News

The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B. The recommendation is based on uncertainty surrounding the long-term efficacy of the…

Roctavian (valoctocogene roxaparvovec-rvox), the first gene therapy approved for adults with severe hemophilia A, is expected to be widely available in the U.S. this month, according to its developer, BioMarin. BioMarin also is working with authorities in Germany, Italy, and France to secure market access to…

Roche is initiating a Phase 3 clinical trial to test the experimental gene therapy SPK-8011 in people with hemophilia A. The company announced the move in its 2023 half-year results. At the same time, however, Roche is discontinuing the development of another investigational hemophilia A gene therapy…

Nuwiq (simoctocog alfa) effectively prevented and controlled bleeds in previously untreated people with severe hemophilia A, according to data from a Phase 3 clinical trial. Full trial results were reported in the study, “Simoctocog alfa (Nuwiq) in previously untreated patients with severe haemophilia A — Final efficacy…

The approval by the U.S. Food and Drug Administration (FDA) of BioMarin Pharmaceutical’s Roctavian (valoctocogene roxaparvovec-rvox) late last month means that eligible adults with severe hemophilia A will soon have access to a gene therapy for the first time. “Adults with severe hemophilia A live with several limitations,…

Preventive treatment with Hemlibra (emicizumab) was not considered cost-effective at its current price compared with standard replacement therapies in adults with mild or moderate hemophilia A without inhibitors, according to a modeling study. The cost of Hemlibra would need to drop by at least 35% to become cost-effective…

Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by individuals with hemophilia A, the company announced in a press release. The one-time treatment from BioMarin Pharmaceutical late last month became the first gene therapy ever approved by…

Dosing has begun in a Phase 2b clinical trial evaluating the safety and efficacy of SerpinPC, Centessa Pharmaceuticals’ investigational therapy for hemophilia. The open-label study, called PRESent-2 (NCT05789524), is part of the company’s registrational program for hemophilia B, which includes multiple clinical trials aiming to…

Switching to a different clotting factor VIII (FVIII) product was not linked to a greater risk of developing neutralizing antibodies against FVIII, or FVIII inhibitors, in people with hemophilia A, a recent study reported. Changing treatment also had no impact on a patient’s immune profile or on therapy efficacy.

Regulatory agencies in the U.S. and Europe are reviewing applications seeking the approval of fidanacogene elaparvovec, an experimental one-time gene therapy being developed by Pfizer for the treatment of hemophilia B in adults. The U.S. Food and Drug Administration (FDA) expects to complete the review process and…