Incyte (Nasdaq:INCY) and Syndax Pharmaceuticals (Nasdaq:SNDX) announced that the U.S. Food and Drug Administration (FDA) has approved Niktimvo™ (axatilimab-csfr) in 9 mg and 22 mg vial sizes. The Companies expect product to be available for order in the U.S. in early February. Niktimvo is approved for the treatment of chronic graft-versus-host disease (GVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg (88.2 lbs). Niktimvo is the first and only FDA-approved prescription treatment for chronic GVHD that targets CSF-1R to reduce the drivers of inflammation and fibrosis.

"We are thrilled to build on our strong commitment to the GVHD community with the U.S. launch of Niktimvo, a first-in-class therapeutic agent that has demonstrated remarkable responses in patients with chronic GVHD whose response was suboptimal after at least two prior lines of systemic therapy," said Hervé Hoppenot, Chief Executive Officer, Incyte. "Our deep understanding of chronic GVHD and our connections in the clinical community will support a successful launch, in partnership with Syndax, of this important medicine for patients."

Niktimvo was approved by the FDA on August 14, 2024. The approval was based on positive data from the global AGAVE-201 trial, which were published in the New England Journal of Medicine in September 2024.¹ The trial met the primary endpoint across all cohorts receiving Niktimvo with 75% of patients who received 0.3 mg/kg every two weeks achieving a response at six months of treatment (N=79).

"As the first and only FDA-approved anti-CSF-1R antibody targeting the drivers of inflammation and fibrosis in chronic GVHD, Niktimvo represents a major breakthrough for patient care," said Michael Metzger, Chief Executive Officer, Syndax. "Together with Incyte, we look forward to executing a robust commercial launch and advancing the treatment paradigm for patients with chronic GVHD who have progressed after at least two lines of systemic therapy."

Serious adverse reactions occurred in 44% of patients who received Niktimvo (N=79). Serious adverse reactions in > 2 patients included infection (pathogen unspecified) (14%), viral infection (14%), and respiratory failure (5.1%). Permanent discontinuation of Niktimvo due to an adverse reaction occurred in 10% of patients and dose reduction due to adverse reaction occurred in 8% of patients. Dose interruptions due to an adverse reaction occurred in 44% of patients. The adverse reactions leading to dose interruption in >2 patients were viral infection, infection (pathogen unspecified), bacterial infection, musculoskeletal pain and pyrexia.

The most common (≥15%) adverse reactions, including laboratory abnormalities, were increased aspartate aminotransferase (AST), infection (pathogen unspecified), increased alanine aminotransferase (ALT), decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased creatine phosphokinase (CPK), increased alkaline phosphatase (ALP), nausea, headache, diarrhea, cough, bacterial infection, pyrexia and dyspnea.

The approved dose of Niktimvo for adults and pediatric patients weighing at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every two weeks. Niktimvo will be available for healthcare providers to order through a network of specialty distributors in both 9 mg vial and 22 mg vial sizes to facilitate patient dosing.