预约演示

更新于：2026-05-26

Infigratinib Phosphate

英菲格拉替尼

更新于：2026-05-26

概要

基本信息

药物类型

小分子化药

别名

Infigratinib、BBP-831、BGJ-398

+ [7]

靶点

FGFR1 x FGFR2 x FGFR3 x FGFR4

作用方式

拮抗剂

作用机制

FGFR1拮抗剂(成纤维细胞生长因子受体-1拮抗剂)、FGFR2拮抗剂(成纤维细胞生长因子受体-2拮抗剂)、FGFR3拮抗剂(成纤维细胞生长因子受体-3拮抗剂)

治疗领域

肿瘤消化系统疾病其他疾病+ [3]

在研适应症

FGFR2融合或重排的胆管癌软骨发育不全膀胱尿路上皮癌+ [8]

非在研适应症

晚期胆管癌膀胱癌子宫内膜癌+ [25]

原研机构

QED Therapeutics, Inc.

在研机构

QED Therapeutics, Inc.

BridgeBio Pharma, Inc.Ohio State University Comprehensive Cancer Center

+ [4]

非在研机构

Helsinn Healthcare SA

Novartis Pharmaceuticals Canada, Inc.

Novartis Pharmaceuticals Corp.

+ [6]

权益机构

Xediton Pharmaceuticals, Inc.

Novartis AG

Kyowa Kirin Co., Ltd.

+ [5]

最高研发阶段批准上市

首次获批日期

美国 (2021-05-28),

FGFR2融合或重排的胆管癌

最高研发阶段(中国)临床申请批准

特殊审评突破性疗法 (美国)、加速批准 (美国)、孤儿药 (美国)、罕见儿科疾病 (美国)、孤儿药 (欧盟)、突破性疗法 (中国)、孤儿药 (澳大利亚)、快速通道 (美国)

登录后查看时间轴

结构/序列

分子式C26H34Cl2N7O7P

InChIKeyGUQNHCGYHLSITB-UHFFFAOYSA-N

CAS号1310746-10-1

关联

项与英菲格拉替尼相关的临床试验

NCT07393373

/ Enrolling by invitation临床2期

Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Hypochondroplasia: ACCEL OLE

Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.

开始日期2026-04-23

申办/合作机构

QED Therapeutics, Inc.

NCT07169279

/ Recruiting临床2期

Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia

This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants < 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children < 3 years old with ACH at the selected dose.

开始日期2025-11-19

申办/合作机构

QED Therapeutics, Inc.

NCT06873035

/ Enrolling by invitation临床2/3期

A Phase 2/3, Multicenter, Open-Label Phase Followed by a Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children With Hypochondroplasia: ACCEL 2/3

ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib, at one of two doses, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).

开始日期2025-04-22

申办/合作机构-

100 项与英菲格拉替尼相关的临床结果

登录后查看更多信息

100 项与英菲格拉替尼相关的转化医学

登录后查看更多信息

100 项与英菲格拉替尼相关的专利（医药）

登录后查看更多信息

303

项与英菲格拉替尼相关的文献（医药）

2026-05-01·Cancer Medicine

Simultaneous Establishment of Autologous Colorectal Cancer and Mesothelial Stromal Cell Lines from Malignant Ascites Reveals a Mesothelial‐Stromal FGFR3 Axis as a Potential Vulnerability in Peritoneal Metastasis

Article

作者: Kanae Echizen ; Yuki Nakanishi ; Takahiro Toyokawa ; Hiroaki Kasashima ; Yuichiro Miki ; Yasuhiro Fukui ; Masatsune Shibutani ; Tatsunari Fukuoka ; Kiyoshi Maeda ; Tatsuro Tamura ; Zizhou Wang ; Mami Yoshii ; Yoshiki Nonaka ; Yukina Kusunoki ; Naoko Ohtani ; Yuki Seki ; Masakazu Yashiro ; Iguru Omori ; Yu Muta ; Kenji Kuroda

ABSTRACT:

