Nilotinib

Nilotinib is an inhibitor of the BCR-ABL kinase. Nilotinib binds to and stabilizes the inactive conformation of the kinase domain of ABL protein.

• Nilotinib capsules are a kinase inhibitor indicated for the treatment of: Adult and pediatric patients greater than or equal to 1 year of age with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase. ( 1.1 ) Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib.ICD-10: C95.90

• Nilotinib is contraindicated in patients with hypokalemia, hypomagnesemia, or long QT syndrome [ see Boxed Warning ]. Nilotinib is contraindicated in patients with hypokalemia, hypomagnesemia, or long QT syndrome.contraindicated

Recommended Adult Dose: Newly diagnosed Ph+ CML-CP: 300 mg orally twice daily. Resistant or intolerant Ph+ CML-CP and CML-AP: 400 mg orally twice daily. ( 2.1 ) Recommended Pediatric Dose: Newly Diagnosed Ph+ CML-CP or Ph+ CML-CP resistant or intolerant to prior TKI therapy: 230 mg/m 2 orally twice daily, rounded to the nearest 50 mg dose (to a maximum single dose of 400 mg). ( 2.1 ) See Dosage and Administration for full dosing instructions and dose-reduction instructions for toxicity. ( 2.1 ) Reduce starting dose in patients with baseline hepatic impairment. ( 2.7 ) Eligible newly diagnosed adult patients with Ph+ CML-CP who have received nilotinib capsules for a minimum of 3 years and have achieved a sustained molecular response (MR4.5) and patients with Ph+ CML-CP resistant or intolerant to imatinib who have received nilotinib capsules for at least 3 years and have achieved a sustained molecular response (MR4.5) may be considered for treatment discontinuation. ( 2.2 , 2.3 , 5.16 ) 2.1 Recommended Dosage Dose nilotinib capsules twice daily at approximately 12-hour intervals on an empty stomach. No food should be consumed for at least 2 hours before the dose is taken and for at least 1 hour after the dose is taken. Advise patients to swallow the capsules whole with water [ see Boxed Warning , Clinical Pharmacology (12.3) ]. For patients who are unable to swallow capsules, the contents of each capsule may be dispersed in 1 teaspoon of applesauce (puréed apple).

Myelosuppression : Monitor complete blood count (CBC) during therapy and manage by treatment interruption or dose reduction. ( 5.1 ) Cardiac and Arterial Vascular Occlusive Events : Evaluate cardiovascular status, monitor and manage cardiovascular risk factors during nilotinib therapy. ( 5.4 ) Pancreatitis and Elevated Serum Lipase : Monitor serum lipase; if elevations are accompanied by abdominal symptoms, interrupt doses and consider appropriate diagnostics to exclude pancreatitis. ( 5.5 ) Hepatotoxicity : Monitor hepatic function tests monthly or as clinically indicated. ( 5.6 ) Electrolyte Abnormalities : Nilotinib can cause hypophosphatemia, hypokalemia, hyperkalemia, hypocalcemia, and hyponatremia. Correct electrolyte abnormalities prior to initiating nilotinib and monitor periodically during therapy. ( 5.7 ) Tumor Lysis Syndrome : Maintain adequate hydration and correct uric acid levels prior to initiating therapy with nilotinib. ( 5.8 ) Hemorrhage : Hemorrhage from any site may occur. Advise patients to report signs and symptoms of bleeding and medically manage as needed. ( 5.9 ) Fluid Retention : Monitor patients for unexpected rapid weight gain, swelling, and shortness of breath. Manage medically. ( 5.13 ) Effects on Growth and Development in Pediatric Patients: Growth retardation has been reported in pediatric patients treated with nilotinib. Monitor growth and development in pediatric patients. ( 5.14 ) Embryo-Fetal Toxicity : Advise females of reproductive potential of potential risk to a fetus and to use effective contraception.

