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 protein missing or defective in people with hemophilia A, called blood coagulation factor VIII (FVIII), is able on its own to stimulate immune responses in a mouse model, a study suggests. This immune response to FVIII is dependent on a set of distinct immune cells within the spleen,…

A significant proportion of bleeds in people with hemophilia A are left untreated, according to an analysis of data from an observational study. Because only treated bleeds are typically reported in clinical trials, these findings suggested the full disease burden is not adequately captured in many studies. As such,…

Gene therapy has the potential to stop hemophilia in its tracks. But such therapy will likely be costly, and can carry risks that have generated some apprehension among patients who could benefit most from it. That’s the message from hemophilia patient advocates who spoke at the 2018 NORD Rare Diseases…

The U.K.’s Medicines and Healthcare products Regulatory Agency (MHRA) has approved Hympavzi (marstacimab) for the prevention of bleeding episodes in people with both hemophilia A and hemophilia B, ages 12 and older. The therapy, already approved in the U.S. and the European Union, is designed to…

Eligible patients with severe hemophilia A in Germany should now have improved access to Roctavian (valoctocogene roxaparvovec-rvox) — a gene therapy intended to reduce bleed risk in certain adults with the bleeding disorder — given a new agreement between its developer BioMarin Pharmaceutical and the German National Association…