FDA Approves Incyte and Syndax’s Niktimvo for Chronic Graft-Versus-Host Disease in 9 and 22 mg Vial Sizes

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The FDA has approved Incyte’s and Syndax’s Niktimvo (axatilimab-csfr) in 9 mg and 22 mg vial sizes for the treatment of chronic graft-versus-host disease (GVHD) in adults and pediatric patients weighing at least 40 kg who have failed at least two prior lines of systemic therapy. According to Incyte, approval was based on data from the AGAVE-201 trial, which demonstrated that a majority of patients treated with Niktimvo achieved a response at six months. The regulatory action marks the first FDA-approved therapy that targets CSF-1R to address inflammation and fibrosis in chronic GVHD.¹

"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 CEO, Incyte, in a press release. "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."

The global, dose-ranging AGAVE-201 trial evaluated the efficacy, safety, and tolerability of Niktimvo in 241 adult and pediatric patients. Enrollment criteria included having experienced disease progression after two or more prior therapies. Patients were randomly assigned into three cohorts to investigate the effects of different doses of Niktimvo, which was administered at 0.3 mg/kg every two weeks, 1.0 mg/kg every two weeks, or 3.0 mg/kg every four weeks. The primary endpoint of the study was the proportion of patients in each dose group who achieved an objective response.

Results found that 75% of participants who received 0.3 mg/kg every two weeks achieved a response within six months of treatment.1 Additionally, the study found that after one year of treatment, 60% of participants maintained a response. Further, 56% of patients achieved a ≥7-point improvement in the modified Lee Symptom Scale score.²

Forty-four percent of patients experienced serious adverse events (AEs), including numerous infections and respiratory failure. An estimated 10% of patients discontinued treatment with Niktimvo due to AEs, while 8% required a dose reduction. The most commonly reported AEs included increased aspartate aminotransferase; increased alanine aminotransferase; decreased phosphate; decreased hemoglobin; viral infection; increased gamma glutamyl transferase; musculoskeletal pain; increased lipase; fatigue; increased amylase; increased calcium; increased creatine phosphokinase; increased alkaline phosphatase; nausea; headache; diarrhea; coughing; and bacterial infection.¹

According to the Fred Hutchinson Cancer Center, 70% of transplant recipients will develop GVHD within the first few months of treatment. Forty percent will be diagnosed with chronic GVHD, which appears around 100 days after the transplant.³

"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, CEO, Syndax, in a press release. "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."

In August 2024, Niktimvo was given a category 2A recommendation in the NCCN Clinical Practice Guidelines. Incyte and Syndax are co-commercializing Niktimvo in the United States through the IncyteCARES program, to assist eligible patients with access, education, and financial support.1 The treatment was first approved in August 2024 as a 50mg vial.²

References

1. Incyte and Syndax Announce U.S. Food and Drug Administration (FDA) Approval of Niktimvo™ (axatilimab-csfr) 9 mg and 22 mg Vial Sizes. Incyte. January 15, 2025. Accessed January 16, 2025. https://investor.incyte.com/news-releases/news-release-details/incyte-and-syndax-announce-us-food-and-drug-administration-fda

2. Incyte and Syndax Announce U.S. FDA Approval of Niktimvo™ (axatilimab-csfr) for the Treatment of Chronic Graft-Versus-Host Disease (GVHD). Syndax. August 14, 2024. Accessed January 16, 2025. https://ir.syndax.com/news-releases/news-release-details/incyte-and-syndax-announce-us-fda-approval-niktimvotm-axatilimab

3. Life with graft-vs.-host disease: When the transplant is just the beginning. Fred Hutch Cancer Center. Accessed January 16, 2025. https://www.fredhutch.org/en/news/center-news/2015/04/tackling-graft-vs-host-disease.html#:~:text=GVHD%20is%20not%20rare.,to%20disabling%20to%20life%2Dthreatening.