By Robb M. Stewart
Australia biotechnology company CSL’s Hemgenix has been authorized by Health Canada as the first gene therapy for the treatment of the rare bleeding disorder hemophilia B.
Hemgenix is a one-time, single-dose treatment for adults who require routine prophylaxis to prevent or reduce the frequency of bleeding episodes.
CSL said the approval is based on the results of an ongoing late-stage trial of 54 participants, the largest gene therapy trial in hemophilia B to date. Data from the Phase III, open label, single-dose, single-arm study found that 96% of patients following infusion of Hemgenix discontinued use of prophylaxis and remained free of previous continuous routine prophylaxis therapy, the company said.
Hemgenix was approved by the U.S. Food and Drug Administration and was granted conditional marketing authorization by the European Commission for the European Union and European Economic Area, and the U.K.’s Medicines and Healthcare products Regulatory Agency.
Write to Robb M. Stewart at [email protected]
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