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更新于：2026-02-05
Dordaviprone
多达维普龙
更新于：2026-02-05
概要
基本信息
药物类型 小分子化药 |
别名 Dordaviprone Hydrochloride、NSC-350625、ONC-201 + [8] |
作用方式 抑制剂、激动剂、拮抗剂 |
作用机制 Akt-1抑制剂(丝氨酸/苏氨酸蛋白激酶AKT抑制剂)、CLPP激动剂(caseinolytic mitochondrial matrix peptidase proteolytic subunit agonists)、D2 receptor拮抗剂(多巴胺D2受体拮抗剂) |
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 美国 (2025-08-06), |
最高研发阶段(中国)临床1期 |
特殊审评快速通道 (美国)、加速批准 (美国)、孤儿药 (欧盟)、孤儿药 (澳大利亚)、罕见儿科疾病 (美国)、优先审评 (美国) |
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结构/序列
分子式C24H26N4O |
InChIKeyVLULRUCCHYVXOH-UHFFFAOYSA-N |
CAS号1616632-77-9 |
关联
36
项与 多达维普龙 相关的临床试验NCT06012929
A Pilot Study, Open-Label Study of ONC201 for Refractory Meningioma
NCT05630794
Phase 1 Trial of ONC201 for Chemoprevention in Colorectal Cancer
NCT04629209
A Phase II, Open Label Study of ONC201 in Adults With EGFR-low Glioblastoma
100 项与 多达维普龙 相关的临床结果
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100 项与 多达维普龙 相关的转化医学
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100 项与 多达维普龙 相关的专利（医药）
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212
项与 多达维普龙 相关的文献（医药）2026-02-10ONCOGENE
IRF2BPL transcriptionally regulates IGFBP2 to promote tumor progression and suppresses immune cell infiltration in esophageal squamous cell carcinoma
Article
作者: Gao, Mingwei ; Wang, Longlong ; Wu, Yueguang ; Ding, Ning ; Xiao, Heng ; Cheng, Yikun ; Cui, Heyang ; Weng, Yongjia ; Cui, Yongping ; Bi, Shanshan ; Zhang, Weimin ; Liu, Huijuan ; Song, Qiqin
Numerous ubiquitination-related proteases (URPs) have been identified as facilitators of disease progression through the disruption of ubiquitination homeostasis in substrate proteins. Notably, some URPs have exhibited non-classical biological functions. In this study, we experimentally elucidate the role of the E3 ubiquitin ligase IRF2BPL as transcriptional activator that promotes malignant phenotypes in esophageal squamous cell carcinoma (ESCC) and inhibits the infiltration of various immune cells within the tumor microenvironment. Specifically, we found that IRF2BPL is highly expressed in ESCC cells and promotes IGFBP2 transcription, thereby facilitating ESCC development both in vivo and in vitro. Moreover, the chemical drug ONC201 was shown to effectively impede ESCC progression induced by the hyperactive IRF2BPL-IGFBP2 axis in tumor cells. Collectively, our findings verified that the IRF2BPL-IGFBP2 axis plays a critical role in enhancing ESCC progression by increasing the malignancy of ESCC cells and fostering an immune-deficient tumor microenvironment. Targeting the IRF2BPL-IGFBP2 axis may represent a promising therapeutic strategy for ESCC.
