Please reply to this message with the % probability that SRPT will get Accelerated Approval by the FDA?
My vote is 55% chance they will get it, so just over 50-50.
My vote is 100% certainty; Here is a quote from Commissioner Peggy Hamburg which shows that SRPT will get a "green light" to file and will be approved even though sample size is small. The commisioner does not seem to be worried about studies that have an "extremely small patient population[ ]." You can find it on the FDA web-page.
Moving New Drugs from Discovery to Delivery
Remarks as Delivered by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
November 10, 2011
"We’ve been working on novel ways to expedite the development and delivery of new drugs. Our drug approval pathways now include Fast Track…Accelerated Approval… Priority Review…and Expanded Access programs. These programs are designed to speed the testing, availability, and approval of drugs in different ways. "
"We’ve also been showing increased flexibility when it comes to clinical trials. Adaptive clinical trial designs like the I-SPY study is one great example. And again, if you look at the list of new drug approvals just released, you can see clinical requirements were streamlined to permit smaller, shorter or fewer studies wherever possible. Many have been surprised to see the number of drugs approved on the basis of single arm studies or using studies with extremely small patient populations."
20% chance FDA suggests a better path than AA - no confirmatory trial!
- there would most likely be requirement to monitor those on drug
50% chance FDA suggests AA
30% chance FDA says forget it, do the phase 3 first
Here is another quote from a speech given by Commissioner Hamburg that bolsters my confidence. She is speaking about her vision of and the importance of a "fully functioning FDA that earns the confidence and support of people who are suffering. This is the same type of transparency that Drs. Margaret Hamburg, Janet Woodcock and Robert Temple have participated in during 2011-12 when they made relevant speeches and in 2013 when they met with the families of children with Duchenne.
The FDA at the Crossroads
Remarks at the Consensus Science Conference as Delivered by Margaret A. Hamburg, M.D.
Commissioner of Food and Drugs
November 15, 2011
"History has shown that both consumers and industry have a tremendous stake in an agency that takes science-based action on behalf of public health…an agency that not only takes action to keep Americans safe and healthy—but also promotes the science, and standards, that keep the engines of innovation running.
With this power, of course, comes great responsibility. And we have a responsibility to take action that is transparent and can inspire trust in the public whose health we are trying to promote and protect.
Unfortunately, public confidence in my agency has taken hits in the last decade. And one thing that I learned early on is that when people do not understand FDA or its actions, they often assume the worst. Only by really explaining what we do, and why, can we foster that trust, even in times of conflict or controversy.
So I have been working with my colleagues to promote a culture of even greater openness at the FDA. We are creating an agency that takes its own leadership role seriously while also trying to listen to all of our relevant stakeholders."
You have our federal overlords who only get criticized if they allow excess risk. What do you think their choice is going to be - the prospects of the kids don't even enter the 'regulatory analysis'.
30% chance of AA with a decline to the high teens followed by a relatively quick recovery to mid twenties if AA is not granted.
I am a subtantial long but plan a partial exit by mid April.
I'm going with 25%. I am long and hope I am wrong - especially for the kids - but not expecting it at this point. Think the drug works and will save a lot of lives. So I really hope I am wrong. Either way I am buying more after we find out whether AA or not. Barring AA and a stock price over $75, I am planning on adding more post-news of the timeline for approval.
There's a lot of people in the same camp as you. The drug works but the FDA won't approve it yet. Think about this for a minute. If the FDA feels the same way (which I believe they will), the new regulations make it much easier to approve a drug that everyone know works well. We don't need Phase 3 trials and hundreds of patients in a trial for approval if you know the drug worksh. The FDA would be doing the public a huge disservice if they knew a drug worked and they didn't approve it because it didn't fit in a nice box of a large Phase 3 trial.