Alice Melão,  —

The U.S. Food and Drug Administration (FDA) has approved an update to the label of Alprolix, based on long-term efficacy and safety data from two Phase 3 studies, the B-YOND extension trial and the Kids B-LONG pediatric study, manufacturer Bioverativ announced. This label extension, which adds pediatric data, further supports…

Administration of once-weekly Hemlibra (emicizumab-kxwh) significantly reduced bleeding rates in patients with Hemophilia A compared to other clotting factor VIII prophylaxis (preventive) therapy, according to the latest results of the Phase 3 HAVEN 3 study announced by Roche. Developed by Chugai Pharmaceutical, Roche, and Genentech,…

An ongoing Phase 1/2 clinical trial evaluating a gene therapy, SPK-9001, in people with hemophilia B is expanding its enrollment by up to five more patients, who will be given a new version of the therapy, its developer announced. One patient in this group has already received SPK-9001 produced under…

The U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to BioMarin Pharmaceutical’s investigative therapy, valoctocogene roxaparvovec to treat patients with hemophilia A. This new status is likely to speed up the development and regulatory review of valoctocogene roxaparvovec, which has already received an Orphan Drug Designation from both…

The U.S. Food and Drug Administration (FDA) has approved BioMarin Pharmaceutical‘s Investigational New Drug (IND) application for its gene therapy candidate to treat severe hemophilia A. FDA’s decision on BMN 270, also known for its generic name valoctocogene roxaparvovec,…

Researchers have found that the high price of recombinant clotting factors for patients with hemophilia B is the main cause of the economic burden of the disease in the United States. However, despite the cost, prophylaxis treatment can provide clinical benefits to patients that may reduce other costs not…

Hemophilia patients are taking heart from a number of innovative treatment approaches that have reached the clinical trials stage. Researchers at the Children’s Hospital of Philadelphia offered a rundown on the advances in an article in the journal Blood titled “Novel approaches to hemophilia therapy: successes and challenges.” They include …

Knowledge about the clinical benefits of treatments and their importance in the outcome of patients, as well as more stable therapies, will likely improve treatment adherence among hemophilia patients in the U.S., according to new research. The study, “Treatment adherence in hemophilia,” was conducted by Dr. Courtney Thornburg, medical…

U.S. hemophiliacs who use patient-reported outcome (PRO) instruments can provide reliable information on their disease burden, finds a new study that appeared in the journal Patient Preference and Adherence. In the study, “Reliability of patient-reported outcome instruments in US adults with hemophilia: the Pain, Functional Impairment and Quality…

The Boston-based Institute for Clinical and Economic Review (ICER) says a “draft scoping document” outlining its planned comparative review of emicizumab‘s clinical effectiveness and value in treating hemophilia A will be available until Sept. 29. ICER’s report will be subject to a public deliberation in March 2018 by the…