An experimental gene therapy using modified blood stem cells helped five adults with severe hemophilia A attain therapeutic levels of clotting factor VIII (FVIII) to prevent and control bleeding, according to a small first-in-human study. Over the course of the Phase 1 clinical study (NCT05265767), none of the…
Sangamo Therapeutics will regain full rights to develop and market giroctocogene fitelparvovec, a gene therapy candidate for adults with moderately severe to severe hemophilia A, after Pfizer decided to end its involvement in the program. Pfizer’s decision not to advance into regulatory submissions came despite positive…
In 2024, Hemophilia News Today provided readers with up-to-date coverage on research, treatments, and clinical trials for hemophilia. We compiled the top 10 most-read stories of the year, each with a brief summary. As we enter 2025, we look forward to continuing to support the hemophilia community. We…
For adults and adolescents with hemophilia A without inhibitors, Altuviiio (efanesoctocog alfa) may work better than other standard or extended half-life factor replacement therapies at preventing bleeding episodes, while requiring less frequent injections, a study found. Researchers made an indirect comparison of data from XTEND-1 (NCT04161495) and published…
Adults and adolescents with hemophilia A or hemophilia B without inhibitors experienced significantly fewer bleeding episodes when using concizumab as a prophylactic (preventive) treatment compared with those who weren’t on any prophylaxis. That’s according to data from explorer8 (NCT04082429), an ongoing Phase 3 clinical study in which…
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…
Hympavzi (marstacimab-hncq) may be available by prescription in the U.S. before the end of the year. The therapy was approved this month as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or…
The U.S. Food and Drug Administration (FDA) has approved Pfizer’s marstacimab under the brand name Hympavzi for use as a routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adults and children, ages 12 and older, with hemophilia A or B without inhibitors. With…
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…
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…
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