FDA Approves First Recombinant von Willebrand Factor

The U.S. Food and Drug Administration today approved Vonvendi, von Willebrand factor (Recombinant), for use in adults 18 years of age and older who have von Willebrand disease (VWD). Vonvendi is the first FDA-approved recombinant von Willebrand factor, and is approved for the on-demand (as needed) treatment and control of bleeding episodes in adults diagnosed […]

Phase I Study Results for Once-Monthly Subcutaneous Hemophilia Treatment

Note: The following is an edited press release from Alnylam. The original release can be read in its entirety here. Alnylam Pharmaceuticals, Inc. announced on December 7, 2015 positive results from its ongoing Phase 1 clinical study with fitusiran (fi-TOO-si-ran), the recommended International Nonproprietary Name (INN) for ALN-AT3. Fitusiran is an investigational RNAi therapeutic targeting […]

New Data Released on Long-Acting Hemophilia B Treatment

Note: The following is the edited version of a press release issued by CSL Behring. The original release can be found here. On December 7, 2015, CSL Behring presented data from its Phase III PROLONG-9FP clinical program evaluating the efficacy and long-term safety of its investigational long-acting fusion protein linking recombinant coagulation factor IX with recombinant […]

Pain and Mobility Are Major Problems for Patients with Hemophilia

Note: The following originally appeared on MDMagazine.com. New research further supports the understanding that patients with hemophilia suffer from pain and mobility issues, and it went on to uncover which joints are most greatly affected by the condition.Christine Kempton, MD, MSc, from Emory University in Georgia, and colleagues used multiple strategies to find how big […]