Therapeutic Response

NPM1 p.W288Cfs*12 status confers therapeutic sensitivity to Revumenib in patients with Acute Leukemias of Ambiguous Lineage.

Statements

Source and description

Revuforj (revumenib) [package insert]. FDA. The U.S. Food and Drug Administration (FDA) granted approval to revumenib for the treatment of adult and pediatric patients 1 year and older with relapsed or refractory acute myeloid leukemia and a susceptible .nucleophosmin 1 (NPM1) variant. The approval defines susceptible NPM1 mutations as those that result in a loss of the nucleolar localization signal and the insertion of a new nuclear export signal leading to the accumulation of mutant NPM1 in the cytoplasm of AML cells; the most common of such NPM1 variants in patients with AML being Types A (c.860_863dupTCTG), B (c.863_864insCATG), and D (c.863_864insCCTG).