A Phase 1b Study of Combination Tovorafenib (DAY101) and Rituximab Treatment in Relapsed or Refractory Classical Hairy Cell Leukemia and Phase 2 Randomized Study Comparing Tovorafenib (DAY101) and Rituximab With Cladribine and Rituximab for Front-Line Treatment of Classical Hairy Cell Leukemia

This phase Ib/II trial tests the safety, side effects, and effectiveness of tovorafenib in combination with rituximab in patients with classical hairy cell leukemia (cHCL) that has come back after a period of improvement (recurrent) or that has not responded to previous treatment (refractory) and compares the effect of tovorafenib and rituximab to current standard treatment of cladribine and rituximab in cHCL patients that have not yet received treatment. Tovorafenib blocks certain proteins made by the mutated BRAF gene, which may help keep cancer cells from growing. It is a type of kinase inhibitor. Rituximab is a monoclonal antibody. It binds to a protein called CD20, which is found on B cells (a type of white blood cell) and some types of cancer cells. This may help the immune system kill cancer cells. Cladribine damages the cell's deoxyribonucleic acid and may kill cancer cells. It is a type of antimetabolite. Giving tovorafenib in combination with rituximab may be safe and tolerable and more effective than cladribine with rituximab in treating patients with untreated, recurrent or refractory cHCL.

开始日期2025-09-30

申办/合作机构

National Cancer Institute

NCT06381570

/ Recruiting早期临床1期IIT

VICTORY: A Pilot Study to Investigate Safety and Efficacy of Weekly Combination of Intravenous Vinblastine With Oral Type II RAF Inhibitor Tovorafenib in Pediatric Patients With Recurrent/Progressive RAF Altered Low Grade Gliomas

This is a Pilot, multicenter, open-label study of patients less than or equal to 25 years, with recurrent or progressive LGG harboring a CRAF or BRAF alteration, including BRAF V600 mutations and KIAA1549: BRAF fusions. Patients with BRAF or CRAF alterations will be identified through molecular assays as routinely performed at Clinical Laboratory Improvement Amendments (CLIA) of 1988 or other similarly certified laboratories.
The study will be conducted in two sequential phases:
Phase A: A Feasibility (combination dose finding) phase, followed by Phase B: An Efficacy phase. The maximum tolerated dose (MTD)/Recommended Phase 2 Dose (RP2D) of the combination as determined in Phase A would be the dose used in Phase B. The patients on Phase A who were below the MTD/RP2D would be eligible for intra-patient dose escalation to MTD/RP2D subject to criteria outlined later

开始日期2024-03-21

申办/合作机构

The Hospital for Sick Children

[+1]

NCT05566795

/ Recruiting临床3期

LOGGIC/FIREFLY-2: A Phase 3, Randomized, International Multicenter Trial of DAY101 Monotherapy Versus Standard of Care Chemotherapy in Patients With Pediatric Low-Grade Glioma Harboring an Activating RAF Alteration Requiring First-Line Systemic Therapy

This is a 2-arm, randomized, open-label, multicenter, global, Phase 3 trial to evaluate the efficacy, safety, and tolerability of tovorafenib monotherapy versus standard of care (SoC) chemotherapy in participants with pediatric low-grade glioma (LGG) harboring an activating rapidly accelerated fibrosarcoma (RAF) alteration requiring first-line systemic therapy.

开始日期2023-02-27

申办/合作机构

Day One Biopharmaceuticals, Inc.

100 项与 Tovorafenib 相关的临床结果

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100 项与 Tovorafenib 相关的转化医学

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100 项与 Tovorafenib 相关的专利（医药）

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项与 Tovorafenib 相关的文献（医药）

2025-12-01·COMPUTATIONAL BIOLOGY AND CHEMISTRY

Identification of different lung adenocarcinoma subtypes in combination with antidiuretic hormone-related genes and creation of an associated index to predict prognosis and guide immunotherapy

Article

作者: Dai, Hong ; Lv, Yuankai ; Zheng, Hao ; Cai, Xiaoping

BACKGROUND:

Lung adenocarcinoma (LUAD) is one of the most aggressive and rapidly lethal tumor types. Previous studies have demonstrated the involvement of antidiuretic hormone (ADH)-related genes in cancer. However, the role of ADH-related genes in LUAD remains unclear. Therefore, investigating the characteristics of these genes in LUAD is essential.

