The FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has announced that it will discuss the new drug application for eteplirsen (Sarepta Therapeutics) on April 25––nearly three months after the original action date. Sarepta is seeking approval for the treatment of Duchenne muscular dystrophy (DMD) in patients who have a confirmed mutation of the DMD gene that is amenable to exon 51 skipping.
On January 15, FDA staff said they were unconvinced about eteplirsen’s efficacy and trial design, a day after the agency rejected rival drug drisapersen (Kyndrisa, BioMarin Pharmaceutical) for insufficient efficacy.
There are no FDA-approved drugs for DMD, and pressure has been mounting on the FDA to quickly approve treatments.
Three years ago, Sarepta’s CEO claimed that the company was making progress in convincing the FDA to accept dystrophin production as a surrogate marker that could be used for early approval. But Sarepta’s data on walking distance in boys were taken from a tiny study, and an internal review by FDA staff dismissed the company’s findings out of hand.
As of now, analysts give eteplirsen little chance of being approved when it comes up for a formal decision.
Sources: Reuters; March 10, 2016; and FierceBiotech; March 10, 2016.
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