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Scemblix

asciminib
STAMP Inhibitor (BCR-ABL1 Allosteric) Novartis FDA Approved 2021
Indications Dosing Forms Contraindications Warnings Adverse Reactions Pharmacology Clinical Studies Tumor Types
1. Indications and Usage

Ph+ CML in chronic phase, previously treated with two or more tyrosine kinase inhibitors; Ph+ CML in chronic phase with the T315I mutation.

2. Dosage and Administration

Without T315I: 80 mg orally once daily or 40 mg twice daily
With T315I: 200 mg orally twice daily
Take with or without food
Avoid concomitant use of strong CYP3A inducers and CYP2C9 substrates with narrow therapeutic index

3. Dosage Forms and Strengths

Tablets: 20 mg, 40 mg

4. Contraindications

None listed.

5. Warnings and Precautions
  • Myelosuppression: Grade 3-4 thrombocytopenia (21%), neutropenia (18%), anemia (7%). Monitor CBC every 2 weeks × 3 months, then monthly.
  • Pancreatic Toxicity: Lipase elevation in 28% (Grade 3-4 in 6%). Monitor lipase monthly × 3 months, then periodically. Withhold for Grade 3+.
  • Hepatotoxicity: ALT/AST elevations. Monitor LFTs monthly × 3 months, then periodically.
  • Cardiovascular Toxicity: Hypertension (17%), QT prolongation (rare). Monitor BP.
6. Adverse Reactions
Most Common Adverse Reactions

Musculoskeletal pain (26%), upper respiratory tract infection (19%), fatigue (17%), hypertension (17%), diarrhea (16%), rash (16%), nausea (15%), headache (13%), arthralgia (13%), abdominal pain (12%), lipase elevation (28%)

Lipase Elevation
28%
Musculoskeletal pain
26%
Upper Respiratory Tract Infection
19%
Fatigue
17%
Hypertension
17%
Diarrhea
16%
Rash
16%
Nausea
15%
Headache
13%
Arthralgia
13%

Consult the complete prescribing information for a comprehensive list of adverse reactions and their frequencies.

12. Clinical Pharmacology
Mechanism of Action

Asciminib is a first-in-class BCR-ABL1 inhibitor that binds specifically to the ABL myristoyl pocket (STAMP — Specifically Targeting the ABL Myristoyl Pocket), a unique allosteric regulatory site. Unlike ATP-competitive TKIs (imatinib, dasatinib, nilotinib), asciminib does not bind the ATP-binding site, which means it maintains activity against most ATP-site resistance mutations including T315I. The myristoyl pocket is essential for maintaining ABL kinase in its autoinhibited conformation; binding of asciminib locks ABL in this inactive state.

Pharmacokinetics

Tmax: 2-3 hours. Half-life: approximately 7-9 hours. Protein binding: ~97%. Metabolized by CYP3A4 and UGT2B7. Steady-state by Day 3-4. Excreted in feces (80%) and urine (11%).

14. Clinical Studies

Clinical efficacy and safety data supporting the approval are available in the full prescribing information and from the clinical trials listed below.

Pivotal Clinical Trials
Additional Resources
Approved Tumor Types