METHODS:

Differentially expressed genes (DEGs) associated with ADH in LUAD were identified using the STRING database. Consensus clustering was performed, and a protein-protein interaction network was constructed for the DEGs between subtypes. Genes extracted from the PPI network underwent univariate, LASSO, and multivariate Cox regression analyses to develop a predictive model for LUAD. A nomogram integrating clinical data and risk scores was created, and its prognostic power for overall survival (OS) in LUAD patients was evaluated. Additionally, LUAD patients were analyzed for targeted therapies, immune landscape, functional enrichment, and mutation profiles. Finally, qRT-PCR was used to examine the expression of signature genes in LUAD cells.

RESULTS:

Based on ADH-related DEGs, LUAD patients were stratified into two clusters (Cluster 1 and Cluster 2) with distinct survival outcomes. A predictive model incorporating nine feature genes was subsequently constructed using DEGs from these two subtypes. The receiver operating characteristic curve demonstrated the model's prognostic accuracy in predicting OS in LUAD patients. Compared to the high-risk group, patients in the low-risk group exhibited higher immune infiltration levels and immunophenoscore, along with lower tumor immune dysfunction and exclusion scores. Enrichment analysis revealed that immune response pathways and ligand-receptor interactions were the primary functional categories distinguishing the high- and low-risk groups. The low-risk group showed a significantly lower gene mutation burden. Drug sensitivity analysis identified several potential targeted therapies, including Dabrafenib, ARQ-680, Vemurafenib, BGB-283, MLN-2480, and GDC-0994, which might act on hub genes. qRT-PCR validation confirmed that DNAH12 was significantly downregulated in tumor tissues, while DKK1, DLX2, IGFBP1, NTSR1, RPE65, and VGF were markedly upregulated.

CONCLUSION:

This study provided potential prognostic biomarkers for LUAD and might facilitate the development of effective immunotherapy strategies for LUAD patients.

2025-06-01·PHARMACOLOGICAL RESEARCH

Properties of FDA-approved small molecule protein kinase inhibitors: A 2025 update

Review

作者: Roskoski, Robert

Because of the deregulation of protein kinase action in many inflammatory diseases and cancer, the protein kinase family has become one of the most significant drug targets in the 21st century. There are 85 FDA-approved protein kinase antagonists that target about two dozen different enzymes and four of these drugs were approved in 2024 and a fifth was approved in 2025. Of these drugs, five target dual specificity protein kinases (MEK1/2), fourteen inhibit protein-serine/threonine protein kinases, twenty-one block nonreceptor protein-tyrosine kinases, and 45 target receptor protein-tyrosine kinases. The data indicate that 75 of these drugs are prescribed for the treatment of neoplasms. Seven drugs (abrocitinib, baricitinib, deucravacitinib, deuruxolitinib, ritlecitinib, tofacitinib, upadacitinib) are prescribed for the management of inflammatory diseases (atopic dermatitis, rheumatoid arthritis, psoriasis, alopecia areata, and ulcerative colitis). Of the 85 FDA-approved agents, about two dozen are used in the treatment of multiple diseases. The following four drugs received FDA approval in 2024 - deuruxolitinib (alopecia areata), ensartinib and lazertinib (non-small cell lung cancer), and tovorafenib (pediatric glioma) while mirdametinib was approved in 2025 for the treatment of type I neurofibromatosis (von Recklinghausen disease). Apart from netarsudil, temsirolimus, and trilaciclib, the approved protein kinase blockers are orally bioavailable. This article summarizes the physicochemical properties of all 85 FDA-approved small molecule protein kinase inhibitors including the molecular weight, number of hydrogen bond donors/acceptors, ligand efficiency, lipophilic efficiency, polar surface area, and solubility. A total of 39 of the 85 FDA-approved drugs have a least one Lipinski rule of 5 violation.

2025-05-08·ONCOLOGIST

Response to a novel type II RAF inhibitor in diffuse leptomeningeal glioneuronal tumor with BRAF fusion

Article

作者: Starkey, Jay ; Abtin, Vicki ; Youngworth, Sandra ; Stull, David ; Kummar, Shivaani ; Chun, Brie M

Abstract:

Background:

Diffuse leptomeningeal glioneuronal tumor (DL-GNT) is a rare disease which is more often diagnosed in children and adolescents than adults. Activation of the MAPK/ERK pathway is implicated in the majority of cases, and BRAF fusions are the most common genetic alteration. BRAF fusions result in dimerization and constitutive downstream MAPK/ERK activity, against which type I RAF inhibitors have limited efficacy. Type II RAF inhibitors stabilize RAF in an inactive conformation and inhibit both dimer protomers, thus inhibiting downstream MAPK/ERK activity in the setting of BRAF fusions.

