Sarepta plunges as FDA questions eteplirsen trial results

Shares of US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) plummeted 46% to $19.70 in pre-market trading on November 12, after the company indicated that the US regulator may require more data on its investigational Duchenne muscular dystrophy (DMD) drug eteplirsen. Before this, the stock had risen 42% so far this year.

Citing recent developments since Sarepta's last meeting with the US Food and Drug Administration, including a failed study with a competitive product and recent natural history data in DMD, the FDA indicated the new data raise "considerable doubt" about both the dystrophin biomarker and the supportive clinical efficacy assessed on the 6-minute walk test (6MWT) in the Phase IIb clinical study of eteplirsen. As a result of these recent data, the FDA stated that it "currently considers an NDA [New Drug Application] filing for eteplirsen as premature."

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