On Monday, an FDA Advisory panel voted down Sarepta Therapeutics (SRPT) Eteplirsen to treat patients with Duchenne Muscular Dystrophy or DMD. The panel noted concerns with the fact that only 12 patients were recruited in the trial. The truth is that proper efficacy can't be concluded just from a small 12 patient trial. 

Biomarin Pharmaceutical (BMRN) ran a 290 patient trial for DMD as well, and they got their drug rejected back In January, this year. That panel concluded that the subsequent efficacy was not enough to warrant approval. Sarepta got shot down because the panel votes were all terrible. Basically, Sarepta got shot down on every single vote that was presented, a total of 7 questions. 

The most important question was the last one which indicated whether or not there was evidence that Eteplirsen's drug worked in DMD patients. On this question the panel voted as follows: 

• 3 panelists voted "Yes" for approval
• 7 panelists voted "No" against approval
• 3 panelists abstained from the question

The vote couldn't have been worse for Sarepta, and now its highly possible patients with DMD won't get the treatment they need. The main doubt most panelists had was why the trial was not done with a placebo compound. I highly believe that if the trial had been done with a placebo could have been a greater success. Instead, Sarepta tried to take the easy way out by only comparing to historical controls. 

There is some slight hope now, because the FDA is set to decide on May 27 whether to approve or not. Even with the FDA chair Janet Woodcock has doubts about the drug, doesn't mean that she and other FDA members will go against the panelists. There have been instances when the FDA goes against the panelists. But the fact that the trial was done with a small sample size, and no placebo, I don't see the FDA going against the panel's recommendation.