U.S. drugmaker Pfizer Inc. said on Tuesday late-stage study data for its experimental hemophilia therapy showed superiority to the current standard of care treatment in reducing bleeding rates in patients.
Tag Archive for: Hemgenix
Today the Dutch drug developer said it would sell a part of the royalty rights for the world’s most expensive treatment Hemgenix to HealthCare Royalty and Sagard Healthcare for up to $400 million in cash.
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.
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.
Bristol Myers Squibb is terminating a seven-year-old gene therapy collaboration to develop potential treatments for congestive heart failure with uniQure N.V. valued at $1 billion.
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.
