FDA, Sarepta
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For Sarepta, the hits keep coming, with several downgrades in the past week after it was forced to temporarily take its top-selling drug off the market.
Sarepta faces an “arduous and treacherous path” to try to get its Duchenne muscular dystrophy therapy Elevidys back onto the market
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.
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.
Analysts downgraded Sarepta as FDA pause on Elevidys raises regulatory uncertainty, leading to significant stock pressure and valuation cuts.
Arrowhead Pharmaceuticals (ARWR) stock gains as company reassures investors about financial obligations required from Sarepta (SRPT) under a 2025 deal. Read more here.
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 Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators.