Asciminib

Asciminib is an ABL/BCR-ABL1 tyrosine kinase inhibitor. Asciminib inhibits the ABL1 kinase activity of the BCR::ABL1 fusion protein, by binding to the ABL myristoyl pocket.

• SCEMBLIX is indicated for the treatment of adult patients with: Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). This indication is approved under accelerated approval based on major molecular response rate [see Clinical Studies (14.1)] .ICD-10: C95.90

• None. None.contraindicated

Recommended Dosage in Ph+ CML in CP: 80 mg orally once daily or 40 mg orally twice daily. ( 2.1 ) Recommended Dosage in Ph+ CML in CP with the T315I Mutation: 200 mg orally twice daily. ( 2.2 ) Avoid food for at least 2 hours before and 1 hour after taking SCEMBLIX. ( 2.5 ) Swallow tablets whole. Do not break, crush, or chew the tablets. ( 2.5 ) 2.1 Recommended Dosage in Patients with Newly Diagnosed or Previously Treated Ph+ CML-CP The recommended dose of SCEMBLIX is 80 mg taken orally once daily at approximately the same time each day or 40 mg orally twice daily at approximately 12-hour intervals. The recommended dose of SCEMBLIX is taken orally without food. Avoid food consumption for at least 2 hours before and 1 hour after taking SCEMBLIX [see Clinical Pharmacology (12.2)] . Continue treatment with SCEMBLIX as long as clinical benefit is observed or until unacceptable toxicity occurs. 2.2 Recommended Dosage in Patients with Ph+ CML-CP with the T315I Mutation The recommended dose of SCEMBLIX is 200 mg taken orally twice daily at approximately 12-hour intervals. The recommended dose of SCEMBLIX is taken orally without food. Avoid food consumption for at least 2 hours before and 1 hour after taking SCEMBLIX [see Clinical Pharmacology (12.2)] . 2.3 Missed Dose Once Daily Dosage Regimen: If a SCEMBLIX dose is missed by more than approximately 12 hours, skip the dose and take the next dose as scheduled. Twice Daily Dosage Regimens: If a SCEMBLIX dose is missed by more than approximately 6 hours, skip the dose and take the next dose as scheduled.

Myelosuppression: Severe thrombocytopenia and neutropenia events may occur. Monitor complete blood counts regularly during therapy and manage by treatment interruption or dose reduction. ( 2.4 , 5.1 ) Pancreatic Toxicity: Monitor serum lipase and amylase. Interrupt, then resume at reduced dose or discontinue SCEMBLIX based on severity. Evaluate for pancreatitis when lipase elevation is accompanied by abdominal symptoms. ( 2.4 , 5.2 ) Hypertension: Monitor blood pressure and manage hypertension as clinically indicated. Interrupt, dose reduce, or stop SCEMBLIX if hypertension is not medically controlled. ( 2.4 , 5.3 ) Hypersensitivity: May cause hypersensitivity reactions. Monitor patients for signs and symptoms and initiate appropriate treatment as clinically indicated. ( 5.4 ) Cardiovascular Toxicity: Cardiovascular toxicity may occur. Monitor patients with history of cardiovascular risk factors for cardiovascular signs and symptoms. Initiate appropriate treatment as clinically indicated. ( 5.5 ) Embryo-Fetal Toxicity: Can cause fetal harm. Advise females of reproductive potential of the potential risk to a fetus and to use effective contraception. ( 5.6 , 8.1 , 8.3 ) 5.1 Myelosuppression Thrombocytopenia, neutropenia, and anemia have occurred in patients receiving SCEMBLIX. Thrombocytopenia occurred in 156 of 556 (28%) patients receiving SCEMBLIX, with Grade 3 or 4 thrombocytopenia reported in 39 (7%) and 53 (10%) of patients, respectively.

The following clinically significant adverse reactions can occur with SCEMBLIX and are discussed in greater detail in other sections of the labeling: Myelosuppression [see Warnings and Precautions (5.1)] Pancreatic Toxicity [see Warnings and Precautions (5.2)] Hypertension [see Warnings and Precautions (5.3)] Hypersensitivity [see Warnings and Precautions (5.4)] Cardiovascular Toxicity [see Warnings and Precautions (5.5)] Most common adverse reactions (≥ 20%) are musculoskeletal pain, rash, fatigue, upper respiratory tract infection, headache, abdominal pain, arthralgia, and diarrhea. ( 6.1 ) Most common select laboratory abnormalities (≥ 20%) are lymphocyte count decreased, leukocyte count decreased, platelet count decreased, neutrophil count decreased, calcium corrected decreased, lipase increased, cholesterol increased, uric acid increased, alanine aminotransferase (ALT) increased, alkaline phosphatase (ALP) increased, hemoglobin decreased, triglycerides increased, creatine kinase increased, amylase increased, and aspartate aminotransferase (AST) increased. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Novartis Pharmaceuticals Corporation at 1-888-669-6682 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 not to breastfeed. ( 8.2 ) 8.1 Pregnancy Risk Summary Based on findings from animal studies and the mechanism of action, SCEMBLIX can cause embryo-fetal harm when administered to a pregnant woman [see Clinical Pharmacology (12.1)] . There are no available data on SCEMBLIX use in pregnant women to evaluate a drug-associated risk. Animal reproduction studies in pregnant rats and rabbits demonstrated that oral administration of asciminib during organogenesis induced structural abnormalities, embryo-fetal mortality, and alterations to growth ( see Data ). Advise pregnant women and females of reproductive potential of the potential risk to a 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. Data Animal Data In embryo-fetal development studies, pregnant animals received oral doses of asciminib at 25, 150, and 600 mg/kg/day in rats and at 15, 50, and 300 mg/kg/day in rabbits during the period of organogenesis. In rats, maternal toxicity at the asciminib dose of 600 mg/kg/day resulted in the early termination of the dose group; a complete embryo-fetal examination was not conducted for this group. Adverse embryo-fetal findings were observed at 25 and 150 mg/kg; these doses did not cause maternal toxicities.

