Takeda and Protagonist Therapeutics have announced the submission of a New Drug Application (NDA) to the US FDA for rusfertide, a first-in-class therapy aimed at adults with polycythemia vera (PV).
The investigational subcutaneous hepcidin mimetic peptide is designed to regulate iron and red blood cell production, controlling hematocrit levels in patients with the rare blood disorder.
“This is an important milestone toward our goal of addressing critical gaps that patients living with polycythemia vera face today,” said Teresa Bitetti, President, Global Oncology Business Unit, Takeda.
“The comprehensive VERIFY study data underscore rusfertide’s strong clinical profile and potential to provide sustained hematocrit control while reducing phlebotomy and symptom burden. Our collaboration with Protagonist exemplifies how partnerships can advance innovative science, with a focus on making a meaningful difference for patients.”
The NDA submission follows positive Phase 3 VERIFY trial results showing that rusfertide, combined with standard care, achieved durable hematocrit control, reduced the need for phlebotomy, and improved patient-reported outcomes. The drug also demonstrated consistent benefits in the earlier Phase 2 REVIVE study.
Rusfertide has already received Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation from the FDA, highlighting its potential to improve on existing PV therapies.
“Rusfertide has the potential to redefine the treatment paradigm for PV by offering patients a novel, first-in-class erythrocytosis-specific therapy that significantly reduces or eliminates the need for frequent phlebotomy,” said Dinesh V. Patel, Protagonist President and CEO.
"Submitting this NDA marks a major inflection point in the decade long journey that started with a hepcidin mimetic program at Protagonist. Rusfertide has practice-changing potential that could become standard of care for patients with PV who currently rely on burdensome and often ineffective therapies.”
