It’s not surprising that, of all the recommendations the U.S. CDC’s vaccine advisory board made at its June meeting, the first one Health and Human Services Secretary Robert Kennedy signed off on would essentially remove thimerosal from influenza vaccines in the U.S. What is surprising is the time it took for him to do so, given his long-held position on the preservative used in multidose vials and his insistence that a thimerosal presentation and vote be added to the agenda.
Apnimed Inc.’s lead oral therapy for obstructive sleep apnea has delivered a second round of positive phase III data, bringing a potential U.S. FDA filing closer. Top-line results taken from the second pivotal late-stage study show AD-109 (aroxybutynin 2.5mg/atomoxetine 75 mg) hit its primary endpoint with clinically meaningful and statistically significant drops in airway obstruction at 26 weeks.
In an about-face, Sarepta Therapeutics Inc. said it would “voluntarily and temporarily” pause all shipments of Duchenne muscular dystrophy gene therapy Elevidys (delandistrogene moxeparvovec) in the U.S. The move comes only a few days after the firm publicly declined a U.S. FDA request to halt shipping of the therapy in the wake of a third patient death, this one linked to a gene therapy using the same adeno-associated virus (AAV) vector as Elevidys.
There are a raft of problems the U.S. FDA wants resolved before Replimune Group Inc.’s BLA for RP-1 (vusolimogene oderparepvec) with nivolumab to treat advanced melanoma goes any further, all of which the company said are a surprise.
Despite Health and Human Services Secretary Robert Kennedy’s oft-repeated vow to rid the FDA of industry influence, FDA Commissioner Marty Makary announced George Tidmarsh, an industry veteran, will be the next director of the agency’s Center for Drug Evaluation and Research (CDER).
Sarepta Therapeutics Inc. is declining a U.S. FDA request to voluntarily halt shipping its gene therapy, Elevidys (delandistrogene moxeparvovec), in the U.S. On July 18, Sarepta said had it received “an informal request” from the FDA to stop the shipments following a third patient’s death, tied to the gene therapy SRP-9004, which uses the same vector as Elevidys.
The U.S. FDA approved 22 drugs in June, tying with March for the highest number of approvals and a jump from 15 approvals in May. The FDA approved 107 drugs in the first half (H1) of 2025, making it the third-highest total for this period in BioWorld’s records, trailing 118 approvals in 2024 and 108 in 2020.
The U.S. FDA on July 15 cleared Biocon Biologics Ltd.’s Kirsty (insulin aspart-xjhz) as the first and only interchangeable biosimilar product referencing Novo Nordisk A/S’ Novolog (insulin aspart), a rapid-acting diabetes medication.
The pharmaceutical industry in Europe has taken the lead in pulling together a coalition of 17 industry groups, academics and clinicians to call for the urgent implementation of the EU Life Science Strategy published earlier this month, in order to rescue the clinical trial ecosystem from “a perilous situation.”
Sarepta Therapeutics Inc. made known a third gene therapy death, this time with SRP-9004 for limb-girdle muscular dystrophy. The patient was a late-stage, non-ambulatory 51-year-old man participating in the phase I Discovery trial, who expired about a month ago of acute liver failure, as did the two previous subjects who passed away after they were treated with Elevidys (delandistrogene moxeparvovec), Cambridge, Mass.-based Sarepta’s gene product for Duchenne muscular dystrophy.
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