Vorasidenib

VORANIGO demonstrated significant improvement in progression-free survival with a favorable safety profile in a pivotal Phase 3 study of patients with Grade 2 IDH-mutant glioma BOSTON, Sept. 22, 2025 /PRNewswire/ -- Servier, an independent international pharmaceutical group governed by a foundation, today announced that the European Commission (EC) has approved VORANIGO® (vorasidenib) 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 decision to approve VORANIGO as the first targeted therapy to treat Grade 2 IDH-mutant glioma follows a positive opinion from the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) in July 2025. Gliomas are types of brain cancer that can hinder normal brain function and cause a variety of symptoms. Diffuse gliomas with IDH mutations represent the most common malignant primary brain tumors diagnosed in adults younger than 50 years of age. Historically, treatment options have been limited, and tumors continue to grow and infiltrate normal brain tissue without treatment.1,2,3 "Today's EU approval of VORANIGO is a landmark moment for people in the EU living with IDH-mutated glioma who have been waiting more than two decades for new treatment options. VORANIGO is the first EMA-approved therapy specifically designed to target mutant IDH enzymes in Grade 2 glioma and represents a long-awaited shift in the treatment paradigm. As a leader in precision medicine, we're grateful to the researchers, patients and advocates who have helped expand our understanding of IDH inhibition and bring this breakthrough to the EU," said Islam Hassan, Global Head of Development-Neuro-Oncology & Senior Director, LS/LCM at Servier. Decisions by the EC are applicable in the 27 member states of the EU, as well as Norway, Liechtenstein, and Iceland. The approval of VORANIGO is supported by results from the pivotal Phase 3 INDIGO clinical trial published in The New England Journal of Medicine and presented during the Plenary Session at the 2023 Annual Meeting of the American Society of Clinical Oncology (ASCO), which showed that VORANIGO significantly extended progression-free survival (PFS) and time to next intervention (TTNI) when compared to placebo. The INDIGO study showed that VORANIGO was well tolerated, and its safety profile was consistent with results from the Phase 1 studies. The most common (≥15%) adverse reactions were fatigue, COVID-19, musculoskeletal pain, diarrhea and seizure.4 VORANIGO has also been granted marketing authorization in the United States, Canada, Australia, Israel, the United Arab Emirates, Saudi Arabia, Switzerland, Brazil, the United Kingdom, and Japan. Servier has submitted marketing authorization applications in several other regions as well and reviews by the respective health authorities are ongoing. Press Contact Sara Noonan: [email protected] About Servier Servier is an independent international pharmaceutical company governed by a nonprofit foundation, committed to making a meaningful social impact for patients and contributing to a sustainable world. Its unique governance model ensures its independence, while supporting long-term innovation, with 100% of its profits reinvested in the Group's development. As a world leader in hypertension and venous diseases and major player in cardiometabolism, Servier drives transformative innovation to support patients with chronic conditions and improve their day-to-day lives through