IDH1 inhibitors(Isocitrate dehydrogenase [NADP] cytoplasmic inhibitors), IDH2 inhibitors(Isocitrate dehydrogenase [NADP], mitochondrial inhibitors), Epigenetic drug

Therapeutic Areas

NeoplasmsNervous System DiseasesOther Diseases

Active Indication

OligodendrogliomaIDH2 mutant GliomaAstrocytoma, IDH-Mutant+ [10]

Inactive Indication

Acute Myeloid LeukemiaLiver InjuryMyelodysplastic Syndromes

Originator Organization

Celgene Corp.

Active Organization

Institut de Recherches Intern ServierInstitut de Recherches Internationales Servier

Les Laboratoires Servier SAS

+ [4]

Inactive Organization

Agios Pharmaceuticals, Inc.

License Organization

Agios Pharmaceuticals, Inc.

Royalty Pharma Plc

Celgene Corp.

+ [2]

Drug Highest PhaseApproved

First Approval Date

Canada (01 Aug 2024),

IDH1-mutant Glioma

RegulationPriority Review (United States), Breakthrough Therapy (United States), Orphan Drug (United States), Priority Review (China), Orphan Drug (Japan), Orphan Drug (South Korea), Orphan Drug (Australia), Priority Review (Australia), Accelerated assessment (European Union), Fast Track (United States)

Structure/Sequence

Molecular FormulaC14H13ClF6N6

InChIKeyQCZAWDGAVJMPTA-RNFRBKRXSA-N

CAS Registry1644545-52-7

Clinical Trials associated with Vorasidenib

NCT07629089

/ Not yet recruitingPhase 1IIT

VALIENT: Phase 1 Clinical Trial of VorAsidenib in Combination With Lomustine In Patients With rEcurrent IDH-mutaNT Glioma Harboring IDH1 and/or IDH2 Mutations

This study will test the hypothesis that vorasidenib in combination with lomustine will be safe and tolerable. The overall goal of this study is to identify the optimal vorasidenib dose that can be tested in a subsequent phase 2 study to determine the efficacy of vorasidenib in combination with lomustine in patients with recurrent IDH mutant gliomas.

Start Date30 Jun 2026

Sponsor / Collaborator

University of Pittsburgh

[+1]

NCT07250633

/ RecruitingPhase 1

A Phase 1, Open-Label, Non-randomized, Single Dose, Safety, Tolerability, and Pharmacokinetic Study of Vorasidenib Administered to Participants With Severe Hepatic Impairment and Matched-Participants With Normal Hepatic Function

The objective of this study is to evaluate the pharmacokinetics, safety, and tolerability of one dose of vorasidenib in participants with severe impaired hepatic function compared to participants with normal hepatic function. The study includes a screening phase, a treatment period, and a follow-up period. During the first part of the treatment period, from Day 1 through Day 4, participants will remain in-house in the clinical research unit. In the second part of the treatment period, from Day 5 through Day 43, participants can go home but may also choose to remain in-house. The entire study, including screening and follow-up, will last up to 77 days. Participants may undergo blood tests, heart tests (electrocardiogram (ECG)), vital sign checks, and physical exams.

Start Date23 Apr 2026

Sponsor / Collaborator

Institut de Recherches Intern Servier

NCT07469735

/ Not yet recruitingNot ApplicableIIT

18F-AGX PET for Evaluation of Vorasidenib Response and Tumor Metabolic Changes in Low-grade IDH-Mutant Glioma

The goal of this prospective, single-arm, open-label clinical trial is to evaluate whether 18F-AGX PET imaging can be used to assess early treatment response and metabolic changes in adult patients with recurrent or residual WHO 2021 grade 2-3 IDH-mutant diffuse glioma receiving Vorasidenib therapy.
IDH-mutant diffuse gliomas often show slow tumor growth, making early treatment response difficult to evaluate using conventional structural imaging such as magnetic resonance imaging (MRI). Clinical endpoints such as progression-free survival (PFS) and overall survival (OS) typically require long follow-up periods to detect treatment effects. Therefore, the development of sensitive and noninvasive imaging methods for early evaluation of therapeutic response is needed.
This study aims to determine whether metabolic changes detected by 18F-AGX PET during Vorasidenib treatment are associated with tumor structural changes and clinical outcomes.
The main questions it aims to answer are:
* Whether early changes in tumor metabolic activity measured by 18F-AGX PET, including percentage change in maximum tumor-to-background ratio (TBRmax), are associated with changes in tumor growth rate (TGR) measured by MRI during treatment.
* Whether early metabolic response detected by 18F-AGX PET imaging after initiation of Vorasidenib treatment can predict subsequent disease progression or tumor growth dynamics.
Participants enrolled in this study will receive oral Vorasidenib once daily for 12 treatment cycles (28 days per cycle), with dosing based on body weight.
Participants will:
* Undergo baseline MRI and 18F-AGX PET imaging following surgery for recurrent or residual disease.
* Receive oral Vorasidenib continuously for 12 cycles.
* Undergo MRI scans at baseline and during treatment cycles 1, 2, 3, 6, 9, and 12 to assess structural tumor changes.
* Undergo 18F-AGX PET/CT scans at baseline and during treatment cycles 1, 2, 3, 6, and 12 to assess metabolic tumor activity.
* Provide serial blood samples for laboratory safety monitoring, including hematologic and biochemical testing.
* Undergo magnetic resonance spectroscopy (MRS) to quantify intratumoral 2-hydroxyglutarate (2-HG) levels as an indicator of IDH mutation-associated metabolic activity.
Participants will be followed for imaging-based disease progression using RANO criteria and for treatment-related adverse events during the study period.
This study will evaluate the feasibility of using 18F-AGX PET imaging as a noninvasive imaging biomarker for early response assessment in IDH-mutant diffuse glioma patients receiving targeted IDH inhibition therapy with Vorasidenib.

