News

New Formula of Recombinant Factor VIII in Kids with Severe Hemophilia A Is Effective, Trial Shows

A new, modified version of recombinant factor VIII (rFVIII), called rVIII-SingleChain, showed great efficacy and safety in treating bleeding events in children with severe hemophilia A. The study, “Safety, efficacy and pharmacokinetics of rVIII-SingleChain in children with severe hemophilia A: results of a multicenter clinical trial,” was published in…

Uniqure’s Hemophilia B Treatment Granted FDA Breakthrough Therapy Designation

The U.S. Food and Drug Administration (FDA) has granted uniQure’s AMT-060, an investigational gene therapy, breakthrough therapy designation for patients with severe hemophilia B. The regulatory agency based its decision on data from the ongoing, dose-ranging Phase 1/2 clinical trial (NCT02396342) evaluating AMT-060 for hemophilia B. The study’s estimated completion…