Takeda and Protagonist seek FDA approval for rusfertide to treat PV

Takeda and Protagonist Therapeutics have jointly submitted a new drug application (NDA) seeking approval from the US Food and Drug Administration (FDA) for rusfertide to treat adults with polycythemia vera (PV).

The first-in-class, subcutaneously administered hepcidin mimetic peptide, rusfertide is intended to regulate iron homeostasis and red blood cell production to help manage haematocrit levels in PV patients.

The NDA follows positive results from the 32-week primary analysis and 52-week evaluation of the Phase III global placebo-controlled, randomised VERIFY trial.

In this trial, rusfertide achieved the primary endpoint and all four key secondary endpoints.

The study found that patients treated with the therapy alongside standard of care (SoC) therapy achieved a notably higher response rate than those given a placebo with SoC.

The application also includes findings from the earlier Phase II REVIVE study.

Rusfertide has received breakthrough therapy designation from the FDA, recognising its potential to offer substantial improvement over existing therapies. It has also secured fast track and orphan drug designations from the agency.

Takeda global oncology business unit president Teresa Bitetti said: “This is an important milestone toward our goal of addressing critical gaps that patients living with polycythemia vera face today.

“The comprehensive VERIFY study data underscore rusfertide’s strong clinical profile and potential to provide sustained haematocrit 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.”

According to the worldwide licence and collaboration agreement signed between Protagonist and Takeda in January 2024, this NDA submission activates a 120-day period after which Protagonist can choose to exercise its opt-out right within an additional 90-day window.

In December 2025, Takeda and Innovent closed their global partnership for next-generation immuno-oncology and antibody-drug conjugate therapies, following the fulfilment of all closing conditions.

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