Despite the COVID-19 pandemic, Catalyst Biosciences has successfully completed a Phase 1 trial of marzeptacog alfa activated (MarzAA), with results supporting the treatment as an on-demand option for bleeding episodes in people with hemophilia A or B. The study, MAA-102 (NCT04072237), was designed to assess…
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The first of my four alarms sounds at 6 a.m. Groggy, I roll over to turn it off, knowing I have 15 minutes until the next one goes off. I am a night owl and often let time slip away, crawling into bed after midnight. The next morning, I pay…
An ultrasound-mediated, non-viral gene therapy safely and effectively increased the levels of factor VIII (FVIII) — the missing clotting factor in hemophilia A — and lessened bleeding in a mouse model of the disease, a study shows. The delivery of a modified, improved version of the disease-associated F8 gene…
A single dose of the experimental gene therapy Roctavian (valoctocogene roxaparvovec) continues to prevent bleeds and the need for preventive treatment, or prophylaxis, over at least two years in men with severe hemophilia A, according to updated data from the Phase 3 GENEr8-1 trial. Notably, the activity of…
Children with severe hemophilia A with blood group O are not at increased risk of developing neutralizing antibodies against factor VIII (FVIII) replacement therapy, a study found. The study, “Blood Group O Does Not Increase the Risk of Inhibitors in Severe Haemophilia A: Data from the…
I’m happy to finally be an official member of the Father’s Day club. I’ve learned much from the way my own family raised me. It’s an uphill battle raising our daughter Cittie with the occasional bleed dragging me down. But seeing her mature so quickly…
The European Medicines Agency (EMA) has validated BioMarin Pharmaceutical’s new application seeking approval of its investigational one-time gene therapy Roctavian (valoctocogene roxaparvovec) for severe hemophilia A. The application was resubmitted to EMA following a request for longer follow-up data from the Phase 3 GENEr8-1 clinical trial (NCT03370913),…
Nearly two-thirds of children with severe hemophilia A given once-weekly Altuviiio (efanesoctocog alfa) in a Phase 3 clinical trial were completely free of bleeds over about one year of treatment. That’s according to new findings from the recently-completed XTEND-Kids study (NCT04759131), which were presented as late-breaking…
Nearly two-thirds of children with severe hemophilia A on preventive, or prophylactic, treatment with once-weekly Altuviiio (efanesoctocog alfa) in the yearlong XTEND-Kids study were free of bleeding episodes needing treatment. More than 80% in this Phase 3 clinical trial also were free of spontaneous bleedings and joint bleeds needing…
Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s…