If she is skeptical on the science of Etep, Janet Woodcock would avoid the close relationship with the DMD boys, parents and advocacy groups. Why get you the political baggage? No one will blame her is she keeps a distance from the DMD parents, boys and ad groups. She chose to, indicating the she is comfortable with the Etep science. At most she is concerned the formality of a confirmatory Phase III. At the end of the day, it is the science that counts.
It will make her look bad that she kisses the baby then turn around rejects accelerated approval without finishing a Phase III. She's been in FDA for a long long time. She is experienced and smart, and knows the implications.
The scary thing for shorts: Etep data good enough to give FDA excuse for AA approval. FDA can would, and should do what the top brass want to do: Janet Woodcock and other top dogs in FDA are getting personal with the DMD kids. Considering the toxicity profile, non-reversible nature of the disease and lack of any treatment, approving Etep without finishing phase III will get the FDA no heat but rave praise.
FDA is a political animal too. Think about it, what the FDA may do:
1. Approving Etep, no political risk or scientific risk. a. If it works in the confirming Phase III, they become heroes. b. If it does not work in the confirming Phase III, they still get political risk because the situation of the DMD boys, and the actions of the US congress.
2. Requiring a Phase III to be finished before approving Etep, tons of political and social heat and Marg Hamburg will get congressional pressure right now. a. If it works in the confirming Phase III trial, some in FDA might be pressured to resign. b. If it does not work in the confirming Phase III trial, no one wins anyway because FDA cannot parade itself of denying the Accelerated Approval. Even many shorts do not argue that Etep does not work, but arguing the Accelerated Approval damage FDA integrity and independence from political pressure.
Sentiment: Strong Buy
I still hold this opinion today. The 1st SA article today is trash. The 2nd SA article is not trash, written by a scientist with some valid points, but his interpretation of data is wrong because he wrongfully determined that Drisa does not work.
In fact, Drisa works in the phase II and would have worked in the phase III is properly conducted. Drisa failed phase III due to poor trial management. It involved patients from too many countries, including some eastern European countries widely known to host messed-up clinical trials.
That said, even if Drisa succeeded in a phase III, it is too toxic to be used in young boys for more than 10 years, thus would not likely to get approved.
Even if Drisa is approved, Etep will destroy its sales for its superior safety profile.
I would like to see Janet Woodcock FIRED.
Condescending arrogance to bereaved parents is justification enough.
THEN,... FDA officials would start to listen.
But probably not until we can bring down one of them.
IMO its very difficult to make a case the FDA are bad guys to this point. They were on board for the P2 study, they have told the advocates they would review this expeditiously, and they invited a NDA filing. Some have found fault with the 4th biopsy request, but it may shed light on a lot of science needed for the other exons, if the parents agree( not that it isn't a lot to ask). The drug is not available yet so even if they approved it today many kids would wait months to get it. I think we need to see how it plays out before we start pointing fault. It is my opinion etep will get AA soon (after NDA is filed) and maybe before the confirm starts. I also believe when the drug is available it will be approved. The advocates have stated they believe the FDA is listening and CG has stated they have been very cooperative. Give them a chance to do whats obviously the right thing to do before we criticize them.
At the end, it's not the science, it's the EVIDENCE/Clinical Validation that counts!! Drug development towards marketing approval is totally different beast from fundamental science. This is the major problem with biotech executives to not able to distinguish between the two. Your 12 patient single center data, based on yet to be validated endpoint and not on clinical end point, is just an exploratory study. We need evidence from a phase III pivotal trial...
The drama won't cut it with the FDA. Cut your BS. NO AA approval. Don't count on it.
The 6met is a validated endpoint. Dystrophin expression is not as yet (will be accepted soon for class approval). Both 6mwt and expression met endpoints. Both the validated clinical endpoint AND the surrogate were stat sig.
You seem to be fixated on something you don't understand. Please before you cost yourself more read the new PDUFA V. The regs are quite clear on rare disease approvals. This is basic regulatory stuff that you should be aware of but seem to be in denial.
You are going to lose your bet here, maybe you can still save something by waking up now. Likely you go down with the ship. How fun to see a hack like you scream bloody ignorant murder.
And you Mr. regulatoryexpert along with the FDA and your delays will be responsible for the death of +/- 75-100 children for every 12 months it gets delayed. You call it "drama" they call it life and death.
Who is the 'WE' who need evidence from a phase III trial? The Accelerated Approval process is used by the FDA. The drug is approved and a confirmatory trial is run. This was done before, Eteplirsen would not be the first drug approved through AA. The data speaks for itself: 100% RT-PCR positive, 100% showing consistent % dystrophin positive fibers, 100% stable in 6mwt after 2+ years on the drug, 100% showing improvements (more energy, less toe walking, better performance in school etc). All this with NO side effects. NO clinical trial for a DMD drug came even remotely close to Eteplirsen's results and there is NO treatment approved for Duchenne.
So cut the #$%$. You don't like it, then take it with the FDA.