Sarepta Therapeutics Inc. SRPT said Wednesday that the U.S. Food and Drug Administration has indicated after talks with the company that it’s working toward granting accelerated approval of SRP-9001, its latest treatment for Duchenne muscular dystrophy, or DMD. The agency said that’s subject to the completion of its Biologics License Application (BLA) review and that it would initially be for use in patients aged 4 to 5 years old. The FDA also told Sarepta that if the confirmatory Phase 3 study of SRP-9001 meets its objectives, it would entertain a non-age restricted expansion of the label. The trial is fully enrolled and top-line data are expected in the fourth quarter. The FDA further said it may require ‘modest additional time” to complete the review, “including final label negotiations and postmarketing commitment discussion.” The agency is expecting the review to be complete by June 22. An FDA advisory panel voted 8-6 in favor of accelerated approval for the therapy earlier this month, despite questions about the therapy’s clinical benefit and safety. DMD is an inherited disorder of progressive muscular weakness that typically affects boys. Symptoms that appear in early childhood include frequent falls, difficulties getting up or running and learning disabilities, and patients have a life expectancy of about 27. The stock fell 12% premarket.
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