Sarepta Therapeutics won one of the most dramatic and controversial drug approvals in the history of the FDA a few years ago. It didn’t have the same luck the second time around.

The FDA late Monday rejected golodirsen (Vyondys 53), which was widely expected to become the second approved Duchenne muscular dystrophy drug from Sarepta (NASDAQ: SRPT), following the agency’s 2016 approval of eteplirsen (Exondys 51).

In a statement, Cambridge, MA-based Sarepta said that the agency has two concerns with golodirsen. One is the risk of infections from the “ports” used to infuse golodirsen into patients. The second is… Read more »