Colorectal cancer (CRC) with peritoneal dissemination remains a major therapeutic challenge because of poor prognosis and limited treatment options. Experimental models that accurately recapitulate tumor–mesothelial interactions are scarce. Here, we report the establishment of a novel autologous paired model comprising a CRC cell line (OMUCR‐1) and matched cancer‐associated mesothelial cells (CAmeso), both simultaneously derived from the malignant ascites of the same patient. Lineage marker analysis using qPCR demonstrated that OMUCR‐1 selectively expressed epithelial markers ( EPCAM, KRT20 ), whereas CAmeso strongly expressed mesothelial‐mesenchymal markers (ACTA2, MSLN) and lacked epithelial marker expression. These mutually exclusive expression patterns confirm that the two cell populations are phenotypically distinct and rule out cross‐contamination. OMUCR‐1 displayed strong tumorigenic capacity across multiple transplantation models. CAmeso enhanced CRC cell migration and invasion in vitro, and co‐transplantation with OMUCR‐1 resulted in larger tumors enriched with αSMA‐positive stromal components. RNA sequencing of co‐injected xenografts revealed increased expression of murine stromal Fgfr3. Treatment with the FGFR inhibitor BGJ398 reduced tumor growth and decreased stromal FGFR3‐positive components, suggesting that stromal FGFR3 may represent a potential microenvironmental vulnerability in CRC with peritoneal dissemination. This autologous CRC‐mesothelial system provides a physiologically relevant platform for dissecting tumor‐stroma interactions in peritoneal metastasis and may advance stromal‐targeted therapeutic strategies.

2026-05-01·CANCER LETTERS

Targeting FGFR1-regulated SPP1 signaling repolarizes immunosuppressive macrophages and sensitizes Hepatocellular Carcinoma to anti-PD-1 therapy

Article

作者: Fu, Jing ; Meng, Zhengyuan ; Gu, Mingye ; Jiang, Youhai ; Wang, Hongyang ; Zong, Yali ; Liu, Xiru ; Chen, Lei ; Liu, Erdong ; Liu, Chunliang ; Li, Jingfeng ; Wang, Xiang ; Chen, Jianan

The response rate to immune checkpoint blockade (ICB) in hepatocellular carcinoma (HCC) remains unsatisfactory, and the mechanisms of resistance are not fully understood. Here, we investigated the role of fibroblast growth factor receptor 1 (FGFR1) in shaping the tumor microenvironment (TME) and mediating ICB resistance. An anti-PD-1-resistant HCC model was established in mice, followed by single-cell RNA sequencing to profile TME alterations. We observed that ICB resistance was associated with FGFR1 upregulation, which activated MAPK signaling and induced SPP1 expression. This cascade promotes macrophage infiltration and M2-type polarization, while simultaneously suppressing T cell recruitment and cytotoxic function, thereby fostering an immunosuppressive microenvironment. SPP1 knockdown or neutralization significantly reduced macrophage accumulation and restored intratumoral T cell infiltration. Importantly, pharmacological inhibition of FGFR1 using BGJ398 synergized with anti-PD-1 therapy, resulting in enhanced antitumor efficacy in preclinical models. Analysis of clinical datasets further revealed that high FGFR1 expression correlated with poor responses to ICB of HCC patients. Collectively, these findings identify FGFR1 as a key mediator of ICB resistance in HCC. Targeting FGFR1 represents a promising strategy to reprogram the immunosuppressive TME and enhance response to immunotherapy, with potential additional value as a predictive biomarker.

2026-04-01·ESMO Open

Phase III trial of infigratinib versus gemcitabine/cisplatin in adults with advanced cholangiocarcinoma with FGFR2 gene fusion or rearrangement: results and reflections on early termination of PROOF 301

Article

作者: Lamarca, A ; Savastano, D M ; Borbath, I ; Goyal, L ; Shroff, R T ; Solanas, J ; Oh, D-Y ; van Veenhuyzen, D F ; Roychowdhury, S ; Tamaş, C ; Freas, E ; Xu, C ; Javle, M M ; Macarulla, T ; Abou-Alfa, G K

BACKGROUND:

Infigratinib, an oral fibroblast growth factor receptor (FGFR) 1-3 inhibitor, showed clinical activity and manageable adverse events in the P2 CBGJ398X2204 study and was conditionally approved for adults with previously treated, unresectable, or metastatic cholangiocarcinoma (CCA) with FGFR2 fusion or other rearrangement. PROOF 301, reported in this article, is the confirmatory phase III trial of infigratinib versus gemcitabine plus cisplatin as first-line treatment of FGFR2-rearranged CCA.

