First gene therapy for hemophilia B granted Direct Access in France

USA-headquartered biotech CSL Behring, a subsidiary of Australia’s CSL Limited (ASX: CSL), has been granted Direct Access for Hemgenix (etranacogene dezaparvovec) by the French Ministry of Health, marking the first treatment to be authorized by this innovative process in France.

The company notes that Hemgenix – which is licensed from Dutch gene therapy company uniQure (Nasdaq: QURE) - is the first and only one-time gene therapy for the treatment of severe and moderately severe hemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors. Through Direct Access, appropriate people living with hemophilia B will now be able to access Hemgenix.

Patients with hemophilia B currently require lifelong treatment with intravenous Factor IX infusions, which can have a significant impact on quality of life and wellbeing. According to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP), there is an “unmet medical need for new therapeutic approaches that might free patients from the burden of frequent infusions, or episodically at the time of a bleeding event.”