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…
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Transplants of embryonic stem (ES) cells that secrete the human factor VIII (FVIII) may be a promising therapeutic approach for patients with hemophilia A, according to an animal study conducted by researchers at the Nara Medical University School of Medicine in Japan. The study, “Therapeutic approaches for treating hemophilia…
Children and adults with hemophilia A in the United States may soon be treated with N8-GP (turoctocog alfa pegol), an engineered formulation of clotting factor VIII developed by Novo Nordisk. The therapy will be available under the brand name Esperoct. The U.S. Food and Drug Administration (FDA) approved N8-GP, or…
Jivi (BAY94-9027), a lab-made version of the blood clotting factor VIII (FVIII), has been approved by the European Commission for the prophylactic (preventative) treatment of hemophilia A in patients older than 12, Bayer announced. The recommended prophylaxis for Jivi administration is every five days, every…
A population-based pharmacokinetic approach for tailored treatment regimens of hemophilia A patients may contribute to an increase in treatment adherence and a decrease in annual bleeding episodes, a study shows. The study, “Clinical outcomes in hemophilia A patients undergoing tailoring of prophylaxis based on population-based pharmacokinetic dosing,” was published…
Long-term prophylactic (preventive) treatment with a fixed dose of Esperoct (turoctocog alfa pegol) — approved in the U.S. and the EU in 2019 — was effective in preventing and stopping bleeding in people with severe hemophilia A, according to data from two Phase 3 clinical trials. Findings from…
Hemlibra (emicizumab) is safe and effective to treat children younger than 12 who have hemophilia type A and are negative for antibodies against synthetic factor VIII, results from a Japanese clinical study show. The trial findings were reported in the study, “A multicentre, open‐label study of emicizumab given…
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…
Orsini Specialty Pharmacy has been selected to distribute the new gene therapy Roctavian (valoctocogene roxaparvovec-rvox) for use by individuals with hemophilia A, the company announced in a press release. The one-time treatment from BioMarin Pharmaceutical late last month became the first gene therapy ever approved by…
Almost two-thirds of men with moderate or severe hemophilia A have joint problems that cause pain and reduce their quality of life, even when they’re on factor replacement therapy to reduce the frequency of bleeding episodes, a real-world study shows. “Overall, the data indicate that joint problems still…