Actionable news
All posts from Actionable news
Actionable news in SRPT: Sarepta Therapeutics, Inc.,

Sarepta's Eteplirsen Gets Thumbs Down Votes By FDA Advisory Panel

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...