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Sarepta Therapeutics, Inc. Message Board

  • simp08801 simp08801 Feb 18, 2013 8:51 AM Flag

    what is going on is in front of you

    "CureDuchenne has a sent a letter to the FDA on behalf of the Duchenne community requesting attention to the risk tolerance that patients and parents have for this fatal disease"

    Fist the November 2012 Temple presentation on BT and AA

    then the February 2013 Woodcock presentation which also addressed BT and AA

    then woodcock and temple privately meet with certain duchnene activists

    now temple will give a webinar on february 20 for duchenne community through MDA, CD and PPMD, on "accelerated pathways" to FDA approval

    much talk here about dr rusty katz at the fda

    katz reports to temple, and temple reports to woodcock (she is #2 at FDA)

    you could write a list from here to Pluto on why the FDA senior officials would want to be in hiding on this issue, publicly, at this precise time

    but instead, it appears the FDA is unusually out in the public on this

    WHY?

    I think the FDA is fact finding on risk awareness and risk tolerance in the duchenne community

    and that is why I believe the above quote was in the CureDuchenne press release announcing the temple webinar

    I believe risk awareness/tolerance will be a key issue when woodcock and temple deal with Srpt

    I would bet Vegas money on an Srpt AA app being filed here

    tea leaves

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    • simp - it is good to see that you are coming around to the common sense issues that will drive the AA decision. The punits/"analysts" that raise issues with the trial size and uncertain connection between dystrophin production levels and clinical effectiveness (as demonstrated by the 6MWT) seem to forget what the accelerated approval regulations were designed for - i.e. to allow a drug to be approved based on a surrogate endpoint. The FDA requires the trial to be "adequate and well controlled", and while there may be arguments on why with respect to the 6MWT results the trial is not large enough or crossover impact has tainted the control arm, but when it comes to the surrogate endpoint of dystrophin production, there is no argument that the adequacy (including patients from earlier trials) or control could possibly impact the level of dystrophin production - i.e. 12 of 12 boys generated meaningul levels of dsytrophin after being on treatment. between 24 and 48 weeks and the 4 placebo patients generated no increase in dystrophin (not that any boy in the history of the disease has probably ever spontaneously started generating dystrophin) after 24 weeks. 100% in 12 treated patients vs 0% in 4 placebo patients - that is "adequate and well controlled". Combine the dystrophin production results with the pristine safety results over 52+ weeks and you have more than enough evidence to file for accelerated approval. If there ever was a "poster child" for the type of disease and groundbreaking treatment that the accelerated approval regulations weredesigned for, DMD and eteplirsen is it.

    • If the inquiry by top FDA officials is "risk tolerance", then the FDA goal may be to get the GSK exon-skipping drug through AA, since Eteplirsin has no risks.

      • 3 Replies to avii_world
      • yo avi world

        as for GSK/Prosensa, they are surely active 24/7 in all ways that they think are in their best interest

        to assume otherwise is foolhardy in my view

        but as to your comment on risk, we need to listen vary clearly to the "risk" word as used by Woodcock and Temple in their recent public presentations

        I think the risk they are talking about is not, per se, for example, about any one drug that produces protein in urine vs. another that does not

        I think it is about understanding the risk of widescale commercial usage of a drug BEFORE it has been previously tested further in a much larger, multiple site, trial

        as we know, there have been an endless number of dead drug candidates who did great in P2 and then failed in P3, and were never approved, and some of them in P3, actually proved harmful

        so the risk I think is mostly on the table here with the dmd drug candidates (particularly with Srpt) relates awareness and acceptance of the risk of widescale administration right off a P2

        the situation with Prosensa is tricky in the total mix here

        its P23 results are due out in 6-7 months

        when you timeline out an AA for srpt and a straight P3 approval for Prosensa, they are not materially diferent in terms of a dmd drug being commercially available for the first time

        Prosensa surely has the protein issue, which seems particulary relevant when you view it in terms of a 3-year facing drug administration for multiple decades

        so yes, there is a "risk" analysis for sure with Prosensa, which risk analysis is not exactly the same as the risk analysis possibly focussed on with respect to Srpt

        which "risk" was CureDuchenne focussed on in its recent submission to FDA?

        my guess would be both

        for me, GSK's decision to not publicly release its P2 results for the stated reason that it in the middle of a P3, is a neon red flag there ... find me any other biotech situation where a company has ever done this before

        but back to the srpt situation .... if I am the FDA and I am faced with an Srpt AA app, one of the things on my checklist would surely be awareness/acceptance of the risks of mass dosing off just a P2

        all you have to do is read the not exceeding 1 page description of the new AA focus in last year's new law

        patient risk acceptance on orphan drugs for rare diseases is expressly mentioned as something to be more considered for FDA accelerated approval

        the new law on this issue is clear and express, so clear and express that I actually think it is being overlooked in the macro analysis on whether a company like Srpt could get an AA off a P2

        I believe that if you read the actual law (all one page of it) on this point, you would be very bullish here

      • Huh?

      • lol..the gsk drug ?...okay i'll be gsk...hey guys we've got a pill that is reversing the dd effects and possibly curing based on the trial data and there are no side effects..hey let's keep this info quiet and test it for another 4 years

 
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