PATIENTS AND METHODS:

Eligible adult patients were randomly assigned in a 2 : 1 ratio to infigratinib 125 mg on days 1-21 of a 28-day treatment cycle versus gemcitabine (1000 mg/m²) + cisplatin (25 mg/m²) on days 1 and 8 of a 21-day cycle. The primary endpoint was progression-free survival (PFS). Secondary endpoints included overall survival, investigator-determined PFS, overall response rate (ORR), best overall response, disease control rate, duration of response, and safety.

RESULTS:

Over 40 months, 1127 patients were pre-screened at 120 sites, and 48 patients were randomly allocated to the study in a 2 : 1 ratio (29 to infigratinib, 19 to chemotherapy). With a target accrual of ∼300 patients, this study was terminated early due to poor accrual. Median PFS (95% confidence interval) by blinded independent central review (BICR) was 7.4 and 8.0 months with infigratinib and chemotherapy, respectively. The ORRs by BICR were 37.9% with infigratinib and 15.8% with chemotherapy. Grade 3-4 adverse events occurred in 79.3% and 58.8% patients treated with infigratinib and chemotherapy, respectively.

CONCLUSION:

Early termination limited the ability to draw definitive conclusions on the efficacy of infigratinib as first-line treatment of FGFR2-rearranged CCA. This study illustrates the challenges of powering confirmatory studies in biomarker-selected subpopulations of rare tumors and highlights the need for regulatory collaboration to develop pragmatic frameworks for assessing novel therapies in ultra-rare malignancies.

