Vorasidenib as Maintenance Treatment After First-line Chemoradiotherapy in IDH-mutant Grade 2 or 3 Astrocytoma: a Placebo-controlled, Triple-blind, Randomized Phase III Study (VIGOR)

The main goal of VIGOR is to demonstrate that vorasidenib maintenance therapy improves locally assessed progression-free survival (PFS) from enrolment compared to placebo in patients with IDH-mutant, CNS5 WHO Grade 2 or 3 astrocytoma following the completion of first-line chemoradiotherapy.
The primary endpoint is Progression-free survival (PFS), as assessed locally from the date of enrolment using the RANO 2.0 criteria.
In this a comparative, randomized (1:1), triple blinded, multicentre phase III superiority trial with one stopping rule for efficacy and futility after end of enrolment, participants in the experimental arm will receive vorasidenib orally once daily at a dose of 40 mg in continuous 28-day cycles while participants in the control arm will receive a matched oral placebo once daily in continuous 28-day cycles

开始日期2025-12-05

申办/合作机构

EORTC

[+1]

NCT05609994

/ Recruiting临床1期IIT

ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas

The purpose of this study is to determine the safety and efficacy of a PEPIDH1M vaccine in combination with vorasidenib, a dual inhibitor of mutant IDH1 and IDH2 enzymes, in adult patients diagnosed with recurrent IDH1 mutant lower grade gliomas.

开始日期2025-07-15

申办/合作机构

Duke University

[+1]

NCT06969352

/ Not yet recruitingN/A

A Multicentre, Retrospective-prospective Real-world Study: to Evaluate the Effectiveness and Safety of Vorasidenib in Patients With Isocitrate Dehydrogenase IDH1/2 Mutant Grade 2 Astrocytoma or Oligodendroglioma (VICTORIA Study)

The goal of this Study is to evaluate the effectiveness and safety of Vorasidenib in Patients with Isocitrate dehydrogenase IDH1/2 mutant Grade 2 astrocytoma or oligodendroglioma, primary purposeis to evaluate the efficectiveness of Vorasidenib in glioma patients treated in routine clinical practice in In China, patients aged 12 and above with grade II or higher astrocytoma or oligodendroglioma with IDH1 or IDH2 mutations. The main question it aims to answer is:
if this trend is consistent with the efficacy observed in the INDIGO study, and there is not any new safety signal compared to previous research data? Researchers will compare to no treatment. Participants is not mandatory for a formal visit as it is a real-word study.However, due to the fact that patients will be treated with new drugs and need to collect data on major efficacy, regular visits should be performed in routine clinical practice.
This study is a multicenter, retrospective-and prospective real-world study, There are treatment group (Vorasidenib) and external control group (untreated after surgery).

开始日期2025-06-01

申办/合作机构

施维雅（天津）制药有限公司

[+3]

100 项与 Vorasidenib 相关的临床结果

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100 项与 Vorasidenib 相关的转化医学

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100 项与 Vorasidenib 相关的专利（医药）

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项与 Vorasidenib 相关的文献（医药）

2025-11-01·Current Drug Safety

Vorasidenib: A Milestone in Targeted Therapy for IDH-Mutant Lower Grade Gliomas - Efficacy, Emerging Safety Concerns, and the Call for Comprehensive Safety Evaluation

Article

作者: Pattanayak, Manisa ; Sah, Sanjit ; Yoosuf, Beema T.

2025-07-22·MINI-REVIEWS IN MEDICINAL CHEMISTRY

Structure-Property Relationships Reported for the New Drugs Approved in 2024

Article

作者: Choi, Kihang

Abstract::

This mini-review summarizes the structure-property relationships of seven smallmolecule drugs approved in 2024, providing insights into effective lead-to-candidate optimization strategies. The analysis focused on aprocitentan, flurpiridaz F-18, inavolisib, vorasidenib, ensitrelvir, golidocitinib, and zorifertinib, highlighting the key structural modifications that enhanced their drug-like properties. Notable optimization strategies included the strategic use of five- and sixmembered nitrogen-containing heterocycles as cyclic bioisosteres and solubilizing groups. For the kinase inhibitor golidocitinib, the unique position of a solubilizing group within the binding pocket achieved dual benefits, i.e., enhanced target selectivity and physicochemical properties. When developing central nervous system-penetrant drugs such as zorifertinib, careful control of rotatable bonds, hydrogen bond donors, and molecular lipophilicity was critical for optimizing blood-brain barrier penetration while remaining suitable for oral administration. These findings on structureproperty relationships offer valuable guidance for future drug development, particularly in addressing challenges related to solubility, bioavailability, and tissue-specific drug distribution.

