Voranigo (vorasidenib) / Servier, Royalty - LARVOL DELTA

LB06: Pharmacist-Led Management of Vorasidenib in Patients with Isocitrate Dehydrogenase (IDH) Mutant Gliomas (HOPA 2026) - "Background: Vorasidenib was recently approved for the treatment of low-grade IDH 1- and 2-mutant gliomas, demonstrating higher central nervous system (CNS) penetration compared to Ivosidenib1. Close monitoring of patients with low-grade gliomas is vital to ensure safe, efficacious, and comprehensive care. The findings from this study highlight the critical role of pharmacist-led interventions in optimizing medication therapy management in this complex patient population."

Clinical • Brain Cancer • Epilepsy • Glioma • Solid Tumor • CYP1A2 • CYP3A4

Pharmacist-Led Management of Vorasidenib in Patients With Isocitrate Dehydrogenase Mutation–Positive Gliomas (HOPA 2026) - "BACKGROUND: Vorasidenib was recently approved for the treatment of low-grade isocitrate dehydrogenase (IDH) 1 and IDH2 mutation–positive gliomas, demonstrating higher central nervous system penetration compared with ivosidenib.1 Increasing use of antidepressants and antiepileptic drugs has been seen in patients with low-grade gliomas.2,3 The vorasidenib prescribing information recommends avoiding concomitant administration of cytochrome (CY) P450 1A2 inhibitors and inducers, as well as CYP3A4 substrates,4 which presents a challenge when starting patients on vorasidenib. Close monitoring of patients with low-grade gliomas is vital to ensure safe, efficacious, and comprehensive care. The findings from this study highlight the critical role of pharmacist-led interventions in optimizing medication therapy management in this complex patient population."

Clinical • Late-breaking abstract • Brain Cancer • Glioma • Solid Tumor • CYP3A4 • IDH2

NanoGlio recapitulates IDH-mutant glioma biology and therapeutic response to vorasidenib (AACR 2026) - "Furthermore, NanoGlio-derived conditioned media, when applied to co-cultures of HER2-specific chimeric antigen receptor (CAR) T cells and HER2-positive SKOV3 tumor cells, enabled functional assessment of soluble factor-mediated immune suppression. Together, these data establish NanoGlio as a scalable, patient-relevant organoid platform for modeling IDH-mutant glioma biology and therapeutic response, with direct applications to drug-response prediction and rational combination strategies in precision oncology."

Brain Cancer • Glioma • Oncology • Solid Tumor • HER-2 • IDH1 • IDH2

Vorasidenib in IDH1- or IDH2-Mutant Low-Grade Glioma. (PubMed, N Engl J Med) - P3 | "In patients with grade 2 IDH-mutant glioma, vorasidenib significantly improved progression-free survival and delayed the time to the next intervention. (Funded by Servier; INDIGO ClinicalTrials.gov number, NCT04164901.)."

Journal • Brain Cancer • CNS Tumor • Glioma • Oncology • Solid Tumor • IDH1 • IDH2

CL1-95032-005: Study of Vorasidenib and Pembrolizumab Combination in Recurrent or Progressive IDH-1 Mutant Glioma (clinicaltrials.gov) - P1 | N=60 | Active, not recruiting | Sponsor: Institut de Recherches Internationales Servier | Recruiting ➔ Active, not recruiting

Enrollment closed • Anaplastic Oligoastrocytoma • Astrocytoma • Brain Cancer • Glioma • Oligodendroglioma • Oncology • Solid Tumor • ATRX • IDH1

Efficacy of vorasidenib in patients with IDH-mutant gliomas previously treated with ivosidenib (ESMO-TAT 2026) - "Treatments received prior to ivosidenib included surgical resection (23, 79%), radiation (16, 55%), temozolomide (15, 52%), PCV (4, 14%) bevacizumab (3, 10%), lomustine (2, 7%) and pembrolizumab (1, 3%). Vorasidenib demonstrated clinical efficacy and tolerability in patients with IDH-mutant gliomas previously treated with ivosidenib. Notably, patients who transitioned due to FDA approval or toxicity maintained disease stability, while a subset of those with progression on ivosidenib achieved stabilization with vorasidenib. These findings support vorasidenib as a viable therapeutic option following ivosidenib exposure."

