As per the conference call by earlier this week, Chris suggested that the information the FDA was looking for would take a short time to provide. I believe 2-3 weeks. Now, it is believed by most that the company met with the FDA sometime in the middle of March, maybe slightly toward the end. Assuming the FDA minutes came out somewhat shortly thereafter (maybe a few weeks later), the company has for known days/weeks prior to the conference what the FDA wants and therefore has already been working on it. Thus, it could be any day or week that the company is able to provide this additional data. If the FDA is satisfied, allowance of AA could happen much sooner than we think. Most believe the FDA is setting this all up as an NDA filing whereby they will likely approve if SRPT applies for AA. From what i heard on the conference call, the FDA is not really looking for a lot so i guess they were pleased with everything they have thus far. If there were a lot problems, i am sure the fda would have suggested that SRPT provide a lot more data than they are now requesting.
zwerp - why do you get caught up into all this hand-wringing over whether the nod from the FDA on allowing an AA submission will happen in 1 month vs 3 months? If you really listened to the call, you would know that they have to have another meeting with the FDA after filing the additional information requested - his hope was that meeting could happen before the end of Q2, based on his perception that the FDA was being very flexible with their scheduling. Given that statement, why would you start speculating whether an AA "allowance" could happen "much sooner" - does a month or two difference in the timing really change your perception of the value of this company? Besides , it is not clear whether they will have a formal answer until after the CMC meeting, which won't happen until Q3.
Why does it matter if we try to speculate when these meetings, approvals etc may take place? Most on this board has done it for a long time such as speculating when data will be out, when the company met with the fda etc. There are some who believe that based on the conference call, there will be a huge delay. I don;t think that is the case which is why i and others talk about timelines. A lot of people here want to get a sense of what the timeline may be. Then there is the argument of whether the FDA is really looking at this as an NDA submission versus just an application to apply for AA and whether the FDA is basically reviewing the data now as if they will deny or approve immediately. Some argue the fda does not want the company to apply for AA if they aren't going to approve. If they give them the green light, some believe it's approved
I actually think the collection of dystrophin data is not as easy as people are making it out to be. As backwards as it is, the clinical outcome benefit was the easier part. Now the company has to provide data to prove dystrophin as an appropriate surrogate endpoint and linked to the disease. However, the boys are not creating full length dystrophin. Is it going to be harder to collect data on less than fully functional dystrophin as an endpoint?