News

Hemophilia gene therapy Beqvez (fidanacogene elaparvovec), marketed as Durveqtix in the European Union, results in stable production of factor IX (FIX), the clotting protein that is missing or faulty in hemophilia B, leading to about three times fewer bleeding episodes compared with prophylaxis, or preventive treatment, with regular infusions…

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,…

Preventive therapy, or prophylaxis, in patients ages 50 and older with severe hemophilia type A  and type B was effective at reducing bleeds, including those in target joints, and improving some aspects of their quality of life. That’s according to findings from a post hoc analysis of four…

A European Medicines Agency (EMA) committee has issued a positive opinion recommending the approval of marstacimab in the European Union for preventing bleeding episodes in people with severe hemophilia A or hemophilia B without inhibitors. The European Commission will now review the recommendation, made by the…

Having a COVID-19 infection had no significant impact on bleeding episodes, nor on the levels of factor replacement therapy used in treatment, for people with hemophilia, a new study from China found. The study did reveal, however, that, compared with adults, children with hemophilia had a significantly lower…

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…

A gene therapy designed to provide hemophilia A patients with a highly functional version of factor VIII (FVIII), the blood clotting protein they lack, could offer more sustained bleed control than existing gene therapies, preclinical research suggests. The current approach in hemophilia A gene therapy is to provide a…

MGX-001, a gene-editing therapy being developed by Metagenomi for hemophilia A, has shown promising safety and durability in an ongoing preclinical study in three nonhuman primates, the company has announced. Results showed that after one year, two animals had normal (82%) or nearly normal activity levels (41%) of…