2025-07-19·ACTA NEUROCHIRURGICA

A scoping review of proton radiation therapy and mutant-isocitrate dehydrogenase-inhibitors in IDH mutated lower-grade gliomas: pushing beyond surrogate end-points.

Article

作者: Brandal, Petter ; Corell, Alba ; Blomstrand, Malin ; Mansouri, Alireza ; Jakola, Asgeir Store ; Harba, Dima

BACKGROUND:

Proton radiation therapy (PRT) and mutant isocitrate dehydrogenase inhibitors (mIDH-inhibitors) are emerging therapies for mIDH lower grade gliomas (LGGs). Despite their substantial theoretical benefits, comparisons with current standards - particularly pertaining to patient-centred outcomes - are limited.

METHODS:

Through PubMed and Scopus, a search strategy based on keywords focusing on PRT and mIDH-inhibitors was applied on December 3, 2024. Studies in English on at least 20 adult patients (≥ 18 years) with mIDH-LGG grade 2 or 3 and published between January 1, 2011 and August 31, 2024 were included. Review articles were excluded.

RESULTS:

Of 6383 identified articles, seven per treatment strategy were included. Overall survival was not reported for mIDH-inhibitors. The lack of high-quality studies comparing PRT to photon radiation therapy precludes conclusions regarding efficacy, effectiveness or even post-PRT radiological manifestations. For the mIDH-inhibitor Vorasidenib (AG-881), the radiological objective response rate was 10.0-42.9%, although lower for contrast-enhancing tumors. Vorasidenib significantly delayed tumor progression (27.7 versus 11.1 months, p < 0.001) and time to next intervention (not reached versus 17.8 months, p < 0.001) when compared to placebo. Adverse events were mostly mild, including elevated liver enzymes (15.6-44.2%) and headache (26.9-46.2%). Only 1/14 studies included assessments related to quality of life (QoL)-domains with inconclusive research on cognitive outcomes.

CONCLUSION:

Studies reporting on patient-centred data including survival, cognition and QoL remain scarce. Larger, comparative prospective studies, preferably randomized controlled trials, with such outcomes are needed to inform clinicians whether the theoretical and radiological benefits can be translated to improved outcomes that matter to patients, i.e. living better and/or longer.

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项与 Vorasidenib 相关的新闻（医药）

2025-07-25

·FiercePharma

Europe's CHMP gives thumbs-up to Lilly's Kisunla and Gilead's long-acting PrEP drug Yeytuo

Europe’s Committee for Medicinal Products for Human Use has reversed its decision on Eli Lilly's Alzheimer's disease treatment Kisunla, giving it a positive opinion in a limited patient population. Four months after giving Eli Lilly’s Alzheimer’s disease treatment a thumbs-down, Europe’s Committee for Medicinal Products for Human Use (CHMP) has recommended it for approval, albeit in a restricted patient population.The positive opinion covers Kisunla's use in patients with early Alzheimer’s symptoms who have one or no copies of the apolipoprotein E ε4 (ApoE4) gene. The drug is not recommended for individuals carrying two copies of the ApoE4 gene. Those patients are more susceptible to amyloid-related imaging abnormalities (ARIA), which are a primary side effect of currently approved Alzheimer’s drugs.The restriction is the same as the one applied by European regulators when it granted marketing authorization to Eisai and Biogen’s Alzheimer’s drug Leqembi three months ago. Those companies went through the same rejection/appeal process as Lilly did with European regulators.Lilly went through the same go-round with regulators in the U.S. before it secured approval of Kisunla in July of last year. Then, earlier this month, the FDA signed off on a new dosing regimen for the treatment, featuring a more gradual titration, which leads to a significantly lower rate of ARIA-related brain swelling. The new dosing regimen also applies to Thursday’s thumbs-up from the CHMP. Endorsement for Gilead’s PrEP injection The CHMP has also provided a positive opinion on Gilead Sciences' HIV pre-exposure prophylaxis (PrEP) Yeytuo (lenacapavir), a twice-a-year subcutaneous injection which has been hailed as one of the most important breakthroughs in the effort to end the epidemic.The FDA blessed the shot last month under its commercial name Yeztugo.The CHMP recommendation applies to adults and adolescents and came under an accelerated pathway, “because it is considered to be of major public health interest in the EU and the rest of the world,” the regulator said.It was also evaluated under the EU-Medicines for all program, which included participation from the World Health Organization and regulators from several African and Asian nations. The program helps speed approvals where “regulatory capacity may be limited,” the CHMP said.“Lenacapavir for PrEP has the potential to become a critical tool for public health, helping to expand prevention options for people who face the highest barriers to care,” Dietmar Berger, M.D., Ph.D., Gilead’s chief medical officer, said in a release. Other CHMP decisions A month after Supernus Pharmaceuticals made a $561 million bet on Zurzuvae, buying out its developer Sage Therapeutics, the CHMP has registered a positive opinion on the treatment for postpartum depression. The Biogen-partnered treatment was endorsed by the FDA in August 2023, along with a rejection from the regulator in a much larger patient population, major depressive disorder.The European regulator has also signed off on rare disease specialist IntraBio’s Aqneursa for the treatment of the lysosomal storage disorder Niemann-Pick disease type C (NPC). The endorsement comes 10 months after the FDA approved two treatments for NPC in consecutive weeks, Aqneursa and Zevra Therapeutics’ Miplyffa, which has yet to win a nod in Europe.Less than three weeks after the FDA approved KalVista Pharmaceuticals' Ekterly for acute attacks of hereditary angioedema (HAE), the CHMP has followed suit. This is the first oral treatment for HAE recommended for approval in Europe, reducing the burden of injection on patients and speeding up the time between onset of attack and administration of the medicine, the CHMP said. Ono Pharmaceuticals has won a CHMP nod for Romvimza, for the treatment of adults with symptomatic tenosynovial giant cell tumor (TGCT), a condition where the tissue surrounding the joints and tendons expands abnormally, forming outgrowths of the joint. The drug, which was acquired last year in Ono’s $2.4 billion buyout of Deciphera Pharmaceuticals, was endorsed by the FDA in February of this year, joining Daiichi Sankyo’s Turalio as the only other marketed treatment for TGCT.Ionis Pharmaceuticals' Tryngolza, for the treatment of adults with familial chylomicronemia syndrome, has gotten a thumbs-up from CHMP. The rare, autosomal recessive inherited disorder is characterized by elevated levels of triglycerides in the blood. The FDA approved the treatment in December of last year.Also receiving a CHMP nod is Servier’s Voranigo for low-grade astrocytoma or oligodendroglioma, two rare malignant brain tumors. The FDA blessed the treatment in August of last year.

