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    • Hemgenix

    - PMLiVE

    First NHS patient treated with CSL Behring’s haemophilia B gene therapy Hemgenix

    The genetic bleeding disorder affects more than 2,000 people in the UK

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    EC approves CSL’s Andembry to prevent hereditary angioedema attacks

    Approximately one in every 50,000 people worldwide are affected by the rare genetic disorder

    - PMLiVE

    CSL Behring announces four-year results for haemophilia B gene therapy Hemgenix

    The genetic bleeding disorder affects an estimated one per every 19,283 male births in the US

    - PMLiVE

    MHRA approves CSL’s Andembry to treat angioedema attacks in HAE patients

    The rare genetic condition occurs in approximately one in every 50,000 people in the UK

    - PMLiVE

    Pfizer’s one-time haemophilia B gene therapy Durveqtix granted EC approval

    More than 42,000 people globally are living with the rare bleeding disorder

    - PMLiVE

    NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix

    More than 2,000 people in the UK are currently living with the genetic bleeding disorder

    - PMLiVE

    Pfizer’s one-time haemophilia B therapy Durveqtix receives CHMP recommendation

    More than 38,000 people worldwide are currently affected by the inherited bleeding disorder

    - PMLiVE

    NICE publishes draft guidance not recommending CSL’s haemophilia B gene therapy

    The company has been seeking approval for use of the therapy in adults with severe or moderately severe haemophilia B without a history of factor IX inhibitors

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    EC approves first gene therapy for adults with haemophilia B

    Typically, haemophilia B patients must adhere to strict, lifelong IV infusion schedules

    - PMLiVE

    Pfizer announces positive phase 3 results for haemophilia B gene therapy

    A one-off, intravenous dose of the therapy candidate reduced bleeding by 71%

    - PMLiVE

    FDA approves first gene therapy for adults with haemophilia B

    CSL’s Hemgenix allowed patients to produce mean factor IX activity of 39% at six months

    - PMLiVE

    FDA removes clinical hold on uniQure’s haemophilia gene therapy

    Clinical hold placed following a case of hepatocellular carcinoma in a patient in the trial

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