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…

In my five-plus years of writing columns for Hemophilia News Today, I’ve missed two weeks. Many times, I feel that I’m repeating myself as I struggle to write something that matters to the bleeding disorders community. I share daily experiences as I navigate bad and good moments with…

A researcher at the University at Buffalo has won a three-year grant worth $1.5 million to develop a way to tame a patient’s immune system and prevent it from rejecting factor VIII as a replacement therapy for hemophilia A. “My interest in hemophilia started when I read a mother’s…

People with hemophilia A who start on preventive treatment in the first years of life generally report better health-related quality of life and joint health, a study reports. “Our results indicate that delayed start of prophylaxis in an older cohort with severe [hemophilia A] can still achieve excellent bleeding…

Clinical trials for hemophilia are essential to advance medical knowledge and develop treatments. But for decades, trials related to hemophilia enrolled only men, given the assumption that women were only carriers of the disease. Consequently, women with the disorder were denied the opportunity to participate in…