This is all good stuff but why is Janet waiting this long to approve the drug? Are there other forces standing between her and AA at FDA? This turned out to be such a big drama for everybody combined (shorts/longs, patients, kids, physicians).
It is the same as a CRL. If FDA grants compassionate use who will pay the cost of the drug? Most parent can't afford it. According to FDA insurance companies won't pay.
Directly from FDA website
"Investigational drugs are expensive to make. Remember that most insurance companies will not pay for access to an investigational drug and that there may be additional cost for administering them and then for monitoring of the IND by your hospital or healthcare provider. It is important that you and your physician consider the cost of the investigational drug and the medical services associated with its use that are not covered by third-party payers such as insurance or Medicare."
This is giving a death sentence to kids and SRPT together. I hope this is not in FDA's agenda.
It does not really matter what FDA says as a disclaimer. This is a safe drug targeting an unmet need with a reasonably likely clinical benefit. The drug will sell itself. Whatever FDA says will not save shorts.
Yes I do. The day before the stock was down more than 5% if my memory does not fail me. Then the news came and it shoot up to $45. I sold at $41 back then. It was my biggest ever gain both in my 401K and trading accounts. Now I hold 9400 shares. 2/3 in my 401K and 1/3 in my trading account. I don't think today's action is a concern at all. They may even take this down further to take all stop losses.
According to a publication I found on the web this is what a medical writer does:
"Medical writing involves writing scientific documents of different types which include regulatory and research-related documents, disease or drug-related educational and promotional literature, publication articles like journal manuscripts and abstracts, content for healthcare websites, health-related magazines or news articles."
Out of all these tasks the most relevant to SRPT would be "disease or drug-related educational and promotional literature". Why would they hire a medical writer when they don't have any drug in the pipeline other than Eteplirsen? If they knew Etep will be rejected they would not hire anybody. They know very well that they will be facing financial challenges in the case of CRL. If it was a CRL they would have to lay off people not hire.
IMO they got a signal from FDA on Friday or late Thursday. This could in the form an email exchange or FDA visiting manufacturing facilities or etc. Let's see what early next week brings.
I don't think today's price action has anything to do with Zika. SRPT is all about DMD now. I am thinking some positive developments might have happened. As Bionerd mentioned a few days ago It could be FDA visiting SRPT manufacturing facilities before they give AA. Call it wishful thinking ...
I think if the question was worded differently there would be more than two people who voted yes. However, this attempt by him won't do anything. ADCOM members will ignore him. He is a portfolio manager after all. What would have been really useful if Christine and other parents did the same plea (not through public media) but by calling or emailing them and asking their vote for a differently worded question. Most of them may not respond but a few that responds would create big publicity and extra pressure on FDA. After what they have done for their kids I don't understand why they don't do this.
Isn't this amazing? The more I hear about how kids are doing the more proud I am being a SRPT investor.
"Just ended Billy's annual IEP meeting....PT & OT are on monthly consult/observation basis only. PT & OT plus supervisor said they have not observed any decline whatsoever and even noted minor improvement.....Billy is 15 1/2 and will enter sophomore year next school year. #FDA $SRPT #Eteplirsen is the reason"
PT on Mobility--"walks independently on level and (uneven surfaces).."he opens/closes doors"...$SRPT #FDA Billy 15.5
"Balances on one foot to kick a small rolling ball" School PT reporting at annual IEP $SRPT #Etep #Fda Billy is 15.5
We have heard from several kids on the ongoing confirmatory trials in the ADCOM that they are benefiting from the drug. What other information are they referring to as interim data when we know the trials will not be completed for another year or two.
FDA bias is very important. Farkas and Hastings tell you only one side of the story. FDA agreed on the historical control. Farkas and Hastings are liars!!! They repeatedly said in the ADCOM that company's statements were misleading when in fact they were the ones misleading the people. They orchestrated the entire ADCOM so that a unanimous negative vote will come out of it. If FDA senior management has some respect for themselves they should fire these people. Never in the history of FDA ADCOMs I have seen so stupid voting questions worded the way they were so that there is no room left for a positive vote. If FDA gives a CRL, as an investor I will be in for suing FDA for not applying the same standards for all drug companies. It will be a class-action suit by patients, investors, and other stakeholders. Let FDA decide what to do.
There were several kids from ongoing confirmatory trials testified in the ADCOM not just one. Their clinicians also testified that the drug works for most if not all. This is no longer an issue of whether the drug works or not imo.
Forbes article is good but if I was approached by somebody who is a portfolio manager and was asked how my answer would change if the question was worded differently I would simply ignore him. However, if patient advocacy groups approached I would take them more seriously. Now is the time for the patient advocacy groups to approach these to doctors (and possibly all other in the panel) and ask them how their vote would change if they were to vote for the following question?
"Has the applicant provided substantial evidence that eteplirsen induces production of dystrophin to a level that is reasonably likely to predict clinical benefit?"
If they publicly say they would vote yes this will put extra pressue on FDA toward AA. I hope Christine or somebody else can see my email? I don't use twitter.
Did not they have enough time already? 4 years + 3 months extension and now they are waiting until the last minute to render a decision when they know very well that every day matters for these kids. I can't believe that these irresponsible people are ruling FDA.
They get honorarium fees plus all their expenses are paid. I don't know about FDA for NSF and NIH the honorarium fee for a day is around $500. I would assume for FDA to be higher.
The problem is with the institutions. They loan their shares to short sellers. Blackrock does this. Vanguard and Fidelity might also do from time to time.
In the unexpected event that SRPT receives a CRL (I give 10% chances for that) the stock may take a hit but it will recover as soon as other alternatives (buy-out, partnership etc.) becomes available. IMO this is the best risk/reward I have seen for a very very long time.
I found this information on the Jett Foundation's blog. Very interesting ... Apologies if this was already shared.
Further Clarification by Dr. Kunkel after his testimony at ADCOM:
I suspect many (in FDA, on the panel, and at AdComm) did not get my point. In the 1988 NEJM paper Eric (Hoffman) showed that most DMD patients made no detectable dystrophin on western blots as predicted by our proposed reading frame hypothesis. The reviewers asked for an estimate of his level of detection. He (Eric) did a dilution series with normal muscle and felt at the time that he was not confident calling dystrophin levels below 3% of normal. So the field has interpreted 3% as the level below which you are DMD.
The real way to look at this in that paper is that dystrophin levels are not detectable in biopsies of DMD at a sensitivity of 3% of normal and that was using 1988 western blotting technology. Today’s technology is 10 times more sensitive consistent with Sarepta’s 0.2% of normal sensitivity. In their data on untreated biopsy samples many of the DMD patients were below that threshold of 0.2%.(1988’s 3% that was detectable.)
These facts are why I am confident that eteplersin is skipping exon 51 and more protein than would be expected for Duchenne patients is being made. Remember our reading frame hypothesis predicts that they should be making no full length dystrophin. Hope this helps.
Yes she voted "Yes" for question 2, AA question but if you consider the level of expertise she has in DMD compared to the experts testified in ADCOM it is still nothing IMO. It is mind boggling how somebody can vote "No" after listening to the experts. This SRPT ADCOM would not be forgotten for years to come believe me. It was historical on every aspect.