上市批准加速审批

2025-07-25

·PRNewswire

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: [email protected] 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 . 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. 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 WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

临床3期孤儿药临床结果快速通道突破性疗法

2025-06-23

·学术经纬

30岁确诊脑癌的她已活过11年：她试过的新疗法已获批上市，能让肿瘤缩小或长期稳定

编者按：恶性脑肿瘤俗称“脑癌”，其中高达80%都是脑胶质瘤。脑胶质瘤具有高致残率、高复发率的特点。脑胶质瘤即使手术也很难彻底切除，术后容易复发甚至进一步恶化。近年研究发现，异柠檬酸脱氢酶（IDH）基因是其诊断与治疗的关键，由此诞生了针对IDH突变的新疗法，比如2024年获美国FDA批准上市的vorasidenib，这是胶质瘤领域20多年来具有里程碑性质的突破性疗法。在今天的故事中，这位患者就曾尝试这款新疗法，最终肿瘤不再继续生长。这背后，是科学家们锲而不舍的探索精神与心血。长久以来，药明康德也一直在支持包括IDH抑制剂在内的脑胶质瘤靶向疗法的开发，用“一体化、端到端”的CRDMO服务赋能助力合作伙伴的新药加速问世，造福广大患者。孕晚期紧急入院抢救，查出脑胶质瘤2008年春天，30岁出头、家住美国新泽西州的玛莉亚·罗森塔尔（Maria Rozentul）已步入孕晚期，正满怀期待地准备迎接宝宝的到来。然而，就在距离预产期还有2个月的时候，意外来临了——玛莉亚突然多次出现了疑似癫痫发作的症状，丈夫列夫（Lev）手忙脚乱地叫来了救护车。医护人员赶到后，用药物暂时控制了她的症状，并将她送往家附近的医院抢救。为确保母子平安，医生首先进行了剖宫产手术，让玛莉亚的女儿阿莉拉（Ariela）提前2个月来到这个世界。作为早产儿，阿莉拉出生后就被送到了新生儿重症监护室观察。图片来源：123RF更让人担忧的是玛莉亚的身体状况——为查找癫痫的“病根”，医生为她进行了颅脑CT扫描等影像学检查，发现玛莉亚竟患有脑肿瘤。出于对病情的审慎考虑，玛莉亚决定前往纪念斯隆-凯特琳癌症中心（Memorial Sloan Kettering Cancer Center），以获取全面的评估与治疗方案。经产科医生推荐，夫妇俩见到了神经外科主任菲利普·古廷（Philip Gutin）。面对权威专家，这位母亲鼓起勇气问道：“可以推迟6个月手术吗？我家宝宝是早产儿，小得可怜，我想让她多喝些母乳。”令她感动的是，古廷医生认真评估后认为半年的等待不会影响治疗窗口期，于是同意了这一请求。进一步检查后，玛莉亚被确诊为弥漫性星形胶质细胞瘤。它属于低级别脑胶质瘤，这类肿瘤大部分生长缓慢，但弥漫浸润性（即缓慢扩散到全脑）的生长方式会导致其与脑组织分界不清，手术很难做到真正意义上的彻底切除，术后残留的肿瘤组织不可避免地会复发、并向恶性程度更高的级别进展。尽管如此，手术仍然是这类肿瘤重要的治疗方法，可以解除或缓解临床症状、延长患者的生存期，并获得足够的肿瘤标本用来明确病理学诊断和进行分子遗传学检测（即基因检测）。此外，放化疗、系统性治疗和支持治疗等也很重要。2008年12月，古廷医生与团队为玛莉亚实施了手术，术中切除了尽可能多的肿瘤。术后，玛莉亚积极康复、坚持行走锻炼，因为护士说“多活动有助于身体恢复”。不久，玛莉亚肿瘤组织的基因检测结果也出来了，原来她的癌细胞存在IDH1基因突变。当时的她还不明白这个结果意味着什么，但命运之窗已经悄悄为她敞开。多次治疗仍难逃复发、恶化，试验新药让她的人生“柳暗花明”术后一年左右，玛莉亚的肿瘤不出意料地复发了，她开始接受化疗。好在她对化疗比较敏感、耐受性还不错，肿瘤暂时停止了生长。作为新手妈妈，玛莉亚忙碌不已，一边定期到医院治疗，一边还要忙着照顾宝宝。同一时期，由于古廷医生工作上的调整，玛莉亚的主治医生换成了脑肿瘤科主任英戈·梅林霍夫（Ingo Mellinghoff）。“他完全理解患者换主治医生后的不安。他很专业、很有耐心，让我发自内心地对他完全信赖。”而梅林霍夫医生则表示，玛莉亚惊人的毅力也让自己深受感染。图片来源：123RF整个化疗过程持续了多年。然而，在完成化疗18个月后，玛莉亚的肿瘤再次进展。不幸中的万幸是，一个难得的机会来临了——梅林霍夫医生推荐她参加一项针对IDH1突变的新药临床试验。听到这串字母，玛莉亚莫名觉得有些熟悉，这才意识到自己曾查出携带有这种突变。她进一步了解到，IDH1和1DH2是代谢酶，正常情况下能帮助分解营养物质、为细胞提供能量；当IDH1和IDH2基因突变后，表达异常的IDH会导致相关代谢产物异常过多、阻止细胞分化或特化为其原本应该成为的细胞类型，进而可能引发细胞生长失控，变为“疯长”的癌细胞。而这项试验所用的新型药物vorasidenib是一款具有脑渗透性与选择性的口服双重抑制剂，能抑制突变的IDH1/2蛋白活性，从而针对这一过程进行精准阻断、抑制肿瘤生长，帮助控制疾病进展。充分理解其中的原理之后，玛莉亚毫不犹豫地加入了临床试验。期间，她每天仅需服用2片试验药物，然后每月去医院做一次血液检查、每2个月做一次影像学检查。