Sarepta Rare Disease Advisory Panel Meeting today, page-2

This statement by Janet Woodcock is interesting "She said “there is agreement that the drug does achieve primary pharmacodynamic effect,” which is increasing a protein needed for muscle production known as dystophin. The amount that it increased that protein was not as much as hoped, at least in the small study, but she acknowledged that there is evidence that even a small amount may help patients." I feel like Woodcock is trying to move her subordinates away from the classical review approach into a new way of thinking about accelerating drugs that are for rare conditions.

If it is safe, then this should be a prime example of erring on the side of a Type 2 error rather than worrying about at Type 1 with such a devastating orphan indication. Problem is, with such a limited sample is it safe? Certainly interesting discourse today with FDA leveling claims of misrepresentation by SRPT.