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…
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In people with hemophilia A, certain immune markers in the blood may help predict patient responses to immune tolerance induction (ITI) — a treatment regimen used for individuals who develop inhibitors after factor replacement therapy — according to a new study by researchers in Brazil. Inhibitors, neutralizing antibodies…
Prophylactic, or preventive, use of Hemlibra (emicizumab) promotes joint health in people with hemophilia A, according to a new analysis of data from a Phase 3 trial. These findings were announced in the oral presentation “Bone and Joint Health Markers in Persons with Hemophilia A (PwHA)…
The U.S. Food and Drug Administration (FDA) has cleared the biotechnology company TeraImmune to launch a Phase 1/2a clinical trial testing TI-168, its regulatory T-cell therapy designed to eliminate inhibitors in people with hemophilia A. The upcoming trial is expected to enroll up to 18 people with congenital (genetic)…
The Phase 1/2 trial looking at the Factor VIII protein SHP656 as a long-acting therapeutic to treat hemophilia A did not meet a pre-defined once-weekly dosing criterion, according to Xenetic Biosciences and Shire, which are jointly developing the drug. The study demonstrated SHP656’s efficacy and pharmacokinetic data (the drug’s behavior once…
Antibody therapy Mim8 (denecimig) was well tolerated and provided effective bleed control in children with hemophilia A regardless of inhibitor status. That’s according to interim data from the now-complete Phase 3 FRONTIER3 study (NCT05306418), which enrolled children ages 1-11. These findings were consistent with recent data from…
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…
More than 80% of the men with severe hemophilia A treated with the investigational gene therapy Roctavian (valoctocogene roxaparvovec) in the Phase 3 GENEr8-1 trial remained bleed-free two years later, and nearly all were off standard preventive therapies. These findings were among new data presented at the recent 15th Annual…
Data from ongoing Phase 3 clinical trials show that Genentech’s Hemlibra (emicizumab-kxwh) prophylaxis — or preventive treatment — greatly outperforms current standard-of-care therapies, leading to significant reductions in the number of bleeds in hemophilia A patients regardless of inhibitor status. Promising results from these pivotal studies may lead…