The U.S. Food and Drug Administration (FDA) has cleared clinical testing in adults of BE-101, a B-cell treatment candidate for hemophilia B being developed by Be Biopharma. The Phase 1/2 trial, called BeCoMe-9, will be a first-in-human study designed to assess the safety and preliminary efficacy of BE-101…

Over seven years of follow-up, a single dose of Roctavian (valoctocogene roxaparvovec-rvox) sustainably reduced the yearly bleeding rate in adults with severe hemophilia A by up to 96%. That’s according to an update on a Phase 1/2 clinical trial (NCT02576795), launched in the U.K. in 2015, that’s…

CSL Behring has issued a voluntary, pharmacy level recall covering one batch of Mononine, its long-standing treatment for preventing and controlling bleedings in people with hemophilia B that is soon to be discontinued. According to the company’s statement, the recall is a precautionary measure due to…

For children with severe hemophilia A who develop inhibitors — neutralizing antibodies that reduce the effectiveness of standard therapies — starting treatment to eliminate inhibitors earlier is associated with a higher chance of success, a recent study reports. The study, “Low-dose immune tolerance induction therapy in…

Cell therapies may be a feasible way of achieving a long-term correction of bleeding disorders, preclinical data shared by Sigilon Therapeutics show. A single implant of Sigilon’s engineered cells given to mice enabled durable production of therapeutic levels of blood clotting factors and normalized bleeding time. The technology uses the…

A single dose of Roctavian (valoctocogene roxaparvovec-rvox) nearly zeroed out the number of bleeds and the need for prophylaxis, or preventive therapy, for as long as seven years — the longest follow-up to date for any hemophilia A gene therapy clinical trial. While most of the 12 patients…

Obizur (susoctocog alfa) is an effective and safe therapeutic option for the treatment of severe bleeding episodes in people with acquired hemophilia A, a study in Japanese patients showed. The study “supports the use of [Obizur] as a novel therapy in the clinical management of patients with [acquired…

The U.K. Medicines and Healthcare products Regulatory Agency (MHRA) has approved efanesoctocog alfa — to be sold under the brand name Altuvoct —  as a treatment for moderate or severe hemophilia A in adults and children, ages 2 and older. Approved in the European Union in June…

Last week, I was on Facebook and a post I had been dreaming about appeared on my timeline. The U.S. Food and Drug Administration has approved a new therapy, Hemlibra (emicizumab-kxwh), for patients with hemophilia A and inhibitors. It’s the first new medicine for inhibitors in almost…