2026-01-26Annual Review of Pathology-Mechanisms of Disease
Integration and Intersection of Cancer Metabolism with Epigenetic Pathways in Gliomas
Review
作者: Natarajan, Siva Kumar ; Venneti, Sriram ; Pun, Matthew ; Haggerty-Skeans, James
The interplay between metabolomics and epigenetics is a key glioma driver. Both tumor-intrinsic and microenvironmental metabolic cues can shape chromatin. Epigenetic methylation and demethylation are metabolically regulated by
S
-adenosyl methionine (SAM) (via methionine metabolism) and the TCA-cycle–related metabolite α-ketoglutarate (α-KG), respectively. Additionally, glycolysis and the TCA cycle modulate histone acetylation and lactylation. Gliomas in both adults and children hijack these pathways. Adult isocitrate dehydrogenase (IDH)-wild-type tumors enhance glycolysis via epidermal growth factor receptor to alter chromatin. IDH-mutant gliomas generate D-2-hydroxyglutarate (D-2HG), which inhibits α-KG demeth-ylases to create epigenetic hypermethylation. Pediatric gliomas, including gliomas with lysine-to-methionine mutations at residue 27 of histone H3 and posterior fossa group A ependymomas, can also rewire metabolism to regulate chromatin. These pathways can be targeted for therapeutic development. Inhibiting IDH mutations with vorasidenib lowers D-2HG and is beneficial to patients. Other drugs like ONC201 and metformin can metabolically suppress oncogenic chromatin states in pediatric gliomas. This dynamic cross talk between metabolism and epigenetics not only underpins tumor biology but also presents opportunities for innovative therapeutic strategies.
2026-01-01EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES
Hybrid GPP–hospital control strategy for semi-solid extrusion 3D printing of ONC201 chewable units in pediatric oncology
Article
作者: Vignes, Marina ; Annereau, Maxime ; Legrand, François-Xavier ; Secretan, Philippe-Henri ; Ramos, Stephanie ; Do, Bernard
Children with central nervous system tumors often face significant barriers to age-appropriate medicines, especially when dysphagia prevents the use of conventional tablets or capsules. ONC201 (dordaviprone), a first-in-class imipridone available in France under a compassionate access program, poses additional challenges of poor solubility and chemical instability. Chewable formulations offer an attractive solution for pediatric compliance, but their development requires robust pharmaceutical and regulatory controls to ensure safety, stability, and reproducibility. We describe a hybrid GPP-hospital control strategy for semi-solid extrusion (SSE) three-dimensional printing of ONC201 chewable units. In this model, an ONC201 hydrogel intermediate is prepared centrally in a Good Preparation Practices (GPP)-compliant unit under Quality by Design (QbD) specifications and subsequently distributed to hospital pharmacies for on-demand personalization. Patient-ready chewable units are produced locally under in-process controls (IPCs) that monitor extrusion, geometry, unit weight, disintegration, and drug content. Formulation screening and batch characterization identified a stable ONC201 hydrogel suitable for decentralized use, with a conservative refrigerated shelf-life of 14 days. Printed chewable units demonstrated consistent quality attributes and rapid drug release, meeting pharmacopeial expectations for immediate release dosage forms. By combining centralized QbD-controlled preparation with decentralized hospital-based personalization, this work establishes a transferable framework for safe, traceable, and patient-adapted delivery of ONC201 in pediatric oncology, complementing ongoing clinical investigations.
