Ya right. The FDA was hoping they could bungle DMD completely, and the parents would just say, "that's fine. Do what you need to do while my son needlessly rots away. Go ahead and prevent Eteplirsen from moving forward because of the shortcomings of a completely different (2-ome) chemistry. Use flawed statistics, and blatantly false data, in order to show Sarepta who's boss. We don't mind if our son loses his ability to walk, or feed himself, as long as the FDA is happy". Give us all a break Starfe. You are a #$%$ idiot.
We are one month past the PDUFA date. It is reasonable to assume that the FDA communicated with SRPT on or prior to the PDUFA data. WB's take three weeks max to analyze. Thus, the quiet period appears to have begun right about the time the company should have had in it's possession the new WB data. I agree with you, that the new WB data is 100% "material". i don't see how any lawyer could argue or accept otherwise. The real question now becomes, how much additional time will the FDA waste this time? They've already wasted an incredible amount of time with their Eteplirsen charade. It would be nice, if for once, they could render a decision in a timely manner. Hope springs eternal.
Leave it to you to not see the forest because the trees are in your way. Those numbers all imply far more dystrophin is being made than .9%. You'd rather focus on the fact that these data conflict with what Dr. Kunkel said. Too funny.
Have you ever considered anti anxiety meds? I think they might do you a world of good. Maybe a little dash of lithium to dull the mania too? You might even enjoy the fact that your thesis is complete garbage if you tweak the drugs just right. Just a friendly suggestion.
Hey, that's awesome! It must mean that the wounds left on the poor kids from receiving Disastersen have finally healed. Great to hear. See what NOT having poison in your body can do for a kid?
While I do not remember that quote by Dr. Woodcock, I'm going to assume you are correct when you quoted her. Even so, she also stated:
""there is agreement that the drug does achieve it's primary pharmacodynamic effect,"
Thus, Dr. Woodcock and the FDA are faced with possibly saying "no" to a drug that achieves it's intended effect but produces less dystrophin than hoped for because .9% might not be enough. That decision, if made, would be protecting these dying children from "some" dystrophin in favor of them enjoying the full brunt of their untreated , relentless disease and zero dystrophin
Ew. That's just ugly. I can't see that decision happening unless the WB's come back with zip for dystrophin and I find that unlikely.
While you're correct about a "flexible and proactive FDA" that "could have"...................
I doubt anyone familiar the the DNP has ever entertained the idea of using flexible and proactive in a sentence where that disaster of a department is concerned. No matter the outcome here, the DNP should be dismantled and rebuilt into an entity that is far more beneficial to the American society. There is a VERY good reason, that the DNP is the worst rated division within the FDA, and their ugly performance at Sarepta's Adcom did absolutely nothing to change things.
"By the most reliable Western blot methods, dystrophin less than ≈3% of normal muscle appears to be associated with the typical DMD phenotype."
If Dr. Woodcock truly believed those words, there is absolutely no reason why she would then request P3 WB data which was not part of the Eteplirsen NDA package. She would simply issue a CRL, and ask them to re-apply once the P3 data is in hand. There is also no reason to expect a jump from .9% WB to 3 %, though it isn't impossible.
The 3% figure also is in direct conflict with the vast majority of DMD experts world wide. Furthermore, in humans, the truth is, nobody knows exactly how much dystrohpin is required to impart a benefit. Certainly, some dystrophin is far better than none. DMD experts worldwide resoundingly endorse that concept. It appears as though the FDA may be getting ready to agree.
Copp, I see your biorhythms are back in sync. Good for you! You've really got it going on today. The way you add to the conversation here is incredible. Thanks for taking the time to share yet another of your pearls here.
His lack of leadership and follow through have destroyed 90% over last couple years. How long before his "overpromise, always deliver late" management style crushes what little is left?
Copp, your biorhythms are seriously out of kilter. But, that is not my fault. I laughed out loud though so thanks. Of all the posters here, accusing me of keeping people "in the dark" only shows what a tool you really are. I've done nothing but share good DD for years and during that time, you've been an angry, obstreperous POS who adds only his sour vitriol to the mix. Pardon me for pointing out that posting what Jeff did without crediting the true author is not cool. If you take enough time to copy someone else's efforts, you ought to also be able to credit them rather than making it seem as though it's your own work. And Copp.......get those biorhythms looked at before you become even angrier for your own shortcomings. That's not healthy.
" it makes me wonder what her motives" ...........................Uh, you don't have to wonder. She was CLEARLY paid to misinterpret the truth. Anyone who knows even half of the truth here will immediately know beyond a shadow of a doubt that she doesn't know what she is talking about. It's funny, that the huge short contingent, believes that they can influence the share price by posting complete garbage here, or in publications like hers. I question what impact, distorting the truth like she has done, actually has. That said, the shorts seem more than willing to continue to pay folks to post ridiculous garbage like Molchan has published.
They may in fact grant approval, but it sure as hell won't be accelerated. The FDA should have simply clearly stated that Sarepta needed to start an FDA sanctioned P3 immediately after the remarkable P2 , 48 week data. Instead, they began a game of charade, and refused to provide critical P3 design guidance, effectively stalling Eteplirsen for 2 years. Then, they found ways to stall every single step forward. What the FDA ought to do is publicly admit that they have created a brand new approval process - Decelerated Approval, where a company can be forced to wait several needless years before possibly staggering across the approval finish line.
If you're going to cut and past everything, you should include credits to the author as well. Saying it's "on Twitter" is kinda lame.
3rd Q? The FDA probably has the D data now. They shouldn't require another 3 months to announce AA. They've had so much extra time here, that a couple weeks ought to be adequate. But Q3? That's just wrong