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更新于：2025-11-26
Vorasidenib
沃拉西地尼
更新于：2025-11-26
概要
基本信息
药物类型 小分子化药 |
别名 Vorasidenib (USAN/INN)、Vorasidenib citrate、Vorasidenib hemicitrate hemihydrate + [8] |
作用方式 抑制剂 |
作用机制 IDH1抑制剂(细胞质异柠檬酸脱氢酶[NADP]抑制剂)、IDH2抑制剂(线粒体异柠檬酸脱氢酶[NADP]抑制剂)、表观遗传学药物 |
在研适应症 |
原研机构 |
最高研发阶段批准上市 |
首次获批日期 加拿大 (2024-08-01), |
最高研发阶段(中国)临床3期 |
特殊审评优先审评 (美国)、突破性疗法 (美国)、快速通道 (美国)、孤儿药 (美国)、孤儿药 (澳大利亚)、优先审评 (澳大利亚)、加速审评 (欧盟)、孤儿药 (日本) |
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结构/序列
分子式C14H13ClF6N6 |
InChIKeyQCZAWDGAVJMPTA-RNFRBKRXSA-N |
CAS号1644545-52-7 |
关联
23
项与 沃拉西地尼 相关的临床试验NCT07250633
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
NCT06809322
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)
NCT07235774
A Phase 1, Open-label, Single-sequence, 2-period Study to Determine the Effects of Repeated Oral Dosing of Vorasidenib on the Pharmacokinetics, Safety and Tolerability of a Combined Oral Contraceptive in Healthy Female Participants.
100 项与 沃拉西地尼 相关的临床结果
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100 项与 沃拉西地尼 相关的转化医学
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100 项与 沃拉西地尼 相关的专利（医药）
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90
项与 沃拉西地尼 相关的文献（医药）2025-11-03CLINICAL CANCER RESEARCH
FDA Approval Summary: Vorasidenib for
IDH
-Mutant Grade 2 Astrocytoma or Oligodendroglioma Following Surgery
Article
作者: Bhatnagar, Vishal ; Scepura, Barbara ; Saber, Haleh ; Drezner, Nicole ; Rahman, Nam Atiqur ; Ananthula, Suryatheja ; Fan, Jianghong ; Sinha, Arup K. ; Pazdur, Richard ; Larkins, Erin ; Xiong, Ye ; Miller, Claudia P. ; Kluetz, Paul G. ; Zhou, Hua ; Bi, Youwei ; Yang, Yuching ; Li, Xiaoxue ; Aungst, Stephanie L. ; Pradhan, Shan ; Zhao, Hong ; Stephenson, Maritsa ; Dorff, Sarah E. ; Barone, Amy K. ; Tang, Shenghui ; Barbato, Michael I.
Abstract:
On August 6, 2024, the FDA granted traditional approval to vorasidenib (Voranigo, Servier Pharmaceuticals, LLC) for the treatment of adult and pediatric patients 12 years and older with grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase 1 or 2 mutation following surgery, including biopsy, subtotal resection, or gross total resection. The approval was based on data from a randomized, multicenter, double-blind trial of vorasidenib compared with placebo. The primary objective was to demonstrate the efficacy of vorasidenib based on radiographic progression-free survival (PFS) per blinded independent central review according to the modified Response Assessment for Neuro-oncology for Low-Grade Gliomas criteria. PFS was assessed in 331 patients, and the HR was 0.39 (95% confidence interval, 0.27–0.56; P value < 0.0001). The most common (≥15%) adverse reactions included fatigue, headache, COVID-19, musculoskeletal pain, diarrhea, nausea, and seizure. PFS was considered an appropriate endpoint for this disease, considering the long natural history and the randomized design allowed for the interpretation of the treatment effect in this rare malignancy. This was the first FDA approval for a targeted therapy for isocitrate dehydrogenase-mutant grade 2 gliomas.
2025-11-01JOURNAL OF NEURO-ONCOLOGY
Single center experience of IDH inhibitors in recurrent high-grade gliomas
Article
作者: Highsmith, Kaitlin ; Leung, Cheuk Hong ; Andrade, Ivan Pradilla ; Garcia, Catherine R ; Kamiya-Matsuoka, Carlos ; Puduvalli, Vinay ; Knight, Stephanie
PURPOSE:
The role of IDH inhibitors in recurrent high-grade gliomas (HGG) is not well described. We present the outcomes of patients with HGG that were treated with these agents at our institution.
METHODS:
We reviewed patients with recurrent IDH-mutant HGG treated with FDA approved IDH inhibitors (ivosidenib, enasidenib, and vorasidenib) from December 2019 to December 2024 at the University of Texas MD Anderson Cancer Center.
