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Niktimvo™ (axatilimab-csfr) adverse reactions and considerations

Nonlaboratory adverse reactions in ≥10% of patients1
Niktimvo
0.3 mg/kg IV Q2W 
(N=79)
All grades 
(%)		Grades 
3-4 (%)
Infections and infestations
Infection (pathogen unspecified)5714
Viral infection4315
Bacterial infection158
Musculoskeletal and connective tissue disorders
Musculoskeletal pain353
General disorders and administration site conditions
Fatigue324
Pyrexia151
Edema131
Gastrointestinal disorders
Nausea233
Diarrhea185
Nervous system disorders
Headache201
Dizziness110
Respiratory, thoracic, and mediastinal disorders
Cough180
Dyspnea153
Immune system disorders
Drug hypersensitivity133
Metabolism and nutrition disorders
Decreased appetite114
Vascular disorders
Hemorrhage111
Skin and subcutaneous tissue disorders
Rash100

Graded according to National Cancer Institute Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.1

  • No fatal or opportunistic infections (invasive fungal, cytomegalovirus, and Epstein-Barr virus) occurred in any patients in the 0.3 mg/kg IV Q2W group2
  • No observed cytopenias1
  • No observed drug-drug interactions1
  • Selected laboratory abnormalities >30% included decreases in phosphate (51%) and hemoglobin (48%), as well as increases in AST (61%), ALT (51%), GGT (39%), lipase (34%), amylase (32%), and calcium (31%)1
  • Transient increases in serum enzyme levels were consistent with the known effect of CSF-1R inhibition on macrophage clearance (31%)3
  • Permanent discontinuation and dose reduction due to an adverse reaction occurred in 10% and 8% of patients, respectively1

For further characterization of each adverse reaction type, please see below Table 2 in section 6.1 (Clinical Trial Experience) of the Full Prescribing Information for Niktimvo.

ALT=alanine aminotransferase; AST=aspartate aminotransferase; CSF-1R=colony stimulating factor-1 receptor; GGT=gamma glutamyl transferase; IV=intravenous; Q2W=every 2 weeks.

References: 1. Niktimvo Prescribing Information. Wilmington, DE: Incyte Corporation. 2. Wolff D, Cutler C, Lee SJ, et al; for the AGAVE-201 Investigators. Axatilimab in recurrent or refractory chronic graft-versus-host disease. N Engl J Med. 2024;391(11):1002-1014. Supplementary appendix available at: https://www.nejm.org/doi/full/10.1056/NEJMoa2401537. 3. Data on file. Incyte Corporation. Wilmington, DE.

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IMPORTANT SAFETY INFORMATION

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

Niktimvo™ (axatilimab-csfr) can cause infusion-related reactions. Infusion-related reactions, including hypersensitivity reactions, occurred in 18% of patients who received Niktimvo in the clinical trial (AGAVE-201), with Grade 3 or 4 reactions in 1.3%.

INDICATIONS AND USAGE

Niktimvo™ (axatilimab-csfr) is a colony stimulating factor-1 receptor (CSF-1R)-blocking antibody indicated 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.

Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to Niktimvo. Monitor patients for signs and symptoms of infusion-related reactions, including fever, chills, rash, flushing, dyspnea, and hypertension. Interrupt or slow the rate of infusion or permanently discontinue Niktimvo based on severity of the reaction.

Embryo-Fetal Toxicity

Based on its mechanism of action, Niktimvo may cause fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to the fetus. Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose.

ADVERSE REACTIONS

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.

Clinically relevant adverse reactions in < 10% of patients who received Niktimvo included:

  • Eye disorders: periorbital edema
  • Skin and subcutaneous skin disorders: pruritus
  • Vascular disorders: hypertension

Immunogenicity: Anti-Drug Antibody–Associated Adverse Reactions

Across treatment arms in patients with cGVHD who received Niktimvo in clinical trials, among the patients who developed anti-drug antibodies (ADAs), hypersensitivity reactions occurred in 26% (13/50) of patients with neutralizing antibodies (NAb) and in 4% (2/45) of those without NAb.

USE IN SPECIFIC POPULATIONS

Lactation

Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment and for 30 days after the last dose of Niktimvo.

Females and Males of Reproductive Potential

Pregnancy Testing

Verify pregnancy status in females of reproductive potential prior to initiating Niktimvo.

Contraception

Females
Advise females of reproductive potential to use effective contraception during treatment with Niktimvo and for 30 days after the last dose of Niktimvo.

DOSAGE AND ADMINISTRATION

Dosage Modifications for Adverse Reactions

Monitor aspartate aminotransferase (AST), alanine aminotransferase (ALT), alkaline phosphatase (ALP), creatine phosphokinase (CPK), amylase, and lipase prior to the start of Niktimvo therapy, every 2 weeks for the first month, and every 1 to 2 months thereafter until abnormalities are resolved. See Table 1 in the Prescribing Information for more recommendations.

Please see Full Prescribing Information for Niktimvo.

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Incyte and the Incyte logo are registered trademarks of Incyte.

Niktimvo (axatilimab) is licensed to Incyte from Syndax.

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