FDA approves Takeda’s MIMRYLO for polycythemia vera, triggering $275 million payment

  • FDA approved Takeda’s MIMRYLO for polycythemia vera.
  • VERIFY reported a 76.9% patient response rate in the Phase 3 trial.
  • $275 million in payments to Protagonist were triggered by the approval.

The U.S. Food and Drug Administration approved Takeda Pharmaceutical Co. Ltd.’s MIMRYLO (rusfertide), a first-in-class treatment for polycythemia vera, a type of blood cancer that causes excessive red blood cell production and raises the risk of thrombotic events. The approval was supported by the global Phase 3 VERIFY study, which enrolled 293 patients and produced a 76.9% patient response rate. MIMRYLO, given with standard care, improved hematocrit control, reduced routine phlebotomy requirements and eased fatigue, while maintaining a favorable safety profile. The treatment is a hepcidin mimetic, an iron-regulating therapy designed to limit red blood cell overproduction and keep hematocrit below 45%. An open-label extension remains ongoing, with further findings planned for medical conferences. Barclays raised Protagonist Therapeutics Inc.’s stock price target to $180 following the approval. Takeda will commercialize the drug under its 2024 worldwide license and collaboration agreement with Protagonist, which discovered rusfertide and led development through Phase 3 before Takeda took responsibility for the New Drug Application submission and commercialization after Protagonist’s opt-out election in April 2026. The approval triggers $275 million in payments to Protagonist, comprising a $200 million opt-out fee and a separate $75 million approval milestone. Protagonist may also receive up to $875 million in additional potential milestone payments and tiered royalties of 14% to 29% on worldwide net sales, equivalent to about 21% on a weighted-average basis at $1.5 billion in annual sales. Takeda shares were down 0.19% at $17.99 on Monday at the time of publication, according to Benzinga Pro data.

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