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Health Canada Approves CSL Behring's Hemgenix Gene Therapy

NEW YORK – Health Canada has authorized CSL Behring's Hemgenix (etranacogene dezaparvovec) as a one-time gene therapy for moderately severe or severe hemophilia B, the biotech company said Thursday.

The approval is based on data from the company's ongoing open-label, single-arm, multicenter Phase III trial of 54 patients with the rare bleeding disorder, dubbed HOPE-B.

On average, patients in the HOPE-B trial experienced an average annual bleeding rate reduction of 58 percent at seven to 18 months post-treatment compared to the six-month lead-in period, and 96 percent of patients discontinued use of prophylaxis therapy. The most common adverse reactions from the Phase IIb and Phase III trials were alanine transaminase elevations, headache, influenza-like illness, and aspartate aminotransferase elevations.

"The approval of Hemgenix in Canada marks an important milestone, and we look forward to collaborating with the hemophilia B community to provide access to this innovative treatment option," Philippe Hebert, CSL Behring's Canada general manager, said in a statement.

Hemophilia B is caused by a mutation in the FIX gene, which results in insufficient production of an eponymous protein that supports blood clotting. Hemgenix, as an adeno-associated virus type 5 vector-based gene therapy, delivers a functional copy of the FIX gene.

Hemgenix, frequently referred to as the most expensive drug in the world, was approved last year in the US where it is priced at $3.5 million. The gene therapy has conditional marketing authorization in the EU and UK, although the UK National Institute for Health and Care Excellence this summer recommended against offering the drug in England.