Metabolism

Asciminib steady-state exposure (AUC and C max ) increase slightly more than dose proportional across the dose range of 10 to 200 mg (0.25 to 5 times the recommended 80 mg daily dosage) administered once or twice daily. Pharmacokinetic parameters are presented as geometric mean (CV%) unless otherwise stated.

• Oct 29, 2021NDANDA215358Novartis

1. 1Fatigue2368.1%
2. 2Death1705.8%
3. 3Nausea1705.8%
4. 4Drug Ineffective1625.6%
5. 5Thrombocytopenia1465.0%
6. 6Headache1234.2%
7. 7Diarrhoea1224.2%
8. 8Drug Intolerance1214.2%
9. 9Rash1043.6%
10. 10Hypertension973.3%
11. 11Pleural Effusion973.3%
12. 12Arthralgia893.1%
13. 13Cytopenia802.7%
14. 14Dyspnoea802.7%
15. 15Malignant Neoplasm Progression772.6%

The 10 most recently updated of 54 ClinicalTrials.gov registrations naming Asciminib as an intervention. Registration is not evidence of efficacy or safety — reference crosswalk only.

• A Real-world Study of Asciminib Effectiveness in Philadelphia Positive Acute Lymphoblastic Leukemia Patients
  Completed · Observational · 37 enrolled · Novartis Pharmaceuticals
  NCT07644351updated 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
• Efficacy of Asciminib in Real-world in Patients With Chronic Myeloid Leukemia in Second or Subsequent Lines
  Not yet recruiting · Observational · 98 enrolled · Gruppo Italiano Malattie EMatologiche dell'Adulto
  NCT07640750updated 2026-06-11
• Asciminib Roll-over Study
  Recruiting · Phase 4 · Interventional · 347 enrolled · Novartis Pharmaceuticals
  NCT04877522updated 2026-06-10
• Asciminib With or Without Sildenafil for Brain Tumors
  Not yet recruiting · Early phase 1 · Interventional · 12 enrolled · Washington University School of Medicine
  NCT07039760updated 2026-06-09
• Phase 1/2 FLAG-IDA, VEN and Asciminib in CML and Ph+ AML
  Not yet recruiting · Phase 1 · Phase 2 · Interventional · 30 enrolled · M.D. Anderson Cancer Center
  NCT07604233updated 2026-06-08
• Phase I/II Study of the Combination of Blinatumomab and Asciminib in Patients With Philadelphia Chromosome-Positive Acute Lymphoblastic Leukemia
  Recruiting · Phase 1 · Phase 2 · Interventional · 40 enrolled · M.D. Anderson Cancer Center
  NCT06308588updated 2026-06-08
• Phase II Study of Asciminib for Second-line Treatment of Chronic Phase Chronic Myeloid Leukemia
  Recruiting · Phase 2 · Interventional · 40 enrolled · M.D. Anderson Cancer Center
  NCT06629584updated 2026-06-08
• 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
• Phase II Study Assessing Efficacy and Safety of Asciminib in Patients With Newly Diagnosed Chronic Myeloid Leukemia in Chronic Phase.
  Recruiting · Phase 2 · Interventional · 50 enrolled · M.D. Anderson Cancer Center
  NCT06236724updated 2026-06-08

Frequently asked questions

How does Asciminib work?

Asciminib is an ABL/BCR-ABL1 tyrosine kinase inhibitor. Asciminib inhibits the ABL1 kinase activity of the BCR::ABL1 fusion protein, by binding to the ABL myristoyl pocket.

What is Asciminib used for?

According to FDA labeling, Asciminib carries indications including: SCEMBLIX is indicated for the treatment of adult patients with: Newly diagnosed Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP). This indication is approved under accelerated approval based on major molecular response rate [see Clinical Studies (14.1)] .. This is a reference summary of labeled uses, not medical advice or a treatment recommendation.

What class of drug is Asciminib?

Asciminib is classified as BCR-ABL tyrosine kinase inhibitors, Bcr-Abl Tyrosine Kinase Inhibitors.

What are the brand names for Asciminib?

Asciminib is marketed under brand names including Scemblix.

What are the contraindications for Asciminib?

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