渐渐地，她的肿瘤生长速度开始减慢，然后就完全停止了生长！到2019年，也就是玛莉亚坚持使用新药3年后，她的肿瘤依然保持着“沉睡”状态，没再进一步生长、恶化。从确诊那一刻开始，玛莉亚已经不知不觉中走过了11个年头，而她的人生依然精彩。在业余时间里，她是一名兼职的摄影师，同时还支持着11岁的女儿积极发展演艺方面的兴趣爱好。图片来源：123RF回顾抗癌历程，她感慨万千。“虽然经历了多轮治疗，但我一直保持着积极的心态，爱人和女儿的陪伴也让我动力满满。要相信，患癌只是人生中的一段小插曲，可能会让你难过、消沉一阵子，但别让它的阴影占据你的全部生活。找到自己的精神支柱，保持对未来的期待，无论发生什么，都要勇往直前。”多款脑胶质瘤靶向疗法获批上市，为无数患者带来曙光5年后，为这款新药付出过努力的人们等来了回音——2024年8月6日，vorasidenib（商品名：Voranigo）获美国FDA批准上市，适用于携带IDH1或IDH2突变的2级星形细胞瘤或少突胶质细胞瘤的12岁及以上成人和儿童患者的手术（包括活检、次全切除术或全切除术）后治疗。IDH1突变型弥漫性胶质瘤在50岁以下成人恶性原发性脑肿瘤中最为常见，此前这类患者在手术后的治疗选择长期以来非常有限，即使有效的药物也仅能延长1-3个月的生存期。而这款新药的获批，意味着大多数年轻的脑癌患者从此有了进一步延长生存的希望。另外，这款新药是小分子药物、每天只需口服一次，为患者带来了更便利的治疗选择。在支持其上市的关键3期临床试验中，331名仅接受手术治疗且出现残留或复发的IDH1/2突变2级胶质瘤患者被1：1随机分为两组，一组每日口服一次40 mg vorasidenib，另一组服用安慰剂，直到疾病进展或出现不可接受的毒性；随机分配到安慰剂组的患者在影像学确认疾病进展后，可转换为vorasidenib治疗。结果显示，vorasidenib组患者的无进展生存期延长到了27.7个月，是安慰剂组（11.1个月）的约2.5倍，患者的疾病进展或死亡风险降低了61%，且耐受性良好。此外，随访至30个月时，vorasidenib组超80%的患者仍不需要后续干预，而安慰剂组在17.8个月时已有半数患者需要后续干预；vorasidenib组每6个月的肿瘤体积平均减少2.5%，而安慰剂组的这一数值平均增加了13.9%。也就是说，vorasidenib有望让患者获得长达2年以上的病情稳定、肿瘤逐渐缩小、不需要二次干预的高质量生活，生存的希望大大增加。还有一个好消息是，这款新药针对同一适应症在中国患者中的3期临床试验也已启动，未来有望惠及中国患者。图片来源：123RF产业界对vorasidenib的探索仍在继续，期望进一步扩大适应症、挖掘它在联合治疗方面的潜力，造福更多患者。目前，vorasidenib联合替莫唑胺用于IDH1/2突变的2-4级脑胶质瘤的1b/2期临床试验、联合PD-1抑制剂帕博利珠单抗用于治疗后复发或进展的IDH1突变脑胶质瘤的1期试验、联合肿瘤新生抗原多肽疫苗用于复发性IDH1突变型低级别脑胶质瘤的1期试验均已启动。长期以来，脑胶质瘤的生存预后并不理想、治疗需求一直未能得到充分满足，直到近年才在靶向治疗方面取得突破性进展。尤其是vorasidenib，作为FDA批准的首款用于IDH突变脑胶质瘤患者群体的靶向疗法，也是胶质瘤领域近25年来的重大突破，具有里程碑意义。同样在2024年获FDA批准的还有tovorafenib，用于治疗携带BRAF重排（包括融合）变异的儿童低级别脑胶质瘤患者。这两款小分子药物的出现，为脑胶质瘤患者提供了新的靶向治疗选择，重塑了治疗格局。过去20多年中，美国FDA批准了至少6款脑胶质瘤相关疗法，其中大部分都是靶向药物。药明康德很高兴能为其中多款疗法赋能、助力这些创新疗法的问世，造福病患。长期以来，药明康德都在为此类疾病创新疗法的开发提供各种服务，通过独特的一体化、端到端CRDMO模式，支持全球合作伙伴从药物发现（R）、开发（D）到商业化生产（M）各个阶段的需求，助力更多药物加速从实验室来到患者身边。以测试业务为例，在一项近期发表的研究中提到，药明康德为某款针对脑胶质瘤的泛RAF激酶抑制剂的临床前体外实验提供了赋能支持，包括对全细胞裂解物进行了超敏电化学发光（MSD）免疫分析与酶联免疫吸附测定（ELISA）实验等。此外，药明康德测试业务平台的药性评价部能为包括IDH抑制剂在内的各类药物的体内、体外药物代谢动力学（DMPK）研究提供支持。在新药研发的漫漫征途上，每一块里程碑都来之不易，上面凝结着无数人的智慧与汗水。未来，我们将陪伴创新者们继续前行，见证更多突破性疗法的诞生，为更多像玛莉亚一样的患者带去绝处逢生的希望。Partnering for Progress: Driving the Future of Brain Tumor TherapiesGliomas are among the most serious and challenging types of brain tumors. Due to their location and invasive nature, gliomas are often difficult to remove completely through surgery, leading to high recurrence rates and limited treatment options.However, advances in our understanding of disease biology—combined with the development