New FDA Approval: NIKTIMVO™ (axatilimab-csfr)
The U.S Food and Drug Administration recently approved approved axatilimab-csfr (Niktimvo, Incyte Corporation), a colony stimulating factor-1 receptor-blocking antibody, for the treatment of chronic graft-versus-host disease (cGVHD) after failure of at least two prior lines of systemic therapy in adult and pediatric patients weighing at least 40 kg.
Read InpharmD's summary below:
| Drug Name |
NIKTIMVO™ (axatilimab-csfr) |
| Active Ingredient |
axatilimab-csfr |
| Date of Approval |
August 14, 2024 |
| Manufacturer |
Incyte and Syndax Pharmaceuticals |
| Approval Pathways and Indications |
Approval Pathway: BLA
Indication: NIKTIMVO™ is indicated 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 |
| Therapeutic Class |
Monoclonal Antibody |
| Formulation |
Injection: 50 mg/mL solution in a single-dose vial. |
| MoA |
Axatilimab-csfr binds to CSF-1R expressed on monocytes and macrophages. Blocking CSF-1R with axatilimab-csfr reduces the levels of these circulating proinflammatory and profibrotic monocytes and monocyte-derived macrophages, as demonstrated by a reduction of nonclassical monocyte counts in nonclinical studies with axatilimab-csfr, and inhibits the activity of pathogenic macrophages in tissues. |
| Dosing and Administration |
The recommended dosage of NIKTIMVO™ is 0.3 mg/kg (maximum 35 mg) every 2 weeks in adult and pediatric patients weighing 40 kg and above.
Administer only as an intravenous infusion over 30 minutes |
| Administered by self or by HCP |
Administered by HCP |
| Place in Therapy |
Chronic GVHD is a serious condition that occurs when donated cells from an allogeneic stem cell transplant attack the recipient's organs. It affects about 42% of transplant recipients, approximately 17,000 people in the U.S., and is a leading cause of morbidity and mortality. Nearly 50% of those affected require at least three lines of treatment, highlighting the need for more options.
NIKTIMVO™ is the first approved anti-CSF-1R antibody that targets the inflammation and fibrosis in chronic GVHD. It offers a new treatment option for patients whose disease has progressed despite prior therapies. Serious adverse reactions include unspecified infections, viral infections, and respiratory failure. |
| Expected Market Launch Date |
First quarter of 2025 |
| New Molecular Entity (NME) or Existing Formulation |
New Molecular Entity |
| Expected Cost |
Data Unavailable |
| Product Discontinuation |
N/A |
| Clinical Trials |
The efficacy of NIKTIMVO™ was assessed in the AGAVE-201 study, a randomized, open-label, multicenter trial involving adult and pediatric patients with recurrent or refractory chronic GVHD who had undergone at least two prior lines of systemic therapy. Eligible patients had a platelet count ≥ 50 × 10^9/L, an absolute neutrophil count ≥ 1 × 10^9/L, ALT and AST levels ≤ 2.5 × ULN (or ≤ 5 × ULN with liver cGVHD), total bilirubin ≤ 1.5 × ULN, and creatinine clearance ≥ 30 mL/minute. NIKTIMVO™ was administered intravenously at a dose of 0.3 mg/kg every two weeks until disease progression, lack of efficacy after 9 months, or unacceptable toxicity.
Efficacy was determined based on the overall response rate up to Cycle 7 Day 1, with a response defined as either a complete or partial response according to the 2014 NIH Consensus Criteria. The median time to first response was 1.5 months (range, 0.9 to 5.1 months). The median duration of response was 1.9 months (95% CI: 1.6 to 3.5 months). Among patients who responded, 60% (95% CI: 43 to 74) did not experience death or require new systemic therapies for chronic GVHD for at least 12 months. Additionally, exploratory analyses of patient-reported symptoms showed that 56% (95% CI: 44 to 67) of patients had at least a 7-point decrease in the modified Lee Symptom Scale score through Cycle 7 Day 1. |
| Sources |
U.S. Food and Drug Administration. NIKTIMVO (axatilimab) [package insert]. Available from: https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/761411s000lbl.pdf
Incyte and Syndax announce U.S. FDA approval of NIKTIMVO™ (axatilimab). Syndax Pharmaceuticals. August 14, 2024. Available from: https://ir.syndax.com/news-releases/news-release-details/incyte-and-syndax-announce-us-fda-approval-niktimvotm-axatilimab
National Center for Biotechnology Information. Gene: CD4. Available from: https://www.ncbi.nlm.nih.gov/gene/1436
Drugs.com. NIKTIMVO (axatilimab). Available from: https://www.drugs.com/niktimvo.html |
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