259

项与英菲格拉替尼相关的新闻（医药）

2026-05-25

·科创数资视界

20亿样本重构新药研发逻辑：Mammal跨模态AI，打通用数据孤岛破局传统制药

藏在各大制药巨头财报最无奈最现实的就是研发支出。砸进去10亿美元耗费10年时间最后换来一个高达90%的失败率。从一个拥有潜力的分子被筛选出来到动物实验再到临床一期、二期、三期，每一步都是一台残酷的碎钞机。如果一家建筑公司造出来的桥有90%都会坍塌，这家公司早就从地球上消失了，但这就是2026年全球制药巨头们每天都在面临的真实困境。资本市场对这种高风险、低回报的盲盒游戏早就已经厌倦，顶级资本正在6疯狂寻找能够彻底改变底层规则的新基础设施。代号为MAMMAL的底层人工智能模型彻底掀翻了传统的医药研发牌桌客观来看人类目前手中掌握的技术不可谓不先进：智能手机已经普及，基因测序技术把人类的生命密码解析得清清楚楚，医药界还有能够精准预测蛋白质结构的AlphaFold 3以及能够读取基因的EvoR，这些都是强悍的单点突破工具。但荒谬的地方在于手里攥着这么多王牌，新药研发依然像是在蒙着眼睛掷飞镖。那么问题到底出在哪里？答案在于现代生物科技陷入了严重的孤岛效应。疾病在人体内的暴发从来不是某个独立文件夹里运行的程序，而是从DNA变异、基因表达异常、蛋白质结构改变，到细胞病变、肿瘤扩散的完整生命链路。可现在的技术工具全都是各自为战：懂化学分子的系统不懂基因序列，懂基因表达的系统看不懂蛋白质的三维结构。时代的医疗技术就像几个互相听不懂对方说话的盲人侦探在摸同一头大象——每个人手里的线索无比珍贵却没有统一的语言体系，拼凑不出完整的真相。这就导致设计药物时往往顾此失彼。开发一款新药，本质是在由10亿个精密零件组成的人体机器里，寻找一把精准的分子钥匙。科学家先找到致病靶点，再设计药物锁死它：小分子药物体积小，能钻进细胞干预蛋白质；抗体药物像精准制导钳子，在细胞表面咬住目标。但人体系统极度复杂，一把钥匙能开多把锁。药物杀死致病蛋白的同时，可能破坏心脏正常运转，这就是致命副作用的来源。传统研发模式下找到安全有效的完美分子几乎是大海捞针。 MAMMAL模型的出现直接重写了整条产业链的底层逻辑它不再是看懂单一图纸的专家，而是吃透整个人体生物学系统的超级通才。这里需要看透一个核心本质：再先进的算法架构、再顶级的模型算力都只是工具外壳！ MAMMAL实现降维突破的核心根基是海量真实个体临床数据的沉淀、验证与训练迭代。所有精准推演、反常识预判、动态结构破解，并非算法凭空运算，而是依托无数人体实战数据，摸透了生命运行的真实底层规律，这也是它碾压传统模型的核心底气。该模型预训练规模极其庞大，直接吸纳超20亿个样本的海量数据，涵盖数十亿抗体序列、已知蛋白质结构、数百万小分子化学结构，最核心的核心是沉淀了海量真实人体基因表达、临床诊疗、病灶变化的实战数据，真正实现同时吃透化学、遗传学、蛋白质学的跨界融合。这背后是变态的工程学难题：小分子化学结构、基因序列、抗体氨基酸链，三者逻辑完全不同，强行整合极易导致系统崩溃。为此研发团队设计了模块化分词器机制，如同超级翻译局，下设化学、遗传学、蛋白质三大翻译科。识别小分子启动化学翻译科将三维结构转为统一底层代码；读取基因按活跃度排序后翻译为相同底层代码；解析蛋白质/抗体读取氨基酸链完成精准翻译。所有数据最终汇入共享多维空间交融。模型不再孤立看待化学、基因、蛋白，而是依托真实临床数据自动领悟跨界元素的隐秘关联，精准复盘小分子结构如何影响蛋白功能、触发基因表达巨变。这种打通生物学的全局视角是过往仅靠实验室静态数据训练的单一工具，永远无法企及的高度。 MAMMAL参与药物发现11项严苛基准测试碾压行业老牌专用顶级模型其中最具商业价值的是血脑屏障穿透性、临床毒性预测两大测试。血脑屏障是人体大脑的终极保安也是神经类药物的百慕大三角。阿尔茨海默症药物穿不透屏障，数亿研发投入直接打水漂；化疗药穿透屏障会造成致命医疗灾难。此前该领域霸主是专攻10亿+小分子数据的极限特训偏科型模型。