of novel targeted therapies—are beginning to transform the treatment landscape. At WuXi AppTec, we are proud to contribute to this progress. Through our unique, integrated CRDMO platform, we have supported the development of several glioma therapies, including some that are now approved and benefiting patients globally. In addition, multiple innovative therapies are also currently in active development with our support.As we observe World Brain Tumor Day on June 8, we are reminded of the urgency and importance of continued progress in the fight against these devastating diseases. We will continue to enable our partners across every stage of the therapeutic development journey. By providing seamless, end-to-end solutions, we help shorten the timeline from bench to bedside—because every day counts when patients are waiting.Driven by our vision that “every drug can be made and every disease can be treated,” we will continue to work alongside our partners to advance brain tumor therapies—and bring renewed hope to patients and families around the world.参考资料（可上下滑动查看）[1] 国家卫生健康委员会医政医管局,中国抗癌协会脑胶质瘤专业委员会,中国医师协会脑胶质瘤专业委员会. (2022). 脑胶质瘤诊疗指南(2022版). 中华神经外科杂志,38(08),757-777.DOI:10.3760/cma.j.cn112050-20220510-00239[2] 中国抗癌协会脑胶质瘤专业委员会, 江涛, 张伟, & 王政. (2022). 中国抗癌协会脑胶质瘤整合诊治指南(精简版). 中国肿瘤临床, 49(19), 1.[3] 黄国浩, 刘国龙, 任鹏, 等.(2024). 成人较低级别胶质瘤患者的生存预后及其影响因素分析. 中华神经外科杂志, 40(8) ,821-826. DOI: 10.3760/cma.j.cn112050-20231020-00132.[4] Flourishing 11 Years after a Brain Tumor Diagnosis: Maria’s Story | Memorial Sloan Kettering Cancer Center . Retrieved June 8, 2025 from https://www.mskcc.org/experience/hear-from-patients/flourishing-11-years-after-brain-tumor-diagnosis-maria-s[5] Glioma Clinical Trials | Memorial Sloan Kettering Cancer Center (mskcc.org) .Retrieved June 8, 2025 from https://www.mskcc.org/cancer-care/types/glioma/glioma-clinical-trials[6] 施维雅VORANIGO®（vorasidenib）获得FDA批准，成为首个针对IDH突变2级胶质瘤的靶向疗法.Retrieved June 8, 2025 from https://mp.weixin.qq.com/s/EiyxHsDMetEqfOLrdog73w[7] FDA approves vorasidenib for Grade 2 astrocytoma or oligodendroglioma with a susceptible IDH1 or IDH2 mutation. .Retrieved June 8, 2025 from https://www.fda.gov/drugs/resources-information-approved-drugs/fda-approves-vorasidenib-grade-2-astrocytoma-or-oligodendroglioma-susceptible-idh1-or-idh2-mutation[8] Mellinghoff, I. K., Van Den Bent, M. J., Blumenthal, D. T., Touat, M., Peters, K. B., Clarke, J., ... & Cloughesy, T. F. (2023). Vorasidenib in IDH1-or IDH2-mutant low-grade glioma. New England Journal of Medicine, 389(7), 589-601.