Biotech Values

[ghmm]

SRPT:

I took it as there is still a lot to know about the disease. While I would agree that sponsors tend to state things in their favor outside of trial issues the FDA arguments were more open ended (e.g. how much dystrophin is meaningful, what clinical endpoints may be most appropriate). While SRPT could be faulted for many things I thought in a disease with a lot of heterogeneity FDA wanting to do comparisons by age (vs. other base line characteristics) may not be the most accurate comparison.

The ITMN panel was the first one I saw at the time I took the FDA's doubt on FVC as indicative it was not a meaningful prognostic indicator on outcome. Added to that ITMN failed to get an SPA at the time I remember Dan Welch saying something to the effect that sometimes the agency is a little slow to catch up when a disease has no treatment. I actually didn't believe him at the time. In retrospect I think he may have been being quite fair in his assessment and certainly in with the ASCEND results they were able to correlate FVC decline with mortality.