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NICE recommends CSL Behring’s haemophilia B gene therapy Hemgenix

June 28, 2024


The agency’s positive opinion, which specifically applies to adults with severe or moderately severe haemophilia B without a history of factor IX (FIX) inhibitors, reverses its previous decision to reject the therapy.

Affecting more than 2,000 people in the UK, haemophilia B is a genetic bleeding disorder resulting from missing or insufficient levels of clotting FIX.

Patients with severe cases of the disease currently require lifelong treatment with intravenous FIX, which can still leave them vulnerable to breakthrough bleeds and pain in the days before infusions.

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