Overview
- The FDA approved rusfertide for adults with polycythemia vera whose disease is not adequately controlled with existing therapies on Monday, August 31, 2026, making the drug available as a new option to lower excess red blood cells.
- Rusfertide is a first-in-class, once-weekly subcutaneous peptide that mimics the hormone hepcidin to limit iron availability and blunt red blood cell production.
- Approval was based mainly on the randomized phase 3 VERIFY trial of 293 phlebotomy-dependent patients, which showed 76.9% of rusfertide recipients avoided phlebotomy between weeks 20 and 32 versus 32.9% with placebo.
- Safety signals in VERIFY included frequent injection-site reactions and increased rates of anemia and fatigue, and two thrombotic events occurred in rusfertide-treated patients, so ongoing monitoring will be important.
- Because the trial allowed placebo patients to switch to rusfertide at week 32 and participants stayed on background therapies such as hydroxyurea or ruxolitinib, real-world use and longer follow-up will be needed to define longer-term benefits and risks.