[9] New Analyses from Pivotal Phase 3 INDIGO Study Reinforce Vorasidenib’s Potential to Change the Treatment Paradigm for IDH-Mutant Diffuse Glioma. Retrieved June 8, 2025 from New Analyses from Pivotal Phase 3 INDIGO Study Reinforce Vorasidenib’s Potential to Change the Treatment Paradigm for IDH-Mutant Diffuse Glioma | Blog (servier.us)[10] 我们的专长-肿瘤-Servier. Retrieved June 8, 2025 from https://www.servier.com.cn/tumour/[11] PDX模型在抗肿瘤药物研发中的拓展应用.Retrieved June 8, 2025 from https://mp.weixin.qq.com/s?__biz=MzU0MzI5ODk4NQ==&mid=2247487667&idx=1&sn=ecce4090305a1afcc8538aac84f0e030&chksm=fb0cd475cc7b5d63d18a40a18f86449527bcc8aecd20f15464ca200fc4e42ae328d32134027d#rd[12] 2024年FDA获批新药亮点：人体药代动力学及药物相互作用特征一览.Retrieved June 8, 2025 from https://mp.weixin.qq.com/s?__biz=MzkzMDE3OTkxNw==&mid=2247561751&idx=1&sn=5d9695453fb88186920610181a07f6cb&chksm=c33004fa61b1da71fb79e597f8eb01e1094394b134ef369f1d92cf4a8d61db9e9a0b3979d14f#rd[13] Phase 3 Study of Vorasidenib (S095032/AG-881) in Asian Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 orIDH2 Mutation. Retrieved June 8, 2025 from https://clinicaltrials.gov/study/NCT06780930[14] Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma. Retrieved June 8, 2025 from https://clinicaltrials.gov/study/NCT06478212[15] Study of Vorasidenib and Pembrolizumab Combination in Recurrent or Progressive IDH-1 Mutant Glioma. Retrieved June 8, 2025 from https://clinicaltrials.gov/study/NCT05484622[16] ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas. Retrieved June 8, 2025 from https://clinicaltrials.gov/study/NCT05609994[17] Study Details | Study of Orally Administered AG-881 in Patients With Advanced Solid Tumors, Including Gliomas, With an IDH1 and/or IDH2 Mutation | ClinicalTrials.gov Retrieved June 8, 2025 from https://clinicaltrials.gov/study/NCT02481154[18] Shubhra Rastogi, Samantha Perino, Madhu Lal-Nag, et al. (2025). Preclinical Activity of the Type II RAF Inhibitor Tovorafenib in Tumor Models Harboring Either a BRAF Fusion or an NF1 Loss-of-Function Mutation. Cancer Research Communications, 5 (4): 668–679. https://doi.org/10.1158/2767-9764.CRC-24-0451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，聚焦全球生物医药健康创新

