Research & Development
CSL's HEMGENIX haemophilia B gene therapy receives US FDA approval
23 November 2022 -

CSL (ASX: CSL), an Australia-based global biotechnology company, announced on Tuesday that it has received approval from the US Food and Drug Administration (FDA) for its HEMGENIX (etranacogene dezaparvovec-drlb), the first and only one-time gene therapy intended to treat appropriate adults with haemophilia B.

The product is approved for the treatment of adults with haemophilia B who presently use factor IX prophylaxis therapy, or have present or historical life-threatening haemorrhage or have repeated, serious spontaneous bleeding episodes. The company said that during the ongoing clinical trial, the product decreased the rate of annual bleeds and 94% of the subjects discontinued factor IX prophylaxis and remained prophylaxis-free.

The approval is supported by outcomes from the ongoing HOPE-B trial. Outcomes from the study revealed that the product enabled patients to produce mean factor IX activity of 39% at six months and 36.7% at 24 months post infusion.

uniQure (Nasdaq: QURE) headed the multi-year clinical development program for HEMGENIX and sponsorship of the clinical trials moved to CSL subsequent to the acquisition of the global rights to commercialise the treatment.

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