FDA questions efficacy of DMD drug

A US Food and Drug Administration (FDA) advisory committee will meet on Thursday to discuss the New Drug Application (NDA) for Translarna (ataluren), submitted by US drugmaker PTC Therapeutics, for the treatment of dystrophinopathies – most notably Duchenne muscular dystrophy – resulting from nonsense mutations in the dystrophin gene.

The FDA has prepared a report for the committee to consider ahead of the meeting, alerting members to the fact that the NDA was filed-over-protest and that the agency has twice refused to file applications for the drug in this indication in the past. The report also refers to a lack of evidence to show the drug’s efficacy.