FDA Delays Decision on Duchenne Muscular Dystrophy Drug

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This is the second extension requested by the FDA.

The FDA will not meet its May 26 deadline for a decision on the Duchenne muscular dystrophy drug eteplirsen, drug manufacturer Sarepta said in a brief statement.

This is the second time that the FDA has delayed a decision on the drug, which is meant to treat Duchenne muscular dystrophy in patients with mutations amendable to exon 51 skipping. Last month, an FDA advisory panel voted 8-5 against the approval of the drug despite the submission of an amendment to previous documents and a very large showing of support from the Duchenne muscular dystrophy community.

The panel has said that the drug does not meet its minimum standards for accelerated approval based on inadequate efficacy. It turned down Biomarin Pharmaceutical’s drisapersen back in November for similar reasons. The NDA was officially denied by the FDA in January. 

“The FDA has communicated that they will continue to work past the PDUFA goal date and strive to complete their work in as timely a manner as possible,” Sarepta said in a statement.