- NDA submission for rusfertide follows positive results from Phase 3 VERIFY study.
- Rusfertide targets erythrocytosis in polycythemia vera by regulating iron homeostasis.
- Protagonist may opt-out within 90 days after a 120-day period, eligible for up to $400 million.
- Rusfertide has Breakthrough, Orphan Drug, and Fast Track Designations from the FDA.
Rusfertide NDA Submission
Takeda and Protagonist Therapeutics have submitted a New Drug Application (NDA) to the U.S. FDA for rusfertide, aimed at treating polycythemia vera (PV). This investigational hepcidin mimetic peptide is designed to regulate iron homeostasis and control hematocrit levels in PV patients.
VERIFY Study Results
The NDA includes data from the Phase 3 VERIFY study, which met its primary and all four key secondary endpoints. The study demonstrated that rusfertide provides durable hematocrit control and reduces the need for frequent phlebotomies, showing a higher response rate compared to placebo.
Regulatory Designations
Rusfertide has received Breakthrough Therapy, Orphan Drug, and Fast Track Designations from the U.S. FDA, highlighting its potential to offer significant improvements over existing therapies for PV patients.
Collaboration and Financials
Under a January 2024 agreement, Protagonist can opt-out within 90 days after a 120-day period post-NDA submission, potentially receiving up to $400 million in opt-out payments, along with enhanced milestone payments and tiered royalties on global sales.
