The U.S. Food and Drug Administration (FDA) has cleared CRYOcheck Chromogenic Factor IX assay, a test intended to be used by clinical labs to detect a deficiency in factor IX (FIX), the cause of hemophilia B. The chromogenic, or color-based, test developed by Precision BioLogic is reported…

The U.S. Food and Drug Administration (FDA) has scheduled its inspection of the facility in California where BioMarin Pharmaceutical would produce the gene therapy Roctavian (valoctocogene roxaparvovec) if it’s approved. The agency is reviewing a biologics license application (BLA) from BioMarin that seeks approval of Roctavian to…

Patients with moderate to severe hemophilia A who switched to Kovaltry (octocog alfa) showed no worsening of their annual bleeding rate, according to real-world data from a Phase 4 study. Adhering to the therapy remained high and patients reported high treatment satisfaction. Overall, the study “confirms and extends clinical trial…

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…

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 “…

Most people with moderate or mild hemophilia A who received preventive treatment with Hemlibra (emicizumab) in the HAVEN 6 clinical trial had no bleeds requiring treatment over a median follow-up of over a year, new data show. The findings were presented at the 30th International Society on Thrombosis and…

An immersive multimedia exhibit, which features portraits by renowned photographer Rankin, seeks to educate about life with hemophilia and the disease community’s hope for the future. The exhibit, called “Portraits of Progress,” is a collaboration between biotherapeutics company CSL Behring and Rankin, a British photographer who has done…

Roche has extended its partnership with the World Federation of Hemophilia (WFH) to expand access to treatments for hemophilia through the end of 2028, the company announced. Under the partnership, Roche will continue to provide its prophylactic (preventive) treatment for hemophilia A to the WFH’s Humanitarian…

The rate of intracranial hemorrhage (ICH) — a bleed in the brain — is higher in children and adults with hemophilia than in the general population, an Italian study suggests. ICH rates were particularly high among adults with mild disease and with high blood pressure. Most patients received only…

More than 80% of the men with severe hemophilia A treated with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) in the Phase 3 GENEr8-1 trial remained bleed-free two years later, and nearly all were off standard preventive therapies. These findings were among new data presented at the recent 15th Annual…