Rusfertide is an investigational first-in-class hepcidin mimetic peptide being developed for adults with polycythemia vera. In March 2026, the FDA accepted its NDA and granted Priority Review with a PDUFA goal date in Q3 2026. It is therefore not yet an approved marketed therapy.
January and March 2026 drug pipeline updates included Phase 3 results, Fast Track designations and Priority Review decisions across multiple disease areas. Mavacamten, teplizumab-mzwv, anifrolumab, AXS-05, gedatolisib and AFTX-201 were among the programs with notable milestones.
Protagonist Therapeutics reported fourth quarter and full year 2025 financial results, with two NDAs submitted and multiple Phase 3 trials advancing. The company holds $646 million in cash and expects regulatory decisions in 2026.
The FDA has accepted New Drug Applications for rusfertide in polycythemia vera with Priority Review and tirabrutinib for relapsed/refractory primary CNS lymphoma under Accelerated Approval, with PDUFA dates set for Q3 2026 and December 2026 respectively.
The FDA has accepted the New Drug Application for rusfertide, a first-in-class hepcidin mimetic peptide for polycythemia vera, and granted Priority Review with a PDUFA goal date in the third quarter of 2026.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT05210790 |
A Phase 3 Study of Rusfertide in Patients With Polycythemia Vera |
ACTIVE_NOT_RECRUITING | PHASE3 |
