were made by the FDA based on Drisa data that they Haven't even SEEN! They will see that data before the next meeting.
The 4th biopsy hasn't taken place yet and now we know why they wanted it.Proof of steady dystrophin production now would go a long ways to change their thinking. All the researchers and experts are on SRPT 's side on this one. The dystrophin data will hold up after further review.
WE know what they were thinking before this meeting but not what they are thinking now or what they will be thinking after SRPT states their case at the next meeting.
The advocates are not out of this by any means, and they are sick and furious.
The fact they are making decisions about Etep based on drisa data is in fact illogical and I am sure this will be pointed out to them.
FDA's two main reasons for this mind change are both based on poor comparisons and CG and ED Kaye I'am sure will and have already begun to educate them on that.
The PR after the next meeting and everything up till then will be very interesting this game is not over.
thig - I agree in concept that the 4th biopsy COULD be a game changer, but when the FDA has punted on what qualifies as a valid measurement of dystrophin, how do you proceed? The FDA hasn't backed
SRPT into a corner - they've kicked them off the playing field - no rules to follow and no idea when the rules will be decided upon. As you pointed out, they haven't even seen the full data set from the drisa trial, yet they have made the assessment that the data raises doubts about SRPT's trial and data, which they haven't seen all of, either. While the AA regs are supposed to provide them the flexibility to think outside the box, they have shown they don't have the intelligence to handle that flexibility - they are stuck in the same mental sandbox that ru and pasteur have been spouting for the past year. I would like to think that if by this time next year all 10 boys are still stable/thriving on Etep that it might change the mind set, but based on how they handled things the past month, it isn't anything you can count on?
FDA is looking really stupid on this one. I mean they give drisapersen breakthrough therapy status and now they don't have a clue on how to proceed with the drug that works?!? And then they propose some crazy schemes for combining endpoints that have no proven credibility whatsoever?!?
SRPT has to get the agency in the same room with some of the thought leaders in DMD research and tell these yahoos that a large, placebo controlled study is fantasyland. You can't find enough kids in the appropriate age range. Europe is out because of the patent issue. Subjecting kids to multiple muscle biopsies under general anesthesia is unethical in the extreme and delaying hope for these kids is criminal.
The patient advocates are going to march on FDA with pitchforks and burning torches like its Frankenstein's castle if these idiots, the FDA decision makers, don't face reality and realize that you can't design the perfect biology experiment in this case. Congress ain't gonna like this BS either. This can't be the way this is going down. Either they clear the snot out of their craniums and go back to the July game plan and/or they agree to a confirmatory trial based pretty much on the same lines as previously outlined
Sentiment: Strong Buy
Just added SRP today at 13 but have been following the stock for over a year...
Today I am indeed sorry for long time holders, but the stock traded at 55 this year for goodness sakes, so the risk should have been fairly obvious what could happen if anything negative occurred....and it did. So that event is now water under the bridge.
As for FDA approval. If the 10 boys who received dystrophin are still doing great, why not show the FDA and all the physicians out there that 250 boys and girls can do great on the drug too. Or 2,000 for that matter.
That is what clinical trials are all about. You want a cancer drug approved with only 10 patients?
How about a major heart drug? 10 patients= approval.
Come on gang. Dry those tears and move on. SRPT needs a partner to fund and manage the Phase III trials
and they should be able to get one. It's not the drug that is at fault here. It's management. Nobody in their right mind should think they could get a drug approved by the FDA with 10 subjects. Crazy.
Hey ignorant, check Ariad drug for cancer. Their AA approval based on greater than 400 patients exposure and yet it was proven toxic in real life i.e. real use in market place. 12 pateints exploratory single center trial????? Come on, give us a break....FDA knows better!
hey mr. pumper and mr. fraud thigrlsk, game over!!!
There is no 4th biopsy!!! stop playing games!
No comparison between Drisa Vs Etep. Drissa was solid with their greater than 50 patients proof of concept p2 data which justified to do a phase III pivotal study but unfortunately failed!
They accepted the failure with dignity and class. Also proved that this class of drugs Drissa, etep may not be the right drugs to treat DMD.
Etep with 12 patient single center data is a fake, false positive data......no big company will invest in phase III....Game over!!! SRPT is a wolf in sheep skin! Check the past of this company AVi or whatever they called themselves of.....
Hey RegulatoryFake you have no business calling anyone a fraud when you are the biggest of them all. You were the one that called the DMD boys fake. You have lost total credibility on this board after that. Now take your little horror show somewhere else...like hell. lmao at you Fraud!!!! This isn't over in any sense of your imagination.
Sentiment: Strong Buy