The United States Food and Drug Administration (FDA) accepted a new drug application (NDA) for rusfertide for the treatment of adults with polycythemia vera. In addition to priority review, the FDA granted Breakthrough Therapy Designation, Orphan Drug Designation, and Fast Track Designation for the medication.
Results from the phase 3 VERIFY study supported the filing, showing that the drug improved hematocrit levels and lowered the frequency of required blood removals. The filing also included four-year safety and efficacy data from the phase 2 REVIVE and THRIVE studies, which evaluated long-term durability of response and maintenance of hematocrit control in patients who received the once-weekly subcutaneous injection for more than 12 months according to the press release byTakeda and Protagonist Therapeutics, Inc.
A decision date was set for the third quarter of 2026 under the Prescription Drug User Fee Act (PDUFA).