Start Date01 Apr 2026

Sponsor / Collaborator

Shanghai Huashan Hospital

100 Clinical Results associated with Vorasidenib

100 Translational Medicine associated with Vorasidenib

100 Patents (Medical) associated with Vorasidenib

104

Literatures (Medical) associated with Vorasidenib

01 Oct 2026·Neurotherapeutics

Current and emerging therapies in IDH-mutant glioma

Review

Author: Youssef, Gilbert ; Wen, Patrick Y ; Nakhate, Vihang

Isocitrate dehydrogenase (IDH)-mutant gliomas constitute a distinct molecular subtype of diffuse gliomas, characterized by unique biology and relatively favorable clinical outcomes. However, despite their more indolent initial course, these tumors ultimately develop treatment resistance and remain incurable. Standard treatment approaches have relied on surgery followed by radiation and alkylating chemotherapy, which provide meaningful disease control but are associated with cumulative neurocognitive toxicities. The oral mutant IDH inhibitor vorasidenib recently became the first targeted therapy available for IDH-mutant glioma. Based on results of the randomized phase 3 INDIGO trial, vorasidenib was approved by the US Food and Drug Administration in 2024 as a first-line treatment option for grade 2 IDH-mutant glioma following surgery. In this review, we summarize the current therapeutic paradigm for IDH-mutant glioma, including the use of radiation and chemotherapy as well as the evolving role of mutant IDH-targeted therapy. We also highlight emerging therapeutic strategies, including approaches targeting key biologically informed vulnerabilities such as DNA damage repair pathways, cell-cycle and metabolic dependencies, tumor-associated hypermethylation, and anti-tumor immune activation. Collectively, these advances reflect a rapidly evolving treatment landscape driven by improved understanding of IDH biology, and hold promise to overcome therapeutic resistance and improve patient outcomes.

01 Jun 2026·CURRENT THERAPEUTIC RESEARCH-CLINICAL AND EXPERIMENTAL

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

Review

Author: Owens, Katie ; Argon, Sophie ; Yu, Jingjing ; Ragueneau-Majlessi, Isabelle ; Wang, Yan

Objective:

This analysis aimed to provide a mechanistic understanding and an evaluation of the clinical relevance of pharmacokinetic drug-drug interactions (DDIs) associated with drugs approved by the Food and Drug Administration in 2024.

Methods:

Drug metabolism, transport, and absorption-based DDI data available in New Drug Applications (NDAs) of small molecular drugs approved (n = 34) was analyzed. The mechanism and clinical outcome of these interactions were characterized based on in vitro, in silico, and clinical data.

Results:

As objects, 11 drugs were identified as clinical substrates. 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. All clinical DDIs with AUC changes ≥ 2-fold triggered labeling recommendations. 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.

Conclusions:

Mechanistic DDI insights from newly approved therapies can be extrapolated to inform the management of commonly co-administered drugs, supporting a safer and more effective use of new drug products in the context of polypharmacy.

01 Jun 2026·CANCER

Cost‐effectiveness of vorasidenib in treatment of isocitrate dehydrogenase–mutated low‐grade glioma

Article

Author: Waddle, Mark ; Kowalchuk, Roman O. ; Yan, Elizabeth ; Laack, Nadia N. ; Breen, William G. ; Kizilbash, Sani ; Lehrer, Eric J. ; Neth, Bryan ; Kim, Hayeon ; Mahajan, Anita ; Schultz, Sydney E. ; Kessel, Adam C. ; Brown, Paul D. ; Sener, Ugur

Abstract:

Background:

Vorasidenib is Food and Drug Administration‐approved for treatment of grade 2 isocitrate dehydrogenase (IDH)–mutant gliomas and has demonstrated a benefit in progression‐free survival compared to a placebo. This study assessed the cost‐effectiveness of this treatment strategy compared to standard treatment: observation or chemoradiation (CRT).

Methods:

The authors used an integrated Markov model to compare quality‐adjusted life‐years (QALY) gained and cost‐effectiveness of patients with IDH‐mutated low‐grade glioma (LGG) treated with vorasidenib, observation, or CRT. Outcomes were expressed as incremental cost‐effectiveness ratios (ICERs) per QALY gained. Treatment is considered cost‐effective if it meets the commonly accepted willingness‐to‐pay threshold of $100,000 per QALY gained.

Results:

Based on model parameters, vorasidenib demonstrated an ICER of $356,756 per QALY when compared to CRT. When compared to observation, vorasidenib demonstrated an improved ICER of $215,191 per QALY. On one‐way sensitivity analysis compared to observation, median cost of vorasidenib and median overall survival were most likely to affect ICER with only marginal changes on analysis of other model factors. For ICER to become ≤$100,000 compared to observation, the cost of vorasidenib would need to drop to below $14,072 monthly or have a median survival gain of at least 11.9 years. The probabilistic sensitivity analysis demonstrated that vorasidenib was favored in none of 5000 Monte Carlo iterations.

Conclusion:

Using the best available data, the current price of vorasidenib does not meet commonly accepted cost‐effectiveness thresholds in treatment of IDH‐mutated LGGs.

