Vorasidenib

Isocitrate Dehydrogenase 1 Inhibitor — ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS

Key facts

Vorasidenib is an onp drug in the Isocitrate Dehydrogenase 1 Inhibitor class catalogued in DrugCentral. It was first approved by the FDA in 2024. Its mechanism of action is classified as Cytochrome P450 3A Inducers, Isocitrate Dehydrogenase 1 Inhibitors and Isocitrate Dehydrogenase 2 Inhibitors. DrugCentral lists 2 molecular targets, including IDH1 and IDH2. Recorded indications include Grade 2 astrocytoma.

• Approval status: onp (first approved 2024 by the FDA).
• Drug class: Isocitrate Dehydrogenase 1 Inhibitor; Isocitrate Dehydrogenase 2 Inhibitor.
• Mechanism of action: Cytochrome P450 3A Inducers; Isocitrate Dehydrogenase 1 Inhibitors; Isocitrate Dehydrogenase 2 Inhibitors.
• Molecular targets: IDH1 and IDH2 (2 listed in DrugCentral).
• Indications: Grade 2 astrocytoma.
• ATC code: L01XM04 (Isocitrate dehydrogenase (IDH) inhibitors).
• Molecular formula: C14H13ClF6N6; molecular weight 414.74 g/mol.

Indications & contraindications

Drug–disease relationships curated by DrugCentral (OMOP / SNOMED concepts).

Indications (1)

• Grade 2 astrocytoma

Mechanism & targets

• Mechanism of action (MoA): Cytochrome P450 3A Inducers; Isocitrate Dehydrogenase 1 Inhibitors; Isocitrate Dehydrogenase 2 Inhibitors
• Established pharmacologic class: Isocitrate Dehydrogenase 1 Inhibitor; Isocitrate Dehydrogenase 2 Inhibitor

Molecular targets (2)

Target	Gene	Action	Activity
Isocitrate dehydrogenase [NADP] cytoplasmic	IDH1	inhibitor	IC50 7.69
Isocitrate dehydrogenase [NADP], mitochondrial	IDH2	inhibitor	IC50 6.95

Regulatory approvals (3)

Date	Agency	Marketed as	Applicant	Orphan
2025-09-19	PMDA	—	Nihon Servier Co., Ltd.	Yes
2025-09-17	EMA	—	Les Laboratoires Servier	Yes
2024-08-06	FDA	—	Servier	Yes

ATC classification

ATC code	Therapeutic hierarchy
L01XM04	ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS › ANTINEOPLASTIC AGENTS › OTHER ANTINEOPLASTIC AGENTS › Isocitrate dehydrogenase (IDH) inhibitors

Pharmacogenomics

Drug-label and guideline annotations from PharmGKB and CPIC.

• EMA label: IDH1, IDH2 · Testing Required
• FDA label: IDH1, IDH2 · Testing Required
• HCSC label: IDH1, IDH2 · Testing Required

Reported adverse-event signals

Strongest FDA FAERS disproportionality signals (log-likelihood ratio). A signal is a statistical association in spontaneous reports, not proof that the drug causes the event.

MedDRA term	LLR
Alanine aminotransferase increased	194.0
Aspartate aminotransferase increased	150.3
Hepatic cytolysis	144.3

Chemical structure

• Molecular formula: C₁₄H₁₃ClF₆N₆
• Molecular weight: 414.74 g/mol
• CAS number: —
• InChIKey: QCZAWDGAVJMPTA-RNFRBKRXSA-N
• PubChem CID: 117817422

C[C@H](C(F)(F)F)NC1=NC(=NC(=N1)C2=NC(=CC=C2)Cl)N[C@H](C)C(F)(F)F

Frequently asked questions

Answers are generated from the structured data on this page, not written by hand.

What is Vorasidenib used for?

DrugCentral lists the following indications for Vorasidenib: Grade 2 astrocytoma.

How does Vorasidenib work?

Vorasidenib is classified as Cytochrome P450 3A Inducers, Isocitrate Dehydrogenase 1 Inhibitors and Isocitrate Dehydrogenase 2 Inhibitors; its molecular targets in DrugCentral include IDH1 (inhibitor) and IDH2 (inhibitor).

When was Vorasidenib first approved?

Vorasidenib was first approved by the FDA in 2024.

What drug class does Vorasidenib belong to?

Vorasidenib is in the Isocitrate Dehydrogenase 1 Inhibitor class.

What is the chemical formula of Vorasidenib?

Vorasidenib has the molecular formula C14H13ClF6N6 and a molecular weight of 414.74 g/mol.

Sources

• DrugCentral drug card 5829 — structure, indications, targets, approvals, pharmacologic classes (CC BY-SA 4.0)
• ChEMBL CHEMBL4279047 — compound record and bioactivity
• PubChem CID 117817422 — chemical identifiers
• DrugBank DB17097 — drug record
• FDA UNII 789Q85GA8P — substance identity
• DailyMed: FDA labels for Vorasidenib — current prescribing information (label search)
• UniProt O75874 (IDH1) — target protein
• UniProt P48735 (IDH2) — target protein
• FDA Adverse Event Reporting System (openFDA FAERS) — adverse-event signals, as aggregated by DrugCentral
• PharmGKB / CPIC — pharmacogenomic label annotations

How this page is built: methodology · editorial policy & corrections.