Asciminib (Scemblix)

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Mechanism of action

From the NCI Drug Dictionary: An orally bioavailable, allosteric Bcr-Abl tyrosine kinase inhibitor with potential antineoplastic activity. Designed to overcome resistance, asciminib binds to the Abl portion of the Bcr-Abl fusion protein at a location that is distinct from the ATP-binding domain, and is a so-called STAMP (Specifically Targeting the ABL Myristoyl Pocket) inhibitor. This binding results in the inhibition of Bcr-Abl-mediated proliferation and enhanced apoptosis of Philadelphia chromosome-positive (Ph+) hematological malignancies.

Diseases for which it is used

History of changes in FDA indication

  • 10/29/2021: Granted accelerated approval for patients with Philadelphia chromosome-positive chronic myeloid leukemia (Ph+ CML) in chronic phase (CP), previously treated with two or more tyrosine kinase inhibitors (TKIs). (Based on ASCEMBL)
  • 10/29/2021: Approved for adult patients with Ph+ CML in CP with the T315I mutation. (Based on CABL001X2101)

Also known as

  • Code name: ABL001
  • Brand name: Scemblix