按常规商业逻辑，专才必然碾压通才，可MAMMAL却以碾压姿态击败老牌霸主。核心逻辑颠覆认知：纯粹的化学算法模拟终究是纸上谈兵，唯有结合真实人体临床数据的全局推演，才能还原药物在人体内的真实反应。同时学习基因、蛋白与临床诊疗数据的全局模型，在真实药理预判上实现了对纯细分算法模型的降维打击。它看透的不是数据公式而是人体真实的生化运行法则，预判不再是经验猜测而是临床数据堆叠出的精准规律推演。药企早期引入该模型能直接避开研发死胡同砍掉数十亿无效损耗。也正因临床真实数据的不可替代性，生物医药板块的底层投资逻辑，正在发生根本性转移。过去风投评估药企核心看的是罕见病靶点储备、独家分子合成工艺；但在AI制药新时代单一靶点专利、单一技术工艺的壁垒正在快速弱化，极易被快速迭代的算法淘汰。真正具备长期垄断潜力的核心资产不再是专利和技术，而是能够持续沉淀、合规积累、标准化打磨的个体临床真实数据。能够依托海量临床数据喂养大模型、实现全链路药物验证的基础设施级平台，才是未来产业的核心赢家。如同半导体行业，最大利润不属于短期芯片设计公司，而属于掌控制程底层工艺的晶圆巨头。在全新生物大工业时代，数据为王的底层逻辑已然彻底浮出水面。真正让医疗界、华尔街胆寒的不是MAMMAL的常规跑分第一，而是它依托真实临床数据，应对人类顶级绝症时展现出的反常识上帝视角——癌症药物重定位。医药圈近年大火的“老药新用”，核心是跳过漫长的临床安全性验证，从获批或失败旧药里挖掘新用途，一旦成功就是百亿级收益。但人体肿瘤细胞超千种，基因突变各不相同，人工试错近乎愚公移山。为验证MAMMAL实力，研究团队设置严苛盲测局：选取卡非佐米、那丹尼布、Infigratinib、Vimirafinib四款结构复杂药物；通过Tanimoto相似度算法核查，其中3款得分＜0.7，对模型而言是完全陌生的分子；再输入800+种实体肿瘤细胞基因图谱与临床病灶数据让模型给出药物杀伤力排名。 MAMMAL仅依靠一维文本序列与海量临床实战数据对照迅速给出精准排名将卡非佐米列为实体肿瘤最强效药物。而医学常识里卡非佐米是获批的血液癌靶向药，全球医学界仅凭过往片面经验公认其对实体肿瘤无效。 AI的结论直接推翻了人类几十年的固有认知。团队随即开展细胞毒性实验，真实实验结果与MAMMAL预测100%重合，805种癌细胞测试中药效排序准确率高达95%。这背后的本质就是人类专家局限于有限的临床经验，而大模型依托海量全域临床数据看透了人类从未发现的药理关联。一个仅读取文本序列、对照亿万临床数据的AI，隔空推翻人类肿瘤专家的经验偏见足以证明：传统医学经验在海量真实临床数据堆叠出的底层规律面前，存在无法弥补的巨大盲区。一旦该模型商用，药企尘封的废弃药物库将依托AI+临床数据的组合赋能变成价值连城的超级金矿，各类绝症特效药会快速井喷。 MAMMAL真正确立行业统治地位的是与诺奖级模型AlphaFold 3的正面硬刚 AlphaFold 3精准预测蛋白质三维结构，团队斩获诺贝尔化学奖，是行业黄金标准。常规逻辑下自然语言模型MAMMAL比拼三维结构预测完全没有胜算！研究团队选取7种复杂疾病靶点让两个模型预测抗体结合能力最终MAMMAL在5个靶点上击败AlphaFold 3。核心原因在于人类对蛋白质的固有认知误区：教科书里的蛋白质是冷冻电镜拍下的静态结构；而人体内30%–40%的蛋白质是固有无序区，无固定形态，持续动态扭动。 AlphaFold 3仅依托实验室静态结构数据训练，面对动态无序蛋白会严重失真； MAMMAL的核心优势是结合了海量人体真实临床动态数据，不依赖固化的三维结构，而是通过20亿+临床与生物数据，吃透氨基酸序列的底层语法，依靠真实人体生化规律推演动态结合概率。这种绕过静态表象依托实战数据直击底层规律的能力让MAMMAL应对复杂病变蛋白时精准度远超AlphaFold 3。这意味着传统耗时数年耗资巨大的抗体筛选流水线，将面临技术性淘汰。药企可批量输入抗体序列依托数据驱动的AI模型，快速匹配未知病毒、罕见病靶点。