FDA, Sarepta Therapeutics
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Sarepta Therapeutics Inc. announced that the company plans to temporarily pause all shipments of its gene therapy to treat Duchenne muscular dystrophy, Elevidys, in a reversal of its prior stance.
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.
After initially refusing to suspend Elevidys distribution after two deaths, Sarepta has now given in to the FDA’s request, noting the need to maintain a good working relationship with the regulator.
Shipments will halt by close of business Tuesday evening, the company said. Sarepta had initially rejected the agency’s request, which was issued Friday.
A brief skirmish between Sarepta Therapeutics and the FDA has ended before escalating into a full-on regulatory clash, as the company has bowed to the agency’s demand. | A brief skirmish between Sarepta Therapeutics and the FDA has ended before escalating into a full-on regulatory clash,
Sarepta Therapeutics said it will voluntarily “and temporarily” pause all shipments of Elevidys® (delandistrogene moxeparvovec-rokl), the marketed Duchenne muscular dystrophy (DMD) treatment linked to the deaths of two patients this year.
The head of the US Food and Drug Administration said the agency will “take a hard look” at a gene therapy from Sarepta Therapeutics Inc. after two patient deaths, adding pressure to the biotech company which also announced a third patient had recently died on a similar treatment.
The regulator had asked Sarepta Therapeutics to halt all shipments of its therapy, Elevidys, after three patients died from liver failure after taking it or a similar treatment.