Roche, Gene and Duchenne muscular dystrophy
Digest more
6hon 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.
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.
Roche Holding AG paused all shipments of the controversial gene therapy Elevidys, Bloomberg News reported on Tuesday.
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.
Explore more
Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, today issued the following statement: