Axatilimab

Axatilimab-csfr is a monoclonal antibody that binds to colony stimulating factor-1 receptors (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.

• NIKTIMVO is 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. NIKTIMVO 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.

• None. None.contraindicated

Administer only as an intravenous infusion over 30 minutes. ( 2.3 ) 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. ( 2.1 ) See Full Prescribing Information for dosage modifications for adverse reactions ( 2.2 ) and preparation and administration instructions. ( 2.3 ) 2.1 Recommended Dosage For patients weighing at least 40 kg, administer NIKTIMVO 0.3 mg/kg, up to a maximum dose of 35 mg, as an intravenous infusion over 30 minutes every 2 weeks until progression or unacceptable toxicity. 2.2 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. For recommended NIKTIMVO dosage modifications due to adverse reactions, see Table 1. Table 1: Recommended NIKTIMVO Dosage Modifications for Adverse Reactions AST = aspartate aminotransferase; ALT = alanine aminotransferase; ULN = upper limit of normal; ALP = alkaline phosphatase; CPK = creatine phosphokinase. Adverse Reaction Severity Graded per National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) v5. Dosage Modification Infusion-related reactions [see Warnings and Precautions ( 5.1 )] Grade 1 or 2 Temporarily interrupt the infusion until resolution or decrease infusion rate by 50%.

Infusion-Related Reactions: Interrupt or slow the rate of infusion or permanently discontinue NIKTIMVO based on severity of reaction. ( 2.2 , 5.1 ) Embryo-Fetal Toxicity: May cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception. ( 5.2 , 8.1 , 8.3 ) 5.1 Infusion-Related Reactions NIKTIMVO 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% [see Adverse Reactions ( 6.1 )] . Premedicate with an antihistamine and an antipyretic for patients who have previously experienced an infusion-related reaction to NIKTIMVO [see Dosage and Administration ( 2.2 )] . 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 [see Dosage and Administration ( 2.2 )] . 5.2 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 [see Use in Specific Populations ( 8.1 , 8.3 )] .

The following adverse reactions are described elsewhere in the labeling. Infusion-Related Reactions [see Warnings and Precautions ( 5.1 )] The most common (≥ 15%) adverse reactions, including laboratory abnormalities, are increased AST, infection (pathogen unspecified), increased ALT, decreased phosphate, decreased hemoglobin, viral infection, increased GGT, musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased CPK, increased ALP, nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Incyte Corporation at 1-855-463-3463 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trial Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared to rates in the clinical trials of another drug and may not reflect the rates observed in practice. Chronic Graft-Versus-Host Disease The safety of NIKTIMVO was evaluated in 79 adult and pediatric patients with cGVHD treated with NIKTIMVO 0.3 mg/kg intravenously every 2 weeks in the AGAVE‑201 trial [see Clinical Studies ( 14 )] . The median duration of treatment was 10.3 months (range: 0.5 to 28.6 months), and 73.4% were treated for more than 6 months. Serious adverse reactions occurred in 44% of patients who received NIKTIMVO. Serious adverse reactions in more than 2 patients included infection (pathogen unspecified), viral infection, and respiratory failure.

Lactation: Advise not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on its mechanism of action, NIKTIMVO may cause fetal harm when administered to pregnant women [see Clinical Pharmacology ( 12.1 )] . There are no available data on the use of NIKTIMVO in pregnant women to evaluate for a drug-associated risk. No animal reproductive and developmental toxicity studies have been conducted with axatilimab-csfr. Targeted mutation of CSF-1R or CSF-1 in rodent models results in prenatal and perinatal death, deficits in growth, and pleiotropic impact on multiple organ systems, including skeletal and reproductive. Regulation by CSF-1R on non-mononuclear phagocytic cells and macrophages plays a role in the innate immune protection of the fetus and in pregnancy maintenance and embryo-fetal development. Human immunoglobulin G (IgG) is known to cross the placenta; therefore, NIKTIMVO has the potential to be transmitted from the mother to the developing fetus. Advise women of the potential risk to the fetus. In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2% to 4% and 15% to 20%, respectively. 8.2 Lactation Risk Summary There are no data on the presence of axatilimab-csfr in human milk or the effects on the breastfed child or milk production.

Metabolism

Axatilimab-csfr pharmacokinetics are presented as geometric mean (coefficient of variation [%CV]) in adult patients with cGVHD following axatilimab-csfr 0.3 mg/kg (maximum 35 mg) every 2 weeks, unless otherwise specified. Axatilimab-csfr AUC increased in a greater than dose-proportional manner following single-dose administration of axatilimab-csfr over a dose range of 0.15 mg/kg to 3 mg/kg (0.5 to 10 times the approved recommended dosage) in healthy subjects.

