A pill that could replace infusions for a rare blood disease?

This trial compares an experimental oral drug, FXS6837, to the standard infusion eculizumab in people with paroxysmal nocturnal hemoglobinuria (PNH), a rare blood disorder that causes red blood cells to break apart. About 90 adults who have not previously used complement inhibitor treatments will take either FXS6837 capsules or receive eculizumab infusions for 24 weeks. The main goal is to see if FXS6837 can raise hemoglobin levels to normal or near-normal without needing blood transfusions, which would suggest it could be a more convenient treatment option.