Clinical • Anaplastic Astrocytoma • Astrocytoma • Brain Cancer • Glioblastoma • Glioma • Oligodendroglioma • Oncology • Solid Tumor • IDH1

VANGUARD: Vorasidenib Guided by AGX PET in Recurrent/Low-grade Glioma (clinicaltrials.gov) - P=N/A | N=10 | Not yet recruiting | Sponsor: Huashan Hospital

New trial • Brain Cancer • Glioma • Oncology • Solid Tumor • IDH1 • IDH2

Preserved IDH mutation and methylation class in vorasidenib nonresponders: A report of 2 cases. (PubMed, Neurooncol Pract) - "At the last follow-up, 3 years after vorasidenib discontinuation, the disease remained stable in 1 patient, while the other one had recurred. Vorasidenib does not appear to select an IDH-wildtype clone that would potentially reduce the tumor's sensitivity to standard therapies typically used in IDH-mutated glioma."

Journal • Brain Cancer • Glioma • Oncology • Solid Tumor • MGMT

Today, the Scottish Medicines Consortium (SMC) have approved vorasidenib – also known by its brand name Voranigo – to be used by NHS Scotland to treat people aged 12 or older with IDH-mutated, low grade gliomas – including astrocytomas and ogliodendrogliomas. (The Brain Tumour Charity)

Reimbursement • Astrocytoma • Low Grade Glioma • Oligodendroglioma

Exploratory Analyses from the Phase Three INDIGO Study Suggest Potential Mechanism of Seizure Control Through Tumor Volume Reduction Under Treatment With Vorasidenib (AAN 2026) - P3 | "Vorasidenib was associated with lower seizure rate and severity than placebo in patients with mIDH1/2 glioma. Smaller tumor volume was associated with lower seizure rate. Our analyses suggest a potential mechanism of seizure control with vorasidenib through tumor size reduction."

Astrocytoma • Brain Cancer • CNS Disorders • Epilepsy • Glioma • Oligodendroglioma • Oncology • Solid Tumor • IDH1

Bridging Neuro-oncology and Gender-affirming Care: Clinical and Treatment Insights From a Multi-site Mayo Clinic Cohort (AAN 2026) - "Tumor-directed treatments included surgery (n=10), radiation therapy (n=6), and systemic therapy (n=7, using temozolomide, lomustine, ivosidenib, vorasidenib, lapatinib, bevacizumab). GAHT was well tolerated and not associated with accelerated tumor progression in most cases. The temporal association between estrogen exposure and symptom fluctuation in an NF2-associated meningioma highlights the need for individualized risk assessment and multidisciplinary care in this understudied population."

Clinical • Astrocytoma • Brain Cancer • CNS Tumor • Glioblastoma • Glioma • Meningioma • Ocular Inflammation • Oligodendroglioma • Ophthalmology • Optic Neuritis • Pituitary Gland Carcinoma • Solid Tumor • BRAF

Beyond Cytotoxic Therapy: A Meta-analysis of IDH-targeted Agents in Lower-grade Gliomas (AAN 2026) - "Objective: This meta-analysis evaluates the clinical potential of mutant isocitrate dehydrogenase (IDH) enzyme inhibitors—ivosidenib and vorasidenib—as an intervention strategy aimed at disrupting glioma pathogenesisBackground: Gliomas harboring IDH mutations present therapeutic challenges due to their infiltrative nature and resistance to conventional treatments. IDH-mutant enzyme inhibitors demonstrate modest efficacy in treatment of gliomas, with substantial disease stabilization and prolonged progression free survival in non-enhancing gliomas. However, their low response rates and frequent toxicities call for careful risk–benefit assessment."

