Investing.com — The U.S. Food and Drug Administration has recommended lifting the voluntary hold on Sarepta Therapeutics (NASDAQ:SRPT)’ gene therapy Elevidys for ambulatory patients with Duchenne Muscular Dystrophy (DMD).
The FDA concluded that the death of an 8-year-old boy was unrelated to the gene therapy product itself, allowing ambulatory patients to once again receive the treatment.
However, the voluntary hold remains in place for non-ambulatory patients following two deaths. The FDA stated it will continue working with Sarepta regarding this patient group.
Elevidys is an adeno-associated virus vector-based gene therapy that uses Sarepta’s AAVrh74 Platform Technology to treat DMD, a rare genetic condition characterized by progressive muscular weakness caused by a defective gene. The treatment is administered as a single intravenous dose.
Trading of Sarepta Therapeutics stock was halted after-hours Monday ahead of this FDA update. Shares had fallen 20% over the past month and were expected to rebound once trading resumes.
The FDA emphasized that the patient community is an important voice, and the agency will continue to listen and respond to thoughts from those impacted by DMD.
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