Sarepta, FDA
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1don MSN
Sarepta Therapeutics laying off dozens in Ohio, won't comply with FDA request to halt therapy
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.
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.
Analysts downgraded Sarepta as FDA pause on Elevidys raises regulatory uncertainty, leading to significant stock pressure and valuation cuts.
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.
Roche Holding AG said on Tuesday it has paused shipments of muscular disorder gene therapy Elevidys in some countries outside the U.S., echoing a similar decision by U.S. partner Sarepta Therapeutics .
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.
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,
Children’s Hospital Los Angeles paused the use of Sarepta Therapeutics Inc.’s gene therapy for Duchenne muscular dystrophy in the wake of rising safety concerns about liver toxicity, adding to mounting challenges for the drugmaker.
1don MSN
Sarepta Therapeutics stock sinks further Tuesday, after halting shipment of bestselling drug Elvidys
Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an unrelated drug.