News (Medical) associated with Vorasidenib

31 May 2026

·www.prnewswire.com

Servier to Present Extended Follow-Up Results from the Phase 3 INDIGO Trial Showing Durable and Sustained Treatment Benefits of VORANIGO® (vorasidenib) at ASCO 2026

Latest long-term analysis, including more than three years of follow-up data of patients with Grade 2 IDH-mutant glioma treated with VORANIGO® (vorasidenib), will be presented in a rapid oral session on May 31 at 4:36 p.m. CDT Results show median progression-free survival among patients treated with VORANIGO was 44.1 months Findings from an exploratory analysis from the INDIGO study evaluating the impact of VORANIGO on seizure rates and quality of life will be shared in a separate poster presentation BOSTON, May 31, 2026 /PRNewswire/ -- Servier today announced updated efficacy and safety results from more than three years of follow-up in the Phase 3 INDIGO trial evaluating VORANIGO® (vorasidenib) versus placebo in patients with Grade 2 mutant isocitrate dehydrogenase 1 or 2 (IDH1/2) glioma following surgical intervention and who are not in immediate need of chemoradiotherapy. These data, which will be presented at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting on May 31 at 4:36 p.m. CDT in Chicago, strengthen the previous findings from the INDIGO pivotal trial and demonstrate the clinical benefits of VORANIGO continue to improve over time. The extended analysis includes 21.3 months of additional unblinded data collected between the March 7, 2023, trial unblinding and the January 17, 2025, data cutoff. As of data cutoff, all 163 patients enrolled in the placebo arm discontinued treatment and 144 crossed over to the VORANIGO treatment arm. Median follow-up was 41.6 months. These results build upon the placebo-controlled, double-blind data previously published in The Lancet Oncology. "The extended Phase 3 INDIGO trial analysis significantly improves our understanding of the clinical benefits of VORANIGO," said Islam Hassan, M.D., MSc., Global Head of Development-Neuro-Oncology at Servier. "With more than three years of follow-up data, these results validate the durable and sustained benefits of long-term treatment with VORANIGO and demonstrate a steady improvement in patient responses over time. We are proud to share these results and remain committed to expanding our understanding of the long-term impact of VORANIGO for the glioma community." A global, randomized, double-blinded, Phase 3 trial of vorasidenib vs placebo in patients with grade 2 glioma with an IDH1/2 mutation (INDIGO): updated efficacy and safety – Sunday, May 31, 4:36 - 4:42 p.m. CDT Key findings, which were presented by Timothy Cloughesy, M.D., David Geffen School of Medicine, Department of Neurology, University of California, Los Angeles, an investigator for the INDIGO trial, include: Median progression-free survival (PFS) per blinded independent review committee (BIRC) was 44.1 months (95% CI, 27.7-not estimable [NE]) in patients treated with VORANIGO. PFS was the primary endpoint of the trial. The median time to next intervention (TTNI) per BIRC for patients treated with VORANIGO was NE (95% CI, 52-NE). The number of next-intervention events remained low among VORANIGO patients (23.8%), indicating that VORANIGO is effectively delaying the need for subsequent treatment. TTNI was a key secondary endpoint of the trial. Patients treated with VORANIGO experienced an objective response rate (ORR) per BIRC of 20.8% (95% CI, 15%-27.8%). ORR improved with longer follow-up, reflecting durable and gradual responses with VORANIGO. An additional 72.6% of patients experienced stable disease. Median duration of treatment among VORANIGO patients was 38.3 months (95% CI, 19.98-43.76). A 72% reduction in the rate of on-treatment seizures was observed in patients treated with VORANIGO. The safety profile of VORANIGO was tolerable with mainly low-grade adverse events (AEs) and consistent with previously reported data. The most commonly reported Grade ≥3 or worse treatment-emergent adverse events (TEAEs) were increased alanine aminotransferase (10.8%), increased aspartate aminotransferase (4.8%), seizures (4.8%), increased gamma-glutamyltransferase (3%) and syncope (1.8%). No new safety signals were detected and fewer than 5% of patients discontinued treatment due to an AE. There were no treatment-related deaths. "Historically, many patients with Grade 2 IDH-mutant gliomas faced a poor long-term prognosis and managed their disease with a 'watch and wait' strategy due to the limited availability of targeted treatment options," said Dr. Timothy Cloughesy. "The latest results from the Phase 3 INDIGO trial—the largest dataset in IDH-mutant glioma to date—provide valuable evidence on how long-term IDH inhibition can delay disease progression and extend the time to next intervention." Impact of vorasidenib vs placebo on seizure rates and quality of life: exploratory analysis from Phase 3 INDIGO study – Monday, June 1, 1:30 - 4:30 p.m. CDT Findings from an exploratory analysis of the INDIGO study evaluating the impact of VORANIGO on seizure rates and quality of life will be shared in a separate presentation. As of data cut off in January 2025, results show: The rate of seizures per person per year in the VORANIGO arm (15.9 seizures per person-year [95% CI, 9.1-27.7]; p=0.0002) demonstrated the sustained, positive, long-term effect of VORANIGO on seizure control. The effects of VORANIGO were more pronounced among people with oligodendrogliomas (6.9 seizures per person-year [95% CI, 2.7-17.7]; p<0.0001) than those with astrocytoma (17.5 seizures per person-year [95% CI, 9.4-32.3]; p=0.6448). VORANIGO had an acceptable safety profile with no new safety signals during extended follow-up. Media Contact Darby Malkin: [email protected] About Servier in Oncology Servier is a global leader in oncology, governed by a non-profit foundation. Servier approaches innovation with a long-term vision, free of influence from fiduciary responsibilities. Servier invests close to 20% of revenue from brand-name medicines in R&D each year. Servier aspires to advance more targeted therapies by identifying mutations and understanding how these mutations impact cancer and its progression. Servier believes we can serve more people by helping the right patients find the right treatment, at the right time. Servier takes a One Innovation Engine approach to R&D and is actively seeking alliances, partnerships and acquisitions at various stages of the portfolio. For more information about working with Servier to bring the promise of tomorrow to the patients it serves, visit Servier.com and Servier.us. About the INDIGO Phase 3 Trial (NCT04164901)1 INDIGO, the pivotal Phase 3 clinical trial, was a registration-enabling Phase 3 global, randomized, double-blind placebo-controlled study of vorasidenib in patients with residual or recurrent Grade 2 glioma with an isocitrate dehydrogenase 1/2 (IDH1/2) mutation who have undergone surgery as their only treatment. As of March 7, 2023, 331 patients were randomized globally to receive VORANIGO 40 mg daily (n=168) or placebo (n=163) continuously in 28-day cycles. Of the 331 patients, 172 had oligodendroglioma (88 VORANIGO; 84 placebo) and 159 patients had astrocytoma (80 VORANIGO; 79 placebo). The extended analysis includes 21.3 months of additional unblinded data collected between the March 7, 2023, trial unblinding and the January 17, 2025, data cutoff. As of data cutoff, all 163 patients enrolled in the placebo arm discontinued treatment and 144 crossed over to the VORANIGO treatment arm. Median follow-up was 41.6 