Biotech Values

[ghmm]

BMRN / SPRT:

Appreciate your observations.

I am still going through the documents though from what I've read the tone sounds as if the data is insufficient
1-It seems FDA is not even accepting the smaller studies as positive
2-The lack of supportive secondary endpoints
3-It would seem even if FDA becomes accepting of some efficacy the review may still be delayed as you noted they still want to see other natural history data and it appears some other analysis (risk/benefit) are needed.

While FDA may want 2-3yr RCT and given the limited benefit and patient variability it may certainly be so, however, I highly doubt that either company would be willing to undertake that given the time to enroll and large number of dropouts one would expect and then there is the ethical aspect of basically asking a fairly young age person to take 10% of their life in a trial possibly on placebo!