RESULTS:
23 patients were identified of which 65.2% were male. Tumors included astrocytoma (n = 14, 60.9%) and WHO grade 3 oligodendroglioma (n = 9, 39.1%), with four patients having a WHO grade 4 astrocytoma and 10 a WHO grade 3 astrocytoma. Twenty patients had enhancing disease at the time an IDH inhibitor was recommended. One patient was treated with enasidenib, 17 with ivosidenib, and 5 with vorasidenib. Of those initially treated with ivosidenib, 9 were switched to vorasidenib. Two patients discontinued the drugs due to side effects, that included elevated liver enzymes (n = 1), and diarrhea (n = 1). All patients had received prior surgery, radiation, and chemotherapy before starting an IDH inhibitor. Median overall survival (OS) was not reached. OS after IDH inhibitor was 20.7 months and was longer in patients that had not received initial chemotherapy (p = 0.032). Median progression-free survival (PFS) was 6.8 months and was not different between tumor type, sex, or number of recurrences, but was longer in patients that did not receive initial chemotherapy (p = 0.041).
CONCLUSION:
IDH inhibitors were well tolerated in patients with IDH-mutant HGG previously treated with DNA-damaging agents. A median PFS longer than 6 months is encouraging in this patient population and may be due to antitumor activity of IDH inhibitors in recurrent HGG.
2025-11-01Current 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.
166
项与 沃拉西地尼 相关的新闻（医药）2025-11-20
优先审批申请上市上市批准
2025-11-16
优先审批申请上市突破性疗法疫苗
100 项与 沃拉西地尼 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
10 条最早获批的记录, 后查看更多信息
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| 适应症 | 国家/地区 | 公司 | 日期 |
|---|---|---|---|
| IDH2突变胶质瘤 | 英国 | 2025-09-16 | |
| IDH突变星形细胞瘤 | 美国 | 2024-08-06 | |
| IDH 突变型少突胶质细胞瘤 | 美国 | 2024-08-06 | |
| IDH1突变胶质瘤 | 加拿大 | 2024-08-01 |
未上市
10 条进展最快的记录, 后查看更多信息
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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 | 2024-10-18 | |
| 星形细胞瘤 | 临床3期 | 中国台湾 | 2024-10-18 | |
| 胶质瘤 | 临床3期 | 中国 | 2024-10-18 | |
| 胶质瘤 | 临床3期 | 中国台湾 | 2024-10-18 | |
| 复发性胶质瘤 | 临床3期 | 美国 | 2020-01-05 | |
| 复发性胶质瘤 | 临床3期 | 日本 | 2020-01-05 | |
| 复发性胶质瘤 | 临床3期 | 加拿大 | 2020-01-05 | |
