The Adcom is over and Eteplirsen was voted down. A final decision still remains for approval. Much was discussed in the Adcom, the key arguments were over the following: 1. Adequacy of dystrophin creation. 2. Efficacy of 201/202 Trial vs Historical Controls Both are intertwined. Adequacy of dystrophin creation: FDA had little faith in […]
Sarepta: Is Eteplirsen Dead?
This was originally going to be titled “Ahead of the AdCom” but that no longers seems fitting. The highly anticipated Advisory Committee meeting originally scheduled for Friday January 22 was canceled due to blizzard conditions. Extremely negative reviews in Briefing documents [link] from the FDA are leading many to expect a denial for Eteplirsen’s drug […]
