Sarepta, FDA and Duchenne
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Sarepta, FDA and The Cambridge
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Arrowhead Pharmaceuticals said on Wednesday it expects to receive near-term milestone payments from Sarepta Therapeutics as part of their licensing agreement, despite recent setbacks at the Cambridge,
Arrowhead Pharmaceuticals has moved to reassure Wall Street about its deal with Sarepta Therapeutics, telling investors that it expects its troubled R&D partner to meet its financial obligations. Sarepta struck a major deal with Arrowhead in November,
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.
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Sarepta Therapeutics' (SRPT) stock continued to sink Tuesday, down more than 4% in pre-market trade, following a rough end to last week, including a previously unreported patient death in a clinical trial of an unnamed therapy and the threat of the FDA taking a different therapy,
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.
The FDA has placed multiple investigational gene therapy clinical trials on hold, signaling broader platform concerns.
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.
Shares of Cambridge, Mass.-based Sarepta have fallen by more than 90% in the last year amid safety concerns about its Elevidys, a gene therapy that treats Duchenne muscular dystrophy, following reports of sudden patient deaths. Sarepta recently halted sales of the treatment.