Pfizer’s Hympavzi (marstacimab) has been approved in the European Union (EU) to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with severe hemophilia A or hemophilia B who weigh at least 35 kg (77 pounds) and haven’t developed neutralizing…

I cannot tell you how many times I’ve heard the term “Bubble Wrap.” A child with hemophilia is born: “How am I going to keep this kid Bubble Wrapped and safe?” A kid with hemophilia falls on the playground and bleeds: “I wish we could Bubble Wrap you…

Metagenomi, which is developing MGX-001, a gene-editing therapy for hemophilia A, is preparing to initiate investigational new drug (IND)-enabling activities that seek to support the therapy’s transition from preclinical to clinical testing. The move is backed by preclinical studies in nonhuman primates that show the treatment led to…

Immunotherapy is an effective treatment strategy to suppress the production of autoantibodies and induce remission in people with acquired hemophilia A, a small study in China suggests. In some cases, however, multiple cycles of immunosuppressive treatment may be required for patients to achieve a good treatment response.The results were published…

Hemlibra (emicizumab) effectively prevented bleeds in patients with acquired hemophilia A (AHA), according to real-world data from a single center study in Japan. The treatment also led to shorter hospital stays, helped patients maintain their daily living activities, and contributed to lower the doses of bypassing agents,…

Note: This story was updated Oct. 15, 2024, to note results from the subset of patients with inhibitors participating in BASIS are now expected next year. A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for…

Obizur (susoctocog alfa) is an effective and safe therapeutic option for the treatment of severe bleeding episodes in people with acquired hemophilia A, a study in Japanese patients showed. The study “supports the use of [Obizur] as a novel therapy in the clinical management of patients with [acquired…

Almost two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce their quality of life, even when they’re on factor replacement therapy to reduce the frequency of bleeding episodes, a real-world study shows. “Overall, the data indicate that joint problems still…