months. About Glioma2 Gliomas are tumors that arise from glial or precursor cells within the central nervous system (CNS). The 2021 World Health Organization (WHO) classification recognizes four general groups of gliomas, one of which is adult-type diffuse gliomas. These diffuse gliomas are the most common primary malignant brain tumors in adults. The pathogenesis and prognosis of these tumors are tightly linked to mutations (or lack thereof) in the metabolic enzyme isocitrate dehydrogenase (IDH), and molecular testing is required for proper diagnosis. As of 2021, adult-type diffuse gliomas are sub-divided into only three categories: Astrocytoma, IDH-mutant (CNS WHO grades 2-4) Oligodendroglioma, IDH-mutant and1p19q-codeleted (CNS WHO grades 2-3) Glioblastoma, IDH-wildtype (CNS WHO grade 4) VORANIGO IMPORTANT SAFETY INFORMATION What is VORANIGO? VORANIGO (40 mg tablets) is a prescription medicine used to treat adults and children 12 years of age and older with certain types of brain tumors called astrocytoma or oligodendroglioma with an isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, following surgery. Your healthcare provider will perform a test to make sure that VORANIGO is right for you. It is not known if VORANIGO is safe and effective in children under 12 years of age. What are the possible side effects of VORANIGO? VORANIGO may cause serious side effects, including: Liver problems. Changes in liver function blood tests may happen during treatment with VORANIGO and can be serious. Your healthcare provider will do blood tests to check your liver function before and during treatment with VORANIGO. Tell your healthcare provider right away if you develop any of the following signs and symptoms of liver problems: yellowing of your skin or the white part of your eyes (jaundice) dark tea-colored urine  loss of appetite  pain on the upper right side of your stomach area  feeling very tired or weak The most common side effects of VORANIGO include: increased liver enzyme levels in the blood lack of energy, tiredness headache COVID-19 muscle aches or stiffness diarrhea nausea seizure Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with VORANIGO if you have certain side effects. VORANIGO may affect fertility in females and males, which may affect the ability to have children. Talk to your healthcare provider if this is a concern for you. These are not all of the possible side effects of VORANIGO. Before taking VORANIGO, tell your healthcare provider about all of your medical conditions, including if you: have liver problems have kidney problems or are on dialysis smoke tobacco are pregnant or plan to become pregnant. VORANIGO can harm your unborn baby Females who are able to become pregnant: Your healthcare provider will do a pregnancy test before you start treatment with VORANIGO You should use effective nonhormonal birth control during treatment with VORANIGO and for 3 months after the last dose. VORANIGO may affect how hormonal contraceptives (birth control) work and cause them to not work well. Talk to your healthcare provider about birth control methods that may be right for you during treatment with VORANIGO Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with VORANIGO Males with female partners who are able to become pregnant: You should use effective birth control during treatment with VORANIGO and for 3 months after the last dose Tell your healthcare provider right away if your partner becomes pregnant or thinks she may be pregnant during your treatment with VORANIGO Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if VORANIGO passes into breast milk.  Do not breastfeed during treatment with VORANIGO and for 2 months after the last dose. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. VORANIGO may affect the way other medicines work, and other medicines may affect how VORANIGO works. Please click  here  for full prescribing information. Disclosures This release contains general information about the Servier Group and its entities (hereinafter "Servier and its Affiliates") and is intended for informational purposes only. The information is thought to be reliable; however, Servier and its Affiliates make no representation as to the accuracy or completeness of the information contained herein or otherwise provided and accept no responsibility or liability, in contract, in tort, in negligence, or otherwise, should the information be found to be inaccurate or incomplete in any respect. Servier and its Affiliates are not acting as an advisor to the recipient of this information, and the ultimate decision to proceed with any transaction rests solely with the recipient of this information. Therefore, prior to entering into any proposed transaction, the recipient of this information should determine, without reliance upon Servier or its Affiliates, the economic risks and merits, as well as the legal, tax, and accounting characterizations and consequences, of the transaction and that it is able to assume these risks. This statement also contains forward-looking statements that are subject to varying levels of uncertainty and risk. Investigational new drugs and indications are subject to further scientific and medical review and regulatory approval. They are not approved for use by the FDA. Any reliance placed on this document is done entirely at the risk of the person placing such reliance. The information contained in this document is neither an offer to sell nor the solicitation of an offer to enter into a transaction. The content of this document is a summary only, is not complete, and does not include all material information about Servier and its Affiliates, including potential conflicts of interest. To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates disclaim all representations, warranties, conditions and guarantees, whether express, implied, statutory or of other kind, nor does it accept any duty to any person, in connection with this document. Without prejudice to the generality of the foregoing, Servier and its Affiliates do not warrant or represent that the information or opinions contained in this document is accurate or complete. To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates shall not be liable for any loss, damage or expense whatsoever, whether direct or indirect, howsoever arising, whether in contract, tort (including negligence), strict liability or otherwise, for direct, indirect, incidental, consequential, punitive or special damages arising out of or in connection with this document, including (without limitation) any course of action taken on the basis of the same. The estimates, strategies, and views expressed in this document are based upon past or current data and information and are subject to change without notice. References 1. Mellinghoff, I. K., van den Bent, M. J., Blumenthal, D. T., Touat, M., Peters, K. B., Clarke, J., Mendez, J., Yust-Katz, S., Welsh, L., Mason, W. P., Ducray, F., Umemura, Y., Nabors, B., Holdhoff, M., Hottinger, A. F., Arakawa, Y., Sepulveda, J. M., Wick, W., Soffietti, R., … Cloughesy, T. F. (2023). Vorasidenib in idh1- or IDH2-mutant low-grade glioma. New England Journal of Medicine, 389(7), 589–601. 2. Louis DN, Perry A, Wesseling P, Brat DJ, Cree IA, Figarella-Branger D, Hawkins C, Ng HK, Pfister SM, Reifenberger G, Soffietti R, von Deimling A, Ellison DW. The 2021 WHO Classification of Tumors of the Central Nervous System: a summary. Neuro Oncol. 2021 Aug 2;23(8):1231-1251. doi: 10.1093/neuonc/noab106. PMID: 34185076; PMCID: PMC8328013. SOURCE Servier Pharmaceuticals 21% more press release views with Request a Demo