Retrospective data • Brain Cancer • Glioma • Solid Tumor

A network-driven computational framework for identifying FDA-approved drug repurposing across heterogeneous brain cancers. (PubMed, Front Mol Biosci) - "As a result, three repurposed drugs were identified as priorities: (i) mefloquine (reference drug: vorasidenib citrate), (ii) clofibric acid (reference drug: carmustine), and armillarisin A (reference drug: lomustine). These results also suggest repurposing candidates for synergistic combinations across different brain tumors. The two applications developed in this work are freely accessible and in the public domain at https://assay.smallmoles.com/escorwin."

FDA event • Heterogeneity • Journal • Brain Cancer • Oncology • Solid Tumor • BRAF • CDK1 • EGFR • KDR • PDGFRA • TERT • TP53

EORTC-2427-BTG: ??Vorasidenib for the treatment of IDH-mutant astrocytoma after standard chemoradiotherapy (clinicaltrialsregister.eu) - P2/3 | N=247 | Recruiting | Sponsor: Europese Organisatie Voor Onderzoek En Behandeling Van Kanker Organisation Europeenne Pour La Recherche Et Le Traitement Du Cancer European Organi

New P2/3 trial • Astrocytoma • Brain Cancer • Oncology • Solid Tumor • IDH1 • IDH2

Multicenter Implementation of the German Compassionate Use Program Vorasidenib in IDH-Mutant Glioma: Feasibility and Early Outcomes (DKK 2026) - P | "A multicentre vorasidenib CUP/EAP was implemented and improved access for patients with IDH-mutant glioma. Early safety and DC data are consistent with those from the trial leading to marketing authorization. No novel findings were observed in this real-world setting."

Clinical • Late-breaking abstract • Astrocytoma • Brain Cancer • Cardiovascular • CNS Disorders • Epilepsy • Glioma • Oligodendroglioma • Solid Tumor • IDH1 • IDH2

Ontario becomes the first province to publicly reimburse the first targeted oral therapy for adults with Grade 2 IDH-mutant glioma. (Yahoo Finance) - "Servier Canada Announces Ontario Coverage of VORANIGO Under the FAST Program."

Reimbursement • Glioma

Understanding Pharmacokinetic-Drug Interactions With Drugs Approved by the US Food and Drug Administration in 2024 to Better Manage the Risk of Drug Interactions With Concomitant Medications: A Review of Clinical Data From New Drug Applications. (PubMed, Curr Ther Res Clin Exp) - "Of these, 7 drugs were substrates of CYP3A, 3 of CYP2C9, one of CYP1A2, and one of CYP2C8, including the sensitive substrates vanzacaftor (CYP3A) and vorasidenib (CYP1A2). As precipitants, 6 drugs (acoramidis, cefepime/enmetazobactam, givinostat, lazertinib, mavorixafor, and resmetirom) were clinical inhibitors of CYP enzymes (2C8, 2C9, 2D6, 2E1, and 3A), with mavorixafor being a CYP2D6 strong inhibitor. Two drugs (elafibranor and tovorafenib) showed weak induction of CYP3A. Regarding transporter data, 3 drugs were substrates of transporters, including seladelpar (BCRP and OAT3), sulopenem (OAT3), and vadadustat (OAT1/3), and 8 drugs (arimoclomol, danicopan, givinostat, lazertinib, mavorixafor, resmetirom, vadadustat, and vazacaftor/tezacaftor/deutivacaftor) were inhibitors of transporters...Several DDIs with an AUC change <2 also had labeling recommendations, pertaining most often to the concomitant use of drugs with a narrow therapeutic index. Mechanistic DDI..."