• Aug 14, 2024BLABLA761411Incyte Corp

1. 1Off Label Use3027%
2. 2Death65.4%
3. 3Liver Function Test Increased65.4%
4. 4Product Dose Omission Issue65.4%
5. 5Diarrhoea43.6%
6. 6Product Use In Unapproved Indication43.6%
7. 7Anaphylactic Reaction32.7%
8. 8Blood Creatine Phosphokinase Increased32.7%
9. 9Condition Aggravated32.7%
10. 10Drug Ineffective32.7%
11. 11Dyspnoea32.7%
12. 12Fatigue32.7%
13. 13Inappropriate Schedule Of Product Administration32.7%
14. 14Infusion Related Reaction32.7%
15. 15Lipase Increased32.7%

The 10 most recently updated of 27 ClinicalTrials.gov registrations naming Axatilimab as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Axatilimab + CAR-T for High-Risk Lymphoma
  Not yet recruiting · Phase 1 · Interventional · 29 enrolled · Northside Hospital, Inc.
  NCT07638982updated 2026-06-10
• Evaluating Combination of Nivolumab and Axatilimab in Patients With Relapsed/Refractory Classical Hodgkin Lymphoma
  Active not recruiting · Phase 2 · Interventional · 9 enrolled · University of Utah
  NCT05723055updated 2026-06-05
• A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256)
  Recruiting · Phase 2 · Interventional · 60 enrolled · Incyte Corporation
  NCT07124078updated 2026-06-02
• A Study to Evaluate Axatilimab and Corticosteroids as Initial Treatment for Chronic Graft-Versus-Host Disease
  Recruiting · Phase 3 · Interventional · 240 enrolled · Incyte Corporation
  NCT06585774updated 2026-06-01
• A Study to Evaluate the Safety and Efficacy of Axatilimab in Combination With Ruxolitinib in Participants With Newly Diagnosed Chronic Graft-Versus-Host Disease
  Active not recruiting · Phase 2 · Interventional · 130 enrolled · Incyte Corporation
  NCT06388564updated 2026-05-22
• A Phase Ib Study of Novel Combination (New) of Low Dose Oral CyclophoSphamide (s) to Potentiate Axatilimab (A) + Retifanlimab (R) in Treating Metastatic Triple Negative Breast Cancer (TNBC)
  Recruiting · Phase 1 · Interventional · 24 enrolled · M.D. Anderson Cancer Center
  NCT06959537updated 2026-05-20
• Axatilimab in Combination With Extracorporeal Photopheresis (ECP) in Chronic Graft-versus-Host Disease
  Recruiting · Phase 2 · Interventional · 49 enrolled · University of Miami
  NCT06663722updated 2026-05-15
• ASTRAEA: ReinvigorAting ReSponse To ImmunotheRApy in MEtAstatic TNBC With Combination Myeloid Inhibition and Radiation
  Not yet recruiting · Phase 2 · Interventional · 34 enrolled · Stephen Shiao
  NCT07015853updated 2026-04-20
• Reinvigorating TNBC Response to Immunotherapy With Combination Myeloid Inhibition and Radiation
  Recruiting · Phase 2 · Interventional · 35 enrolled · Stephen Shiao
  NCT05491226updated 2026-04-20
• Axatilimab Plus Standard of Care Therapy for the Prevention of Graft Versus Host Disease Following Allogeneic Hematopoietic Stem Cell Transplantation in Patients With Hematologic Cancer, ABRAXAS Trial
  Not yet recruiting · Phase 2 · Interventional · 72 enrolled · Mayo Clinic
  NCT07349771updated 2026-04-16

Frequently asked questions

How does Axatilimab work?

Axatilimab-csfr is a monoclonal antibody that binds to colony stimulating factor-1 receptors (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.

What is Axatilimab used for?

According to FDA labeling, Axatilimab carries indications including: NIKTIMVO is 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. NIKTIMVO 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.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Axatilimab?

Axatilimab is classified as Monoclonal antibodies, Colony Stimulating Factor-1 Receptor Blocker, Colony Stimulating Factor Receptor Type 1 (CSF-1R) Inhibitors, Decreased Immunologic Activity.

What are the brand names for Axatilimab?

Axatilimab is marketed under brand names including Niktimvo.

What are the contraindications for Axatilimab?

Axatilimab labeling lists contraindications including: None. None.. Always consult the full prescribing information and a clinician.