News

Treatment with marstacimab (PF-06741086), an experimental antibody-based therapy developed by Pfizer, reduced the number of bleeds in patients with severe hemophilia who took part in a Phase 1b/2 clinical study. This held true regardless of whether patients had hemophilia A or hemophilia B, or had inhibitors…

If approved for hemophilia A, the one-time gene therapy Roctavian (valoctocogene roxaparvovec) would provide substantial cost savings per patient compared with other preventive treatments, specifically Hemlibra. That’s according to a draft evidence report issued by pricing watchdog the Institute for Clinical and Economic Review (ICER). An independent non-profit…

The U.S. Food and Drug Administration (FDA) has granted orphan drug status to SerpinPC for treating hemophilia B. Orphan drug status is awarded to therapeutics intended to treat rare conditions, defined as those affecting fewer than 200,000 people in the U.S. The designation provides financial incentives to support clinical…

A participant in a Phase 3 clinical trial evaluating Roctavian (valoctocogene roxaparvovec), a gene therapy for hemophilia A, has been diagnosed with leukemia. A genetic assessment of the case, conducted by the therapy’s developer, BioMarin Pharmaceutical, suggested the cancer diagnosis was not linked to Roctavian. Details of…

Hemophilia A patients who develop inhibitors — neutralizing antibodies made by immune cells against clotting factors — have more expression, or activity, of genes involved in activating the immune system, a small study reports. “The results of our study reveal that there is an upregulation of genes involved with…

Switching to Hemlibra (emicizumab) was safe and effective in children with hemophilia A, including in those who had been minimally treated before or who had not been treated at all, a real-world study reported. Researchers noted that studies involving a larger number of patients are still warranted to…

Prophylactic, or preventive, treatment with an experimental replacement therapy called TQG202 safely maintained a low bleeding rate in people with severe hemophilia A. That’s according to new data from a Phase 3 clinical trial reported in a recent study, which showed TQG202 worked to control bleeds in testing against…

The U.S. Food and Drug Administration (FDA) has granted priority review for the approval of efanesoctocog alfa, with a decision on the potentially longer-lasting hemophilia A treatment due early next year. The regulatory agency accepted an application from developers Sobi and Sanofi requesting approval of the…

The European Commission has granted conditional marketing authorization to BioMarin’s one-time gene therapy Roctavian (valoctocogene roxaparvovec) as a treatment for adults with severe hemophilia A who have neither inhibitors nor detectable antibodies against adeno-associated virus serotype 5 (AAV5). The decision, which comes a couple of months after a…

Adults with hemophilia and ankle joint damage taking part in a yearlong pilot study largely reported that two injections of hyaluronic acid, which acts to lubricate the joint, eased ankle pain and improved movement. The treatment, given six months apart in this U.K. trial, also enabled most of these people…