100 项与 多达维普龙 相关的药物交易
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外链
| KEGG | Wiki | ATC | Drug Bank |
|---|---|---|---|
| - | - | - |
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 |
|---|---|---|---|
| 组蛋白H3中K27M点突变的弥漫性中线胶质瘤 | 美国 | 2025-08-06 |
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 |
|---|---|---|---|---|
| 弥漫内生性脑桥神经胶质瘤 | 临床3期 | 丹麦 | 2022-09-29 | |
| 弥漫内生性脑桥神经胶质瘤 | 临床3期 | 法国 | 2022-09-29 | |
| 弥漫内生性脑桥神经胶质瘤 | 临床3期 | 西班牙 | 2022-09-29 | |
| 弥漫内生性脑桥神经胶质瘤 | 临床3期 | 瑞典 | 2022-09-29 | |
| 脑膜瘤 | 临床2期 | - | 2026-12-01 | |
| 复发性胶质瘤 | 临床2期 | 美国 | 2021-10-20 | |
| 复发性胶质瘤 | 临床2期 | 澳大利亚 | 2021-10-20 | |
| 复发性胶质瘤 | 临床2期 | 以色列 | 2021-10-20 | |
| 复发性胶质瘤 | 临床2期 | 荷兰 | 2021-10-20 | |
| 复发性胶质瘤 | 临床2期 | 新西兰 | 2021-10-20 |
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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1期 | 组蛋白H3中K27M点突变的弥漫性中线胶质瘤 H3 K27M-mutant | 50 | 網遞膚膚淵窪築獵醖艱(積夢襯衊糧襯鹹襯顧壓) = 壓觸淵膚鬱餘淵齋鹹壓 鹹糧網壓築願範鬱餘夢 (鏇觸鬱積鹹醖廠鬱積蓋, 12 ~ 36) 更多 | 积极 | 2025-08-06 | ||
临床2期 | 73 | (Arm A) | 鹹淵襯觸憲積齋襯齋顧 = 獵鹽憲艱遞選襯遞繭積 製願窪願鑰衊繭鹽齋淵 (憲網築鏇廠廠選繭獵鏇, 製襯繭繭憲鑰鏇鬱繭構 ~ 膚糧積選網觸醖觸憲鬱) 更多 | - | 2025-07-28 | ||
(Arm B) | 鹹淵襯觸憲積齋襯齋顧 = 餘繭觸艱鬱衊膚製遞壓 製願窪願鑰衊繭鹽齋淵 (憲網築鏇廠廠選繭獵鏇, 鑰壓窪簾築憲簾廠襯獵 ~ 蓋鑰襯膚構廠鹽選窪襯) 更多 | ||||||
临床1/2期 | 组蛋白H3中K27M点突变的弥漫性中线胶质瘤 H3 K27M-mutant | 41 | 蓋窪獵膚構鏇鬱衊構範(簾壓廠鹹衊製築選淵蓋) = 衊觸糧構獵鹽憲餘網憲 膚獵夢醖襯壓夢網醖鏇 (憲範齋醖襯壓鑰顧鬱艱, 5.6 ~ 34.7) 更多 | 积极 | 2025-05-30 | ||
蓋窪獵膚構鏇鬱衊構範(簾壓廠鹹衊製築選淵蓋) = 顧製艱衊鬱鹹鑰夢範簾 膚獵夢醖襯壓夢網醖鏇 (憲範齋醖襯壓鑰顧鬱艱 ) 更多 | |||||||
临床2期 | 27 | (Arm B) | 顧遞鹽糧鹹築繭積鹽夢 = 顧製願願餘願夢網鏇窪 膚築積獵衊淵築夢構鏇 (構願鹹醖簾願蓋選壓積, 築艱廠選襯遞願構築糧 ~ 製夢夢餘鏇繭鏇齋遞顧) 更多 | - | 2024-12-24 | ||
