From J&J news - Article from MF doubting AA for SRPT but changes mind
Unprecedented -- until it isn't
I've argued that Sarepta Therapeutics Inc (NASDAQ:SRPT) is unlikely to gain approval of its Duchenne muscular dystrophy drug, eteplirsen, because the most recent trial only treated eight patients with the drug. And two of the patients weren't included in the clinical efficacy calculations because they had deteriorated too far.
I know of no drug that has been approved with data from less than 10 patients. Twenty or 30 patients? Sure, the FDA will accept that few if the drug treats an orphan disease where there aren't that many patients. There's no precedence for approving a drug with eight patients that I know of.
Except, now it sounds like this data could be enough for an approval. Maybe. Possibly. Perhaps.