Today the company announced that Canada’s health regulator approved its gene therapy for the treatment of a rare inherited bleeding disorder called hemophilia B ahead of a U.S. decision.
Tag Archive for: hemophilia B
2022 was a big year for uniQure with the U.S. and European approval of world’s first gene therapy for hemophilia B. In its 2022 financial report Monday, the Lexington, Mass. and Amsterdam-based company provided updates on the progress of its Huntington’s disease and ALS programs.
End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.
U.S. drugmaker Pfizer Inc. said on Thursday its experimental gene therapy for the treatment of hemophilia B, a rare inherited blood disorder, met its main goal in a late-stage study.
If approved, the treatment, branded Hemgenix, will be the first gene therapy in the European Union for the condition that is usually treated by regular injections of factor IX, the European Medicines Agency said.
BioMarin Pharmaceutical announced the FDA no longer plans to hold an advisory committee meeting to review its Biologics License Application (BLA) for Roctavian (valoctocogene roxaparvovec), an AAV gene therapy for adults with severe hemophilia A.
Hemgenix is an adeno-associated virus vector-based gene therapy for the treatment of adults with Hemophilia B (congenital Factor IX deficiency) who currently use Factor IX prophylaxis therapy, or have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.
The Institute for Clinical and Economic Review (ICER) concluded Tuesday that gene therapies for hemophilia A and B are worth it even at a hefty lifetime cost of $2.5 million.