A gene therapy-based factor VIIa (FVIIa) given as prophylactic, or preventative, treatment was seen to produce specific factor levels needed to reduce the frequency of spontaneous bleeding episodes in a rat model of hemophilia A. According to scientists, this work may be a first step in developing therapies that…

BioMarin Pharmaceuticals is moving forward with BMN 270, an experimental gene therapy for the treatment of hemophilia A, and is preparing new clinical trials expected to begin this year. The European Medicines Agency (EMA)’s Committee for Advanced Therapies (CAT) and its Committee for Medicinal Products for Human Use (CHMP) agreed that BMN 270…

Shire has acquired worldwide rights to the antibody-based therapy that Novimmune developed as a treatment for hemophilia A. Geneva-based Novimmune creates therapies for inflammatory diseases, autoimmune diseases and cancer. It uses antibodies that target tumor-associated proteins and the immune checkpoint protein CD47. Shire, which is based in Dublin, is a global…

Measuring levels of YKL-40 — a protein associated with inflammation and tissue remodeling — may help detect hemophilic arthropathy, the joint damage caused by repeated bleeding, in people with severe hemophilia A, a study found. The researchers said the results “suggest that YKL-40 has potential as a biomarker, with its…

Efanesoctocog alfa, a long-lasting factor VIII (FVIII) replacement therapy, has now been approved in the European Union for hemophilia A. It will be marketed under the brand name Altuvoct. Altuvoct can be used as an on-demand treatment for bleeding episodes, a once-weekly prophylactic (preventive) therapy to reduce the…

MRI screening can identify subclinical joint bleeding in some patients with severe hemophilia A on preventive treatment, according to a study in the Netherlands. Subclinical bleeds occur without symptoms and usually cannot be detected by standard physical and ultrasound examinations. “These MRI findings support the hypothesis that subclinical…

Chugai Pharmaceutical recently published data of a Phase I clinical study on the antibody emicizumab. The study, “Factor VIII–Mimetic Function of Humanized Bispecific Antibody in Hemophilia A,” was published in The New England Journal of Medicine. The study revealed possible benefits from emicizumab  for the prevention of bleeding in…

The incidence of acquired hemophilia A (AHA) was nearly twice as high in Hong Kong than in Caucasian populations previously observed, a study found. Patients in Hong Kong tended to be older and have co-existing medical conditions, with age being a significant predictor of mortality and failure to achieve…

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…

Small vesicles in the saliva of people with severe hemophilia A may explain why these patients rarely have bleeds in their mouth and throat. The vesicles contain a protein complex that’s able to induce blood coagulation in these patients, thereby reducing bleeding. The findings may help “develop innovative approaches…