Severe hemophilia A patients using Esperoct (turoctocog alfa pegol) as a preventive treatment do well, with fewer bleeds or breakthrough bleeds, lower costs, and better life quality compared with other extended half-life (EHL) therapies, a modeling analysis suggested. The…
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Beqvez (fidanacogene elaparvovec-dzkt) was a gene therapy that was approved in the U.S. for the treatment of certain adults with moderate to severe hemophilia B. The one-time therapy, given via an into-the-vein or intravenous infusion, was marketed by Pfizer, who made the decision to halt its development and commercialization.
First in a series. Some people in the bleeding disorders community seem like they’re larger than life — not because they seek attention, but because their lives intersect with so many pivotal moments in our shared history. Lee Hall is one of those people. I know Hall through his advocacy…
The U.K.’s National Institute for Health and Care Excellence (NICE) has issued draft guidance recommending that England’s National Health Service (NHS) should not cover the gene therapy Hemgenix (etranacogene dezaparvovec) for adults with hemophilia B. The recommendation is based on uncertainty surrounding the long-term efficacy of the…
The U.S. Food and Drug Administration (FDA)’s request for two years of additional clinical trial data on Roctavian, a potential gene therapy for hemophilia A, took its developer — BioMarin — and others in the hemophilia community by surprise. That decision, announced in a complete response letter of…
A single dose of the investigational gene therapy SPK-8011 is safe and leads to durable production of factor VIII (FVIII) for up to three years, effectively lowering the frequency of spontaneous bleeds in men with hemophilia A, preliminary data from a Phase 1/2 trial show. The findings were…
Preventive treatment with factor VIII (FVIII) — the missing blood clotting protein in hemophilia A — was not fully effective at preventing MRI-detected joint changes in boys with severe disease, according to a Canadian study. Regular evaluation of joints by MRI or ultrasounds, however, may help in identifying those…
New Phase 3 trial results of CSL Behring’s hemophilia B therapy candidate Idelvion show promise in terms of reduction of bleeds, prolonged usage, and effectiveness at low doses. The poster detailing the data, “Long-term safety and efficacy of recombinant fusion protein linking coagulation factor IX with albumin (rIX-FP) in previously treated patients…
Women who are carriers for hemophilia — meaning they can pass the disease-causing mutation to their children — think a more individually tailored, information-rich healthcare approach could improve the experience of getting or being pregnant, a small study found. “Healthcare professionals could use these insights to adapt their consultations to…
Despite the COVID-19Â pandemic, BioMarin Pharmaceutical‘s application to the U.S. Food and Drug Administration (FDA) seeking approval of valoctocogene roxaparvovec, an experimental gene therapy for adults with severe hemophilia A, remains on track. During a conference call, the company also said that if approved, the…