Knight Therapeutics Announces Regulatory Submission of NIKTIMVO(TM) (Axatilimab) in Brazil

Executive Summary

• Knight Therapeutics’ Brazilian affiliate, United Medical Ltda., has filed a marketing‑authorization application with ANVISA for NIKTIMVO® (axatilimab) to treat chronic graft‑versus‑host disease (GVHD) in Brazil.
• The filing follows an amended agreement with Incyte expanding Knight’s exclusive rights to distribute retifanlimab and axatilimab across Latin America.
• NIKTIMVO® already holds U.S. FDA approval (August 2024); the Brazilian submission moves the product toward market entry in a region with ~1,400–1,800 allogeneic transplants annually.

Key Details

• Regulatory Action: United Medical Ltda. submitted a marketing‑authorization application to Brazil’s health regulator ANVISA for NIKTIMVO® (axatilimab).
• Indication: Chronic graft‑versus‑host disease after failure of ≥ two prior systemic therapies in patients ≥ 6 years old (adults and pediatric).
• Agreement Expansion (Aug 4 2025): Knight amended its Incyte agreement to give Knight exclusive rights to distribute retifanlimab (ZYNYZ®) and axatilimab (NIKTIMVO®) throughout Latin America; Incyte remains responsible for development, manufacturing, and supply.
• Prior Agreements: Knight/Incyte have existing exclusive supply/distribution agreements for tafasitamab (MONJUVI®) and pemigatinib (PEMAZYRE®) in Latin America (Sept 2021).
• U.S. FDA Approval: NIKTIMVO® received FDA approval in August 2024 for the same chronic GVHD indication, for patients weighing ≥ 40 kg.
• Clinical Data Reference: Efficacy and safety were demonstrated in the AGAVE‑201 Phase 2 trial (randomized, open‑label, multicenter).
• Market Context – Brazil: Approximately 1,400–1,800 allogeneic transplants performed annually; chronic GVHD incidence ~29.5% (2‑year cumulative), affecting 30‑70% of recipients globally.
• Future Development: Ongoing Phase 2 combination trial with ruxolitinib (NCT06388564) and Phase 3 steroid combination trial for chronic GVHD; Phase 2 trial in idiopathic pulmonary fibrosis (NCT06132256).

Notable Quotes

“For patients living with chronic graft‑versus‑host disease, every new treatment option can mean hope for a better quality of life,” said Samira Sakhia, President and CEO, Knight Therapeutics. “The submission of axatilimab in Brazil brings us closer to making that hope a reality.”