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Sarepta Therapeutics, Inc. Message Board

  • hwsimpsonshero hwsimpsonshero Mar 29, 2013 12:06 PM Flag

    SRPT is not applying for Breakthrough Therapy. They will apply for Accelerated Approval.

    Just to clarify, some investors falsely believe that eteplirsen will be going for Breakthrough Therapy designation. It won't, because it is already well passed that stage. Breakthrough Therapies are for drugs in early stages of development, such as SRPT's other exon-skipping drugs. Eteplirsen is no longer in early stage development, so it has no need to apply for Breakthrough designation. To understand what Breakthrough Therapies are, type this into google to go to the FDA's page explaining them:

    "frequently asked questions: breakthrough therapies"

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    • HW, I'm sure that few can equal your brilliance. That said, your contention that a drug can not be granted breakthrough once it is further along than phase 1 just seems incorrect.

      To qualify for “Breakthrough” status, a sponsor must apply for a designation to FDA. The criteria are broad: the drug must be intended for a serious or life threatening disease and be supported by “preliminary clinical evidence [that] indicates that the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints, such as substantial treatment effects observed early in clinical development.” ............................

      Sure sounds just like SRPT to me. And unless I forgot how to read, "early in clinical development" in no way excludes SRPT....... NO WAY......... The goal is to bring promising drugs to underserved, rare disease populations particularly children and the FDA would appear to have great flexibility in determining where and when to offer breakthrough designation towards this end.

      - serious and life threatening? yup
      - preliminary clinical evidence that it works? yup again
      - -substantial improvement over everything else? uh huh
      - one or more clinically significant endpoints? you bet
      - further along in drug development than phase 1? yes, so what?

    • Pcyc just received breakthrough designation for ibrutinib and that is in late stage.

    • they will only apply for aa if the fda hints they should,if they apply against what the fda wishes it would be suicide. go serepta

      Sentiment: Strong Buy

    • Thanks for the clarification, hwsimpsonhero. You're right:

      In the FDA's FAQ page that you mention at the end of your post, there's this passage:

      "Note that a drug that has received a breakthrough therapy designation or a fast track designation can be eligible for the accelerated approval pathway, if the relevant criteria are met."

      So accelerated approval is better than breakthrough therapy designation. If we get accelerated approval, there is no need to have breakthrough therapy designation.

      Sentiment: Strong Buy

    • One example of a drug recently granted as a Breakthrough Therapy is Novartis's compound LDK378. It has only completed Phase 1, and just initiated its Phase 2 trials.

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