Clinical ResultPhase 3ASCO

21 May 2026

·www.prnewswire.com

Servier Presentations at ASCO 2026 Spotlight Expanding Rare Oncology Portfolio

Updated efficacy and safety data from a long-term analysis of the Phase 3 INDIGO trial of VORANIGO® (vorasidenib) will be presented in a rapid oral presentation Results from an ongoing Phase 1 trial evaluating investigational compound Emi-Le in adenoid cystic carcinoma will be shared in an oral presentation BOSTON, May 21, 2026 /PRNewswire/ -- Servier today announced that it will present new and updated data at the 2026 American Society of Clinical Oncology (ASCO) Annual Meeting May 29 - June 2 in Chicago. Presentations will span across a range of rare cancers, including IDH-mutated glioma and adenoid cystic carcinoma (ACC). "Servier's upcoming presentations at ASCO—which include updated long-term data in IDH-mutant glioma as well as emerging data from our newer oncology programs—underscore our dedication to delivering transformative medicines to patients with high unmet needs," said Becky Martin, PhD, Chief of Medical, Servier Pharmaceuticals. "Together, these programs demonstrate the continued evolution of Servier's oncology portfolio and our growing presence in rare oncology." Notably, Servier will present updated efficacy and safety data from a long-term analysis of the Phase 3 INDIGO trial of VORANIGO® (vorasidenib) in a rapid oral presentation on May 31 at 4:36 p.m. CDT. Key findings from more than three years of follow-up data further support the robust progression-free survival (PFS) and time to next treatment intervention (TTNI) results observed with VORANIGO in previous analyses and confirm the durable and sustained treatment benefit of VORANIGO in Grade 2 IDH-mutant glioma. Additional data from an exploratory analysis of the INDIGO trial demonstrating long-term treatment with VORANIGO generally led to a sustained decrease in seizure frequency and severity and preservation of quality of life will be presented as well. Day One Biopharmaceuticals, now part of Servier Group, will also share results from an ongoing Phase 1 trial evaluating investigational compound Emi-Le (emiltatug ledadotin) in an oral presentation on June 1 at 8:24 a.m. CDT. The data reveal that Emi-Le demonstrates favorable tolerability and promising antitumor activity in patients with aggressive ACC who have no available treatment options and a poor prognosis. Further clinical development is ongoing. In addition, a placebo-controlled trial of vorasidenib in IDH-mutated newly diagnosed Grade 3 astrocytoma led by the Alliance for Clinical Trials in Oncology will be featured in a trial in progress presentation. A full list of ASCO abstracts can be found here. Please visit the Servier booth (#12079) and the Day One booth (#36155) onsite to learn more. Servier will also present research updates at the European Hematology Association (EHA) 2026 Congress June 11-14 in Stockholm. Media contact Darby Malkin: [email protected] About Servier Servier is an independent international pharmaceutical group governed by a foundation. With its governance model, the Group is committed to therapeutic progress to serve patients and integrates the patient voice at every stage of the medicine life cycle. As a leading global player in cardiology and venous diseases, Servier aims to become a leading innovator in oncology and neurology. The Group intends to offer targeted therapeutic solutions, particularly in rare cancers and neurological diseases, and invests nearly 20% of its brand-name sales in R&D. Headquartered in France, Servier relies on its more than 20,000 employees and a solid geographic presence with medicines distributed in more than 130 countries. In the 2024/25 financial year, the Group achieved revenues of €6.9 billion. More information on the Group website: servier.com Follow us on social media: LinkedIn, Facebook, X, Instagram About Servier in Oncology Servier is a global leader in oncology, governed by a non-profit foundation. Servier approaches innovation with a long-term vision, free of influence from fiduciary responsibilities. Servier invests close to 20% of revenue from brand-name medicines in R&D each year. Servier aspires to advance more targeted therapies by identifying mutations and understanding how these mutations impact cancer and its progression. Servier believes we can serve more people by helping the right patients find the right treatment, at the right time. Servier takes a One Innovation Engine approach to R&D and is actively seeking alliances, partnerships and acquisitions at various stages of the portfolio. For more information about working with Servier to bring the promise of tomorrow to the patients it serves, visit Servier.com and Servier.us. About Emi-Le Emi-Le is a B7-H4-directed Dolasynthen ADC with a precise, target-optimized drug-to-antibody ratio (DAR 6) and a proprietary auristatin-F HPA payload with controlled bystander effect. This candidate is under evaluation in an ongoing Phase 1 clinical trial (previously with Mersana Therapeutics). The U.S. Food and Drug Administration has granted Fast Track designations to Emi-Le for the treatment of 1) adult patients with advanced or metastatic triple-negative breast cancer, and 2) advanced or metastatic HER2 low / HER2 negative breast cancer post-topo-1 ADC (including TNBC and certain HR+ breast cancers). It has also received Breakthrough Designation from the FDA for the treatment of patients with locally advanced, recurrent or metastatic adenoid cystic carcinoma with solid histology or high-grade transformation. For more information about the trial, visit clinicaltrials.gov (NCT05377996). VORANIGO IMPORTANT SAFETY INFORMATION What is VORANIGO? VORANIGO (40 mg tablets) is a prescription medicine used to treat adults and children 12 years of age and older with certain types of brain tumors called astrocytoma or oligodendroglioma with an isocitrate dehydrogenase-1 (IDH1) or isocitrate dehydrogenase-2 (IDH2) mutation, following surgery. Your healthcare provider will perform a test to make sure that VORANIGO is right for you. It is not known if VORANIGO is safe and effective in children under 12 years of age. What are the possible side effects of VORANIGO? VORANIGO may cause serious side effects, including: Liver problems. Changes in liver function blood tests may happen during treatment with VORANIGO and can be serious. Your healthcare provider will do blood tests to check your liver function before and during treatment with VORANIGO. Tell your healthcare provider right away if you develop any of the following signs and symptoms of liver problems: yellowing of your skin or the white part of your eyes (jaundice) dark tea-colored urine loss of appetite pain on the upper right side of your stomach area feeling very tired or weak The most common side effects of VORANIGO include: increased liver enzyme levels in the blood lack of energy, tiredness headache COVID-19 muscle aches or stiffness diarrhea nausea seizure Your healthcare provider may change your dose, temporarily stop, or permanently stop treatment with VORANIGO if you have certain side effects. VORANIGO may affect fertility in females and males, which may affect the ability to have children. Talk to your healthcare provider if this is a concern for you. These are not all of the possible side effects of VORANIGO. Before taking VORANIGO, tell your healthcare provider about all of your medical conditions, including if you: have liver problems have kidney problems or are on dialysis smoke tobacco are pregnant or plan to become pregnant. VORANIGO can harm your unborn baby Females who are able to become pregnant: Your healthcare provider will do a pregnancy test before you start treatment with VORANIGO You should use effective nonhormonal birth control during treatment with VORANIGO and for 3 months after the last dose. VORANIGO may affect how hormonal contraceptives (birth control) work and cause them to not work well. Talk to your healthcare provider about birth control methods that may be right for you during treatment with VORANIGO Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with VORANIGO Males with female partners who are able to become pregnant: You should use effective birth control during treatment with VORANIGO and for 3 months after the last dose Tell your healthcare provider right away if your partner becomes pregnant or thinks she may be pregnant during your treatment with VORANIGO Tell your healthcare provider if you are breastfeeding or plan to breastfeed. It is not known if VORANIGO passes into breast milk. Do not breastfeed during treatment with VORANIGO and for 2 months after the last dose. Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements. VORANIGO may affect the way other medicines work, and other medicines may affect how VORANIGO works. Please click here for full prescribing information. Disclosures This release contains general information about the Servier Group and its entities (hereinafter "Servier and its Affiliates") and is intended for informational purposes only. The information is thought to be reliable; however, Servier and its Affiliates make no representation as to the accuracy or completeness of the information contained herein or otherwise provided and accept no responsibility or liability, in contract, in tort, in negligence, or otherwise, should the information be found to be inaccurate or incomplete in any respect. Servier and its Affiliates are not acting as an advisor to the recipient of this information, and the ultimate decision to proceed with any transaction rests solely with the recipient of this information. Therefore, prior to entering into any proposed transaction, the recipient of this information should determine, without reliance upon Servier or its Affiliates, the economic risks and merits, as well as the legal, tax, and accounting characterizations and consequences, of the transaction and that it is able to assume these risks. This statement also contains forward-looking statements that are subject to varying levels of uncertainty and risk. Investigational new drugs and indications are subject to further scientific and medical review and regulatory approval. They are not approved for use by the FDA. Any reliance placed on this document is done entirely at the risk of the person placing such reliance. The information contained in this document is neither an offer to sell nor the solicitation of an offer to enter into a transaction. The content of this document is a summary only, is not complete, and does not include all material information about Servier and its Affiliates, including potential conflicts of interest. To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates disclaim all representations, warranties, conditions and guarantees, whether express, implied, statutory or of other kind, nor does it accept any duty to any person, in connection with this document. Without prejudice to the generality of the foregoing, Servier and its Affiliates do not warrant or represent that the information or opinions contained in this document is accurate or complete. To the maximum extent permitted by applicable laws and regulations, Servier and its Affiliates shall not be liable for any loss, damage or expense whatsoever, whether direct or indirect, howsoever arising, whether in contract, tort (including negligence), strict liability or otherwise, for direct, indirect, incidental, consequential, punitive or special damages arising out of or in connection with this document, including (without limitation) any course of action taken on the basis of the same. The estimates, strategies, and views expressed in this document are based upon past or current data and information and are subject to change without notice. SOURCE Servier Pharmaceuticals 21% more press release views with Request a Demo

