SRPT:
I'm thinking this drug gets approved. Too many concessions are being made by the FDA.
- Company runs trial that is clearly not sufficient for AA.
- Company doesn't have a complete sample set from said trial and therefore can't do a proper analysis of their surrogate
- To make case for AA, company dips into samples from an ongoing trial to serve as controls for the completed trial (also acknowledging, at the time, that 3 patient samples from ongoing trial had already been practically used up and therefore unavailable for analysis)
- Now company advised to provide surrogate analysis from 13 before / after samples from the currently running trial in order to satisfy AA
Basically the FDA was not able to conclude anything from the studies submitted for AA... yet the door is still open for approval.
From a biotech investment standpoint, hopefully investors don't take the wrong lesson. This isn't how the drug approval process works, and if they bet on similar treatment for other companies, their SRPT profits will disappear pretty quickly.