突破性疗法上市批准

100 项与 Vorasidenib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
D11834	-	-	-

研发状态

批准上市

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适应症	国家/地区	公司	日期
IDH突变星形细胞瘤	美国	Servier Pharmaceuticals LLC	2024-08-06
IDH突变星形细胞瘤	美国	Les Laboratoires Servier SAS	2024-08-06
IDH 突变型少突胶质细胞瘤	美国	Les Laboratoires Servier SAS	2024-08-06
IDH 突变型少突胶质细胞瘤	美国	Servier Pharmaceuticals LLC	2024-08-06
IDH1突变胶质瘤	加拿大	Servier Canada, Inc.	2024-08-01

未上市

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适应症	最高研发状态	国家/地区	公司	日期
星形细胞瘤	临床3期	中国	Institut de Recherches Intern Servier	2024-10-18
星形细胞瘤	临床3期	中国台湾	Institut de Recherches Intern Servier	2024-10-18
复发性胶质瘤	临床3期	美国	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	日本	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	加拿大	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	法国	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	德国	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	以色列	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	意大利	Institut de Recherches Intern Servier	2020-01-05
复发性胶质瘤	临床3期	荷兰	Institut de Recherches Intern Servier	2020-01-05

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研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


INDIGO trial (ASCO2025)	临床3期	胶质瘤	30	Vorasidenib 40 mg/day	顧鑰築壓鑰淵衊艱艱齋(顧憲醖鑰鏇築積鹹製觸) = 37% (n = 11) 蓋糧鹽顧簾窪衊糧鏇壓 (淵夢齋窪壓簾鏇窪願蓋 ) 更多	积极	2025-05-30
NCT04164901 (INDIGO, SNO2024) 人工标引	临床3期	胶质瘤 IDH2 Mutation \| IDH1 Mutation	331	Vorasidenib 40 mg	醖夢選衊窪糧鏇齋獵顧(憲艱製製醖襯鹽觸夢蓋) = 鏇製製膚壓齋淵窪壓選觸憲製遞積遞鹽衊願糧 (簾糧艱衊壓衊範簾鬱顧, 22.1 ~ NE) 更多	积极	2024-11-11
				Placebo	醖夢選衊窪糧鏇齋獵顧(憲艱製製醖襯鹽觸夢蓋) = 願鑰齋遞積獵構築鬱獵觸憲製遞積遞鹽衊願糧 (簾糧艱衊壓衊範簾鬱顧, 11.1 ~ 13.9) 更多
NCT05592743 (SNO2024) 人工标引	N/A	胶质瘤 IDH1 Mutation \| IDH2 Mutation	71	Vorasidenib	醖簾艱顧壓選衊願築簾(範製壓齋鬱鬱淵窪憲糧) = 膚遞襯廠網蓋廠衊艱繭製蓋網醖鑰選範壓鏇糧 (餘膚鏇製蓋築膚鏇構窪 ) 更多	-	2024-11-11
