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Sarepta Therapeutics, Inc. Message Board

system_architect_enterprise 9 posts  |  Last Activity: May 23, 2016 8:42 AM Member since: Apr 28, 2006
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  • system_architect_enterprise system_architect_enterprise May 23, 2016 8:42 AM Flag

    Fair valuation is based on $1.2B in annual sales from 3,500 patients at $350K per patient per year (already agreed to by insurance companies). Since this is a life threatening situation, not an elective one, the ramp up will be rapid. Projecting that forward puts fair valuation at more than $120/share, not including any follow on exon skipping treatments that may be derived.

    This does not consider external factors such as short squeeze, strategic value for follow on drugs, validation of the whole exon skipping approach or (on the other side) cost of follow up tracking or additional trials that may be required wi conditional approval.

  • Reply to

    motley article on ptct

    by rxkell May 19, 2016 4:57 PM
    system_architect_enterprise system_architect_enterprise May 19, 2016 9:39 PM Flag

    SRPT has problems ahead of it to be sure, but the fact that the FDA would not even FILE the NDA with PTCT is astounding. The FDA is sending out a signal loud and clear that companies should not invest in these types of orphan drugs. they are too far outside the mainstream of what the FDA knows how to handle.

  • There are many problems and implications if the FDA does not approve. Companies like BMRN, SRPT, PTCT, and many others will simply give up trying to create meds for these orphan drug type situations because the FDA simply makes it too expensive, and the likelihood of approval is simply too low, to make it a worthwhile investment.

    So while this decision may doom 4,000-12,000 boys to a death sentence, it may prevent countless more from ever having a chance.

  • system_architect_enterprise by system_architect_enterprise May 10, 2016 6:38 AM Flag

    The Food and Drug Administration will decide this month whether to approve an innovative drug for a fatal form of muscular dystrophy, and one question is whether the agency’s management will rebuke the dubious science propagated by its staff reviewers. Here’s another: Will FDA defy a law that directs the agency to pick up the pace on treatments for rare diseases?

    Eteplirsen by Sarepta Therapeutics produces dystrophin, the protein missing in boys with Duchenne muscular dystrophy, a condition that destroys every muscle in a decline from walking to wheelchair to death. Eteplirsen shows zero safety risks and four years of striking results in a clinical trial of 12 boys. FDA reviewers assert that the study is too small and maybe the drug doesn’t work, though Duchenne experts world-wide say otherwise.

    As it happens, Congress in 2012 passed a law that should settle the issue. The Food and Drug Administration Safety and Innovation Act grants FDA considerable flexibility to accelerate approval for life-threatening disease drugs dealing with an unmet medical need. FDA can sign off on a drug that “has an effect on a surrogate endpoint that is reasonably likely to predict clinical benefit.” For those without decoder glasses, that means: The drug delivers on some promised action, and in a way that likely helps the patient.


    The FDA vs. Austin Leclaire
    Mental Dystrophy at the FDA
    The trial turned up evidence that eteplirsen makes good on pumping out dystrophin, a feat no treatment has managed. It’s reasonable, even obvious, to predict that dystrophin would benefit a kid whose body can’t produce it. Definitive proof is not required, and the law stipulates that FDA can yank a drug from the market if later trials fail. Sarepta will have results from more trials in two years or so. If that sounds soon enough to delay approval, consider the hundreds of boys who by then won’t walk.


  • system_architect_enterprise by system_architect_enterprise May 10, 2016 6:10 AM Flag

    Interesting article in the WSJ today.

  • system_architect_enterprise by system_architect_enterprise May 8, 2016 10:54 AM Flag

    I have to think it is less than 50/50 because the panel voted against it. Further, the arguments that said that the questions were wired against Eteplirsen makes me wonder how - if they can wire the questions - can we overcome a substantial bias against the drug?

    I hope and pray for approval, but am very concerned about how corrupt the FDA is.

    What do you think? Please reply (just from one ID please) and let me know what your odds are for approval.

  • system_architect_enterprise system_architect_enterprise May 1, 2016 12:28 PM Flag

    That's the whole point though. The FDA shouldn't be "happy" or "unhappy" at all. The FDA needs to look at the facts to see if the drugs benefits outweigh the risks.

    The FDA needs to stop worrying about whether it fits their narrow version of reality or if it fits what THEY want, they need to focus on the benefit for the kids in need. Enough of all this #$%$ about appeasing the FDA, it needs to be about patients, not FDA and their holier than thou attitude.

  • system_architect_enterprise system_architect_enterprise Apr 30, 2016 9:37 PM Flag

    You state that if the FDA grants AA it will only be for the children. Well, I hope so. I hope it isn't denied to further the career of the FDA panel members, or their financial interests in other companies or against this one.

    It should be ALL about helping the children. Period.

  • system_architect_enterprise by system_architect_enterprise Apr 29, 2016 10:18 AM Flag

    As with the government, nothing is ever clear nor certain. I expect Janet will give them some kind of 1/2-baked approval that leaves everybody wondering what it means. Something like they can have approval for kids just until they can complete a bigger more definitive study, then the FDA will get another crack at it, so we will have to go through all the turmoil again.

    For now, I hope and pray, for the kids sake, that the FDA finds some way to approve in some fashion.

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