NIKTIMVO

Dosage and Administration ( 2.3 ) 06/2026

1 INDICATIONS AND USAGE 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. ( 1 )

2 DOSAGE AND ADMINISTRATION 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%. Initiate symptomatic treatment (e.g., antihistamines and antipyretics). For subsequent infusions, premedicate and resume the infusion at 50% of the prior infusion rate. Grade 3 or 4 Permanently discontinue NIKTIMVO. Elevation of AST or ALT (on the day of dosing) [see Adverse Reactions ( 6.1 )] Grade 3 with total bilirubin ≤ Grade 1 Withhold NIKTIMVO until recovery to Grade 2, then resume NIKTIMVO at 0.2 mg/kg (maximum 23 mg) every 2 weeks. Elevation of AST or ALT (regardless of the time of the reaction) [see Adverse Reactions ( 6.1 )] ALT or AST ≥ 3 times ULN with total bilirubin ≥ 2 times ULN and ALP 4 weeks from the planned infusion, permanently discontinue NIKTIMVO. Grade 4 Permanently discontinue NIKTIMVO. 2.3 Preparation and Administration NIKTIMVO 9 mg and 22 mg are available in different concentrations: 9 mg/0.18 mL 9 mg/0.9 mL 22 mg/0.44 mL 22 mg/2.2 mL (10 mg/mL) Prior to preparation, check the vial labels to confirm which concentration is being used to avoid dosing errors, including underdosage and overdosage [see Dosage Forms and Strengths ( 3 )] . Preparation Use aseptic technique to prepare NIKTIMVO. Visually inspect the vial for particulate matter and discoloration prior to dilution. NIKTIMVO is a slightly opalescent, pale brownish yellow solution. Discard the vial if the solution is cloudy, discolored, or contains visible particles. Do not shake the vial. Determine the dose [see Dosage and Administration ( 2.1 )] and total volume of NIKTIMVO solution needed. Dilution Withdraw the calculated volume of NIKTIMVO solution from the vial and add it into an intravenous infusion bag made of polyvinyl chloride (PVC), polyolefin, polyolefin with polyamide, or ethylene vinyl acetate (EVA) containing 0.9% Sodium Chloride Injection to achieve a final concentration between the range of 0.24 mg/mL and 0.75 mg/mL. Discard vial with any unused portion. Mix diluted solution by gentle inversion. Do not shake. Parenteral drug products should be inspected visually for particulate matter and discoloration prior to administration, whenever solution and container permit. The diluted solution is a clear to slightly opalescent, colorless solution that may contain trace amounts of translucent to white particles. Discard if the solution is cloudy, discolored, or contains extraneous particulate matter other than trace amounts of translucent to white particles. Storage of diluted NIKTIMVO solutio …

3 DOSAGE FORMS AND STRENGTHS Injection: slightly opalescent, pale brownish yellow solution available as: 9 mg/0.18 mL in a single-dose vial. 9 mg/0.9 mL in a single-dose vial. 22 mg/0.44 mL in a single-dose vial. 22 mg/2.2 mL (10 mg/mL) in a single-dose vial. Injection : 9 mg/0.18 mL solution in a single-dose vial. ( 3 ) 9 mg/0.9 mL solution in a single-dose vial. ( 3 ) 22 mg/0.44 mL solution in a single-dose vial. ( 3 ) 22 mg/2.2 mL (10 mg/mL) solution in a single-dose vial. ( 3 )

