Regulatory
While FDA Commissioner Marty Makary emphasizes learning and humility, the FDA has systematically removed the very experience that would make change possible.
The decision follows last month’s vote of the CDC’s revamped vaccine advisory committee. Thimerosal, mostly removed from vaccines decades ago, has long been a target of anti-vaccine advocates for unfounded links to autism and adverse health outcomes.
The FDA will select at most five companies that align with national priorities, including lower drug prices and increased domestic investment.
Sarepta Therapeutics faces serious FDA action after news broke of a third patient death, the FDA gets a new top drug regulator in George Tidmarsh, a handful of new drugs get turned away from the market and pharma companies continue to commit billions to reshoring manufacturing.
Sarepta Therapeutics’ stock has dropped precipitously as questions swirl around the safety of its gene therapies. Meanwhile, the Duchenne patient community fears losing access to Elevidys while the regulator considers more drastic action.
Tidmarsh, an adjunct professor at Stanford’s medical school, brings decades of industry experience to the table. Serving as director of the Center for Drug Evaluation and Research will be his first government position.
Otsuka and Lundbeck’s data are insufficient to establish significant efficacy of Rexulti plus sertraline in PTSD, according to the FDA’s outside experts.
In light of recent patient deaths, the FDA has also revoked its platform designation for Sarepta’s AAVrh74 technology. The designation, granted last month, was the first of its kind to be announced publicly.
Earlier this summer the FDA asked Moderna for more efficacy data on its flu vaccine before it could review an mRNA-based combination shot that targets both influenza and COVID-19. Now, the entire vaccine sector is sizing up a new regulatory world, companies’ next steps uncertain.
The rollercoaster week for Sarepta Therapeutics continued, with shares of the embattled gene therapy-turned-siRNA biotech down 37% Friday afternoon as media outlets reported FDA plans to request a stop to all shipments of the Duchenne muscular dystrophy therapy Elevidys following a third patient death linked to the underlying platform.
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