Case Presentation:

A previously-healthy 33 year old man was diagnosed with DL-GNT, which harbored a pathogenic BRAF:KIAA1549 gene fusion. He was initially treated with a MEK inhibitor but developed drug-related cardiotoxicity. Without treatment, he developed significant functional limitations due to leptomeningeal disease. A compassionate use indication was pursued for an investigational CNS-penetrant type II BRAF inhibitor, tovorafenib. Within 3 months of initiating the medication, the patient experienced notable gains in functional status and with over 12 months of treatment has been able to rejoin recreational activities.

Conclusions:

This case highlights the importance of tumor molecular characterization, particularly in rare tumors, whereby identification of the BRAF:KIAA1594 gene fusion led to an appropriate selection of a type II BRAF inhibitor.

168

项与 Tovorafenib 相关的新闻（医药）

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

2025-06-19

·医学新视点

30岁确诊脑癌的她已活过11年：她试过的“救命药”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.推荐阅读62%患者肿瘤退缩，双靶向CAR-T疗法挽救复发性脑瘤！成果登上《自然-医学》针对隐藏在大脑中的 “定时炸弹”，创新蛋白降解分子有望填补致命肿瘤的治疗空白！《科学》：创新CAR-T消除致命脑瘤，减轻炎症反应，2大改造有望变革治疗格局参考资料（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，传递医学新知

突破性疗法上市批准

2025-06-18

·GlobeNewswire-Health Care

XOMA Royalty Declares Quarterly Preferred Stock Dividends

EMERYVILLE, Calif., June 18, 2025 (GLOBE NEWSWIRE) -- XOMA Royalty Corporation (Nasdaq: XOMA) today announced its Board of Directors has authorized the following cash dividends to holders of XOMA Royalty’s Series A and Series B Cumulative Preferred Stock: Holders of the 8.625% Series A Cumulative Perpetual Preferred Stock (Nasdaq: XOMAP) shall receive a cash dividend equal to $0.53906 per share. Holders of depositary shares, each representing 1/1000 of a share of XOMA Royalty’s 8.375% Series B Cumulative Perpetual Preferred Stock (Nasdaq: XOMAO), shall receive a cash dividend equal to $0.52344 per depositary share. The preferred dividends will be paid on or about July 15, 2025, to respective holders of record at the close of business on July 3, 2025. About XOMA Royalty CorporationXOMA Royalty is a biotechnology royalty aggregator playing a distinctive role in helping biotech companies achieve their goal of improving human health. XOMA Royalty acquires the potential future economics associated with pre-commercial therapeutic candidates that have been licensed to pharmaceutical or biotechnology companies. When XOMA Royalty acquires the future economics, the seller receives non-dilutive, non-recourse funding they can use to advance their internal drug candidate(s) or for general corporate purposes. The Company has an extensive and growing portfolio of assets (asset defined as the right to receive potential future economics associated with the advancement of an underlying therapeutic candidate). For more information about the Company and its portfolio, please visit www.xoma.com. EXPLANATORY NOTE: Any references to “portfolio” in this press release refer strictly to milestone and/or royalty rights associated with a basket of drug products in development. Any references to “assets” in this press release refer strictly to milestone and/or royalty rights associated with individual drug products in development. As of the date of this press release, the commercial assets in XOMA Royalty’s milestone and royalty portfolio are VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), MIPLYFFA™ (arimoclomol), XACIATO™ (clindamycin phosphate) vaginal gel 2%, IXINITY® [coagulation factor IX (recombinant)], and DSUVIA® (sufentanil sublingual tablet). All other assets in the milestone and royalty portfolio are investigational compounds. Efficacy and safety have not been established. There is no guarantee that any of the investigational compounds will become commercially available. Investor contact:Media contact:Juliane SnowdenKathy VincentXOMA Royalty CorporationKV Consulting & Management+1-646-438-9754+1-310-403-8951juliane.snowden@xoma.comkathy@kathyvincent.com

100 项与 Tovorafenib 相关的药物交易

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外链

KEGG	Wiki	ATC	Drug Bank
-	-	-	DB15266

研发状态

批准上市

10 条最早获批的记录,

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适应症	国家/地区	公司	日期
BRAF 融合或重排阳性的低级别胶质瘤	美国	Day One Biopharmaceuticals, Inc.	2024-04-23
BRAF V600 突变的低级别胶质瘤	美国	Day One Biopharmaceuticals, Inc.	2024-04-23
低级神经胶质瘤	美国	Day One Biopharmaceuticals, Inc.	2024-04-23

