Sarepta, FDA
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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.
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 .
Analysts downgraded Sarepta as FDA pause on Elevidys raises regulatory uncertainty, leading to significant stock pressure and valuation cuts.
While Sarepta has now consented to the FDA’s request to stop selling Elevidys, the company’s brief standoff with the agency could still carry major consequences for the Duchenne community.
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,
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.
Sarepta Therapeutics refused a FDA request to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy
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,
2don MSN
Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an unrelated drug.