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Insmed Incorporated Message Board

  • blasedp31 blasedp31 Apr 26, 2014 2:03 PM Flag

    Breakthrough Therapy Designation

    Is Insmed 30 days away or less before the FDA gives an answer? I would think patients would like to see it this week!

    [ The Company also intends to apply for Breakthrough Therapy Designation for ARIKAYCE in the United States based upon the culture conversion results. ARIKAYCE has already received Orphan Drug, Qualified Infectious Disease Product (QIDP) and Fast Track designations from the U.S. Food and Drug Administration ]

    [ The Secretary may approve an application for approval of a product for a serious or life-threatening disease ... upon a determination that the product has an effect on ... a clinical endpoint ... that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. ]

    ARIKAYCE converted 11 out of 44 patients suffering from drug-resistant pulmonary mycobacterial infections to culture-negative within twelve weeks - the majority within four weeks.

    1. Is conversion to culture-negative clinically significant?

    Yes - it warranted FDA approval of bedaquiline via the Breakthrough Therapy pathway.

    2. Could the FDA view efficacy in treating pulmonary infection by Non-tuberculous Mycobacteria as being "reasonably likely to predict" a clinical benefit in pulmonary infection by Mycobacterium Tuberculosis?

    a) In the treatment of MDR-TB, which currently requires the patient to suffer two years of systemic toxicity -

    In my opinion it would be reasonable to do so.

    b) In the treatment of normal TB, which currently requires the patient to suffer six months of systemic toxicity -

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    • Blasé, what about the placebo patients that continued treatment after 10/13 (when the NTM trial ended)? Thought Checks posted some progress on those patients too (not sure) and IF that was the case, would this data also be added to the 11/44? That would push the decision and timeline even further UP?

      Sentiment: Buy

      • 1 Reply to justarook04
      • Rook. I don't know about the timeline, but if the news was good or average, I would think it would be added to the 11/44.

        Is Insmed 30 days away or less before the FDA gives an answer? I would think patients would like to see it this week!

        [ The Company also intends to apply for Breakthrough Therapy Designation for ARIKAYCE in the United States based upon the culture conversion results. ARIKAYCE has already received Orphan Drug, Qualified Infectious Disease Product (QIDP) and Fast Track designations from the U.S. Food and Drug Administration ]

        [ The Secretary may approve an application for approval of a product for a serious or life-threatening disease ... upon a determination that the product has an effect on ... a clinical endpoint ... that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit, taking into account the severity, rarity, or prevalence of the condition and the availability or lack of alternative treatments. ]

        ARIKAYCE converted 11 out of 44 patients suffering from drug-resistant pulmonary mycobacterial infections to culture-negative within twelve weeks - the majority within four weeks.

        1. Is conversion to culture-negative clinically significant?

        Yes - it warranted FDA approval of bedaquiline via the Breakthrough Therapy pathway.

        2. Could the FDA view efficacy in treating pulmonary infection by Non-tuberculous Mycobacteria as being "reasonably likely to predict" a clinical benefit in pulmonary infection by Mycobacterium Tuberculosis?

        a) In the treatment of MDR-TB, which currently requires the patient to suffer two years of systemic toxicity -

        In my opinion it would be reasonable to do so.

        b) In the treatment of normal TB, which currently requires the patient to suffer six months of systemic toxicity -

 
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