SCEMBLIX asciminib

Drug Class

STAMP inhibitor

Mechanism

Binds specifically to the myristoyl pocket of ABL kinase (STAMP), inducing an inactive conformation to block BCR-ABL1 proliferation signals.

Approved Indication

Treatment of adult patients with Philadelphia chromosome-positive chronic myeloid leukemia in chronic phase (Ph+ CML-CP), previously treated with two or more tyrosine kinase inhibitors (TKIs), or adult patients with Ph+ CML-CP with the T315I mutation.

What This Means For Patients

Scemblix (asciminib) is a targeted STAMP inhibitor designed for patients with chronic myeloid leukemia (CML). It targets a unique binding pocket on the BCR-ABL1 protein, making it effective in patients who have failed other therapies. It is available under the special-import policy in the Hainan Boao Lecheng pilot zone.

Regulatory Approvals

• FDA — approved (2021) — label
• EMA — approved (2022) — source
• NMPA — approved (2023) — source

Pivotal Clinical Evidence

• ASCEMBL — Réa et al., Blood 2021 (NCT03106779) — DOI

Sources

• https://www.accessdata.fda.gov/drugsatfda_docs/label/2021/215358s000lbl.pdf
• https://www.ema.europa.eu/en/medicines/human/EPAR/scemblix
• https://www.nmpa.gov.cn/