Marisa Wexler, MS,  senior science writer—

The U.S. Food and Drug Administration (FDA) has accepted a resubmitted biologics license application (BLA) from BioMarin Pharmaceutical requesting approval of the gene therapy Roctavian (valoctocogene roxaparvovec) to treat adults with severe hemophilia A. The FDA is now expected to decide by the end of March 2023 on whether…

The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital (genetic)…

Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy being developed by Pfizer and Sangamo Therapeutics. Pfizer, the study’s sponsor, had placed a voluntary pause…

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…

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…

Treatment with Hemlibra (emicizumab) improved joint health in people with hemophilia A participating in the Phase 3 HAVEN 3 trial, with the most pronounced improvements seen in those younger than 40 or in patients who had joints with frequent bleeds. Findings were detailed in the study “…

Older men with hemophilia report higher rates of anxiety, depression, and diabetes compared with the general U.S. population, a recent study reported. However, rates of heart-related health problems, such as myocardial infarction (heart attack) and coronary heart disease, were lower in hemophilia patients than in the general population. The…

Switching to Hemlibra (emicizumab) can add more than $100,000 a year to healthcare costs for people with hemophilia A in the U.S. who were previously using preventive replacement therapies, according to a new analysis. “Increased use of [Hemlibra] may cause an increase in economic burden for payers without…