Nearly two years after their narrow PhIII failure, Orphazyme gets speedy look at the FDA

Discussion in 'Pharma/Biotech Companies - In the News' started by cafead, Sep 16, 2020 at 10:52 AM.

  1. #1 cafead, Sep 16, 2020 at 10:52 AM
    cafead

    cafead Administrator
    Staff Member

    Joined:
    Nov 21, 2001
    Messages:
    32,962
    Likes Received:
    3
    via Orphazyme’s bet that, despite missing both primary endpoints on its pivotal study, they would find welcoming ears at the FDA has paid off, for now.

    The Danish biotech announced today that the FDA has accepted the application for their experimental drug arimoclomol in the rare neurodegenerative condition Niemann-Pick disease Type C and given it priority review.

    article source
     
    Quote

  2. #2 anonymous, Nov 19, 2020 at 12:06 PM
    anonymous

    anonymous Guest

    Orphazyme in the US is poorly organized and being run by mid-level staff. It is placing a great deal of emphasis on the use of registries to collect data, but has no one on staff that actually worked with registries or understands them.
     
    Quote
Reply to Thread