NCT03343197 (SNO2024) 人工标引	临床1期	IDH1突变胶质瘤 IDH1 R132H	46	Vorasidenib 50 mg QD (after surgery)	簾鏇範衊獵膚夢廠憲積(願齋獵範憲糧淵鑰淵窪) = 製醖襯選蓋膚範廠願網夢艱廠繭鬱獵顧齋壓願 (夢鬱積構醖鹽餘築鹽鹹 ) 更多	积极	2024-11-11
				Ivosidenib 500 mg BID (after surgery)	簾鏇範衊獵膚夢廠憲積(願齋獵範憲糧淵鑰淵窪) = 窪繭鹽醖簾餘艱廠夢艱夢艱廠繭鬱獵顧齋壓願 (夢鬱積構醖鹽餘築鹽鹹 ) 更多
NCT04164901 (INDIGO, FDA) 人工标引	临床3期	弥漫性星形细胞瘤 \| 少突神经胶质瘤 IDH1 Mutation \| IDH2 Mutation	331	Vorasidenib	艱構製壓繭窪襯網鑰鑰(憲鏇願繭觸鹽築遞繭網): HR = 0.39 (95% CI, 0.27 ~ 0.56), P-Value = <0.0001 更多	积极	2024-08-06
				Placebo
NCT04164901 (INDIGO, FDA_CDER) 人工标引	临床3期	IDH突变星形细胞瘤 \| IDH 突变型少突胶质细胞瘤 IDH1 Mutation \| IDH2 Mutation	331	VORANIGO	壓窪構網窪廠憲壓鬱鑰(鏇願鏇網膚齋膚鹹壓鹽): HR = 0.39 (95% CI, 0.27 ~ 0.56), P-Value = <0.0001 更多	积极	2024-08-06
				Placebo
NCT04164901 (INDIGO, AAN2024)	临床3期	胶质瘤 mIDH1/2	331	Vorasidenib	鹽鏇壓壓願衊膚網願齋(廠積襯積憲襯簾網夢獵) = 網夢簾範鹹遞醖壓鹽鏇遞構艱製夢繭積襯餘簾 (鬱糧鏇夢鬱衊鬱積醖蓋, 25.05) 更多	积极	2024-04-09
				Placebo	鹽鏇壓壓願衊膚網願齋(廠積襯積憲襯簾網夢獵) = 壓選鹹製選繭窪鏇蓋膚遞構艱製夢繭積襯餘簾 (鬱糧鏇夢鬱衊鬱積醖蓋, 23.60) 更多
NCT04164901 (KS02.6.A INDIGO \| INDIGO \| RTID-05, CTgov)	临床3期	复发性胶质瘤 \| 复发性 WHO II 级胶质瘤 \| WHO II级胶质瘤	331	Vorasidenib (Vorasidenib)	鑰顧鹹襯蓋願襯齋壓襯(憲鏇鏇窪鑰憲衊壓齋襯) = 選壓製鹽膚選糧夢窪壓構醖鬱繭築構簾願鹹觸 (鬱簾築醖憲糧顧遞網選, 願糧醖糧願獵窪夢願壓 ~ 齋壓築襯觸糧範淵願衊) 更多	-	2023-11-24
				Matching Placebo (Matching Placebo)	鑰顧鹹襯蓋願襯齋壓襯(憲鏇鏇窪鑰憲衊壓齋襯) = 襯醖積餘襯淵鏇膚鏇遞構醖鬱繭築構簾願鹹觸 (鬱簾築醖憲糧顧遞網選, 憲鬱鑰鹹積願蓋鹽簾製 ~ 築艱遞廠糧鑰憲壓願鏇) 更多
NCT04164901 (INDIGO, Literature) 人工标引	临床3期	胶质瘤 IDH1 Mutation \| IDH2 Mutation	331	vorasidenib	鬱餘夢餘製鑰窪遞艱壓(築壓膚觸襯鏇蓋壓積鏇) = 齋顧淵憲顧膚遞鬱糧願艱糧淵壓範廠淵廠鏇窪 (壓顧糧簾餘壓襯窪壓簾, -4.7 ~ -0.2)	积极	2023-11-18
				Placebo	鬱餘夢餘製鑰窪遞艱壓(築壓膚觸襯鏇蓋壓積鏇) = 廠蓋鏇構鹹網繭餘憲醖艱糧淵壓範廠淵廠鏇窪 (壓顧糧簾餘壓襯窪壓簾, 11.1 ~ 16.8)
NCT04164901 (KS02.6.A INDIGO, EANO2023)	临床3期	胶质瘤 IDH1m \| IDH2m	331	Vorasidenib 40 mg	築範壓艱構築顧網鏇顧(窪夢築鏇膚觸襯願鬱糧) = 網範遞觸繭襯鹹蓋獵淵鹹願蓋夢蓋選糧餘顧艱 (艱窪獵廠網鏇餘蓋餘壓 ) 更多	积极	2023-09-08
				Placebo	築範壓艱構築顧網鏇顧(窪夢築鏇膚觸襯願鬱糧) = 鏇壓餘憲築網憲遞範憲鹹願蓋夢蓋選糧餘顧艱 (艱窪獵廠網鏇餘蓋餘壓 ) 更多