Replies to post #195527 on Biotech Values

[poorgradstudent]

SRPT:

Admittedly I've enjoyed watching this story and honestly have no idea how to handicap approval prospects. I don't know where the FDA will place their emphasis.

[iwfal]

SRPT

The safety data from on-going studies may be enough to help reduce the risk of approving a drug with what may be modest efficacy.

I doubt they have much safety data from on-going studies since a few months ago they pushed out the end dates by years. And last I knew one exon arm was halted due to safety concerns from an animal study (but haven't checked recently).

BTW - I cannot emphasize enough that Sarepta now, at the last minute, appears to be taking seriously the critiques levied against their data packages. This is a huge step forward - and a huge improvement over what I know they, abetted by the SRPT Moms, were doing previously, which was using the critique to find a way to argue or politic around it. (I stopped posting detailed critique because I would routinely see the political spin on whatever I posted about start within a day or two of my posts - and I find that unethical because it will hurt people). But, of course, it is much too late to fix key items.

(Update) hadn't gotten to the part of their charts that talks about safety data. Although most of the already submitted data is, in fact, from the 12 patients, they also reference that the safety update will include a large number of new patients. Given the lack of progress in their existing trials I think it is a safe bet that much of this is from non-trial participants (I.e. Without controls).