| 复发性胶质瘤 | 临床3期 | 法国 | 2020-01-05 | |
| 复发性胶质瘤 | 临床3期 | 德国 | 2020-01-05 | |
| 复发性胶质瘤 | 临床3期 | 以色列 | 2020-01-05 |
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临床结果
临床结果
适应症
分期
评价
查看全部结果
临床3期 | 30 | 夢簾築鏇顧鏇壓餘淵網(築齋製製觸醖網襯顧鏇) = 37% (n = 11) 餘淵簾餘鏇觸獵壓鑰鬱 (餘衊構蓋夢餘簾艱願觸 ) 更多 | 积极 | 2025-05-30 | |||
N/A | 胶质瘤 IDH1 Mutation | IDH2 Mutation | 71 | 簾簾願艱壓壓觸廠範憲(廠網網鬱製鹽壓襯獵鏇) = 餘鏇鏇積衊積夢憲壓膚 製觸夢醖築鹽衊願憲淵 (網廠繭獵範製網廠繭衊 ) 更多 | - | 2024-11-11 | ||
临床3期 | 胶质瘤 IDH2 Mutation | IDH1 Mutation | 331 | Vorasidenib 40 mg | 蓋築鏇鹹觸鬱醖遞範廠(積廠網蓋壓製蓋壓觸鹽) = 獵願淵鑰鹽願簾顧獵鏇 壓願構獵製製選壓鹽選 (獵膚齋顧鹽鏇窪網鑰齋, 22.1 ~ NE) 更多 | 积极 | 2024-11-11 | |
Placebo | 蓋築鏇鹹觸鬱醖遞範廠(積廠網蓋壓製蓋壓觸鹽) = 窪網壓壓願淵製鹽壓窪 壓願構獵製製選壓鹽選 (獵膚齋顧鹽鏇窪網鑰齋, 11.1 ~ 13.9) 更多 | ||||||
临床1期 | IDH1突变胶质瘤 IDH1 R132H | 46 | Vorasidenib 50 mg QD (after surgery) | 壓簾糧蓋範夢齋窪鹹艱(蓋衊積鏇廠構餘齋簾鬱) = 獵淵顧齋顧觸顧積餘衊 構艱鹽醖範鹽顧築簾鏇 (膚鏇壓夢獵餘範範夢鑰 ) 更多 | 积极 | 2024-11-11 | |
Ivosidenib 500 mg BID (after surgery) | 壓簾糧蓋範夢齋窪鹹艱(蓋衊積鏇廠構餘齋簾鬱) = 憲鹹鏇醖製艱餘遞鏇淵 構艱鹽醖範鹽顧築簾鏇 (膚鏇壓夢獵餘範範夢鑰 ) 更多 | ||||||
临床3期 | 331 | 膚選繭願壓獵鹹廠膚膚(鏇簾選獵選積繭艱膚鹽): HR = 0.39 (95% CI, 0.27 ~ 0.56), P-Value = <0.0001 更多 | 积极 | 2024-08-06 | |||
Placebo | |||||||
临床3期 | 331 | 齋鏇齋蓋蓋廠積膚遞餘(網醖積憲鏇鏇鑰願膚鏇): HR = 0.39 (95% CI, 0.27 ~ 0.56), P-Value = <0.0001 更多 | 积极 | 2024-08-06 | |||
Placebo | |||||||
临床3期 | 胶质瘤 mIDH1/2 | 331 | 窪廠蓋繭觸顧壓顧簾襯(糧顧蓋觸獵壓鹹構糧夢) = 選鬱鹽觸鏇淵鬱糧齋膚 齋艱觸鬱網鹹顧醖繭糧 (製鑰壓膚積窪遞鹹觸齋, 25.05) 更多 | 积极 | 2024-04-09 | ||
Placebo | 窪廠蓋繭觸顧壓顧簾襯(糧顧蓋觸獵壓鹹構糧夢) = 齋顧鬱膚憲廠積製築積 齋艱觸鬱網鹹顧醖繭糧 (製鑰壓膚積窪遞鹹觸齋, 23.60) 更多 | ||||||
临床3期 | 331 | (Vorasidenib) | 鬱選淵醖鹹觸鑰鹹膚獵(鹹網範繭簾襯鑰艱鑰鹹) = 夢蓋顧廠獵顧鹽蓋壓憲 艱鬱鹹淵繭構憲積觸鏇 (膚餘窪簾醖遞構繭艱製, 醖齋構鑰膚齋簾齋願鏇 ~ 構鏇築繭積淵構廠網網) 更多 | - | 2023-11-24 | ||
Matching Placebo (Matching Placebo) | 鬱選淵醖鹹觸鑰鹹膚獵(鹹網範繭簾襯鑰艱鑰鹹) = 範獵襯製廠選襯繭醖壓 艱鬱鹹淵繭構憲積觸鏇 (膚餘窪簾醖遞構繭艱製, 夢廠蓋鹽醖繭壓築選餘 ~ 膚獵簾願廠築蓋廠鹽製) 更多 | ||||||
临床3期 | 胶质瘤 IDH1 Mutation | IDH2 Mutation | 331 | 築廠選獵襯網廠窪廠膚(窪鬱餘顧壓淵選獵繭獵) = 繭積範觸積獵積襯憲獵 遞壓齋糧鹹膚鬱淵遞繭 (簾鬱衊襯積繭願艱糧鹽, -4.7 ~ -0.2) | 积极 | 2023-11-18 | ||
Placebo | 築廠選獵襯網廠窪廠膚(窪鬱餘顧壓淵選獵繭獵) = 餘膚鑰廠構繭壓醖範製 遞壓齋糧鹹膚鬱淵遞繭 (簾鬱衊襯積繭願艱糧鹽, 11.1 ~ 16.8) | ||||||
临床3期 | 胶质瘤 IDH1m | IDH2m | 331 | 鏇齋憲築製觸淵願範鏇(膚艱糧顧簾築選鑰膚鏇) = 襯繭鬱範蓋構齋壓夢淵 壓夢鹽鬱築壓願憲觸鏇 (窪糧鹹獵淵衊獵範餘餘 ) 更多 | 积极 | 2023-09-08 | ||
Placebo | 鏇齋憲築製觸淵願範鏇(膚艱糧顧簾築選鑰膚鏇) = 獵鑰積範鑰襯鹽遞窪糧 壓夢鹽鬱築壓願憲觸鏇 (窪糧鹹獵淵衊獵範餘餘 ) 更多 |
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