I don't think Mendell necessarily communicated the wrong %, I think what was communicated was not western blot, it was the other method. I don't think there was any shady business, I just don't think the FDA understood exactly what was being told to them. I'm sure someone here can provide more info though.
Wording of the questions, that's it. If it said do you believe dystrophin is being produced and it's benefiting the patient. It would have been overwhelmingly positive. All the questions had their own little specific piece that never gave the vote a chance of positive approval.
While I do think approval is less that 50% at this given time, maybe it's the perfect situation for the FDA to gain some compassion points. I mean the FDA has already beat SRPT in all scoring aspects of this so called "game". Think of what the headlines would be if FDA approved even after all that's taken place. There might be some negative articles, but for the most part I think they would receive a warm applause and some great PR. FDA has already publicly called out SRPT's major failures as a company, sent a shot across the bow of all future companies trying to gain accelerated approval, and has come out of this as looking like the FDA who puts ego ahead of children. If they approve it's a major Win in all categories for them. SRPT will still be the screw ups, the kids get the drug, and the FDA is not longer seen as "against the people". Seriously though, it's a perfect ending for the FDA if you think about it.
I'd be ok with that if it means this drug get's approved and patients of other orphan diseases can get access to drugs quicker and at more affordable rates. I don't need the FDA determining whether or not I have the right to buy "snake oil".
I get the purpose of the FDA, I really do, but common sense needs to be implemented in the regulatory process. Not everything is black and white.
Good point, like when the DMD experts discuss why this drug is working, will it just go over their heads? It needs to be known that the DMD clinicians etc are the experts, not the FDA.
I find it ridiculous that the entire adcom can be based on how 1 group words the questions. Shouldn't both parties be allowed to submit questions, or maybe the panel creates the questions. Not the FDA? It's bogus.
What is the likelihood that the questions can be changed or additional questions added the day of the conference?
I find it encouraging that questions were included, but at the same I find it discouraging that all the questions seem specifically driven to what the FDA considers inadequate. I'm concerned that that the FDA is going to say "we included questions and the panel voted no, so we've done our due diligence in providing all options for this drug to be approved." yet the questions suck and in my opinion are extremely biased. At the same time, panel members are going to have to vote on the exact language of the questions so even if they think their is benefit and the drug should be approved, they won't be allowed to support that belief.
Any thoughts from others on this?
I agree, the question handcuffs the panel. Clearly the FDA doesn't think their was a well controlled trial, and honestly I think most people would say the trial has major flaws for regulatory approval. The panel just needs to be convinced that the trial data does shoe efficacy and that the historical group is a valid control. I just find it so lame how the FDA worded the questions.
While I agree, their clarification on "well controlled study" make it much harder for the panel to vote yes. I think SIMP outlined this in one of his posts.
This whole process is freaking lame. Just have a question that says, does this drug show benefit in treating DMD. Does the benefit outweigh the risk. Would you approve this drug. Ok done.
While I agree with your statement, I still don't necessarily believe that ETEP would have been rejected based on the first set of BD's. BD's aren't the say all be all, as ridiculous as they were I still think ETEP was going to get approved by the higher ups at the FDA. As horrible as it's been, I think a lot of this has been show in order for the FDA to de-risk the liability of approving and say they did all they could to ensure the drug was safe and effective. We shall see.
Can't, I'm long, like I already stated. How is what I posted not a possibility? For how many times you've been wrong on this stock I suggest taking a more humble approach on your commenting.
Anyone else worried that the snow delay has allowed the FDA enough time to even strengthen the argument against SRPT? I know the first BD's were a joke, but maybe this extra time has allowed the FDA to dedicate significant resources in proving ETEP's lack of efficacy.
I believe in SRPT and the data, but I'm taking a devil's advocate approach and the fact that the FDA has screwed the company and kids during this whole process, I can't help but think of this as a potential outcome. Logic tells me no, but logic left the building a few years ago. FYI - not soft bashing here, check my history.
This is great and all, but hasn't our local FDA spokesman already confirmed that we're getting a CRL for exon 51 and 45/53 will never start because of pre-clinical issues? I don't see any reason to follow SRPT now that he's relayed the FDA message to us.
Couldn't the same be said about every drug that's been developed/approved. I mean there's always the possibility of an AE, even for drugs with a pristine safety profile. Could you imagine if drugs were never approved based on the possibility of something happening down the line, even though all existing data suggests otherwise.
Mars I get that you're just stating the FDA's position and I really appreciate all your posts because they're always factual, unfortunately the FDA's facts frustrate the hell out of me. I just wish the FDA would use common sense once and a while, this fiasco has become really sad.