Sarepta, FDA and gene
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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.
Roche Holding AG said on Tuesday it has paused all shipments of muscular disorder gene therapy Elevidys outside the U.S., echoing a similar decision by U.S. partner Sarepta Therapeutics on Tuesday.
Roche Holding AG has temporarily halted shipments of the gene therapy Elevidys in some countries outside the U.S. following a similar move by U.S. partner Sarepta Therapeutics. The decision comes after the FDA requested a pause following the death of a patient undergoing the treatment.
The Sarepta saga continues, with the FDA slapping a clinical hold across all of the company’s investigational limb girdle muscular dystrophy (LGMD) trials, while also revoking the biopharma’s gene therapy platform technology designation.
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.
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These were the most active stocks ahead of Friday's market open: Sarepta Therapeutics Inc.'s stock tumbled 27% after the company said another patient died following gene-therapy treatment, Bloomberg reported,
Shares of Sarepta Therapeutics declined 16.5% to $18.32 in premarket trading on Friday after a Bloomberg report that a patient enrolled in an early-stage study of one of the company's gene therapies has died from acute liver failure.