Olutasidenib: Potentially Longer Complete Remissions in AML with a Second-Generation IDH1 Inhibitor
Olutasidenib (Rezlidhia), discovered by Forma Therapeutics and marketed by Rigel, is an oral, brain-penetrant, selective mIDH1 inhibitor. It was granted Orphan Drug Designation and approved in December 2022 by the FDA for adults with relapsed/refractory acute myeloid leukemia (AML). Approval was granted based on a Ph. I/II trial showing a 35% CR+CRh rate and a favorable 25.9-month median duration.
Compared to the first approved mIDH1 inhibitor, ivosidenib (Tibsovo), olutasidenib stands out with its favorable efficacy profile. This article describes the discovery and development of olutasidenib, how it is distinct from other mIDH inhibitors, and its potential in treating gliomas.