a holistic approach, which includes making patient adherence and control a priority across the globe. Its ambition is to become a leading player in rare cancers, which is why the Group invests heavily in oncology, allocating close to 70% of its R&D budget to this field. By leveraging precision medicine, Servier develops therapies that are more targeted and more effective. Bolstered by its success in oncology, Servier has expanded into neurology, a key driver of future growth. The Group is focused on a select number of neurodegenerative diseases, where accurate patient profiling enables targeted therapeutic responses through precision medicine. To open up wider access to high-quality, affordable care, Servier also offers an extensive range of generic medicines, building on well-established brands in France, Eastern Europe, and Brazil. In all its activities, and at every stage of the medicine life cycle, the Group integrates the patient's voice. Headquartered in France, Servier operates in around 140 countries. In 2023-2024, the Group, which employs over 22,000 people worldwide, achieved revenues of €5.9 billion. More information on the Group website: servier.com Follow us on social media: LinkedIn, Facebook, X, Instagram About INDIGO Phase 3 Trial (NCT04164901)4 INDIGO is a global, randomized, double-blind, placebo-controlled Phase 3 study that was designed to obtain registration for vorasidenib grade 2 glioma with a mutation in the gene encoding isocitrate dehydrogenase 1 or 2 (IDH1 or IDH2) where the only treatment provided was surgery. The pivotal Phase 3 INDIGO study met its primary endpoint of progression-free survival (PFS) according to the Blinded Independent Review Committee (BIRC) and its key secondary endpoint of time to next intervention (TTNI) at the second pre-specified interim analysis. The primary endpoint, PFS per BIRC, was statistically significant and clinically meaningful in favor of the vorasidenib group. Median PFS was 27.7 months for the vorasidenib group compared with 11.1 months for the placebo group (hazard ratio [HR]: 0.39; 95% confidence interval [CI]: 0.27, 0.56; one-sided P=0.000000067. Although not detailed in the SmPC, results for the key secondary endpoint of time to next intervention (TTNI) at the second pre-specified interim analysis and an exploratory endpoint, tumor growth rate (TGR), were favorable for vorasidenib. TTNI was statistically significant (HR: 0.26; 95% CI: 0.15 to 0.43; P=0.000000019). The median TTNI was not obtained with vorasidenib and was 17.8 months with placebo. Vorasidenib also reduced tumor volume by an average of 2.5% (Tumor Growth Rate -2.5%; 95% CI -4.7% to -0.2%) every 6 months, while tumor volume increased by an average of 13.9% (TGR 13.9%; 95% CI 11.1% to 16.8%) every 6 months in patients randomized to placebo, as measured by an BIRC. The safety profile in the INDIGO Study was consistent with the results of the Phase 1 trials. Overall, the most common adverse reactions, including laboratory abnormalities, were ALT increased, AST increased, GGT increased, fatigue and diarrhea. About Glioma5 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 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 Mandonnet E, Delattre JY, Tanguy ML, et al. Continuous growth of mean tumor diameter in a subset of grade II gliomas. Ann Neurol 2003;53:524-528. Rees J, Watt H, Jäger HR, et al. Volumes and growth rates of untreated adult low-grade gliomas indicate risk of early malignant transformation. Eur J Radiol 2009;72:54-64. Miller JJ, Gonzalez Castro LN, McBrayer S, et al. Isocitrate dehydrogenase (IDH) mutant gliomas: a Society for Neuro-Oncology (SNO) consensus review on diagnosis, management, and future directions. Neuro Oncol 2023;25:4-25. 