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Asciminib: First Approval

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Abstract

Asciminib (Scemblix®) is an orally administered, small molecule, selective allosteric inhibitor that targets the myristoyl pocket of the BCR-ABL1 tyrosine kinase and is being developed by Novartis for the treatment of haematological malignancies, including Philadelphia chromosome-positive (Ph+) chronic myeloid leukaemia (CML). The drug is active against a number of the single catalytic-site mutations, such as T315I, that confer resistance to conventional tyrosine kinase inhibitors (TKIs) that bind to the ATP-binding site of BCR-ABL1. In October 2021, asciminib monotherapy was granted accelerated approval for the treatment of adults with Ph+ CML in chronic phase (CML-CP), previously treated with ≥ 2 TKIs, and full approval for the treatment of adults with Ph+ CML-CP with the T315I mutation. The drug is under regulatory review for use as monotherapy in CML in the EU, and is in phase 1–3 development exploring its potential in first-line, later-line and paediatric patients with CML. This article summarizes the milestones in the development of asciminib leading to this first approval for the treatment of adults with Ph+ CML-CP, previously treated with ≥ 2 TKIs, and Ph+ CML-CP with the T315I mutation.

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    Emma D. Deeks

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Correspondence to Emma D. Deeks.

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During the peer review process the manufacturer of the agent under review was offered an opportunity to comment on the article. Changes resulting from any comments received were made by the authors on the basis of scientific completeness and accuracy. Emma Deeks is a contracted employee of Adis International Ltd/Springer Nature, and declares no relevant conflicts of interest. All authors contributed to the review and are responsible for the article content.

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This profile has been extracted and modified from the AdisInsight database. AdisInsight tracks drug development worldwide through the entire development process, from discovery, through pre-clinical and clinical studies to market launch and beyond.

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Deeks, E.D. Asciminib: First Approval. Drugs 82, 219–226 (2022). https://doi.org/10.1007/s40265-021-01662-3

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