The hemophilia A treatment Nuwiq (simoctocog alfa) is safe and effective for patients with severe disease, both when used on demand or as a preventative treatment, researchers determined in a recent report. The researchers, who analyzed available data on Nuwuq, detailed their finding in the article, “Efficacy and…

The European Medicines Agency (EMA) Pharmacovigilance Risk Assessment Committee (PRAC) has initiated a review of factor VIII-containing medicines, to evaluate the risk of inhibitor protein development in patients starting treatment for hemophilia A, according to a press release. This review, which will cover all medicines containing factor VIII authorized in the…

Scientists found the immune signaling protein BAFF can promote the formation of neutralizing antibodies, or inhibitors, against blood clotting factor VIII (FVIII), which can lower the effectiveness of FVIII replacement therapies used to treat people with hemophilia A.  They also discovered that hemophilia A…

In boys and men with severe hemophilia A, starting prophylaxis — preventive treatment given regularly to reduce bleeding — early and maintaining regular physical activity were each linked to a lower risk of hemophilic arthropathy, a progressive form of joint damage caused by repeated bleeding, according to a real-world…

Health Canada, the agency that oversees health decisions and regulations in Canada, has approved Afstyla (lonoctocog alfa)  for the treatment and prevention of bleeding in adults and children with hemophilia A. In May, the U.S. Food and Drug Administration (FDA) approved Afstyla for the same indications. Developed by CSL Behring, Afstyla is…

Preventive treatment with Hemlibra (emicizumab) improves health-related quality of life (HQRoL) in children with hemophilia A who develop inhibitors, or neutralizing antibodies, against engineered factor VIII (FVIII), an essential blood-clotting protein, a new analysis of a Phase 3 trial shows. Such Hemlibra therapy also reduces the burden on…

A single infusion of the experimental gene therapy GS001 safely increased the levels of factor VIII (FVIII) — the missing clotting protein in hemophilia A  — for almost three years, effectively reducing bleeds and the need for replacement therapies at doses much lower than those used in currently approved…

Treatment of hemophilia A with bypassing agents is associated with significantly higher costs than with factor replacement therapy, according to a real-world study in the U.S. The study, “Healthcare costs among patients with hemophilia A treated with factor replacement or bypassing agents,” appeared in…

Sigilon Therapeutics’ candidate cell therapy for hemophilia A, called SIG-001, delivers sustained production of factor VIII for over six months and corrects bleeding in a hemophilia A mouse model. These results were shared at the 2019 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting, in a…

Long-term prophylaxis, or preventive treatment with Adynovate continues to safely reduce bleeding events over multiple years in children and adults with severe hemophilia A without causing neutralizing antibodies, a Phase 3b trial found. The study, “Long‐term safety and efficacy results from the phase…