US panel says Sarepta’s trial DMD drug not proven effective

pharmafile | April 26, 2016 | News story | Manufacturing and Production, Research and Development DMD, US FDA, drug trial, sarepta 

A US advisory committee has concluded Sarepta Therapeutics’ (Nasdaq: SRPT) trial drug to treat muscle wasting condition has not proved effective.

The experimental drug for Duchenne muscular dystrophy, a degenerative disorder mostly affecting boys, failed to gain favour for a standard accelerated approval with a 7-3 vote. The committee indicated the trials in 12 patients did not provide substantial evidence of efficacy for patients with a specific genetic mutation.

Edward Kaye, Sarepta’s interim chief executive said: “We appreciated the opportunity to present our data to the advisory committee panel and will continue to work with FDA as they complete their review of the eteplirsen NDA.” 

The US Food and Drug Administration is not obliged to follow the advice of its advisory panels but does rely on its committees for guidance.

The FDA is set to announce a final decision on the drug application by May 26.

The closely watched meeting evaluated the drug Eteplirsen, which produces the missing protein dystrophin in DMD patients.

The disorder affects one in 3,500-5,000 boys worldwide. The patients lose the ability to walk by age 12. Progressive muscle weakness in the lower limbs spreads to the arms, neck and other areas. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The condition is universally fatal, and death usually occurs before the age of 30.

Sarepta’s drug is targeted at treating about 13% percent of these patients with a specific gene mutation.

The FDA committee, however, raised questions if Sarepta’s clinical trial pool, with only 12 patients, was sufficient to showcase any significant results.

Anjali Shukla

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