Altuviiio (efanesoctocog alfa) has been approved in Japan and Taiwan as a treatment to prevent and control bleeding in people with hemophilia A. The Japanese Ministry of Health, Labor, and Welfare (MHLW) granted Altuviiio marketing authorization on Sept. 25, according to Sanofi, the company that markets…

About a month ago, my world was turned upside down when my kneecap dislocated while I was simply shifting my leg on a recliner. This unexpected incident brought back memories of my teenage years, when I underwent two surgeries to keep my kneecap in place. To add complexity to…

A person in Germany with severe hemophilia A has been treated with Roctavian (valoctocogene roxaparvovec-rvox), the first time the gene therapy has been used commercially in Europe, according to BioMarin Pharmaceutical, its developer. “Today represents an important milestone for the hemophilia community and for patients and…

Novo Nordisk’s Alhemo (concizumab injection) has been approved in Canada for hemophilia A patients, 12 years or older, who test positive for factor VIII inhibitors. The under-the-skin injectable therapy was recently approved by Health Canada for hemophilia B patients in the same age group with factor…

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…

Nearly a year has passed since Hurricane Ian wreaked havoc on southwest Florida, leaving my hometown of Punta Gorda and its neighboring communities in disrepair. As recovery efforts continue, the renovation of my condo has been stalled due to a failure by our association to reach an agreement with…

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…

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…

The U.S. Food and Drug Administration (FDA) has approved the one-time gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for eligible adults with severe hemophilia A.  Roctavian now becomes the first gene therapy to win FDA approval for the treatment of hemophilia A in the U.S. “Today’s approval of Roctavian builds…