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…

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…

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…

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…

A gene therapy from uniQure in early testing, called AMT-180, has the the potential to treat all hemophilia A patients, including those with inhibitors, according to the company. Hemophilia A is caused by missing or defective factor VIII (FVIII), a clotting protein. About 30% of patients with severe hemophilia A develop inhibitors, or…

Hemophilia A patients undergoing total hip or knee replacement/reconstruction surgeries have lesser blood loss and require a lower transfusion amount if treated with Cyklokapron (tranexamic acid), a study reports. They also are likely to experience less joint pain or swelling and better joint function, and have lower levels…

A natural anticoagulant protein called tissue factor pathway inhibitor (TFPI) is critical for defining the bleeding severity profiles of people with hemophilia A and B, researchers in France report. This discovery, which was found not to hold for healthy individuals, adds support to TFPI as an important element…