Vorasidenib (AG-881) is a brain-penetrant allosteric inhibitor of mutant isocitrate dehydrogenases 1 and 2 (mIDH1/2) from Agios and Celgene that made headlines summer 2023 due to its stunning efficacy for treatment of glioma in patients with mIDH1/2. Daily oral dosing of vorasidenib increased progression-free survival by a median time of more than 2 years (27.7 months versus 11.1 months with placebo).

This Featured Case Study reviews how it was discovered, how it works, and why it matters.