Fast TrackPhase 1Phase 3ASCOBreakthrough Therapy

06 Mar 2026

·www.fiercebiotech.com

Servier to widen rare cancer offerings with $2.5B buyout of Day One and glioma drug Ojemda

The crown jewel of a transaction with Servier is no doubt Day One Biopharmaceuticals\' Ojemda (tovorafenib), a type 2 RAF inhibitor approved in 2024 to treat patients 6 months and older with relapsed or refractory pediatric low-grade glioma. \n As nonprofit-governed Servier continues to make gains with its IDH-mutant glioma med Voranigo, the drugmaker is wading deeper into the rare oncology arena with a new M&A play. Servier on Friday unveiled a definitive agreement to acquire Day One Biopharmaceuticals—a commercial-stage company developing targeted therapies for pediatric cancers and other diseases—for $21.50 per share in cash. The total value of the deal, which is expected to close in the second quarter, comes to roughly $2.5 billion, the companies said in a March 6 release. The crown jewel of the transaction is no doubt Day One’s Ojemda (tovorafenib), a type 2 RAF inhibitor approved by the FDA in April 2024 to treat patients ages 6 months and older with relapsed or refractory pediatric low-grade glioma (pLGG), a more advanced stage of the common childhood brain tumor. That drug will likely mesh well with Servier’s Voranigo, also approved in 2024 under a different glioma niche in patients 12 years and older with a susceptible IDH1 or IDH2 mutation. Servier acquired that drug by way of transaction, too, picking Voranigo up in its 2021 purchase of Agios Pharmaceuticals for $2 billion. Over on the R&D side of things, Servier will also be able to tap into Day One’s early- to late-stage pipeline, which includes additional programs for tovorafenib in pediatric low-grade gliomas, plus assets running through development in indications like adenoid cystic carcinoma and adult and pediatric solid tumors. Servier says it plans to fund the deal through a mix of existing cash and investments. The company’s share price offer reflects a premium of roughly 68% over Day One’s closing price March 5, according to the companies’ press announcement. “In less than a decade, we have built a durable oncology platform in the United States,” David Lee, CEO of Servier’s U.S. arm, told Fierce in an emailed statement Friday. “That didn’t happen through short-term thinking—it came from sustained investment in science, infrastructure and people.”He reiterated Servier’s ambition to “lead in rare cancers,” noting that for these patients, “progress often depends on a small number of people and organizations choosing to commit when others will not.” Lee continued, “When the science is promising and the need is urgent, we believe it is our responsibility to build the capabilities that can turn discovery into real outcomes for patients.”Servier has long leaned on its patient-centric ethos—credited to its unique governance model under the nonprofit Fondation Internationale de Recherche Servier—as one of the company’s key strengths and distinguishing features. Lee remarked similarly on the company’s governance model allowing Servier “to do things that other companies can’t” and “invest in diseases that other people would not be able to make a business case for” during an interview with Fierce Pharma at the J.P. Morgan Healthcare Conference in 2025. At the time, Lee noted that the landscape for brain cancer treatment remained “pretty open,” adding that, in terms of potential external opportunities for Servier, “anything mutation-driven is still good for us … but we’re open to any [glioblastomas], gliomas or astrocytomas.”Taking a look at the population Ojemda addresses, the FDA has specifically cleared the RAF inhibitor in relapsed or refractory pLGG patients with a BRAF fusion or rearrangement, or BRAF V600 mutation. PLGG is the most common type of childhood brain tumor, with BRAF alterations making up about 1,100 of 1,500 new diagnoses each year. Outside the U.S., Ojemda also won a positive recommendation for approval in the same indication in Europe late last month, a region where Ipsen holds licensing rights to the Day One drug. Servier is making its latest oncology play as the drugmaker seeks to achieve 10 billion euros ($11.5 billion) in annual sales by 2030. After a successful showing in its 2024-25 financial year, which ended in September with 16.2% growth to 6.9 billion euros ($8.2 billion), the group’s president, Olivier Laureau, said in January that Servier was “one important step closer” to meeting that target. Servier didn’t mince words about Voranigo’s contribution to its earnings haul last year, noting that its overall oncology business grew 55% in the 2024-25 fiscal period, with the unit’s contribution toward Servier’s overall revenue rising to 32% from 24% in the previous period. Servier has been busy on the oncology dealmaking front, last year licensing a phase 1 asset from Black Diamond Therapeutics for RAF/RAS-mutant solid tumors, acquiring a phase 1/2 leukemia candidate from BioNova Pharmaceuticals and obtaining ex-U.S. rights to Ideaya Biosciences’ potential first-in-class PKC inhibitor darovasertib under development in uveal melanoma—not to mention an oncology drug discovery pact with artificial intelligence biotech Insilico Medicine made in January. As of late, the company has also been working to bolster its offerings in neurology.

