In the latest twist surrounding Sarepta Therapeutics, the Food and Drug Administration has delayed a decision on whether to approve a drug for Duchenne muscular dystrophy until May 26. At the same time, the FDA accepted an unusual extra submission from the drug maker as a “major amendment” to documents that the agency and its outside experts will review.
The drug, known as eteplirsen, is designed to combat DMD, a rare disease that causes muscles in boys to stop working and eventually results in death because they can no longer breathe. Clinical data was supposed to have been reviewed last month at an FDA expert panel meeting in advance of a Feb. 26 deadline for approving the drug. But that was postponed due to a snow storm and has not been rescheduled.
Meanwhile, Sarepta took the uncommon step last month of submitting another nine-page document in order to rebut an FDA analysis of its drug. Typically, the agency will post on its web site a review of a drug by its own medical staffers prior to an expert panel meeting. The agency also posts documents from the drug company, but in this case, also accepted the after-the-fact Sarepta filing.