Vorasidenib

Clinical data
Trade names	Voranigo
License data	US DailyMed: Vorasidenib
Routes of administration	By mouth
ATC code	None
Legal status
Legal status	US: ℞-only[1]
Identifiers
IUPAC name 6-(6-Chloropyridin-2-yl)-2-N,4-N-bis[(2R)-1,1,1-trifluoropropan-2-yl]-1,3,5-triazine-2,4-diamine
CAS Number	1644545-52-7
PubChem CID	117817422
IUPHAR/BPS	10663
DrugBank	DB17097
ChemSpider	64835242
UNII	789Q85GA8P
KEGG	D11834
ChEMBL	ChEMBL4279047
Chemical and physical data
Formula	C14H13ClF6N6
Molar mass	414.74 g·mol−1
3D model (JSmol)	Interactive image
SMILES C[C@H](C(F)(F)F)NC1=NC(=NC(=N1)C2=NC(=CC=C2)Cl)N[C@H](C)C(F)(F)F
InChI InChI=1S/C14H13ClF6N6/c1-6(13(16,17)18)22-11-25-10(8-4-3-5-9(15)24-8)26-12(27-11)23-7(2)14(19,20)21/h3-7H,1-2H3,(H2,22,23,25,26,27)/t6-,7-/m1/s1 Key:QCZAWDGAVJMPTA-RNFRBKRXSA-N

Vorasidenib, sold under the brand name Voranigo, is an anti-cancer medication used for the treatment of certain forms of glioma.^[1][2] Vorasidenib acts to inhibit the enzymes isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2).^[1][2]

The most common adverse reactions include fatigue, headache, increased risk of COVID-19 infection, musculoskeletal pain, diarrhea, nausea, and seizures.^[2]

Vorasidenib was approved for medical use in the United States in August 2024.^[2][3] It is the first approval by the US Food and Drug Administration (FDA) of a systemic therapy for people with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation.^[2]

Vorasidenib is indicated for the treatment of people aged twelve years of age and older with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation, following surgery including biopsy, sub-total resection, or gross total resection.^[2]

The most common adverse reactions include fatigue, headache, increased risk of COVID-19 infection, musculoskeletal pain, diarrhea, nausea, and seizures.^[2] The most common grade 3 or 4 laboratory abnormalities include increased alanine aminotransferase, increased aspartate aminotransferase, GGT increased, and decreased neutrophils.^[2]

Efficacy was evaluated in 331 participants with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation following surgery enrolled in INDIGO (NCT04164901), a randomized, multicenter, double-blind, placebo-controlled trial.^[2] Participants were randomized 1:1 to receive vorasidenib 40 mg orally once daily or placebo orally once daily until disease progression or unacceptable toxicity.^[2] Isocitrate dehydrogenase-1 or isocitrate dehydrogenase-2 mutation status was prospectively determined by the Life Technologies Corporation Oncomine Dx Target Test.^[2] Participants randomized to placebo were allowed to cross over to vorasidenib after documented radiographic disease progression.^[2] Participants who received prior anti-cancer treatment, including chemotherapy or radiation therapy, were excluded.^[2]

Vorasidenib was approved for medical use in the United States in August 2024.^[2]

The FDA granted the application for vorasidenib priority review, fast track, breakthrough therapy, and orphan drug designations.^[2]

• Clinical trial number NCT04164901 for "Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)" at ClinicalTrials.gov
• Clinical trial number NCT02481154 for "Study of Orally Administered AG-881 in Patients With Advanced Solid Tumors, Including Gliomas, With an IDH1 and/or IDH2 Mutation" at ClinicalTrials.gov
• Clinical trial number NCT03343197 for "Study of AG-120 and AG-881 in Subjects With Low Grade Glioma" at ClinicalTrials.gov