Takeda and Protagonist Therapeutics announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) seeking approval of rusfertide for the treatment of adults with polycythemia vera (PV). Rusfertide is an investigational first-in-class subcutaneously administered hepcidin Mimetic peptide designed to regulate iron homeostasis and red blood cell production to control hematocrit levels in patients with PV. This NDA submission follows the positive 32-week primary analysis and 52-week results from the Phase 3 global randomized, placebo-controlled VERIFY study (NCT05210790), in which rusfertide met the primary endpoint and all four key secondary endpoints, as well as the Phase 2 REVIVE study (NCT04057040).
In the VERIFY study, patients receiving rusfertide plus standard of care therapy demonstrated a substantially higher response rate compared to placebo plus standard of care, including durable hematocrit control, a reduction in phlebotomy requirements and improvement in pre-specified patient reported outcome (PRO) endpoints. Rusfertide has received Breakthrough Therapy Designation, a regulatory milestone recognizing the potential of Rusfertide to offer a substantial improvement over available therapies, as well as Orphan Drug Designation and Fast Track Designation from the U.S. FDA. Rusfertide is a first-in-class investigational subcutaneous treatment that mimics the action of hepcidin, a natural hormone that regulates iron homeostasis and red Blood cell production.
By targeting the underlying mechanism of iron dysregulation in polycythemia vera, rusfertide aims to reduce excess red blood cell production and help patients achieve sustained hematocrit control. Rusfertide is administered once weekly via subcutaneous self-injection and has been generally well-tolerated in clinical trials to date.
