Sarepta, Elevidys and FDA
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1don MSN
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.
Sarepta Therapeutics' (SRPT) stock has been hammered in the past week after the company was forced to pause shipments of its top-selling Duchenne muscular dystrophy (DMD) therapy Elevidys — throwing the future of the company into question.
The Swiss company, which markets Elevidys abroad, said it believes the benefit-risk balance to treatment remains positive in Duchenne patients who can still walk, however.
Analysts downgraded Sarepta as FDA pause on Elevidys raises regulatory uncertainty, leading to significant stock pressure and valuation cuts.
Roche is pausing shipments of Elevidys in some countries, following partner Sarepta Therapeutics’ move in the U.S., as safety concerns mount over the Duchenne muscular dystrophy gene therapy. | Roche is pausing shipments of Elevidys in some countries,
Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.
As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head, the biotech is standing firm behind its approved Duchenne muscular dystrophy (DMD) treatment. | As the controversy over the safety of Sarepta Therapeutics’ gene therapies comes to a head,
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
1don MSN
Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an unrelated drug.