News

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…

Combining gene therapy with minimally invasive, robot-assisted hip replacement surgery safely and effectively treated severe joint damage for a 27-year-old man in China with hemophilia A, according to a case report. The approach significantly reduced the need for replacement therapy…

The U.S. Food and Drug Administration (FDA) has approved Novo Nordisk’s concizumab, which will be sold under the brand name Alhemo, as a daily treatment to prevent or reduce the frequency of bleeding episodes in certain adults and adolescents with hemophilia. Eligible patients are those with hemophilia…

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…

Preventive treatment with Altuviiio (efanesoctocog alfa) provided better bleed prevention than Hemlibra (emicizumab) in adults and adolescents with hemophilia A, according to an indirect comparison of Phase 3 clinical trial data. “While it should be borne in mind that this is an indirect comparison and, as such,…

Nearly two-thirds of hemophilia A patients given a single infusion of the investigational gene therapy giroctocogene fitelparvovec were free of bleeding episodes a median of almost three years after being treated, according to full data from the Phase 3 AFFINE clinical trial. These new data add to top-line…

The first-in-human Phase 1/2 clinical trial testing BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B, is now enrolling patients at two sites in the U.S. BE-101, a first-in-class therapy, aims to address the persistent unmet needs of hemophilia B patients — particularly the burden of ongoing treatment…

Bone mineral density, or the amount of mineral in bone tissue, is decreased in 63.1% of people with hemophilia, according to a study from Germany. Study findings also indicated the degree of loss depended on hemophilia severity. Patients with severe hemophilia had lower bone mineral density in the left…

Men with hemophilia and infected with the human immunodeficiency virus (HIV) are at a high risk of developing certain types of cancer that are more likely in people with HIV, according to a recent study in Japan. These cancers, called non-acquired immunodeficiency syndrome-defining malignancies or NADMs, included liver cancer,…