High-Ranking Meddling Behind Sarepta Duchenne Drug Delay?
Executive Summary
Involvements of senior FDA officials may be the reason the agency put off its much-anticipated decision last week on Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy drug eteplirsen, analysts said.
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Sarepta Therapeutics Inc. investors panicked over the FDA's June 2 release of revised expanded access guidances – worried the action was in preparation for the agency to reject the company's Duchenne muscular dystrophy drug eteplirsen, whose verdict was curiously delayed last week.
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Seeing no clear path to approval in the US or Europe for its Duchenne muscular dystrophy drug Kyndrisa (drisapersen), BioMarin Pharmaceutical Inc. has decided to shelve the experimental medicine, along with three other first-generation follow-on products. But it said its Duchenne pursuit is not completely over, declaring it continues to "explore" its early-stage next-generation oligonucleotides as potential treatments for the disease, which generally kills patients before they reach 30 years.
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While investors were hopeful the FDA's delay in issuing its verdict for Sarepta Therapeutics Inc.'s Duchenne muscular dystrophy medicine eteplirsen may mean the agency is not prepared to reject it, the postponement may create more doubt and uncertainty about the path forward in rare disease drug development in general.