News

After five years of follow-up, Hemgenix (etranacogene dezaparvovec-drlb) was associated with durable factor IX (FIX) levels, lower bleeding rates, and a substantial reduction in the need for regular FIX infusions, with a generally favorable long-term safety profile in men with severe or moderately severe hemophilia B. These data…

In 2025, Hemophilia News Today delivered readers timely reporting on research, therapeutic options, and clinical trials for hemophilia. Below is a list of the top five most-read stories we published this year. As we enter 2026, we look forward to continuing to support and serve as a source…

BBM-H901 (dalnacogene ponparvovec), a gene therapy that’s approved in China for adults with moderate to severe hemophilia B, was shown to significantly reduce bleeding rates among hemophilia B patients in clinical trials, a new study shows. Hemophilia B is a genetic disorder caused by mutations in the gene that…

Hympavzi (marstacimab) reduced bleeding rates and improved quality of life in adults and adolescents with hemophilia A or B who have inhibitors (antibodies against standard replacement therapies). That’s according to final data from the Phase 3 BASIS study (NCT03938792), which demonstrated the treatment met its main goal of…

A newborn in China was diagnosed with hemophilia B after a ruptured liver caused significant abdominal bleeding, according to a case report. Due to early detection, timely surgical repair, and immediate factor-replacement therapy, the infant made a full recovery. This case shows that “neonatal hepatic rupture may be…

A 32-year-old man was diagnosed with hemophilia A after a minor injury caused disproportionate right eye bleeding, with a buildup of pressure inside the eye and vision loss, a new U.S. case study reports. According to the researchers, this case demonstrates that an eye bleed can be a rare…

Beqvez (fidanacogene elaparvovec-dzkt), a discontinued gene therapy for hemophilia B, was shown to help reduce joint bleeding in Phase 3 clinical testing. That’s according to new analysis results from an ongoing global study that assessed the treatment’s effectiveness and found positive results. Specifically, “through 15 months, 12 of…

Starting Hemlibra (emicizumab) early is safe and helps to prevent bleeding in babies with severe hemophilia A who are untreated or minimally treated, though doctors should still monitor for self-reactive antibodies that can make other treatments less effective, a study suggested. The study, “Emicizumab in Previously Untreated…

Data from non-human primates suggest gene-editing therapy MGX-001 may be able to restore clinically meaningful levels of factor VIII (FVIII) after a single dose, supporting its potential as a one-time treatment for hemophilia A. Developer Metagenomi expects to meet with health authorities later this year to develop a strategy for…

Hemlibra (emicizumab-kxwh) safely supports joint health in children with hemophilia A without inhibitors through three years of treatment, according to interim data from a post-marketing study in Japan. The ongoing study, AOZORA (jRCT1080224629), is evaluating the long-term safety of Hemlibra and its impact on joint health in children ages…