FDA delays Sarepta MD drug
The FDA says Sarepta needs to put its experimental Duchenne's muscular dystrophy drug eteplirsen through additional testing. The company announced Tuesday that the agency wants the drug to be tested in a larger trial and compared to placebo. The regulator also wants a more diverse patient pool and different targets.
The FDA's letter to the drugmaker noted the data on-hand excluded “both younger boys who cannot perform such a demanding test, and older boys who are no longer ambulatory, we are concerned that seemingly avoidable limitations on enrollment could undermine study feasibility.”
The regulator noted that recent experience with a competitor's MD drug candidate, which had a similar mechanism of action, influenced its decision. Reuters notes that this drug was GlaxoSmithKline's drisapersen, which failed to show a significant gain in walking distance, much like Sarepta's drug. Walking is a common test in the MD space.
Tuesday's news was a further blow to the company. Reuters notes that Sarepta has been slogging through three decades without getting a drug through FDA review and onto the marketplace.