The following clinically significant adverse reactions can occur with nilotinib and are discussed in greater detail in other sections of labeling: Myelosuppression [ see Warnings and Precautions (5.1) ] QT Prolongation [ see Boxed Warning , Warnings and Precautions (5.2) ] Sudden Deaths [ see Boxed Warning , Warnings and Precautions (5.3) ] Cardiac and Arterial Vascular Occlusive Events [ see Warnings and Precautions (5.4) ] Pancreatitis and Elevated Serum Lipase [ see Warnings and Precautions (5.5) ] Hepatotoxicity [ see Warnings and Precautions (5.6) ] Electrolyte Abnormalities [ see Boxed Warning , Warnings and Precautions (5.7) ] Hemorrhage [ see Warnings and Precautions (5.9) ] Fluid Retention [ see Warnings and Precautions (5.13) ] The most commonly reported non-hematologic adverse reactions (≥ 20%) in adult and pediatric patients were nausea, rash, headache, fatigue, pruritus, vomiting, diarrhea, cough, constipation, arthralgia, nasopharyngitis, pyrexia, and night sweats. Hematologic adverse drug reactions include myelosuppression: thrombocytopenia, neutropenia, and anemia. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Novadoz Pharmaceuticals LLC at 1-855-668-2369 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch. 6.1 Clinical Trials 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.

Lactation : Advise women not to breastfeed. ( 8.2 ) Additional pediatric use information is approved for Novartis Pharmaceuticals Corporation’s TASIGNA (nilotinib) capsules. However, due to Novartis Pharmaceuticals Corporation’s marketing exclusivity rights, this drug product is not labeled with that pediatric information. 8.1 Pregnancy Risk Summary Based on findings from animal studies and the mechanism of action, nilotinib can cause fetal harm when administered to a pregnant woman [ see Clinical Pharmacology (12.1) ] . There are no available data in pregnant women to inform the drug-associated risk. In animal reproduction studies, administration of nilotinib to pregnant rats and rabbits during organogenesis caused adverse developmental outcomes, including embryo-fetal lethality, fetal effects, and fetal variations in rats and rabbits at maternal exposures (AUC) approximately 2 and 0.5 times, respectively, the exposures in patients at the recommended dose (see Data). Advise pregnant women of the potential risk to a fetus. The background risk of major birth defects and miscarriage for the indicated population is unknown. All pregnancies have a background risk of birth defect, loss, or other adverse outcomes. In the U.S.

Metabolism

Steady-state nilotinib exposure was dose-dependent with less than dose-proportional increases in systemic exposure at dose levels higher than 400 mg given as once or twice daily dosing. In adult patients with resistant or intolerant Ph+ CML given nilotinib 400 mg twice daily, the steady-state mean (% CV) C max and AUC 0-12h were 2260 ng/mL (35%) and 18000 ng∙h/mL (33%), respectively.

Overdose with nilotinib has been reported, where an unspecified number of nilotinib capsules were ingested in combination with alcohol and other drugs. Events included neutropenia, vomiting, and drowsiness. In the event of overdose, observe the patient and provide appropriate supportive treatment.

• Oct 29, 2007NDANDA022068Novartis
• Jan 5, 2024ANDAANDA203640Apotex
• Nov 7, 2024NDANDA219293Azurity
• Feb 19, 2025NDANDA218922Cipla
• Nov 19, 2025ANDAANDA218544Msn
• Nov 19, 2025ANDAANDA209651Hetero Labs Ltd V
• Nov 19, 2025ANDAANDA218350Dr Reddys
• Dec 15, 2025ANDAANDA218532Torrent

1. 1Death3,21611%
2. 2Fatigue2,0947.2%
3. 3Rash1,6445.6%
4. 4Nausea1,5275.2%
5. 5Headache1,4745.1%
6. 6Dyspnoea1,3734.7%
7. 7Pain1,3364.6%
8. 8Diarrhoea1,2044.1%
9. 9Drug Ineffective1,1884.1%
10. 10Pruritus1,1283.9%
11. 11Malaise1,1263.9%
12. 12Pyrexia1,1243.9%
13. 13Arthralgia1,0673.7%
14. 14Asthenia1,0453.6%
15. 15Vomiting1,0453.6%