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. 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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日前，美国癌症研究协会（AACR）发布了2025年抗癌进展报告（AACR Cancer Progress Report 2025）。这一长达282页的综合性报告回顾了过去一年在抗癌路上获得的众多进展。其中值得一提的是，近年来癌症死亡率显著下降。除早期筛查和生活方式改变外，创新癌症疗法的持续开发也是癌症患者预后改善的主要原因。本文将与读者分享这一报告的精彩内容。点击文末“阅读原文”即可访问AACR网站查看报告原文。 癌症死亡率下降 分析显示，1991—2023年，总体癌症死亡率下降34%，相当于避免超过450万例癌症死亡。这一下降主要得益于公共卫生措施，尤其是预防、筛查与疫苗接种。统计显示，1975—2020年，控烟、筛查项目及疫苗接种共计避免约594万例癌症死亡；同时，部分癌种治疗的持续进展也为降低癌症死亡率作出了重要贡献。 图片来源：参考资料[1] 报告同时指出，约40%的癌症与可改变的危险因素相关，包括烟草使用、超重/肥胖、体力活动不足、过度紫外线暴露、饮酒、环境暴露以及致癌性病原体感染。尽管近年吸烟率显著下降，但儿童与成人肥胖及饮酒上升仍构成重要公共卫生隐患；如不及时干预，过去50年取得的抗癌进展可能因此受损。 ▲≥30岁成年人癌症病例中可归因于所选因素的百分比（图片来源：参考资料[1]） ▲与肥胖、运动不足相关的癌症种类（图片来源：参考资料[1]） 另一方面，随着早期检测研究持续进展，美国FDA相继批准多项基于人工智能的设备和软件，用于更早、更准确地检测癌症。微创筛查也在加速推进，包括用于结直肠癌筛查的血液与粪便检测，以及支持居家自取样的宫颈癌筛查装置。同时，影像学创新与对癌症风险相关遗传学的更深入理解，共同提升了早期检测的精准性与可及性。这些技术的进展共同提升了早期检测的精准性与可及性，并推动了多种癌症死亡率的显著下降。 ▲1975—2020年因筛查与去除癌前病变而避免的癌症死亡占总数的比例（图片来源：参考资料[1]） 此外，尽管整体抗癌取得显著成效，但不同癌种的抗癌进展不均：五年总生存率方面，胰腺癌为13%、多形性胶质母细胞瘤为6%，而乳腺癌达92%、前列腺癌达98%。这仍是学术界与产业界需要共同应对的关键挑战。 ▲不同时期部分癌症的5年总生存率比较（图片来源：参考资料[1]） 抗癌疗法监管里程碑进展 自2024年7月1日至2025年6月30日，FDA共批准20款用于多种癌症类型的全新疗法和1款用于肺癌治疗的新装置，并将8款已批准疗法的适应症拓展至新的癌种。值得一提的是，在1990至2022年间，在全球范围内共有568款创新抗癌疗法上市。 ▲1990—2022年期间不同国家和地区的上市创新抗癌疗法数量（图片来源：参考资料[1]） 免疫治疗近年来取得多项突破。例如，“first-in-class”工程化T细胞受体（TCR）T细胞疗法Tecelra（afami-cel）于去年8月获批，成为首个用于治疗实体瘤的工程化T细胞疗法，也是十余年来滑膜肉瘤领域首个有效治疗选择。靶向胃癌细胞中创新靶点Claudin18.2的治疗性抗体Vyloy（zolbetuximab）亦于去年11月获批，惠及胃癌或胃食管结合部（GEJ）腺癌患者。自2011年首份AACR抗癌进展报告发布以来，FDA批准的免疫检查点抑制剂（ICI）已由仅用于黑色素瘤的1种，增长至15种，现用于治疗20余种癌症类型。 ▲美国FDA在2024年7月1日至2025年6月30日期间所批准的抗癌疗法（图片来源：参考资料[1]） 除免疫疗法外，靶向治疗同样持续推进。今年5月，组合疗法Avmapki Fakzynja Co-pack（avutometinib与defactinib）获批上市，用于治疗复发性低级别浆液性卵巢癌（LGSOC）成年患者，适用于既往接受过系统性治疗且肿瘤携带KRAS突变的人群，该方案此前已获FDA突破性疗法认定。Voranigo（vorasidenib）则于去年8月获批，成为首个在脑肿瘤中抑制IDH1与IDH2突变蛋白的靶向疗法，惠及2级星形细胞瘤或少突胶质细胞瘤患者。同时，双特异性抗体Ziihera（zanidatamab）亦于去年11月获加速批准，为胆道癌患者带来新的治疗可能。 ▲2011年与2025年，FDA批准抗癌免疫疗法、双特异性T细胞接合器以及抗体偶联药物的数量（图片来源：参考资料[1]） 此外，创新治疗模式的推进同样显著。根据报告，FDA在2025年已批准9款双特异性T细胞接合器和13款抗体偶联药物（ADC），其中11款ADC针对独特靶点，进一步丰富了个体化治疗版图。综上，过去一年在FDA批准与适应症拓展的推动下，免疫与靶向等个体化疗法加速落地，为多类难治性癌症提供更精准、更有效的治疗选项。 血液肿瘤领域的进展 在本次报告中，AACR特别提及血液肿瘤领域的抗癌进展。血液肿瘤起源于造血组织（如骨髓），主要包括白血病、淋巴瘤和多发性骨髓瘤。2025年全球预计新发近140万例、死亡约74.5万例。在研究与技术创新的推动下，血液肿瘤死亡率显著下降，更多患者生存期延长、生活质量提升。1991—2023年间，慢性髓系白血病（CML）、非霍奇金淋巴瘤（NHL）以及多发性骨髓瘤的死亡率分别下降71%、43%和31%。 ▲2015—2025年，FDA批准针对白血病的创新靶向疗法（图片来源：参考资料[1]） 血液肿瘤死亡率下降的主要原因之一，在于对其分子机理的深入研究与理解。这也促成了早期靶向疗法之一Gleevec（imatinib，伊马替尼）的问世并获批用于CML治疗。该药物的批准不仅改变了CML的临床照护，也引领了肿瘤精准医学的革命。近期，首个靶向menin的疗法Revuforj（revumenib）获批，为急性髓系白血病（AML）患者提供了新的治疗选择。2015—2025年间，FDA已批准25款用于血液肿瘤的创新靶向疗法和21款创新免疫疗法。 ▲部分再获FDA批准应用于其他癌症种类的抗癌疗法（图片来源：参考资料[1]） 同时，源自白血病与淋巴瘤领域的多种疗法正被评估，并在部分情况下用于实体瘤；相关发现也逐步应用于自身免疫性疾病和罕见遗传性疾病，进一步推动跨疾病领域的转化与应用。 结语 2025年AACR抗癌进展报告显示，危险因素预防与早期筛查的加强，以及免疫疗法与靶向疗法等创新疗法的相继获批，正持续推动癌症死亡率下降；尤其在血液肿瘤领域，精准医学驱动的突破显著，并带动疗法跨疾病应用。尽管挑战仍在，但在跨学科协作与持续科技创新的推动下，患者照护有望迎来更具革命性的改变，获得更长久、更高质量的生存。 参考资料： [1] AACR Cancer Progress Report 2025: Contents. Retrieved September 18, 2025 from https://cancerprogressreport.aacr.org/progress/cpr25-contents/ 免责声明：本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，传递医学新知