The Chicago Entrepreneur

: Sarepta Therapeutics stock halted premarket ahead of FDA panel meeting on new Duchenne muscular dystrophy treatment

Sarepta Therapeutics Inc. stock SRPT was halted premarket Friday, ahead of a meeting of a Food and Drug Administration advisory panel to discuss the company’s SRP-9001 gene therapy for the rare genetic disorder Duchenne muscular dystrophy, or DMD. DMD 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. About 20,000 children are diagnosed with DMD globally every year, according to the Muscular Dystrophy Association. The FDA was leaning toward rejecting the therapy, according to an April report from specialty healthcare news service STAT,  before a top official intervened and directed staff to schedule an advisory committee meeting. FDA Advisory Committees make recommendations to the FDA on whether to approve a drug, although the agency is not bound to the committee’s decision. Sarepta already markets three treatments for DMD. The stock has gained 90% in the last 12 months, but is down 7% in the year to date, while the S&P 500 SPX has gained 8%.

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