Andrea Lobo, PhD,  science writer—

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to BE-101, Be Biopharma’s B-cell treatment candidate for hemophilia B. This designation is given to therapies that are meant to treat rare diseases, defined as conditions affecting fewer than 200,000 people in the U.S. It offers…

A European Medicines Agency (EMA) committee has issued a positive opinion recommending conditional marketing authorization of gene therapy Durveqtix (fidanacogene elaparvovec), developed by Pfizer, to treat adults with severe and moderately severe hemophilia B. The European Commission will review the recommendation from the EMA’s Committee for Medicinal…

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…

A generic version of DDAVP (desmopressin acetate), a therapy approved to control bleeding episodes in people with mild hemophilia A, is available in the U.S. Avenacy planned to begin shipping the therapy the week of April 15, according to a company press release. The generic is available…

When used long term, Novoeight (turoctocog alfa) safely and effectively prevented and treated bleeding episodes in people with hemophilia A in Japan, according to a real-world study. In fact, Novoeight successfully treated 91.4% of the reported bleeds, and kept bleeding under control in 85.7% of surgical procedures. Moreover,…

Intellia Therapeutics and Regeneron are planning to begin, by mid-year, the first-in-human trial testing a CRISPR/Cas9-based Factor 9 (F9) gene-editing therapy for people with hemophilia B. The announcement of the planned launch of the Phase 1 clinical trial, in an Intellia financial report, follows the recent approval by…

The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Baudax Bio’s investigational regulatory T-cell therapy TI-168 for the treatment of hemophilia A with inhibitors. Orphan drug status is given to therapies intended to treat rare conditions, defined as those affecting less than 200,000 people in…

The U.S. Food and Drug Administration (FDA) has cleared for laboratory use CRYOcheck Factor VIII Deficient Plasma with VWF, a way of detecting factor VIII (FVIII) deficiency in blood samples. Now available in the U.S., Precision Biologic’s manufactured plasma — the liquid part of blood that…

The National Hemophilia Foundation (NHF) has changed its name to the National Bleeding Disorders Foundation (NBDF) to reflect its support for people with inheritable blood and bleeding disorders other than hemophilia. The new name includes a new visual identity and logo. The foundation also unveiled a new tagline…

Dosing has begun in a Phase 2b clinical trial evaluating the safety and efficacy of SerpinPC, Centessa Pharmaceuticals’ investigational therapy for hemophilia. The open-label study, called PRESent-2 (NCT05789524), is part of the company’s registrational program for hemophilia B, which includes multiple clinical trials aiming to…