Door not closed yet for DMD drug, despite a 'no' from FDA panel

A US Food and Drug Administration (FDA) Advisory Panel voted against approving PTC Therapeutics’ Translarna (ataluren) as a treatment of nonsense mutation Duchenne muscular dystrophy on Thursday.

Despite this vote, the committee’s call for more efficacy did not entirely shut the door on the drug’s approval hopes, and since the meeting, PTC’s share price has enjoyed a double-digit percentage increase. Previously the FDA has approved drugs for this rare disease despite a panel voting against them.