News

Marstacimab up for approval for hemophilia in US, Europe

The once-weekly injection therapy marstacimab is being considered for approval in both the U.S. and Europe for people with hemophilia A and hemophilia B who don’t have inhibitors. The U.S. Food and Drug Administration (FDA) is expected to decide on it in late 2024, while a decision from the European…

German health insurance fund to reimburse Roctavian for hem A

Eligible patients with severe hemophilia A in Germany should now have improved access to Roctavian (valoctocogene roxaparvovec-rvox) — a gene therapy intended to reduce bleed risk in certain adults with the bleeding disorder — given a new agreement between its developer BioMarin Pharmaceutical and the German National Association…

Antibody test for Roctavian cleared under new, stringent EU rules

AAV5 DetectCDx, a companion diagnostic test used to determine patient eligibility for the hemophilia A gene therapy Roctavian (valoctocogene roxaparvovec-rvox), has been cleared for use under more stringent European Union rules coming into effect in a few years. Specifically, the test gained the Conformité Européenne (CE) mark under…

Genevant, Novo Nordisk partner on gene-editing treatment for hem A

Genevant Sciences is teaming up with Novo Nordisk to develop a gene-editing treatment for hemophilia A that combines Genevant’s proprietary lipid nanoparticle (LNP) platform with cutting-edge mRNA-based megaTAL technology. The partners will seek to advance a treatment involving gene editing — used to correct, add, or delete…