In a real-world study in South Korea, preventive treatment with Adynovate (rurioctocog alfa pegol) was shown to be effective at preventing bleeding episodes among most children and adults with hemophilia A  — findings that line up with those from clinical testing. In fact, “76.1% of patients on prophylactic…

Switching to Hemlibra (emicizumab) was safe and effective in children with hemophilia A, including in those who had been minimally treated before or who had not been treated at all, a real-world study reported. Researchers noted that studies involving a larger number of patients are still warranted to…

A daily infusion of low-dose factor VIII (FVIII) improves trough levels in patients with severe hemophilia A — compared to infusions performed every other day — without increasing factor VIII consumption, or the amount of medication a patient must take, researchers have found. Trough levels are the lowest concentrations of a…

Genentech recently presented positive Phase 1/2 extension results for its investigational hemophilia A medicine, emicizumab, at the 2016 World Federation of Hemophilia (WFH) World Congress in Orlando, Florida. Emicizumab is being developed by Genentech (a Roche company) and Chugai Pharmaceutical. The latest data analysis continued to show a promising profile…

https://www.youtube.com/watch?v=0_EmvUb171I This video from Medic Tube gives an overview of the genetic causes behind hemophilia. The narrator explains that it’s very rare for females to have hemophilia because they need to inherit the faulty X gene from both of their parents, whereas males only need to inherit one…

Prophylactic (preventive) treatment with Hemlibra (emicizumab) safely and effectively lowers bleeding rates in children and adolescents with severe hemophilia A, according to a real-life study from Israel. The therapy’s effectiveness was independent of the presence of inhibitors (antibodies) against synthetic formulations of factor VIII (FVIII) — the missing…

An infant was diagnosed with severe hemophilia A after developing splenic injury (injury to the spleen), a rare condition among newborns. For the first time, however, a newborn with these conditions was successfully treated with recombinant factor VIII replacement therapy without the need for surgical intervention, doctors reported. The report, titled…

Bayer has filled a Biologics License Application (BLA) with the U.S. Food and Drug Administration (FDA) requesting marketing approval for its long-acting human Factor VIII therapy, BAY94-9027, to treat hemophilia A. The therapy is designed to allow for dosing at intervals as long as once every seven days. The…

The U.S. Food and Drug Administration granted breakthrough therapy designation to Genentech’s Hemlibra (emicizumab-kxwh) for people with hemophilia A without factor VIII inhibitors. Hemlibra is designed to bring together the proteins required to activate the natural coagulation cascade and restore the blood clotting process for patients with…

The European Medicines Agency has recommended marketing authorization for Shire’s Adynovi (BAX-855) as a treatment for adults and adolescents with hemophilia A. Approval would mean the therapy could be used twice a week to prevent bleeding episodes and to counter bleeding when episodes do occur. The U.S. Food and Drug Administration…