Health Canada approves Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes – CSL Behring

Health Canada has approved the gene therapy Hemgenix (etranacogene dezaparvovec) for treating adults with hemophilia B who rely on routine prophylactic therapies to prevent or reduce bleeding episodes. Hemgenix is now the first and only gene therapy available to this patient group.

“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, Canada’s general manager at CSL Behring, Hemgenix’s developer, said in a company press release. “We are proud to add this treatment to our portfolio of coagulation [blood clotting] therapies and look forward to patients benefiting from this therapy.”

Hemgenix is approved in the U.S. for adults on prophylaxis, have had life-threatening bleeds, or have repeated and serious spontaneous bleeds. It is conditionally approved in the U.K. and European Union for adults with moderate to severe hemophilia B who do not have a history of neutralizing antibodies against factor IX (FIX). FIX is the blood clotting protein thatis missing in hemophilia B due to mutations in the F9 gene that encodes its production. This makes patients susceptible to prolonged or spontaneous bleeding.

Patients typically rely on routine infusions of FIX replacement therapies to temporarily provide the missing clotting factor and prevent or reduce bleeds.