Clinical data • FDA event • Journal • NDA • PK/PD data • Review • CYP1A2 • CYP2C9

Voranigo: “The Committee confirmed that all issues previously identified in this application had been addressed”; Astrocytoma or oligodendroglioma (European Medicines Agency) - CHMP Final Minutes of the meeting on 21 - 24 Jul 2025: “The Committee adopted a positive opinion recommending the granting of a marketing authorization by consensus together with the CHMP assessment report and translation timetable”

CHMP • Astrocytoma • Oligodendroglioma • Oncology

IDH-mutant inhibitors enhance the sensitivity of IDH1-mutant gliomas to cysteine-methionine deprivation and ferroptosis. (PubMed, bioRxiv) - "In addition, treatments with the IDH-mutant inhibitors vorasidenib and ivosidenib further sensitize the cells to ferroptosis. Furthermore, dietary cysteine-methionine deprivation alone or in combination with convection-enhanced delivery of RSL3 or ivosidenib in vivo significantly prolongs survival of IDH1-mutant tumor-bearing mice. Our findings suggest that targeting cysteine and methionine metabolism in combination with IDH-mutant inhibition provides promising therapeutic strategies for IDH1-mutant gliomas."

Journal • Brain Cancer • Glioma • Oncology • Solid Tumor • IDH1

Royalty Receipts was $856 million in the fourth quarter of 2025, an increase of 17% compared to $729 million in the fourth quarter of 2024. (The Manila Times) - "The increase was primarily driven by Voranigo, Trelegy, Tremfya and the cystic fibrosis franchise, which was partially offset by a decline from Promacta due to U.S. generic competition which launched in May 2025."

Commercial • Aplastic Anemia • Asthma • Astrocytoma • Chronic Obstructive Pulmonary Disease • Crohn's disease • Cystic Fibrosis • Glioma • Immune Thrombocytopenic Purpura • Immunology • Oligodendroglioma • Psoriasis • Psoriatic Arthritis • Ulcerative Colitis

Isocitrate Dehydrogenase Inhibitors in Acute Myeloid Leukemia. (PubMed, Chem Biodivers) - "Inhibitors of mutated IDH1 and IDH2, vorasidenib, ivosidenib, olutasidenib, and enasidenib, respectively, were recently approved by the FDA for relapsed/refractory AML. In this review, we mainly focus on IDH inhibitors in leukemia therapy, including the discovery, structure optimization, activity of IDH inhibitors, and applications, which provided the reference for the discovery of new anticancer agents."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1 • IDH2

VIGOR: Vorasidenib Maintenance for IDH Mutant Astrocytoma (clinicaltrials.gov) - P3 | N=468 | Recruiting | Sponsor: European Organisation for Research and Treatment of Cancer - EORTC | Not yet recruiting ➔ Recruiting

Enrollment open • Astrocytoma • Brain Cancer • Oncology • Solid Tumor • IDH1 • IDH2

18F-DOPA-PET and advanced MRI improve treatment response assessment in IDH1/2-mutant gliomas treated with IDH inhibitors. (PubMed, Clin Cancer Res) - "These results highlight the potential of ¹⁸F-DOPA-PET and advanced MRI sequences as valuable complements to standard RANO 2.0 MRI evaluations for assessing treatment response in glioma patients undergoing IDHi therapy."

Journal • Astrocytoma • Brain Cancer • Glioma • Oligodendroglioma • Oncology • Solid Tumor • IDH1 • IDH2

Reasons driving choice and clinical course of patients with CNS WHO grade 3 IDH mutant glioma receiving vorasidenib after surgery: a pilot experience. (PubMed, J Neurooncol) - No abstract available

Journal • Astrocytoma • Brain Cancer • Glioma • Oligodendroglioma • Oncology • Solid Tumor

INDIGO: A global, randomized, double-blinded, phase 3 study of vorasidenib versus placebo in patients with residual or recurrent grade 2 glioma with an IDH1/2 mutation. (ASCO 2023) - P3 | "This is the first prospective, randomized phase 3 study of a targeted therapy in grade 2 mIDH glioma. VOR significantly improved PFS by BIRC compared with PBO with a manageable safety profile. These data demonstrate the clinical benefit of VOR in this pt population for whom chemotherapy and radiotherapy are being delayed."

Clinical • Late-breaking abstract • P3 data • Astrocytoma • Brain Cancer • CNS Tumor • Fatigue • Glioma • Infectious Disease • Novel Coronavirus Disease • Oligodendroglioma • Oncology • Pain • Solid Tumor • IDH1 • IDH2