The 10 most recently updated of 229 ClinicalTrials.gov registrations naming Nilotinib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• Targeted Therapy Directed by Genetic Testing in Treating Patients With Locally Advanced or Advanced Solid Tumors, The ComboMATCH Screening Trial
  Recruiting · Phase 2 · Interventional · 2,900 enrolled · National Cancer Institute (NCI)
  NCT05564377updated 2026-06-12
• Impact of Nilotinib on Safety, Tolerability, Pharmacokinetics and Biomarkers in Dementia With Lewy Bodies
  Completed · Phase 2 · Interventional · 43 enrolled · Georgetown University
  NCT04002674updated 2026-06-12
• Impact of Nilotinib on Safety, Tolerability, Pharmacokinetics and Biomarkers in Parkinson's Disease
  Completed · Phase 2 · Interventional · 75 enrolled · Georgetown University
  NCT02954978updated 2026-06-12
• A Study on the Tolerability, Safety and Effectiveness of Asciminib in Patients With Philadelphia Chromosome-positive Chronic Myeloid Leukemia in the Chronic Phase in Germany
  Recruiting · Observational · 380 enrolled · Novartis Pharmaceuticals
  NCT07549516updated 2026-06-12
• Asciminib Roll-over Study
  Recruiting · Phase 4 · Interventional · 347 enrolled · Novartis Pharmaceuticals
  NCT04877522updated 2026-06-10
• A Study of Oral Asciminib Versus Other TKIs in Adult Patients With Newly Diagnosed Ph+ CML-CP
  Active not recruiting · Phase 3 · Interventional · 405 enrolled · Novartis Pharmaceuticals
  NCT04971226updated 2026-06-08
• Asciminib as Initial Therapy for Patients With Chronic Myeloid Leukemia in Chronic Phase
  Recruiting · Phase 2 · Interventional · 100 enrolled · University of Alabama at Birmingham
  NCT05143840updated 2026-06-02
• Combination Nilotinib and Paclitaxel in Adults With Relapsed Solid Tumors
  Completed · Phase 1 · Interventional · 47 enrolled · National Cancer Institute (NCI)
  NCT02379416updated 2026-06-01
• Intravenous and Intraperitoneal Paclitaxel and Oral Nilotinib for Peritoneal Carcinomatosis
  Completed · Phase 2 · Interventional · 21 enrolled · National Cancer Institute (NCI)
  NCT05185947updated 2026-05-29
• A Study to Investigate Tolerability and Efficacy of Asciminib (Oral) Versus Nilotinib (Oral) in Adult Participants (≥18 Years of Age) With Newly Diagnosed Philadelphia Chromosome Positive Chronic Myelogenous Leukemia in Chronic Phase (Ph+ CML-CP)
  Active not recruiting · Phase 3 · Interventional · 568 enrolled · Novartis Pharmaceuticals
  NCT05456191updated 2026-05-27

CPIC-curated drug–gene pairs for Nilotinib. Levels describe the strength of curated evidence and guideline status — never a recommendation to test or to adjust therapy.

• UGT1A1CPIC B/C (provisional)ClinPGx 3FDA label: Informative PGx

Frequently asked questions

How does Nilotinib work?

Nilotinib is an inhibitor of the BCR-ABL kinase. Nilotinib binds to and stabilizes the inactive conformation of the kinase domain of ABL protein.

What is Nilotinib used for?

According to FDA labeling, Nilotinib carries indications including: Nilotinib capsules are a kinase inhibitor indicated for the treatment of: Adult and pediatric patients greater than or equal to 1 year of age with newly diagnosed Philadelphia chromosome positive chronic myeloid leukemia (Ph+ CML) in chronic phase. ( 1.1 ) Adult patients with chronic phase (CP) and accelerated phase (AP) Ph+ CML resistant to or intolerant to prior therapy that included imatinib.. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Nilotinib?

Nilotinib is classified as BCR-ABL tyrosine kinase inhibitors, Kinase Inhibitor, Bcr-Abl Tyrosine Kinase Inhibitors, Cytochrome P450 2B6 Inducers, Cytochrome P450 2C8 Inducers, Cytochrome P450 2C8 Inhibitors, Cytochrome P450 2D6 Inhibitors, P-Glycoprotein Inhibitors, UGT1A1 Inhibitors, Cellular Proliferation Alteration.

What are the brand names for Nilotinib?

Nilotinib is marketed under brand names including Danziten, Tasigna.

What are the contraindications for Nilotinib?

Nilotinib labeling lists contraindications including: Nilotinib is contraindicated in patients with hypokalemia, hypomagnesemia, or long QT syndrome [ see Boxed Warning ]. Nilotinib is contraindicated in patients with hypokalemia, hypomagnesemia, or long QT syndrome.. Always consult the full prescribing information and a clinician.