Sarepta, FDA and Duchenne
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Sarepta Therapeutics' licensing partner Arrowhead Pharmaceuticals said on Wednesday it expects to receive near-term milestone payments from the drugmaker despite recent setbacks, including the death of a trial patient reported last week.
Sarepta faces an “arduous and treacherous path” to try to get its Duchenne muscular dystrophy therapy Elevidys back onto the market
Sarepta Therapeutics shares were lower in premarket trading on Tuesday, as the drugmaker relented to a Food and Drug Administration request to halt shipments of a gene therapy.
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.
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After the FDA request, Cambridge, Massachusetts-based Sarepta said in a statement that it will continue to ship the therapy to ambulatory people but maintain a halt it implemented June 15 for non-ambulatory patients after reporting to the FDA a case of acute liver failure in a patient who could not walk.
Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.
Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an unrelated drug.
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
Sarepta Therapeutics traded lower on Monday, extending a slide as the company took the unusual step of refusing a Food and Drug Administration request to stop selling a drug tied to three deaths.
The FDA will request Sarepta Therapeutics stop all shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy, following two deaths tied to the product.