Jivi was safe and effective at preventing and treating bleeds for more than five years in boys under age 12 with severe hemophilia A, according to long-term data from a Phase 3 trial’s extension study. Data analysis also showed that most of…
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Sometimes I feel like I don’t belong. I’m a misfit, an imposter. I feel fine and haven’t had a bleed in almost a month. Do I really have hemophilia? Do I really need to infuse every three…
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…
The U.S. Food and Drug Administration (FDA) has granted fast track designation to efanesoctocog alfa, an investigational factor VIII (FVIII) replacement therapy for treating hemophilia A, Sanofi, one of the therapy’s developers, announced in a press release. This designation is given to accelerate the development and review of medications…
When given as a prophylactic, or preventive therapy, Advate worked better than on-demand treatment at lowering the number of spontaneous bleeds in people with moderate or severe hemophilia A, according to seven-year data from a real-world study. A separate analysis of the study, called AHEAD International (NCT02078427),…
A few years ago, I went to a tattoo convention. Before I could enter, I was required to sign a liability release. The form said that people with bleeding disorders (and a long list of other conditions) should not get tattoos. Needless to say, I am not always a…
A single dose of the experimental gene therapy SPK-8016 showed promising safety and efficacy in lowering the frequency of bleeds and the need for infusions in men with severe hemophilia A. These preliminary findings from an ongoing Phase 1/2 trial (NCT03734588) were presented by Spencer Sullivan, MD,…
The investigational medication marzeptacog alfa activated (MarzAA) showed promise at stopping bleeding in animal models of hemophilia. The findings were presented in two posters at the Annual Congress of the European Association for Haemophilia and Allied Disorders (EAHAD) by researchers from Catalyst Biosciences, which is developing MarzAA. MarzAA is…
Kovaltry (octocog alfa) is effective and safe as a preventive treatment for children with hemophilia A, a European real-world study reports. The study, “Real-world evidence on Kovaltry (81-8973) in children with moderate or severe hemophilia A in Europe: a…
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…