US FDA votes against Sarepta's eteplirsen for Duchenne muscular dystrophy

US RNA-based drug developer Sarepta Therapeutics (Nasdaq: SRPT) saw its shares plunge by a further 40% with the news that a Food and Drug Administration panel had voted against approving its Duchenne muscular dystrophy (DMD) drug eteplirsen.

The New Drug Application (NDA) was reviewed under the FDA accelerated approval pathway, which allows early approval of a drug for a serious or life-threatening illness that offers a benefit over current treatments.

But the Peripheral and Central Nervous System Advisory Committee (PCNSC) voted against granting the approval, after briefing documents from FDA staffers handed to the panel members expressed concerns about the strength of Sarepta’s marketing application.