Servier Receives Positive CHMP Opinion for VORANIGO® (vorasidenib) for the Treatment of Adults and Adolescents with Grade 2 IDH-mutant Diffuse Glioma

• Recommendation is based on positive results from the pivotal Phase 3 INDIGO trial of VORANIGO
• If approved, VORANIGO would be the first and only targeted therapy for Grade 2 IDH-mutant glioma in the EU

BOSTON, July 25, 2025 /PRNewswire/ -- Servier today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion recommending the approval of VORANIGO^® (vorasidenib) in the European Union (EU) for the treatment of predominantly non-enhancing Grade 2 astrocytoma or oligodendroglioma with an isocitrate dehydrogenase-1 (IDH1) R132 or isocitrate dehydrogenase-2 (IDH2) R172 mutation in adult and adolescent patients aged 12 years and older and weighing at least 40 kg who only had surgical intervention and who are not in immediate need of radiotherapy or chemotherapy.  

The marketing authorization application for VORANIGO will now be reviewed by the European Commission (EC), which has the authority to approve medicines for use in the 27 member states of the EU. Decisions by the EC are also applicable in Norway, Liechtenstein, and Iceland.

"Today's recommendation for EU approval brings us one step closer to offering VORANIGO to patients in the EU with Grade 2 IDH-mutant glioma who have historically had limited treatment options for this relentless disease," said Susan Pandya, M.D., Vice President Clinical Development and Global Head of Oncology LS/LCM, Servier. "We look forward to continuing conversations with the EMA and other regulatory bodies around the world to introduce VORANIGO as a potential new standard of care for patients with IDH-mutant gliomas."

The CHMP opinion is based on the positive results of the Phase 3 INDIGO trial, a global Phase 3 randomized, double-blind placebo-controlled study of vorasidenib in patients with residual or recurrent Grade 2 glioma with an IDH1/2 mutation who have undergone surgery as their only treatment. Results were presented during the plenary session at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting and published simultaneously in The New England Journal of Medicine.

VORANIGO was approved by the United States Food and Drug Administration (FDA) in August 2024 after being granted Fast Track, Breakthrough and Orphan Drug Designations and receiving Priority Review. VORANIGO has also been granted marketing authorization in Canada, Australia, Israel, the United Arab Emirates, Saudi Arabia, and Switzerland. Servier has also submitted marketing authorization applications in the United Kingdom, Japan and various other regions, and reviews by the appropriate health authorities are ongoing.   

The use of VORANIGO is investigational in the EU and is not yet approved.  

Media contact
Sara Noonan: sara.noonan@servier.com I Tel. +1 857 262 3855

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 is the leader in IDH-mutant targeted therapies and devotes more than 65% of its research and development budget to Oncology. 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.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). 

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)  

About VORANIGO
VORANIGO is an isocitrate dehydrogenase-1 (IDH1) and isocitrate dehydrogenase-2 (IDH2) inhibitor and is indicated for the treatment of adult and pediatric patients 12 years and older with Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation following surgery including biopsy, sub-total resection, or gross total resection. VORANIGO offers glioma patients the ability to actively manage their disease with the convenience of a once-daily pill.

VORANIGO is the first and only approved therapy designed to target mutant IDH enzymes in Grade 2 glioma and is licensed for use in the U.S., Canada, Australia, Israel, the United Arab Emirates, Saudi Arabia, and Switzerland.

For more information about VORANIGO in the U.S., please visit www.voranigo.com.

VORANIGO IMPORTANT SAFETY INFORMATION FOR U.S. PATIENTS

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. https://doi.org/10.1056/nejmoa2304194

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. 

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