Steve Bryson, PhD, science writer —

People in France with severe hemophilia A who switched to Elocta (efmoroctocog alfa) — an extended half-life replacement therapy — had fewer infusions and a reduced rate of joint bleeds, a real-world study reports. Notably, those who switched to Elocta had a higher bleeding rate, including joint bleeds,…

One year of treatment with Elocta (efmoroctocog alfa) — an extended half-life replacement therapy known as Eloctate in the U.S. — reduced the frequency of bleeds in the elbows and knees of adults with severe hemophilia A, a Spanish study showed. Reduced pain intensity in the elbows also…

The gene therapy BBM-H901 increased factor IX activity in 10 men with moderate to severe hemophilia B, while reducing bleeds and the use of FIX replacement therapy over one year, according to results of a pilot Phase 1 trial. “The study was the first clinical trial conducted by Belief BioMed…

The U.S. Food and Drug Administration (FDA) has accepted — under priority review — a marketing application for EtranaDez (etranacogene dezaparvovec), an investigational gene therapy for adults with hemophilia B. The FDA grants priority review to investigational therapies designed to treat serious medical conditions. If approved, the treatment…

Treatment with abatacept, a medication approved for rheumatoid arthritis, prevented the formation of inhibitors against hemophilia A replacement therapies in a rat model, a study demonstrates. These findings support the use of abatacept to prevent immune responses against human proteins in a preclinical setting, the researchers noted. The study,…

The U.S. Food and Drug Administration (FDA) has lifted a clinical hold on the Phase 3 trial testing the safety and effectiveness of SB-525 (giroctocogene fitelparvovec), an experimental gene therapy for hemophilia A. Pfizer, which is developing the therapy along with Sangamo Therapeutics, expects to begin dosing…

Eptacog beta was safe and almost 100% effective at stopping bleeds within 24 hours in boys ages 12 and younger with hemophilia A or B, according to final data from the Phase 3 PERSEPT 2 trial. According to researchers, this bypass agent may be an important option for…

Factor VIII (FVIII) levels in the bloodstream of men with severe hemophilia A treated with the investigational gene therapy Roctavian appear to be influenced by regulatory molecules involved in F8 gene activity and FVIII protein folding and release, a study reveals. Researchers also found a dose-dependent relationship between…

Hemophilia B patients who switched to Rebinyn (nonacog beta pegol), an extended half-life factor IX (FIX) replacement therapy, had similar or better responses than with their previous standard or extended half-life FIX replacement therapies, a real-world study in Canada suggested. The study, “Switching to nonacog beta…

Elevated levels of the immune signaling protein BAFF were found in the bloodstream of boys who developed neutralizing antibodies against FVIII replacement therapy, a standard treatment in hemophilia A, a study reported for the first time. Changes in the gene that encodes the BAFF protein, involved in the growth…