Incyte and Syndax Pharmaceuticals announced the U.S. Food and Drug Administration (FDA) approval of Niktimvo™ (axatilimab-csfr) in 9 mg and 22 mg vial sizes. The companies expect the product to be available for order in the United States by early February. Niktimvo is indicated for the treatment of chronic graft-versus-host disease (GVHD) in adult and pediatric patients weighing at least 40 kg (88.2 lbs) who have failed at least two prior lines of systemic therapy. It is the first FDA-approved prescription treatment for chronic GVHD that targets CSF-1R to address the drivers of inflammation and fibrosis.
Breakthrough Treatment for Chronic GVHD
“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 of 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.”
FDA approval of Niktimvo was granted on August 14, 2024, based on positive data from the global AGAVE-201 trial. Published in the New England Journal of Medicine in September 2024, the trial demonstrated a response rate of 75% among patients receiving 0.3 mg/kg of axatilimab every two weeks over six months (N=79). The approval marks a significant milestone in chronic GVHD treatment by offering a novel mechanism of action targeting CSF-1R.
Michael Metzger, Chief Executive Officer of Syndax, emphasized the significance of this innovation: “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. 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.”
Clinical Trial Insights and Safety Profile
The AGAVE-201 trial provided the foundation for Niktimvo’s approval. Serious adverse reactions occurred in 44% of the 79 patients treated, with infection (pathogen unspecified) and viral infection each affecting 14% of patients. Respiratory failure occurred in 5.1% of cases. Adverse reactions led to permanent discontinuation in 10% of patients, while dose reductions and interruptions due to adverse reactions were observed in 8% and 44% of patients, respectively. The most frequent adverse reactions (occurring in ≥15% of patients) included increased aspartate aminotransferase (AST), infections, increased alanine aminotransferase (ALT), and fatigue, among others.
The recommended dosage of Niktimvo for both adults and pediatric patients weighing at least 40 kg is 0.3 mg/kg, up to a maximum dose of 35 mg, administered as an intravenous infusion over 30 minutes every two weeks. To simplify dosing, the drug will be available through specialty distributors in 9 mg and 22 mg vial sizes.
Supporting Patients and Providers
Incyte and Syndax are committed to removing barriers to access for patients prescribed Niktimvo. Through the IncyteCARES program (Connecting to Access, Reimbursement, Education, and Support), eligible U.S. patients receive comprehensive support, including financial assistance, education, and other resources. More information is available by visiting IncyteCARES or calling 1-855-452-5234.
Recognition in Clinical Guidelines
On August 30, 2024, axatilimab-csfr (Niktimvo) was added to the National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology as a category 2A recommendation. This designation indicates a uniform NCCN consensus on its appropriateness based on lower-level evidence for treating chronic GVHD after failure of at least two prior lines of systemic therapy. These guidelines are available at NCCN.
A Collaborative Commercial Strategy
In the U.S., Incyte and Syndax are co-commercializing Niktimvo to maximize its reach and impact. Beyond U.S. borders, Incyte holds exclusive commercialization rights. This partnership reflects both companies’ dedication to transforming care for patients with chronic GVHD.
Advancing Chronic GVHD Care
Niktimvo’s approval introduces a transformative option for patients with chronic GVHD, a condition that arises when donor cells attack the recipient’s tissues following a stem cell transplant. This treatment offers a targeted approach to managing inflammation and fibrosis, improving quality of life for those with limited options. The robust clinical evidence supporting its efficacy underscores its potential to redefine standards of care.
Read more: Incyte and Syndax Announce FDA Approval of Niktimvo\u2122 (Axatilimab) in 9 mg and 22 mg Vials
