Niktimvo™

Understanding Niktimvo™
Niktimvo™ (axatilimab‑csfr) injection, for intravenous use, is a prescription monoclonal antibody classified as a colony‑stimulating factor‑1 receptor (CSF‑1R)–blocking antibody. It is indicated for the treatment of chronic graft‑versus‑host disease (cGVHD) in adult and pediatric patients weighing at least 40 kg who have received at least two prior lines of systemic therapy.

How Niktimvo™ Works:

• Targeted CSF‑1R pathway inhibition: Niktimvo™ is a CSF‑1R–blocking monoclonal antibody that binds to the colony‑stimulating factor‑1 receptor on monocytes and macrophages. By inhibiting CSF‑1R signaling, it targets immune pathways associated with inflammation and fibrosis, which contribute to the manifestations of chronic GVHD.
• Reduction of inflammation and fibrotic activity in cGVHD: In the pivotal AGAVE‑201 trial, Niktimvo™ demonstrated durable responses across all organs studied, with a 75% overall response rate at the approved dose level. These results were observed in patients with recurrent or refractory cGVHD who had received at least two prior systemic therapies.

FDA Approval:

2024 – Niktimvo™ (axatilimab‑csfr) was approved by the U.S. Food and Drug Administration on August 14, 2024 for the treatment of chronic graft‑versus‑host disease after failure of at least two prior systemic therapies in adult and pediatric patients weighing at least 40 kg.

For more information, please visit the Niktimvo™ patient website. Speak with your healthcare provider to determine if Niktimvo™ is the right treatment option for you.