News

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,…

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…

Hemophilia patients who received intensive factor VIII (FVIII) replacement therapy for better joint health were nearly five times more likely to see a reduction or complete resolution of synovial hypertrophy — swelling and thickening of the connective tissue that lines the inside of a joint — compared with those…

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…

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…

Centessa Pharmaceuticals is discontinuing the clinical development of SerpinPC, its experimental under-the-skin therapy for people with hemophilia A and hemophilia B with or without inhibitors. The therapy was being evaluated in several Phase 2 clinical trials. The decision follows the recent approval of Pfizer’s Hympavzi…

A delayed diagnosis is common for people with acquired hemophilia A (AHA), according to a new study that found that it took more than a month to get a diagnosis after the first bleeding episode for about 1 in 3 patients. Despite these delays, all AHA patients in the…