Biotech Values

[ghmm]

SRPT / BMRN:

Appreciate your comments. I would say the gap in our views is diminishing .

I would say the risk in a SRPT approval is increased post briefing docs/panel but without seeing the docs and in particular the questions its hard for me to quantify. I think baring strong pressure from the DMD community the FDA's preferred solution will be to nicely say lets wait and see the results of the confirmatory study (I'd even venture that is one of the questions put to the panel).

Even if I were not long SRPT I am curious to see the briefing docs to see their points of concern (aside from the obvious ones investors expect, small n, dystrophin quantity/measurement, single site, etc.).

While it could be the company (SRPT) is misleading investors I get a much better tone with Ed Kaye and would expect just around the edges differences and not major ones so that leaves me puzzled with:
1-Why did the FDA ask for a historical control if they don't put much value on it (based on briefing docs/panel)
2-Why did FDA even accept package if data were insufficient shouldn't they have not accepted file to begin with? Is it only to satisfy outside pressure and have public review or does pressure go beyond that?
3-To your point about FDA wanting 2-3yr RCT if you check the confirmatory study while the randomized period is 48weeks they do have 96 week measurements so I am guessing it was similar to RNA's agreement with perhaps a bigger compromise and saying we will collect 2 year data. It may actually turn out to help SRPT as I agree that 48 weeks seems hard to show a treatment benefit though we may end up in the same place with comparison to historical control for the second year.