25 May 2016 - The agency again delays a decision on a therapy for a deadly disease.

Boys with lethal Duchenne muscular dystrophy have waited years for the FDA to approve a safe and innovative treatment, and they’ll have to hold out longer. On Wednesday the FDA delayed a decision on eteplirsen by Sarepta Therapeutics, but the agency can still choose the correct scientific and legal outcome.

Sarepta said FDA had notified the company that a decision would not be issued on Thursday as scheduled. The agency offered no clues beyond promising to finish “internal discussions” and the broader review “in as timely a manner as possible.” An FDA advisory panel voted against approval in April, though agency bosses can accept or disregard the input.

The press has spun this story as hysterical parents mowing over scientific standards, but eteplirsen deserves approval on all the evidence. A clinical trial treated 12 boys; 10 still walk after four years, unlike one of 11 in a control group. FDA says the trial is too small or maybe something bugged the results, but more than 35 Duchenne experts dismantled the agency’s claims in a February letter and at the April meeting.

For more details, go to: http://www.wsj.com/articles/muscular-dystrophy-day-at-the-fda-1464219520