这还只是MAMMAL的分析能力，它最颠覆的是依托海量临床数据迭代，从零生成全新生命大分子的造物主级能力。抗体的核心是CDR互补决定区（CDRH3为最难、变异最高的魔鬼区域），决定抗体能否精准锁定疾病靶点也是传统AI的技术盲区。研究团队抹除已知抗体的CDR序列让MAMMAL凭空生成适配靶点的氨基酸序列。全程盲测的语言模型，完美完成序列生成，CDRH3区域准确率较传统顶级模型暴涨19%。在AI基准测试中19%的提升属于碾压级突破，证明模型并非简单概率游戏、死记数据，而是依托海量临床实战，真正吃透了生命底层运行法则。综合所有测试，MAMMAL推翻专家偏见、动态预测击败诺奖模型、自主设计全新抗体，正式宣告盲盒制药的旧时代彻底终结。这是真正的生物学基础大模型（Foundation model for biology）依托真实临床数据打通化学、基因、蛋白质全链路。如果MAMMAL这套依托临床数据驱动的架构全面落地商用，整个医药产业链将迎来全方位重构。过去百亿投入、十年深耕、九成亏损的盲盒研发模式会彻底淘汰，取而代之的是算力推演前置、临床数据验证、精准靶向研发的全新模式。未来的新药研发会先通过AI完成数万种药物、病灶、基因的千万次模拟碰撞，只有经过数据验证、无毒副作用、疗效确定的分子才会进入实验室实操，新药研发成本周期将断崖式下跌。通俗来说普通人的就医治病将彻底告别“千人一方”的刻板模式。未来绝症罕见病患者只需提供个人基因与血液样本，AI依托海量临床数据对照，几分钟就能锁定个人专属病灶成因，快速定制独一无二的专属抗体药物，让精准救病、高效治癌从高端科研，变成普通患者可触及的现实。站在产业与资本的长远视角来看这场变革从来不是简单的AI技术升级，而是整个医药产业价值逻辑的彻底洗牌。对于深耕医疗赛道的企业决策者、创业者而言，当下的行业看似平稳无波，实则暗流涌动。过去靠专利壁垒、技术差异、赛道红利就能安稳盈利的时代已经尾声，隐形的行业危机早已埋下：不布局临床数据沉淀、不拥抱数据驱动AI研发的企业，会慢慢丧失核心竞争力，逐步被高效、精准、低成本的新产业模式替代。而随之而来的是新一轮确定性极强的时代红利。未来产业红利不再集中在单一新药研发、单点技术突破上，真正的机会藏在临床数据合规沉淀、大模型算力适配、个性化生物合成、精准医疗落地的全产业链条中。越早布局数据资产、打通AI研发链路的玩家，越能抢占新产业规则下的头部席位。医疗革命的深层逻辑：当AI Agent与Blockchain重构 “人的价值” 技术永远是工具，真实的人体临床数据才是未来生物医药产业最稀缺、最核心、不可复制的终极资产。这场由数据驱动的医药革命正在重塑所有人的未来。信息来源:（真实性参考，可溯源） 1. MAMMAL大模型原始论文： https://www.biorxiv.org/content/10.1101/2026.03.12.584927v1 2. AlphaFold 3诺奖成果官方报道：https://deepmind.google/discover/blog/alphafold-3-advancing-protein-structure-prediction-to-new-heights/ 3. 血脑屏障与实体瘤药物重定位行业研究（Nature子刊）： https://www.nature.com/articles/s41591-025-03872-9 4. CDRH3抗体序列预测技术突破（Cell Reports Methods）： https://www.cell.com/cell-reports-methods/fulltext/S2667-2375(26)00123-4 5. 华尔街关于AI重塑医药研发赛道分析（Bloomberg）： https://www.bloomberg.com/news/articles/2026-04-15/ai-drug-discovery-model-mammal-upends-pharma-r-d-paradigm

