The European Medicines Agency (EMA) has granted orphan medicinal product status to SB-525, a clinical stage gene therapy candidate under development for the treatment of hemophilia A, according to Sangamo Therapeutics and Pfizer. The announcement coincides with the opening of patient enrollment for a Phase 1/2…

Jivi (damoctocog alfa pegol) has received approval from the U.S. Food and Drug Administration (FDA) that now extends to children with hemophilia A as young as 7 years old. Jivi had previously been authorized for hemophilia A patients 12 and older. The therapy, which is sold by Bayer,…

Researchers have found  that N8-GP (turoctocog alfa pegol) treatment improved the quality of life and satisfaction of children with severe hemophilia A. The study titled “Health-Related Quality of Life and Satisfaction of Children with Severe Hemophilia a Treated with Turoctocog Alfa Pegol (N8-GP),” appeared in the journal Blood.

A programmable mini-pump able to provide regular doses after surgery of FVIIa to hemophilia A patients with inhibitors — those resistant to standard FVIII replacement therapy — successfully maintained expected levels of blood clotting factors, a small study reports. Attending surgical nurses also voiced satisfaction with the pump’s use, reporting a…

Adults with mild and moderate hemophilia A have a substantial number of bleeds per year as well as joint problems and surgeries, revealing an unmet medical need in these patients, according to an interim analysis of the CHESS II study. The findings were shared at…

Ease of use, fear of the unknown, and healthcare providers’ advice are all major factors that play a role in whether people with hemophilia want to switch to new treatment options, a small study suggests. The study, “Patient Perspectives on Novel Treatments in Haemophilia: A Qualitative Study,”…

Have you ever seen a German playground? They’re infamous on the internet for appearing dangerous in an age of “safe” play spaces. Unlike most playgrounds we know of today, which are padded, carpeted, and lack sharp corners, German playgrounds are industrial looking structures built on regular ground.

The U.S. Food and Drug Administration (FDA) has accepted Bayer’s Biologics License Application (BLA) filing for BAY94-9027 to treat hemophilia A in adolescents starting at age 12, and in adults. A BLA is a marketing application that covers biological products, as opposed to chemically synthesized ones, and its…

Treatment with Esperoct led to stabilization and/or improvement of health-related quality of life (HRQoL) in patients with severe hemophilia A from all age groups, a study suggests. The study, “Health-related quality-of-life and treatment satisfaction of individuals with hemophilia A treated with turoctocog alfa pegol (N8-GP): a new recombinant…