FDA, Sarepta
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2don 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.
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.
Analysts downgraded Sarepta as FDA pause on Elevidys raises regulatory uncertainty, leading to significant stock pressure and valuation cuts.
Sarepta Therapeutics is pausing shipments of its gene therapy for muscular dystrophy following several patient deaths that have attracted attention from regulators.
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.
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,
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 .
Sarepta Therapeutics stock sinks further Tuesday after halting shipment of bestselling drug Elevidys
Sarepta stock tanked Friday after a new patient death was uncovered, spurring an FDA request to halt shipments of an unrelated drug.
Sarepta Therapeutics refused a FDA request to halt shipments of Elevidys, its gene therapy for Duchenne muscular dystrophy
A Cambridge-based company developing gene therapies for rare diseases is laying off more than a third of its workforce.