Knight seeks Brazilian regulatory okay for Niktimvo

Executive Summary

• Knight Therapeutics’ Brazilian affiliate, United Medical Ltda., has submitted a marketing authorization application to ANVISA for Niktimvo (axatilimab) to treat chronic graft-versus-host disease (GVHD) in adult and pediatric patients (6+ years) who have failed at least two prior lines of systemic therapy.
• This submission follows an amended agreement announced on August 4, 2025, between Knight Therapeutics and Incyte, which grants Knight exclusive rights to distribute retifanlimab (ZYNYZ) and axatilimab (Niktimvo) in Latin America.
• Under the amended agreement, Incyte retains responsibility for the development, manufacture, and supply of both drugs, while Knight handles regulatory approvals and distribution in the region.

Key Details

• Regulatory Submission: Marketing authorization application submitted to ANVISA (Brazilian health regulatory agency) for Niktimvo (axatilimab-csfr).
• Indication: Treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients six years and older.
• Partnership Amendment:
  • Parties: Knight Therapeutics Inc. and Incyte.
  • Effective Date: August 4, 2025.
  • Scope: Exclusive rights for Knight to distribute retifanlimab (commercialized as ZYNYZ in the US and Europe) and axatilimab (commercialized as Niktimvo in the US) in Latin America.
  • Responsibilities: Incyte is responsible for development, manufacture, and supply; Knight is responsible for seeking regulatory approvals and distribution in Latin America.
• Historical Context: Knight and Incyte previously entered an exclusive supply and distribution agreement for tafasitamab (Monjuvi) and pemigatinib (Pemazyre) in Latin America in September 2021.
• Prior Regulatory Status: Niktimvo received FDA approval in August 2024 for the treatment of chronic GVHD after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kilograms.

Notable Quotes

• "For patients living with chronic graft-versus-host disease, every new treatment option can mean hope for a better quality of life... The submission of axatilimab in Brazil brings us closer to making that hope a reality. At Knight, our mission goes beyond expanding our portfolio. We are committed to ensuring that patients and their families have access to therapies that truly make a difference. This milestone reflects the strength of our partnership with Incyte and our shared dedication to improving lives across Latin America." — Samira Sakhia, President and CEO, Knight Therapeutics