Life when you’re married to someone with severe hemophilia B often feels unpredictable. One moment, everything seems fine, and the next, a spontaneous bleed or unexpected health concern throws everything into chaos. Over the years, I’ve learned to stay on high alert, always ready to shift gears at a…
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The European Medicines Agency (EMA) has accepted BioMarin Pharmaceutical’s request for accelerated assessment of Roctavian, the company’s investigational gene therapy for severe hemophilia A, for a second time. Last year, BioMarin’s regulatory applications for Roctavian (valoctocogene roxaparvovec) got rejected both in Europe and in the…
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Data Supports Potential for Personalized Hemophilia A Therapy with Nuwiq, WFH Presentations Show
Since every hemophilia A patient responds differently to therapies, researchers are working to develop personalized preventive (prophylaxis) strategies with Nuwiq (antihemophilic factor [recombinant].) Octapharma, the treatment’s manufacturer, presented data showing that an individual’s pharmacokinetic (PK) profile (how the therapy works once inside the body) can be used to possibly…
A report describes — for the first time, according to study authors — the case of a girl who has both hemophilia A and C. To date, only 10 such cases had been documented in medical literature, all occurring in men. The case report authors highlight the importance of…
Sometimes I forget I have hemophilia. I know that sounds silly, particularly after years of struggle to obtain a diagnosis, yet it’s true. This forgetting is great when I’m healthy and have no issues. There are times, however, when it’s imperative that I remember I have hemophilia. Sometimes I…
Health Canada has approved Altuviiio (efanesoctocog alfa) to treat children, adolescents, and adults with hemophilia A, with the aim of preventing and controlling bleeds, including those occurring around the time of surgery. “This approval represents important progress in the treatment of patients with hemophilia A in Canada,” Stephanie…
Men with moderate-to-severe hemophilia A are again being enrolled in the Phase 3 AFFINE trial, which is testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy being developed by Pfizer and Sangamo Therapeutics. Pfizer, the study’s sponsor, had placed a voluntary pause…
Recognizing risk factors of thrombosis and carefully monitoring medication doses are crucial for minimizing thrombotic complications in older patients with acquired hemophilia and serious illnesses like cardiovascular disease, a report describing four cases says. The study, “Acquired Haemophilia A. Which is the best therapeutic choice in older adults? Single…
The U.S. Food and Drug Administration (FDA) has given regenerative medicine advanced therapy (RMAT) designation to Roctavian, an investigational gene therapy by BioMarin Pharmaceutical for severe hemophilia A. The designation is “a critical program to advance the efficient development and regulatory review of regenerative medicine products that have the…
BioMarin Pharmaceuticals presented positive interim data from an open-label Phase 1/2 clinical trial of BMN 270, an investigational gene therapy for severe hemophilia A. The promising results, presented at the XXXII International Congress of the World Federation of Hemophilia (WFH), showed that patients had improved and sustained clotting function. BMN 270 is designed to alter…