Biotech Values

[iwfal]

SRPT

I don't think we should be too surprised that the FDA's major sticking point looks like the dystrophin quantitation data

At this instant, yes. But in an AdCom I'll bet 10:1 other items pop up as big as this. E.g. :

1) comparisons to historicals and general natural history data. The company excerpt from the FDA letter notes they need good comparison data from institutions (I.e. They don't have it now)

2) how is the company going to justify the basis of their claim (which appears to be the justification for approval?) that dystrophin maps to function when the two kids who went non-amb had increases in dystrophin.

3) inconsistencies in 6MWD or other measurements. (6MWD being a finicky test to run well)

4) other biomarkers - e.g. missing creatine or missing safety data.

At this point the FDA has probably seen very little of the total package and because of the politics isn't going to be completely blunt about it until they have very crisp data on it. Playing politics isn't good for open dialog.