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DRUG CLASS:

IDH2 inhibitor

5d
New P1 trial
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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IDH2 mutation • IDH1 R132 • IDH2 R172
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lomustine • Voranigo (vorasidenib)
5d
Pharmacokinetic Study of Vorasidenib in Severe Hepatically Impaired and Matched-Control Participants (clinicaltrials.gov)
P1, N=20, Recruiting, Institut de Recherches Internationales Servier (I.R.I.S.) | Not yet recruiting --> Recruiting | Trial completion date: Jul 2026 --> Nov 2026 | Trial primary completion date: Jul 2026 --> Nov 2026
Enrollment open • Trial completion date • Trial primary completion date
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Voranigo (vorasidenib)
6d
Trial primary completion date
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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IDH2 mutation • IDH1 R132 • IDH2 R172
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Voranigo (vorasidenib)
21d
Imaging diagnosis of adult-type diffuse gliomas in the era of WHO CNS5 and cIMPACT-NOW updates 8-11: a narrative review. (PubMed, Jpn J Radiol)
The recent INDIGO trial, which demonstrated efficacy of the IDH1/2 inhibitor vorasidenib in residual grade 2 IDH-mutant glioma, has further increased the importance of preoperative imaging assessment of IDH mutation status...Third, we discuss the implications of cIMPACT-NOW Updates 8-11 for neuroradiologic diagnosis. In the WHO CNS5 era, imaging plays a central role in supporting integrated diagnosis and prioritizing appropriate molecular testing.
Review • Journal
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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IDH wild-type
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Voranigo (vorasidenib)
29d
A Drug-drug Interaction Study of Vorasidenib and a Combined Oral Contraceptive in Healthy Female Participants (clinicaltrials.gov)
P1, N=28, Completed, Institut de Recherches Internationales Servier (I.R.I.S.) | Recruiting --> Completed
Trial completion
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Voranigo (vorasidenib)
1m
Enasidenib in Combination With Cobimetinib for the Treatment of Relapsed or Refractory Acute Myeloid Leukemia (clinicaltrials.gov)
P1, N=3, Completed, City of Hope Medical Center | Active, not recruiting --> Completed | Trial completion date: May 2027 --> Mar 2026 | Trial primary completion date: May 2027 --> Mar 2026
Trial completion • Trial completion date • Trial primary completion date
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KRAS (KRAS proto-oncogene GTPase) • BRAF (B-raf proto-oncogene) • NRAS (Neuroblastoma RAS viral oncogene homolog) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • HRAS (Harvey rat sarcoma viral oncogene homolog) • NF1 (Neurofibromin 1) • PTPN11 (Protein Tyrosine Phosphatase Non-Receptor Type 11) • RIT1 (Ras Like Without CAAX 1)
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KRAS mutation • IDH2 mutation • RAS mutation
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Cotellic (cobimetinib) • Idhifa (enasidenib)
1m
Targeted therapies in adolescent and young adult patients with central nervous system tumors. (PubMed, Neurooncol Adv)
The IDH-mutant inhibitor vorasidenib has been demonstrated to prolong progression-free survival in grade 2 IDH-mutant glioma; however, there is a lack of evidence in patients younger than 18...Craniopharyngioma is a rare CNS tumor in the AYA population, and BRAFV600E mutations in papillary craniopharyngioma represent a targetable alteration. Solutions to improving the care of AYA should include appropriate representation in clinical trials and specialized care by experienced clinicians.
Review • Journal
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SHH (Sonic Hedgehog Signaling Molecule)
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BRAF V600E • BRAF V600 • IDH mutation + BRAF V600E
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Voranigo (vorasidenib)
1m
A Phase 1, Open-Label, Single Ascending (Two Levels) Dose Study to Evaluate the Pharmacokinetics of Vorasidenib in Healthy Japanese and Non-Asian Participants. (PubMed, J Clin Pharmacol)
In conclusion, vorasidenib was generally safe and well tolerated, and plasma exposures were generally similar between Japanese and non-Asian participants following single oral 10- and 50-mg vorasidenib doses. These results enabled vorasidenib clinical development in Japan and supported inclusion of Japanese sites in subsequent vorasidenib clinical trials without dose adjustments.
P1 data • PK/PD data • Journal
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1)
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Voranigo (vorasidenib)
1m
Multi-Omics and Machine Learning Analyses Reveal PIK3CG, PRKCD, and TRIM22 as Potential Markers of Poor Prognosis and Immune Activation in Glioblastoma. (PubMed, J Korean Med Sci)
This study identifies PIK3CG, PRKCD, and TRIM22 as potential biomarkers and therapeutic targets in IDH-wildtype GBM. Their paradoxical association with poor survival and immune activation may inform personalized treatment strategies that combine conventional chemotherapy with immune-based therapies. While our findings are robust across both mixed and IDH-wildtype-focused cohorts, further mechanistic validation is warranted.
Journal • PD(L)-1 Biomarker • IO biomarker
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PD-L1 (Programmed death ligand 1) • CD8 (cluster of differentiation 8) • PIK3CG (Phosphatidylinositol-4,5-Bisphosphate 3-Kinase Catalytic Subunit Gamma) • CD4 (CD4 Molecule)
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PD-L1 expression • IDH wild-type
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linsitinib (ASP7487) • AGI-6780
2ms
New trial
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Voranigo (vorasidenib)
2ms
Ivosidenib and Vorasidenib decrease intratumoral 2-hydroxyglutarate and total choline levels in patients with lower-grade glioma: an in vivo MR Spectroscopy study. (PubMed, Clin Cancer Res)
The striking reductions in tumor 2HG and tCho levels early following initiation of targeted therapy using an IDH inhibitor suggest that MRS may provide an important tool for monitoring treatment response of lower-grade gliomas.
Preclinical • Journal
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IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2)
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Tibsovo (ivosidenib) • Voranigo (vorasidenib)
2ms
Isocitrate Dehydrogenase-Mutant Astrocytomas: Risk Stratification and Therapeutic Advance. (PubMed, MedComm (2020))
Therapeutic advances are highlighted, with particular emphasis on brain-penetrant IDH inhibition (vorasidenib) and on emerging strategies including vaccines, checkpoint combinations, epigenetic modulation, metabolic and microenvironment targeting, and novel delivery platforms...Finally, future directions in trial design, survivorship-oriented endpoints, and biomarker-driven monitoring are outlined. A trajectory-based paradigm is emphasized in which neurocognitive preservation, time to radiotherapy or chemotherapy, and patient-reported outcomes are prioritized while durable disease control is pursued across decades-long survivorship.
Review • Journal
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TP53 (Tumor protein P53) • IDH1 (Isocitrate dehydrogenase (NADP(+)) 1) • IDH2 (Isocitrate Dehydrogenase (NADP(+)) 2) • CDKN2A (Cyclin Dependent Kinase Inhibitor 2A) • CDKN2B (Cyclin Dependent Kinase Inhibitor 2B) • ATRX (ATRX Chromatin Remodeler)
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CDKN2A deletion
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Voranigo (vorasidenib)