Nobody can convince you to cover, which is pretty evident by the fact that you decided to make an antagonizing post.
There is no set timetable for AdCom. It usually comes within two months of the PDFUA date, but when is highly variable. Assuming both BMRN and SRPT have their NDAs filed by the FDA, we'll see a dual AdCom for both drisa and etep.
Not entirely true, winter. A panel will most definitely listen to DMD patients and their families. The latest VRTX AdCom proved that. Most of the members voted against efficacy, but overwhelmingly voted in favor of the drug, due to CF's desperate need for a treatment.
What winter said, but I would also add that even if eteplirsen is granted approval, it does not have to be an accelerated approval. Approving a drug prior to a P3 readout does not equal the same thing as an accelerated approval.
Also, nothing is 90% certain with the FDA. That is probably the most ignorant thing I've ever heard, and I'm amazed that people continue to make such assumptions given all that has happened.
Gotcha. It's semantics. While I think the FDA will certainly consider anecdotal evidence, they won't consider it efficacy since it isn't science-based. But I understand your point.
Generally agree with you, but...
1. There will not be any 24-week efficacy data because there is no way to prove efficacy at 24 weeks. That was true of the P2 as well, so I'm not sure why you think that P3 will somehow have efficacy data that early. 36 weeks is needed to show efficacy.
2. Unless the FDA accepts dystrophin as a surrogate marker--and that is very questionable--it would be a regular approval, meaning other exons would still have to go through the same rigorous trials. If dystrophin is accepted, then it could be an accelerated approval and open up the door to quickly approving eteplirsen for other exons.
The trial won't be terminated at 48 weeks. Please be realistic. There's absolutely nothing unethical about continuing the trial beyond that because it is a NON-PLACEBO trial.
You can't point out call buying without also pointing out put buying; otherwise, you aren't being objective. Yahoo had nearly 500% more puts opened yesterday than the daily average. Just saying...
Well then you should prepare to be disappointed. Besides, you don't actually expect that. You're just trying to make a quick 10% on a short sale. Let's be honest here.
Because experts should weigh in. If anything, it'll strengthen eteplirsen's case. You need to at least try to be objective once in awhile.
A completely new treatment for a previously untreatable disease should ALWAYS have an AdCom. Always. No matter how good it is. Let's be reasonable.
Actually, the VRTX news is very positive for SRPT. The panel was so-so at best about the efficacy of VRTX's drug (3 yes, 4 no, 6 can't determine), but it was the patient testimonials that swayed them for a near unanimous recommendation for approval. So, if the FDA files SRPT's NDA, you're going to see eteplirsen receive approval for the very same reason, regardless of its noisy data. The upside of SRPT at today's price is much higher than that of VRTX. But it all comes down to whether or not the FDA files SRPT's NDA. VRTX is a much safer play though.
That strategy clearly hasn't worked, and there comes a point when you need to retreat because of the very fact that it isn't working. Now is one of those times. Doing so is inadvertently hurting etep's chances of faster approval.
Pearsby, you're my favorite weirdo poster on the board! Your posts always make me chuckle.