News

Brazilian hemophilia A patients who are 19 or older had significantly higher median care costs per patient per year than younger patients, a study reported. The study also revealed an increase in median costs for patients with more severe disease — that is, those who have less than 1%…

In an effort to cut costs and boost profitability, Biomarin Pharmaceutical is limiting commercial development of hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox) to three countries: the U.S., Germany, and Italy. The company has struggled to turn a profit from the gene therapy, which was approved in…

Prophylactic treatment for hemophilia can help people with the disease avoid unemployment due to disability, a study reports. Hemophilia has a negative effect on a person’s employability. A 2022 global report by the World Federation of Hemophilia indicates about 18% of hemophilia patients see their employment status impacted…

A 39-year-old man from Arizona became the first patient in Nevada to receive Hemgenix (etranacogene dezaparvovec), the first gene therapy to be approved for hemophilia B. The infusion was administered at the Cure 4 The Kids Foundation, under the supervision of Aimee Foord, director of the foundation’s bleeding…

Durveqtix (fidanacogene elaparvovec), a gene therapy for adults with severe and moderately severe hemophilia B, has been granted conditional marketing authorization by regulators in the European Union. The decision by the European Commission follows a positive recommendation on the …

Note: This story was updated July 30, 2024, to clarify patients participating in AFFINE had moderately severe to severe disease, and that a secondary study goal involved the mean treated annualized bleeding rate. Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well…

Nearly two-thirds of children with severe hemophilia A on preventive, or prophylactic, treatment with once-weekly Altuviiio (efanesoctocog alfa) in the yearlong XTEND-Kids study were free of bleeding episodes needing treatment. More than 80% in this Phase 3 clinical trial also were free of spontaneous bleedings and joint bleeds needing…

The Canadian Agency for Drugs and Technologies in Health’s (CADTH) Canadian Drug Expert Committee (CDEC) recommended reimbursement for hemophilia B gene therapy Hemgenix (etranacogene dezaparvovec), according to the therapy’s developer, CSL Behring Canada. Hemgenix in 2023 became the first gene therapy in the country approved for hemophilia…

TU7710, an experimental treatment for managing bleeds in people with hemophilia who have inhibitors, demonstrated an average half-life that ranged from 10.4 to 16.6 hours across different doses in a Phase 1a study with healthy volunteers. This extended half-life, that is, the time it takes for a medication’s levels…

In a reversal of a previous recommendation, the U.K.’s National Institute for Health and Care Excellence (NICE) has recommended that the National Health Service (NHS), England’s publicly funded health insurance program, should offer reimbursement for Hemgenix (etranacogene dezaparvovec) to eligible adults with hemophilia B. Reimbursement would come through…