaneous autoinjector, is the first RNA-targeted treatment for HAE. The EU approval is based on positive results from the phase 3 OASIS-HAE and OASISplus studies. In OASISplus, patients who switched to Dawnzera from prior prophylactic treatments experienced a further 62% reduction in mean monthly HAE attack rate at week 16 compared with baseline.
Otsuka holds exclusive rights to Dawnzera in Europe and the Asia-Pacific region. The EU nod triggered a $15 million milestone payment to Ionis, which is also entitled to tiered royalties of up to 30% on net product sales.
Organon said the FDA has approved the company's pregnancy prevention implant, Nexplanon, for longer duration of use. The the birth control implant can now be used for up to five years, versus three years previously.
In a clinical trial to assess the contraceptive for extended use beyond three years, no pregnancies or new safety findings were reported. In a Jan. 16 press release, Organon also touted the study's enrollment of women with a range of body mass index (BMI) values, including 38.1% who are considered to have obesity.
But together with the extension, the FDA also added a new Risk Evaluation and Mitigation Strategy (REMS) program to mitigate complications due to improper insertion and removal. This REMS program builds on existing training requirements installed by Organon. Nexplanon will only be available in the U.S. through the REMS program, which is expected to be available starting on February 23, 2026.
ImmunityBio has not given up on pursuing an FDA approval for Anktiva in BCG-unresponsive papillary non-muscle invasive bladder cancer after a refuse to file letter from the FDA. The issue centered on whether ImmunityBio can use uncontrolled data to seek an approval in the indication. The company's rival Johnson & Johnson previously indicated to Fierce that, after a long conversation with the FDA, it decided to use a randomized phase 3 study to pursue the papillary population.
But in a Jan. 20 announcement, ImmunityBio said that the FDA recommended the company provide “additional information” to potentially support a resubmission. This “does not include the initiation or design of any new clinical trials”, ImmunityBio said. The company plans to submit the new information within 30 days.
"This submission follows a productive face-to-face meeting with senior FDA officials, during which the regulatory path forward for Anktiva in papillary NMIBC was collaboratively defined," ImmunityBio said.
UPDATED: Tuesday, January 13 at 11:00 a.m. ET
The FDA is requesting Novo Nordisk and Eli Lilly update the labels of their popular weight loss medicines to remove warnings about suicidal behavior or ideation, Bloomberg reports.
The move affects Lilly's Zepbound and Novo's Wegovy and Saxenda, according to the news service.
In making the request, the FDA said it had looked into the potential for the drugs to cause suicidal behavior or ideation, but that the data don't point to an increased risk.
The regulator reviewed data from dozens of studies and other sources to draw its conclusion, according to Bloomberg.
A study last year reached a similar conclusion.
UPDATED: Tuesday, January 6 at 11:15 a.m. ET
GSK's Exdensur has been endorsed in Japan as a treatment for severe asthma and chronic rhinosinusitis with nasal polyps (CRSwNP), making it the first ultra-long-acting biologic in the country in the two indications.
Japan's Ministry of Health, Labour and Welfare approved the drug for severe or refractory patients with asthma whose symptoms cannot be controlled with existing treatments and for patients with CRSwNP whose disease is inadequately controlled with standard treatment.
GSK's Swift and Anchor phase 3 studies supported the approval, GSK said in a press release. The trials showed that twice-yearly doses of the drug plus standard of care resulted in significant reductions in asthma exacerbations and nasal polyp size and nasal obstruction in the respective indications compared with placebo and standard of care.
The Japan nod comes amid a regulatory winning streak for the medicine, which recently picked up approvals in the U.S. and U.K.
UPDATED: Monday, January 5 at 9:05 a.m. ET
China's National Medical Products Administration (NMPA) has approved GSK's Nucala as an add-on maintenance treatment for adults with chronic obstructive pulmonary disease (COPD) whose condition is characterized by raised blood eosinophils.
With the approval, Nucala becomes the first monthly biologic in the country for a large proportion of patients with inadequately controlled COPD, GSK said in a Jan. 5 release. The nod covers patients with blood eosinophil counts (BEC) as low as 150 cells/µL.
The approval was based on data from GSK's MATINEE and METREX phase 3 studies. In the trials, Nucala showed its ability to reduce the rate of moderate/severe COPD exacerbations versus placebo and standard of care in a "wide spectrum" of COPD patients with an eosinophilic phenotype, GSK said.
Roughly 100 million people in China have COPD, the company said. Deaths from COPD in the country represent nearly a third of all COPD deaths worldwide, according to the drugmaker.
"The approval of Nucala offers patients in China a monthly add-on maintenance treatment to reduce exacerbations, including those leading to emergency department visits and/or hospitalisations which account for a large proportion of annual direct medical costs," Kaivan Khavandi, GSK's global head of respiratory, immunology and inflammation R&D, said in a statement.
UPDATED: Friday, January 2 at 9:40 a.m. ET
After receiving prior FDA rejections in 2023 and mid-2025, Outlook Therapeutics capped off the year by announcing that ONS-5010 had been turned away by the U.S. regulator once again.
In a Dec. 31 announcement, the company said the FDA spurned Outlook's Biologics License Application resubmission based on a lack of confirmatory evidence.
ONS-5010, also known as Lytenava, is an ophthalmic reformulation of bevacizumab, a cancer drug from Roche that has long been used off-label as a treatment for wet age-related macular degeneration (wet AMD).
In its Complete Response Letter, the FDA cited an ongoing need for "confirmatory evidence of efficacy," the company said, caveating that the agency "has not indicated what type of confirmatory evidence would be acceptable."
After the FDA rejected Outlook's prior filing last summer, the company resubmitted its application in November with data from its Norse clinical trial program. The company contends its data package "provides the required evidence to support approval of the ONS-5010" in the U.S.
Before the latest round of regulatory interactions, the FDA rejected the drug back in 2023 over manufacturing and data shortfalls. In Europe, officials approved the drug last year as a treatment for wet AMD.
Before the U.S. market opened on Friday, Outlook's stock was trading down about 61% to 61 cents per share.