Takeda and Protagonist say FDA accepts rusfertide NDA, sets Q3 2026 PDUFA date

Takeda Pharmaceutical Company and Protagonist Therapeutics said the U.S. Food and Drug Administration has accepted the New Drug Application for rusfertide and granted the investigational therapy Priority Review, setting a PDUFA goal date in the third quarter of 2026. The joint announcement was datelined OSAKA, Japan; CAMBRIDGE, Massachusetts; and NEWARK, California and distributed via Business Wire on March 2, 2026.

Rusfertide is described by the developers as a first-in-class hepcidin mimetic peptide therapeutic intended for adults with polycythemia vera, a blood cancer marked by overproduction of red blood cells. The drug is presented as subcutaneously administered and is designed to regulate iron homeostasis and red blood cell production to control hematocrit, the standard measure clinicians use to reduce the elevated risk of life-threatening thrombotic events in these patients. Maintaining hematocrit below 45 percent is the primary treatment objective in polycythemia vera.

The NDA is supported primarily by data from the phase 3 VERIFY trial (NCT05210790), which evaluated rusfertide in 293 patients with polycythemia vera, and includes results from the phase 2 REVIVE study (NCT04057040) and the long-term extension THRIVE trial (NCT06033586). Takeda and Protagonist said the filing and Priority Review follow earlier FDA designations for the program, which include Breakthrough Therapy, Orphan Drug and Fast Track status.

Protagonist chief executive Dinesh V. Patel, Ph.D., framed the milestone as the culmination of a long development effort. “Rusfertide exemplifies Protagonist’s end-to-end expertise, from exploring a novel hepcidin mimetic mechanism to address unmet needs in polycythemia vera to discovering the peptide and driving its clinical development through NDA filing. We are very pleased with the FDA granting rusfertide Priority Review and look forward to its potential approval in 2026,” he said in the companies’ release. Patel added, “We have identified a great partner in Takeda as rusfertide progresses toward this milestone, thereby bringing a successful closure to our more than decade-long journey from concept-to-commercialization.”

Takeda emphasized the unmet clinical need in PV and the potential patient impact if the drug is approved. Andy Plump, MD, PhD, Takeda’s president of research and development, said in the company statement, “There is an urgent need for innovative treatment options in polycythemia vera, where patients currently [have] limited therapeutic choices to control their hematocrit and significant symptom burden. The FDA’s acceptance of our NDA brings us closer to potentially offering a first-in-class therapy that could meaningfully improve clinical outcomes and quality of life.”

Developers have highlighted a potential practical benefit of rusfertide: reducing or eliminating the frequent phlebotomy sessions many PV patients undergo to lower hematocrit. If the FDA maintains the PDUFA timetable and grants approval, rusfertide would join a small class of targeted therapies for PV and could alter standard approaches to hematocrit management.

Takeda trades on the Tokyo Stock Exchange and New York Stock Exchange (TSE:4502 / NYSE:TAK) and Protagonist is listed on NASDAQ (PTGX). The FDA review now moves to its Priority Review timeline, with a regulatory decision expected by the third quarter of 2026.