Biotech Values

[ghmm]

BMRM Adcomm:

I am long BMRN (& SRPT) and I would approve it. That being said I don't think it gets approved with current data and I don't think BMRN does another trial (unless approved in EU and need to). I think the documents and panel (the questions in particular) were almost setup to get a no sentiment.

I actually came away from the presentation thinking the data is not as bad as what I read from the documents. Maybe FDA was trying to be less confrontational but the questions from panel on the smaller studies especially made me think they weren't as negative as the docs made it out to be.

For those that say in any study some will get better some will get worse regardless of treatment the one thing I find a bit harder to sign off on is several families claiming their son would deteriorate during the period drug was suspended and then stabilize after drug was started (sometime afterward). From what I've learned in DMD that is unusual. I wonder if there is some subset of patients that benefit for some (as yet) unknown reason?