财报

2026-05-21

·FiercePharma

With Voxzogo under pressure, BioMarin touts trial win in label expansion bid

Voxzogo accounted for 29% of the company’s revenue in 2025, but it's facing competitive pressure in its original indication. With phase 3 results that have “exceeded” BioMarin’s expectations, according to R&D chief Greg Friberg, M.D., the California biotech appears set up to gain a label expansion for its top-selling product, dwarfism drug Voxzogo.The Canopy-HCH-3 study in children with hypochondroplasia (HCH) has met its primary endpoint, with Voxzogo providing “highly statistically significant” improvements in annualized growth velocity (AGV), the company said. In a trial of 80 patients, ranging from ages 3 to 17, those on Voxzogo had an AGV of 2.33 centimeters more than those on placebo at week 52. In addition, Voxzogo provided a statistically significant increase in two other height measurements—standing height and height Z-score—and in arm span, which is key in providing patients with a longer reach and allowing them to gain independence, the company said.The results are “tremendously encouraging,” according to Andrew Dauber, the lead investigator of the study and the chief of endocrinology at Children's National Hospital in Washington, D.C.“Seeing these improvements in growth is a milestone we have hoped for after so many years without treatment options,” Dauber added in a release. “These data suggest we may be approaching a new era in how we care for children with hypochondroplasia.”HCH is a rare genetic condition that limits bone growth, resulting in short-limbed dwarfism. It is caused by a mutation in the FGFR3 gene, which slows the conversion of cartilage into bone. Those with HCH typically have an average-sized torso but disproportionately short arms, legs, hands, and feet. The condition can lead to otolaryngologic (ear, nose and throat) issues and neurological complications. HCH is a milder form of achondroplasia (ACH). In 2021, the FDA approved CNP analog Voxzogo as a treatment for ACH and two years later expanded the nod to children aged 5 and younger.With the results—which the company will present in full at a medical conference later this year—BioMarin said it expects to file for approval of Voxzogo in HCH in the third quarter of this year, followed by submissions to the European Medicines Agency and other regulators.“While this is an important win for the Voxzogo franchise and provides incremental expansion beyond achondroplasia (ACH), we remain cautious that success in HCH alone is unlikely to fully offset growing competitive pressures emerging in ACH,” analysts from Leerink Partners wrote in a note to clients.Two months ago, Ascendis gained FDA approval for its ACH treatment, Yuviwel. The approval covers children aged 2 and older who have open growth plates, which are areas where cartilage develops at the end of bones, allowing them to lengthen. The advantage Yuviwel offers is that it is dosed weekly as opposed to daily for Voxzogo. In February, BridgeBio revealed the success of a phase 3 trial of its FGFR1-3 selective tyrosine kinase inhibitor infigratinib in children with ACH. BridgeBio and its partner Kyowa Kirin plan to apply for approval of infigratinib in the second half of this year.“We think it will be important to understand how BioMarin plans to help drive disease awareness and enhance the diagnosis journey as HCH is not diagnosed at the same rate as ACH,” Leerink added. “Based on (KOL) discussions, we think there is a fairly wide spectrum of severity in HCH where some patients might already be on Voxzogo, while other patients may be milder.” With sales of $927 million, which were up 26%, Voxzogo accounted for 29% of the company’s revenue in 2025. The Leerink team said it projects “significantly lower peak sales in HCH vs. ACH.”In March, BioMarin announced that it had discontinued phase 2 trials for Voxzogo across three potential indications: Turner syndrome, SHOX-deficiency and aggrecan (ACAN)-deficiency. Still underway are phase 2 trials of Voxzogo for kids with Noonan syndrome and idiopathic short stature (ISS) without ACAN-deficiency.

