Sarepta, FDA and gene
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10hon 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.
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.
Shares of Sarepta Therapeutics plunged more than 30% on Friday as the future of its approved gene therapy appeared at risk. Sarepta has reported three patient deaths related to its gene therapies.