In an unexpected turn, Catalyst Biosciences is discontinuing the clinical development of marzeptacog alfa activated (MarzAA), its experimental under-the-skin therapy for hemophilia A and B patients with inhibitors, which was being evaluated in an international Phase 3 clinical trial. “Based on several factors including a recently updated feasibility assessment,…
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The extended half-life replacement therapies Eloctate and Alprolix can be used to effectively manage bleeding during surgery in people with hemophilia, according to a new study based on Phase 3 clinical trial data. These findings highlight the “efficacy and safety of these products in a broad age range of…
The Loma Linda University Health (LLUH) Hemophilia Treatment Center has become the first clinic in California offering Hympavzi (marstacimab) for people with hemophilia B. “For the first time, patients with hemophilia B in Southern California have access to a therapy that is not just effective, but also drastically…
Hemophilia A patients at a single center in France reported high rates of satisfaction with Hemlibra (emicizumab) after a year of real-world use, a study has found. Patients reported improvements in health and life quality, as well as reductions in pain and bleeding with the therapy, but problems…
People with hemophilia A in China see major health challenges, such as frequent joint bleeding, joint malformation, and high rates of hospital visits, primarily owing to the limited use of preventive therapies, a real-world study shows. After a bleeding episode, most patients rely on on-demand treatment, which is less…
Jivi (damoctocog alfa pegol) has received approval from the U.S. Food and Drug Administration (FDA) that now extends to children with hemophilia A as young as 7 years old. Jivi had previously been authorized for hemophilia A patients 12 and older. The therapy, which is sold by Bayer,…
BioMarin Pharmaceutical has resubmitted an application to the U.S. Food and Drug Administration (FDA) seeking approval of Roctavian (valoctocogene roxaparvovec), potentially the first gene therapy for adults with severe hemophilia A. The FDA delayed the company’s initial approval request in 2019, with the agency requiring two more…
Qfitlia (fitusiran) is an RNA-based therapy that is approved to prevent or reduce the frequency of bleeds in patients with hemophilia A or B, with or without inhibitors. It is given via under-the-skin, or subcutaneous, injections that can be self-administered.
When my youngest son needed daily infusions, keeping up with factor and supplies was not difficult. There was never any question as to when an infusion would take place, and ordering what was needed was the same every month. At one point when his treatment was extremely complicated, each month…
The U.S. Food and Drug Administration (FDA) has granted orphan drug status to Sigilon Therapeutics’ candidate cell therapy, called SIG-001, for hemophilia A. Orphan drug status aims to encourage therapies for rare and serious diseases, through benefits such as seven years of market exclusivity and exemption from FDA application…