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Asciminib

Deterministic view of the source YAML entity. Clinical authority remains with the cited source IDs and reviewer sign-off state.

IDDRUG-ASCIMINIB
TypeDrug
Aliases
ScemblixАсцимініб
Statusreviewed 2026-04-25 | pending_clinical_signoff
DiseasesDIS-CML
SourcesSRC-ELN-CML-2020 SRC-NCCN-MPN-2025

Drug Facts

ClassBCR-ABL1 STAMP inhibitor (Specifically Targeting the ABL Myristoyl Pocket; allosteric)
MechanismAllosteric inhibitor binding the ABL1 myristoyl pocket — distinct from the ATP-binding site targeted by all classical TKIs (imatinib, dasatinib, nilotinib, bosutinib, ponatinib). This unique mechanism avoids cross-resistance with most ATP-site mutations including T315I (at higher dose 200 mg BID). First-in-class STAMP inhibitor; approved 3L+ for CML-CP after ≥2 prior TKIs (ASCEMBL trial) and for T315I-mutated CML.
Typical dosingCML-CP after ≥2 prior TKIs (no T315I): 80 mg PO once daily OR 40 mg PO BID (with or without food, but consistency required; high-fat meal reduces exposure). T315I-positive CML-CP: 200 mg PO BID. Continue until progression / unacceptable toxicity.
Ukraine registeredFalse
NSZU reimbursedFalse
Ukraine last verified2026-04-27

Notes

Pivotal: ASCEMBL (Réa et al., Blood 2021) phase-3 — asciminib 40 mg BID vs bosutinib 500 mg daily in CML-CP after ≥2 prior TKIs; MMR at 24 weeks 25.5% vs 13.2%. Vascular safety profile substantially better than ponatinib. T315I activity at higher dose (200 mg BID) per phase-1 (Hughes et al., NEJM 2019). Ukraine: NOT registered — access via named-patient import / EAP Novartis / cross-border. Dose-and-take consistency (with food vs without) matters: high-fat meal substantially reduces exposure. Lipase + ECG monitoring; vascular safety profile much better than ponatinib but cardiovascular events still observed.

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