Giroctocogene fitelparvovec, an investigational gene therapy for adults with hemophilia A, is generally well tolerated and superior to standard replacement therapy in reducing bleeds, according to new top-line results from a Phase 3 clinical trial. The open-label trial, dubbed AFFINE (NCT04370054) and sponsored by Pfizer, has…
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…
Hemgenix (etranacogene dezaparvovec), the first approved hemophilia B gene therapy, has been successfully administered to two patients in France, marking the first time the treatment has been given in a real-world setting in Europe. The gene therapy, originally developed by uniQure and marketed by CSL Behring, was…
Most caregivers or patients with hemophilia who were on preventive treatment were satisfied or very satisfied with the Florio Haemo smartphone app, even after prolonged use, a survey study suggests. The app lets patients track their infusions, bleeds, pain, and activities, and helps them manage their medication stock.
Novo Nordisk acquired the hemophilia A program and gene-editing technology rights of 2seventy Bio, with 2seventy staffers involved in the program joining Novo Nordisk and continuing to develop the technology to treat a range of diseases other than cancer. 2seventy could receive payments of up to $40…
Loma Linda University Health (LLUH) is now offering the gene therapies Hemgenix (etranacogene dezaparvovec) to people with hemophilia B and SPK-8011 (dirloctocogene samoparvovec), an experimental treatment for hemophilia A. LLUH’s Center of Excellence Hemophilia Program is structured to ensure that financial constraints don’t prevent access to the therapy,…
Note: This article was updated June 26, 2024, to clarify that the analysis of fitusiran in surgery was conducted in patients receiving prophylactic treatment with the therapy. The U.S. Food and Drug Administration (FDA) is reviewing a request to approve fitusiran, an under-the-skin injection therapy, as a treatment for adults…
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