"Further, Dr. Mendell has stated that the FDA is wrong."
When and what was this stated? After the approval?
They are not the SAME shorts. Shorts cover, and new shorts come in. There may very well be SOME long-term shorts -- but I doubt shorts hold stocks really long-term without a stop loss.
Only the longs here (excluding me) continue to believe in the argument of 10/12 boys walking as a sign of provable clinical benefit. The entire FDA report of what happened over last 4-5 months doesn't even mention that. Neither do they even mention safety.
All they focussed on was: is 0.3% increase in dystrophin reasonably likely to predict clinical benefit?
Is it? No one really knows. Maybe, 0.9% was (due to some evidence of clinical benefit in 202 study). Since the burden of proof lies with the sponsor, I would say --- we should just be plain grateful to JW for her generosity and compassion! I am.
I agree. I wouldn't say that the longs have been right, and the nay-sayers wrong. I for one am happy to admit that I was very wrong in judging the probability of approval. Longs (including me) just got very lucky, perhaps.
Read through the entire report. Its mind boggling. JW vs the REST of the department (DNP + OND + .. you name it).
In effect, Unger (representing DNP + OND) reached the CRL conclusion on July 18th, for very elaborate scientific reasons. You can't blame just Farkas; it was the WHOLE TEAM of reviewers ---- they all in unison concurred to reject the drug.
But, JW concluded differently -- and called the shots. Eventually, she prevailed (I think marginally).
It seems that its a miracle that the drug got approved. In all likelihood, the drug would have been rejected -- and that would have meant that SRPT (and Etep) would have been kaput .... that would have been so unfortunate.
FDA (and biotech business) is TOUGH.
I was WRONG on being so confident (at times I was 99% confident) about the changes of approval. We are just lucky.
Nope, JW is not unscientific --- but her reasons to approve were not entirely scientific either (which is OK -- sometimes you need unscientific common sense to see through the scientific smoke).
Don't be so sure about who was smart/inane. Read the pages 15-17, and you will see (if you have an open and unbiased mind) that JW really went out of the way to approve Etep, and Unger had very valid points (study 301 results were very disappointing). So, count ourself very lucky (or rather, "rescued" by JW's non-scientific passion and/or wisdom)!
Most people didn't bother to find out. Pretty disappointing WB results:
****an increase from 0.22% to 0.32% of normal***
We should all considered ourselves semi-lucky that Etep was approved. Read pg 17-18 of the commissioner's report -- and you will realize that Unger had a valid point, which is:
**Dr. Woodcock never provides a rational argument – based on reliable
data – to support the concept that ‘…low-level increases in dystrophin
production are reasonably likely to predict clinical benefit.’ She provides
no rationale – no link between a mean increase in dystrophin of 3 parts per
thousand and clinical benefit”****
"my favorite part was where unger cited that woodcock issued her approval letter BEFORE he even issued his CRL recommendation on behalf of DNP to her!"
Where did you read that? Where is the FDA COMMISSIONER's report?
No, you need to dig a bit deeper. Yes, the dystrophin measurement techniques and results were all #$%$ up. Beyond that I don't find the company much at fault --- the trial design was fine. Its just that it was NOT supposed to be a pivotal trial, but ended up being that due to seemingly high benefit.
Agency was leaning toward approval, Farkas filed a DPO -- and was ruled over. He felt that his authority had eroded, and that he didn't command sufficient respect among peers. So, he decided to leave.
I think it is unlikely that he was fired -- that wouldn't make sense because: (i) Its not easy to fire someone in a govt job, (ii) Plus, its unfair to fire someone just because he has a difference of opinion (incompetency is non-trivial to prove in this case), (iii) Finally, he had a job even before he left --- so likely he knew he was going to leave beforehand (rather than being abruptly fired; though its also possible that he was given 1-2 months to leave).
#1 is the most likely, of obvious reason.
The key and main part of his data analysis was comparing Etep and EC data matching with age and 6mwt at NON-BASELINE points. This is a very questionable comparison --- and that too without any statistical/numerical backing. This formed the basis of 10-15 figures in the briefing documents.
And, on top of it, he refused to back down from the above analysis ... in BD-2, and possible thereafter.
If I were his boss and had the power to fire him, I would fire him purely on the basis of above.