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…

My husband, Jared, is a capable person. He has a degree in business management and excelled in elementary and high school, consistently ranking at the top of his class. He’s an accomplished swimmer, an excellent cook, a decent handyman, and a wonderful dad. I’m not biased as his…

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…

When dealing with health issues, does when you were born matter? For those living with hemophilia, the answer is a resounding yes. Having personally experienced the challenges of living with bleeding disorders and survived multiple life-threatening situations, I can’t help but reflect on how different my medical journey might…

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…

One fall evening in 1998, I looked around my dining room table in Houston and saw the faces of men and women who’d become some of my dearest friends over the past year. Laughter flooded the room as we stood in awe at what we’d accomplished. I stood, raised a…

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 first adult with hemophilia B in the U.S. has been treated with commercially available Hemgenix (etranacogene dezaparvovec), a recently approved and first gene therapy for the disease, CSL Behring announced. “As part of our longtime promise to patients, CSL Behring is thrilled to mark this latest milestone…

The U.S. Food and Drug Administration (FDA) has agreed to review Medexus Pharmaceuticals’ request to approve Ixinity (trenonacog alfa) for children with hemophilia B younger than 12. Ixinity is already approved by the FDA for adults and adolescents 12 and older. The therapy is indicated for use…