vorasidenib

Vorasidenib is a first-in-class, oral, small molecule dual inhibitor of mutant isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2). It is highly brain-penetrant and selectively targets both wild-type and mutant forms of IDH1 and IDH2 enzymes. By inhibiting these enzymes, vorasidenib suppresses the production of the oncometabolite D-2-hydroxyglutarate (2-HG), which accumulates in tumors with IDH mutations and contributes to oncogenesis by blocking cellular differentiation. Vorasidenib was developed to overcome resistance mechanisms seen with single isoform inhibitors and to improve blood-brain barrier penetration for effective treatment of central nervous system tumors. The drug was first approved by the FDA in August 2024 for use in adult and pediatric patients aged 12 years or older with Grade 2 astrocytoma or oligodendroglioma harboring susceptible IDH1 or IDH2 mutations following surgery[3][4][5][6][7][8].

Brand names

Voranigo

Other names

vorasidenib