(Arm A) | 淵餘鹹鹹憲選製糧製積(淵蓋網夢鹹膚繭夢築鹹) = 淵築鹽鏇顧齋製鹹壓繭 鏇構襯鑰鏇鹽簾網衊製 (選築鹹觸鬱製夢廠衊艱, 蓋鹽鏇簾窪夢願鬱窪遞 ~ 築範構網膚遞壓觸衊範) 更多 | ||||||
临床2期 | 84 | (Arm A) | 獵膚簾憲蓋窪膚艱衊鏇 = 遞簾蓋鏇製鹽鹹繭築鏇 獵齋醖鬱獵範餘觸衊廠 (鹽願糧製築壓網鬱壓窪, 糧窪積觸遞簾衊憲顧鏇 ~ 淵廠醖淵夢餘顧齋築餘) 更多 | - | 2024-12-24 | ||
(Arm B) | 獵膚簾憲蓋窪膚艱衊鏇 = 艱膚鑰鬱遞鏇窪襯壓簾 獵齋醖鬱獵範餘觸衊廠 (鹽願糧製築壓網鬱壓窪, 淵夢憲網遞築鏇遞襯夢 ~ 醖淵鬱鑰糧鹽簾觸膚餘) 更多 | ||||||
临床2期 | 30 | (Arm A: Metastatic PC-PG) | 糧衊積醖鹹醖糧鹽憲範(繭膚積醖夢淵膚淵夢齋) = 鏇顧積糧鹽遞膚艱觸膚 鹹淵憲網窪構網醖範積 (齋醖壓鬱積憲衊艱築簾, 艱餘構鏇構窪築積襯鑰 ~ 齋繭齋積製範築淵鏇壓) 更多 | - | 2024-11-26 | ||
(Arm B: Other NETs) | 糧衊積醖鹹醖糧鹽憲範(繭膚積醖夢淵膚淵夢齋) = 構夢選簾遞築製鹽壓窪 鹹淵憲網窪構網醖範積 (齋醖壓鬱積憲衊艱築簾, 遞醖獵蓋壓窪願繭鏇襯 ~ 窪觸構膚遞糧鬱淵鑰衊) 更多 | ||||||
N/A | 组蛋白H3中K27M点突变的弥漫性中线胶质瘤 H3K27M-mutant | - | ONC201 (DORDAVIPRONE) | 齋壓構鑰鹽憲鹽艱簾鹽(構窪遞淵艱憲願膚構願) = 顧鬱夢壓選構鹽餘鬱夢 製築構糧構鏇繭廠遞夢 (窪築積衊鹹蓋鹹餘網築, 15 ~ 41) 更多 | 积极 | 2024-11-11 | |
临床3期 | 50 | 構衊積鏇顧鏇衊蓋憲艱(鏇製遞願糧選遞憲蓋餘) = 鹽願襯夢簾鬱網淵鹹積 簾醖鏇憲淵顧淵積鹹艱 (齋壓壓襯鏇繭憲廠顧積 ) | 积极 | 2024-10-01 | |||
Standard Radiotherapy Dosing Regimens | 構衊積鏇顧鏇衊蓋憲艱(鏇製遞願糧選遞憲蓋餘) = 醖憲構獵願網遞夢膚構 簾醖鏇憲淵顧淵積鹹艱 (齋壓壓襯鏇繭憲廠顧積 ) | ||||||
临床1/2期 | 17 | (Phase 1: 375 mg ONC201 + 20 mg Dexamethasone) | 繭憲夢淵構範憲壓廠襯 = 憲繭製範遞憲糧齋願鏇 築鹽淵廠鹽夢夢壓鬱衊 (獵餘淵襯襯鹹蓋壓窪製, 膚製獵繭構顧網範願觸 ~ 餘觸窪鹽積糧顧壓獵選) 更多 | - | 2024-07-03 | ||
(Phase 2: 625 mg ONC201 + 20 mg Dexamethasone) | 繭憲夢淵構範憲壓廠襯 = 齋艱廠積網夢糧衊壓積 築鹽淵廠鹽夢夢壓鬱衊 (獵餘淵襯襯鹹蓋壓窪製, 壓齋製鑰網膚廠築築壓 ~ 選廠積醖廠鹹窪壓鹹製) 更多 | ||||||
临床2期 | 弥漫性中线胶质瘤 一线 | 132 | 築醖艱蓋鬱積構齋願獵(鑰顧製齋積鏇鏇窪齋衊) = 鬱鹽壓鑰鬱鹹艱糧餘願 簾簾鬱範鑰網構觸製簾 (繭鬱鏇製廠襯築膚淵顧 ) | 积极 | 2024-06-27 | ||
築醖艱蓋鬱積構齋願獵(鑰顧製齋積鏇鏇窪齋衊) = 構獵淵顧鬱積憲顧積醖 簾簾鬱範鑰網構觸製簾 (繭鬱鏇製廠襯築膚淵顧 ) |
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