Drug ApprovalAcquisitionPhase 1

100 Deals associated with Vorasidenib

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R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Oligodendroglioma	India	Les Laboratoires Servier SAS	10 Dec 2025
IDH2 mutant Glioma	United Kingdom	Les Laboratoires Servier SAS	16 Sep 2025
Astrocytoma, IDH-Mutant	United States	Servier Pharmaceuticals LLC	06 Aug 2024
IDH-mutant oligodendroglioma	United States	Servier Pharmaceuticals LLC	06 Aug 2024
IDH1-mutant Glioma	Canada	Servier Canada, Inc.	01 Aug 2024

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Astrocytoma	Phase 3	China	Institut de Recherches Intern Servier	18 Oct 2024
Astrocytoma	Phase 3	Taiwan Province	Institut de Recherches Intern Servier	18 Oct 2024
Glioma	Phase 3	China	Institut de Recherches Intern Servier	18 Oct 2024
Glioma	Phase 3	Taiwan Province	Institut de Recherches Intern Servier	18 Oct 2024
Recurrent Glioma	Phase 3	United States	Institut de Recherches Intern Servier	05 Jan 2020
Recurrent Glioma	Phase 3	Japan	Institut de Recherches Intern Servier	05 Jan 2020
Recurrent Glioma	Phase 3	Canada	Institut de Recherches Intern Servier	05 Jan 2020
Recurrent Glioma	Phase 3	France	Institut de Recherches Intern Servier	05 Jan 2020
Recurrent Glioma	Phase 3	Germany	Institut de Recherches Intern Servier	05 Jan 2020
Recurrent Glioma	Phase 3	Israel	Institut de Recherches Intern Servier	05 Jan 2020

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04164901 (INDIGO, ASCO2026)	Phase 3	WHO Grade II Glioma IDH1 Mutation \| IDH2 Mutation	331	Vorasidenib 40 mg	cnsjtuqdtp(iggrogbwlo) = jutprpmdta sqanenlvbb (elmecsywuy, NE - NE) View more	Positive	29 May 2026
				Placebo	aolwwjyshw(pubwxgdhzb) = rwurbkwfwl xdcmnlugku (popaywjbdw, 11.1 - 13.9) View more
NCT04164901 (INDIGO, ASCO2026)	Phase 3	isocitrate dehydrogenase 1/2 mutations	110	Vorasidenib 40 mg	lwmqlnjidk(oxmhselmbk) = lwlpydmtnr rfkrwhlszy (uankhzwvwu, 5.8 - 29.8) View more	Positive	29 May 2026
				Placebo	lwmqlnjidk(oxmhselmbk) = sigmpnagnf rfkrwhlszy (uankhzwvwu, 26.5 - 159.1) View more
ASCO2026	Not Applicable	Glioma IDH-mutant	186	Vorasidenib (1L VORA)	jidejbqfbg(vfqzhohpnv) = wancaeozsv socsijrxrd (baklkrjkqn ) View more	Positive	29 May 2026
				Vorasidenib (prior CT/RT)	jidejbqfbg(vfqzhohpnv) = fwsrazrlix socsijrxrd (baklkrjkqn ) View more
VORAFET (ASCO2026)	Not Applicable	Glioma IDH-mutant	26	Vorasidenib	mdmyegeqwg(orgpgowelv) = kbbypxlggt njsphbyrys (evyncjnfmq ) View more	Positive	29 May 2026
EANO2025	Not Applicable	IDH2 mutant Glioma IDH mutations	45	Vorasidenib	udxjkmtsgq(uxealyepoj) = eekphhcsvf aoclitpwwi (sbdziafliv ) View more	Positive	16 Oct 2025
NCT04164901 (INDIGO, EANO2025)	Phase 3	WHO Grade II Glioma IDH1 Mutation \| IDH2 Mutation	331	Vorasidenib 40 mg	nbbnlowlkl(afrwtxnayl) = gijslsnpjm bzeedrxifr (ulpgpsuddd, 8.4 - 39.5)	Positive	16 Oct 2025
				Placebo	nbbnlowlkl(afrwtxnayl) = qvnzupmfgh bzeedrxifr (ulpgpsuddd, 22.9 - 114.8)
INDIGO trial (ASCO2025)	Phase 3	Glioma	30	Vorasidenib 40 mg/day	hsoajiyfln(obfdpgrlyk) = 37% (n = 11) kxlpvrjdex (vohzzcdlbx ) View more	Positive	30 May 2025
NCT03343197 (SNO2024) Manual	Phase 1	IDH1-mutant Glioma IDH1 R132H	46	Vorasidenib 50 mg QD (after surgery)	emnbeovwou(wqbhlwmmbj) = qhzrdwmtqm rzdecixslm (ayncvyhrsd ) View more	Positive	11 Nov 2024
				Ivosidenib 500 mg BID (after surgery)	emnbeovwou(wqbhlwmmbj) = tfpthcbbbf rzdecixslm (ayncvyhrsd ) View more
NCT04164901 (INDIGO, SNO2024) Manual	Phase 3	Glioma IDH2 Mutation \| IDH1 Mutation	331	Vorasidenib 40 mg	bhgophggkh(uhjirdsctu) = oqwoxkcogj enjbkwkuaw (aebcvkscmg, 22.1 - NE) View more	Positive	11 Nov 2024
				Placebo	bhgophggkh(uhjirdsctu) = aqydqnjres enjbkwkuaw (aebcvkscmg, 11.1 - 13.9) View more
NCT05592743 (SNO2024) Manual	Not Applicable	Glioma IDH1 Mutation \| IDH2 Mutation	71	Vorasidenib	vhgnkydskf(tuejixynsd) = npimwaklja nydyzptcsf (ajabpkdnur ) View more	-	11 Nov 2024

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