Recombinant Influenza vaccine quadrivalent(Protein Sciences Corp.)

The FDA has hit back against Moderna after the biotech decried the agency's refusal to review its mRNA-based influenza vaccine, according to a report from The Hill. On Tuesday, Moderna said it received a refusal-to-file (RTF) letter from the FDA's Center for Biologics Evaluation and Research (CBER) regarding its application for mRNA-1010, and blasted the US for providing "inconsistent" feedback.The controversy stems from Moderna's use of a licensed seasonal flu vaccine as the comparator in Phase III testing. While the FDA in 2024 had said the company should instead use a vaccine recommended by the Centers for Disease Control and Prevention (CDC) for adults 65 and up, such as Fluzone HD, Fluad or Flublok, the agency acknowledged that Moderna's choice of comparator "would be acceptable."CEO Stéphane Bancel denounced the FDA's refusal to review the mRNA shot, arguing that "it should not be controversial to conduct a comprehensive review of a flu vaccine submission that uses an FDA-approved vaccine as a comparator in a study that was discussed and agreed on with CBER prior to starting."However, during a press briefing on Wednesday, the FDA placed the blame for the RTF at Moderna's feet."The FDA rejected Moderna’s application for filing because the company refused to follow very clear FDA guidance from 2024 to test its product in a clinical trial against a CDC-recommended flu vaccine to compare safety and efficacy," FDA Commissioner Marty Makary said in a statement. "Moderna exposed participants, age 65 and over, to increased risk of severe illness by giving them a substandard of care against the recommendation of FDA career scientists."Moderna has requested a Type A meeting with the agency to understand the basis for the RTF, and find a path forward for its experimental flu shot. According to The Hill, an unnamed FDA official suggested that Moderna could resubmit the same data if "they come back, maybe even show some humility."

The European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) signed off on six new drugs as well as a clutch of approved drugs that received positive opinions for new indications. In the latest bunch of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP), Novo Nordisk’s semaglutide for severe liver disease could be soon off to the European market under a new name, while Eli Lilly’s Mounjaro didn’t quite make the cut for a new cardiovascular indication and Amgen’s approved Tavneos is up for a second look as concerns emerge on the data integrity of the drug’s pivotal trial.Novo’s Wegovy (semaglutide) made headlines last summer as the second drug in the U.S. approved to treat adult metabolic dysfunction-associated steatohepatitis (MASH) in certain patients. The FDA’s conditional nod made for Wegovy’s third indication, along with obesity and cardiovascular risk reduction, validating Novo’s continued pursuance of the tough-to-treat condition after previous let downs.The CHMP has now agreed with the FDA, recommending a conditional marketing authorization for the med, which will be branded separately from Wegovy in Europe under the name Kayshild. In other GLP-1 decisions, Eli Lilly’s Mounjaro also received news from the CHMP, as the agency has finalized its assessment of Lilly’s bid to extend the drug’s use to cover chronic heart failure with preserved ejection fraction (HFpEF) in adults with obesity.Although the EMA did not ultimately recommend a separate indication for the new use, it agreed to include “relevant data” from the Mounjaro study in this indication in the drug’s label, ensuring “up-to-date data on the effects of Mounjaro in adults with chronic HFpEF and obesity.” The regulator opted against a wholly new indication based on “uncertainty” remaining as to whether the positive results seen in the study is a “weight-loss-independent effect of Mounjaro,” the EMA explained in a release (PDF).Elsewhere, six new medicines, including Kayshild, earned positive CHMP opinions. UCB’s thymidine kinase 2 deficiency (TK2d) drug Kygevvi stood out as a positive opinion issued under “exceptional circumstances,” since the rarity of the disease means that the company had to use a single arm, 39-patient study and a retrospective chart review in its drug application. If approved, the drug would be the first treatment option for the rare genetic disease, and UCB would have to comply with yearly post-marketing obligations.“This positive CHMP opinion marks a turning point in the treatment of TK2d, offering a new chapter of hope and a meaningful step forward for patients, families, and clinicians alike,” UCB’s chief medical officer, Donatello Crocetta, commented in a company release. Sanofi, meanwhile, came out with two wins with a CHMP recommendation for its recombinant, quadrivalent flu vaccine Supemtek and for its Rezurock, which would be a new treatment option for adults and children with chronic graft-versus-host disease (GVHD). Sanofi had requested a re-review of Rezurock after a negative opinion was adopted by CHMP in October. The French pharma is making a commitment to conduct a new post-approval confirmatory study given the “limited late-line treatment options available for EU patients living with chronic GVHD and the body of Rezurock clinical evidence generated,” EVP of Sanofi’s general medicines unit, Olivier Charmeil, explained in a company release.The prior negative opinion was made because it “was difficult to quantify” the effect Rezurock had on patients relative to other treatments, the CHMP said at the time. Rezurock is already approved in 20 countries, including the U.S.Rounding out the wave of positive opinions, the CHMP also signed off on a hormone replacement therapy for menopause called Fylrevy (estetrol) made by Hungarian pharma Gedeon Richter and a radiolabeling product made by Shine that’s called Ilumira.Johnson & Johnson’s Akeega, Neurocrine’s Efmody, ARS’s Eurneffy (sold as Neffy in the U.S.), Incyte’s Inclusig, AstraZeneca’s Imfinzi, Bayer’s Kerendia, Merck’s Noxafil, Bristol Myers Squibb’s Opdivo and Incyte’s Zynyz all are up for label expansions based on positive CHMP opinions.Zynyz in particular would be Europe’s first immunotherapy-based treatment for squamous cell carcinoma of the anal canal (SCAC), the most common type of anal cancer. If approved, the drug could be a “new standard-of-care for patients living with this rare and difficult-to-treat cancer,” head of Incyte International Lee Heeson said in a company statement.All of CHMP’s recommendations are now off to the European Commission for final approval. Tavneos re-reviewTucked in with the positive opinions was negative news for Amgen’s complement inhibitor Tavneos, which CSL Vifor markets in Europe. Amgen took on the drug through its ChemoCentryx buyout in 2022. In January of that year, European regulators signed off on the drug to treat granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA), two rare inflammatory blood vessel conditions. The initial approval hinged on results from ChemoCentryx’s Advocate study, which compared Tavneos to high-dose corticosteroids in 331 patients and found that the med was as effective at prompting remission as the corticosteroids. But concerns about the trial have been mounting since 2021, when the FDA flagged several efficacy and safety issues in the study. Although Tavneos eventually got its FDA nod with a narrower label than anticipated, investors with money tied up in the debacle filed a lawsuit in 2022 alleging that the company knew about the FDA’s concerns but downplayed the situation to its shareholders. As the legal feud continued, additional evidence emerged last year pointing to a larger data integrity issue in the trial. Nonetheless, ChemoCentryx emerged victorious in a securities fraud complaint last summer and the FDA hasn’t taken action that would impact its U.S. approval. The EMA, however, will now “review all available data” to assess if the “emerging information” impacts the drug’s risk/benefit profile, the agency said in a Jan. 30 alert. The review was initiated at the request of the European Commission.“The concerns relate to how the company handled the data for the Advocate study before Tavneos was authorised, which may have impacted the findings on the medicine’s effectiveness,” the EMA explained.After the review, the agency will decide if the drug's marketing approval should be kept, suspended, amended or pulled altogether, it said. Despite the years-long legal back-and-forth, Tavenos garnered $256 million in revenue for Amgen during 2024, in 111% growth year over year.