未上市

10 条进展最快的记录,

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适应症	最高研发状态	国家/地区	公司	日期
毛细胞白血病	临床2期	-	National Cancer Institute	2025-09-30
难治性朗格汉斯细胞组织细胞增生症	临床2期	美国	National Cancer Institute	2024-03-28
难治性朗格汉斯细胞组织细胞增生症	临床2期	澳大利亚	National Cancer Institute	2024-03-28
难治性朗格汉斯细胞组织细胞增生症	临床2期	加拿大	National Cancer Institute	2024-03-28
膀胱癌	临床2期	美国	Day One Biopharmaceuticals, Inc.	2021-07-15
膀胱癌	临床2期	澳大利亚	Day One Biopharmaceuticals, Inc.	2021-07-15
膀胱癌	临床2期	比利时	Day One Biopharmaceuticals, Inc.	2021-07-15
膀胱癌	临床2期	加拿大	Day One Biopharmaceuticals, Inc.	2021-07-15
膀胱癌	临床2期	法国	Day One Biopharmaceuticals, Inc.	2021-07-15
膀胱癌	临床2期	西班牙	Day One Biopharmaceuticals, Inc.	2021-07-15

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临床结果

适应症

分期

评价

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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


NCT04775485 (FIREFLY-1, ASCO2025)	临床2期	BRAF V600 突变的低级别胶质瘤 BRAF-altered	137	Tovorafenib	遞衊願壓鹹鑰簾觸簾鏇(鹹觸艱糧獵壓築鹽網餘) = 繭繭網範鏇壓醖選淵憲襯齋鬱鬱襯觸鹽願鏇簾 (憲製鹹衊醖蓋鬱廠顧觸 ) 更多	-	2025-05-30
ASCO2025	临床1/2期	BRAF	167	Tovorafenib	選網淵積獵繭艱獵選繭(夢壓鏇齋齋繭製遞淵選) = 齋遞製壓構製齋鑰襯醖齋鏇醖網衊範醖遞遞襯 (齋築齋衊觸遞鏇夢齋鑰, 0.4 ~ 2.2)	-	2025-05-30
NCT04985604 (ESMO2024)	临床1/2期	实体瘤 \| 复发性黑色素瘤	-	Tovorafenib 600 mg	廠壓獵範獵鹹夢製網襯(觸淵鏇鹽選鬱鏇網選齋) = 39% 餘鑰齋齋製淵遞齋範蓋 (築範繭鏇醖範齋廠選餘 ) 更多	-	2024-09-14
NCT03429803 (ASCO2024) 人工标引	临床1期	低级神经胶质瘤 MAPK pathway activated	35	Tovorafenib 530 mg/m2	願膚遞蓋鑰鹽窪憲選齋(夢鏇憲壓壓遞鬱鏇顧糧) = 構鏇壓願獵餘簾選遞廠觸夢願壓壓艱選窪蓋膚 (餘衊繭構觸鑰窪積願蓋 ) 更多	积极	2024-05-24
NCT04775485 (FIREFLY-1, FDA_CDER) 人工标引	临床2期	BRAF V600 突变的低级别胶质瘤 \| BRAF 融合或重排阳性的低级别胶质瘤 BRAF Rearrangement \| BRAF V600E	137	OJEMDA	窪廠膚獵窪憲糧選製願(糧願膚鹽糧獵蓋繭糧獵) = 遞鑰鏇繭廠齋鬱鹽餘糧窪齋鬱製艱築網繭簾鏇 (艱廠繭顧鹹醖憲膚獵憲, 40 ~ 63) 更多	积极	2024-04-23
NCT04775485 (FIREFLY-1, ASCO2023) 人工标引	临床2期	低级神经胶质瘤	77	Tovorafenib 420 mg/m2	糧糧壓膚簾鹹積獵膚遞(蓋鏇製簾獵醖壓膚選網) = 淵顧鑰製鑰醖範網齋範鹹願齋壓顧齋壓艱繭憲 (鏇鹽壓壓簾蓋獵襯艱醖 ) 更多	积极	2023-05-31
NCT04775485 (FIREFLY-1, Corporate Publications) 人工标引	临床2期	低级神经胶质瘤	22	Tovorafenib	糧繭醖鏇構積鏇鬱淵窪(鏇製觸鹽遞簾醖艱蓋願) = 選糧築構廠餘構鹽鹽醖構膚襯餘積製憲衊製繭 (簾鏇鏇觸壓蓋憲遞顧鑰 ) 更多	积极	2022-06-12
SNO2020	临床1期	复发性儿童急性淋巴细胞白血病 MEK/ERK pathway alterations \| KIAA1549:BRAF fusions \| SRGAP3-RAF1 gene fusion ... 更多	9	DAY101 280 mg/m^2	窪齋選觸餘積顧構範鹹(製齋簾艱壓窪餘壓餘積) = one grade 3 elevation is CPK 艱廠獵選鏇構鹽積憲餘 (構範鑰築糧願膚衊壓鹽 ) 更多	积极	2020-11-09