4 CONTRAINDICATIONS None. None. ( 4 )

6 ADVERSE REACTIONS 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. 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 more than 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 AST, infection (pathogen unspecified), increased ALT, decreased phosphate, decreased hemoglobin, viral infection, increased gamma glutamyl transferase (GGT), musculoskeletal pain, increased lipase, fatigue, increased amylase, increased calcium, increased CPK, increased ALP, nausea, headache, diarrhea, cough, bacterial infection, pyrexia, and dyspnea. Table 2 summarizes the nonlaboratory adverse reactions in AGAVE-201. Table 2: Adverse Reactions in ≥ 10% of Patients With cGVHD Who Received NIKTIMVO in AGAVE-201 Graded according to NCI CTCAE v5.0. Adverse Reaction NIKTIMVO 0.3 mg/kg intravenously every 2 weeks (N = 79) All Grades (%) Grades 3-4 (%) Infections and infestations Infection (pathogen unspecified) Includes abscess jaw, atypical pneumonia, bacteremia, bronchitis, conjunctivitis, cystitis, device-related infection, enterocolitis infectious, gastroenteritis, gastrointestinal infection, groin abscess, hordeolum, liver abscess, nasopharyngitis, otitis media, otitis media acute, pneumonia, respiratory tract infection, rhinitis, sepsis, sinusitis, tooth infection, upper respiratory tract infection, urinary tract infection, and wound infection. 57 14 Viral infection Includes adenoviral upper respiratory infection, BK virus infection, COVID-19, coronavirus infection, enterovirus infection, gastroenteritis astroviral, gastroenteritis viral, herpes simplex, herpes zoster, influenza, metapneumovirus bronchiolitis, metapneumovirus infection, norovirus infection, oral viral infection, parainfluenza viral bronchitis, parainfluenza virus infection, respiratory syncytial virus infection, rhinovirus infection, viral infection, and viral upper respiratory tract infection. 43 15 Bacterial infection Includes bacterial diarrhea, bacterial vaginosis, campylobacter gastroenteritis, campylobacter infection, cellulitis, clostridium difficile colitis, clostridium difficile infection, …

8 USE IN SPECIFIC POPULATIONS 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. Maternal IgG is known to be present in human milk. 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. 8.3 Females and Males of Reproductive Potential NIKTIMVO may cause fetal harm when administered to a pregnant woman [see Use in Specific Populations ( 8.1 )] . Pregnancy Testing Verify pregnancy status in females of reproductive potential prior to initiating NIKTIMVO [see Use in Specific Populations ( 8.1 )] . 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. 8.4 Pediatric Use The safety and effectiveness of NIKTIMVO for the treatment of cGVHD after failure of at least two prior lines of systemic therapy have been established in pediatric patients weighing at least 40 kg. Use of NIKTIMVO in pediatric patients weighing at least 40 kg is supported by evidence from clinical trials that included 3 children (ages 6 to less than 12 years old) and 5 adolescents (ages 12 to less than 17 years old) [see Clinical Studies ( 14 )] . The safety and effectiveness of NIKTIMVO have not been established in pediatric patients weighing less than 40 kg. Compared to adult and pediatric patients weighing 40 kg and above, patients weighing less than 40 kg had lower maximum concentration, trough concentration, and average concentration at the same weight-based dosage. Based on findings of thickening of the growth plate and metaphysis and/or degeneration of the growth plate in the femur in animals, monitor bone growth and development in pediatric patients [see Nonclinical Toxicology ( 13.2 )] . 8.5 Geriatric Use Of the 79 patients with cGVHD treated with NIKTIMVO, 21 (26.6%) were 65 years and older, and 2 (2.5%) were 75 years and older [see Clinical Studies ( 14 )] . No overall differences in the safety or effectiveness of NIKTIMVO have been observed between patients 65 years of age and older and younger patients.

12.1 Mechanism of Action 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.

11 DESCRIPTION Axatilimab-csfr is a CSF-1R–blocking antibody. Axatilimab-csfr is a humanized IgG4 (kappa light chain) monoclonal antibody produced in Chinese hamster ovary cells. Axatilimab-csfr has an approximate molecular weight of 150 kDa. NIKTIMVO (axatilimab-csfr) injection is a sterile, preservative-free, slightly opalescent, pale brownish yellow solution for intravenous use. The solution is free from visible particles. Each single-dose vial contains 9 mg or 22 mg of axatilimab-csfr at a concentration of 10 mg/mL or 50 mg/mL. Each mL of solution contains citric acid monohydrate (3.6 mg), glycine (9.38 mg), polysorbate 80 (0.5 mg), sodium citrate (8.49 mg), sucrose (42.79 mg) and Water for Injection, USP. The pH is 5.0.

16 HOW SUPPLIED/STORAGE AND HANDLING NIKTIMVO (axatilimab-csfr) injection is a slightly opalescent, pale brownish yellow solution. It is supplied in a carton containing one single-dose vial as: 9 mg/0.18 mL (NDC 50881-034-12) 9 mg/0.9 mL (NDC 50881-036-17) 22 mg/0.44 mL (NDC 50881-023-11) 22 mg/2.2 mL (10 mg/mL) (NDC 50881-047-18) Store refrigerated at 2°C to 8°C (36°F to 46°F) in the original carton to protect from light. Do not freeze or shake.