临床3期临床结果上市批准临床2期加速审批

2026-05-20

·Firstwordpharma

BioMarin moves to expand Voxzogo into hypochondroplasia with pivotal win

BioMarin Pharmaceutical said Voxzogo (vosoritide) cleared the main goal of a Phase III trial in children with hypochondroplasia — delivering notable growth rate improvement over placebo — setting up a filing to the FDA in the third quarter. The modified C-type natriuretic peptide (CNP) is already approved for the treatment of achondroplasia.Greg Friberg, chief R&D officer at BioMarin, said the readout "exceeded…expectations," underscoring "Voxzogo's potential to fundamentally change the treatment landscape for this condition." Along with a submission to the FDA, the company will make filings in the EU and other markets.The CANOPY-HCH-3 study enrolled 80 children aged 3 to 17 years, who were randomised to receive either Voxzogo or placebo. Top-line results showed that the study met its primary endpoint, with the Voxzogo arm demonstrating a highly significant 2.33 cm/year improvement in annualised growth velocity (AGV) from baseline compared with placebo at week 52.Additionally, children receiving Voxzogo showed significant improvements on secondary goals of standing height and height Z-score versus placebo after one year of treatment. Another key prespecified secondary endpoint of arm span also reached significance.BioMarin noted that the safety profile of Voxzogo in the trial was consistent with its established profile in achondroplasia; no new signals were identified. Detailed findings from CANOPY-HCH-3 will be presented at an upcoming medical meeting.Besides achondroplasia and hypochondroplasia, the company has bigger ambitions of securing four more indications for Voxzogo by 2031. However, earlier this year, the company was forced to halt Phase II studies in Turner syndrome, SHOX-deficiency and ACAN-deficiency after the emergence of safety issues — cases of slipped capital femoral epiphysis — in separate investigator-sponsored trials. Studies in Noonan syndrome, as well as those living with idiopathic short stature without ACAN-deficiency, will continue unaffected. Meanwhile, BridgeBio Pharma's infigratinib — an oral FGFR3 inhibitor — has emerged as the main contender to Voxzogo, recently showcasing upbeat late-stage data in achondroplasia. At the time, BridgeBio also noted it will rapidly advance the asset through Phase III testing for hypochondroplasia.

临床3期临床结果临床2期上市批准

100 项与英菲格拉替尼相关的药物交易

登录后查看更多信息

外链

KEGG	Wiki	ATC	Drug Bank
D11589 D11611	英菲格拉替尼	L01XE	DB11886

研发状态

批准上市

10 条最早获批的记录,

后查看更多信息

适应症	国家/地区	公司	日期
FGFR2融合或重排的胆管癌	美国	Helsinn Healthcare SA	2021-05-28

未上市

10 条进展最快的记录,

后查看更多信息

适应症	最高研发状态	国家/地区	公司	日期
胆管癌	申请上市	瑞士	Helsinn Birex Pharmaceuticals Ltd.	2022-01-30
软骨发育不良	临床3期	美国	-	2025-04-22
软骨发育不良	临床3期	澳大利亚	-	2025-04-22
软骨发育不良	临床3期	加拿大	-	2025-04-22
软骨发育不良	临床3期	法国	-	2025-04-22
软骨发育不良	临床3期	挪威	-	2025-04-22
软骨发育不良	临床3期	葡萄牙	-	2025-04-22
软骨发育不良	临床3期	新加坡	-	2025-04-22
软骨发育不良	临床3期	西班牙	-	2025-04-22
软骨发育不良	临床3期	瑞典	-	2025-04-22

登录后查看更多信息

临床结果

适应症

分期

评价

查看全部结果

研究	分期	人群特征	评价人数	分组	结果	评价	发布日期


ESMO_SRC2026	N/A	中枢神经系统肿瘤	9	infigratinib	餘範簾繭齋醖鬱築廠壓(製選鑰淵夢膚網遞繭構) = Toxicities included fatigue, visual changes, hyperphosphatemia and stomatitis. Two patients required dose reductions and four required discontinuation due to toxicity (visual changes - 1, hyperphosphatemia - 1 and fatigue - 2) 餘窪憲鹹壓襯遞夢築網 (憲餘齋網鹽廠簾糧鹽齋 )	积极	2026-03-09
NCT04424966 (CTgov)	早期临床1期	多形性胶质母细胞瘤 \| 复发性恶性胶质瘤	7	Infigratinib	醖壓遞獵鏇網獵範衊簾(簾齋獵襯選鏇製製衊顧) = 鏇遞積齋願築遞衊窪築壓壓顧餘鹽憲積蓋膚鹹 (鬱艱淵鏇鏇蓋顧範鹹鹹, 醖鏇壓鏇衊醖構築膚鬱 ~ 夢艱構築廠顧遞蓋膚顧) 更多	-	2025-10-24
NCT04233567 (CTgov)	临床2期	难治恶性实体肿瘤 \| 转移性实体瘤 \| 晚期恶性实体瘤 ... 更多	17	Infigratinib	選構願蓋鑰製顧鏇範餘 = 築壓鹽製範壓選壓繭選壓築壓構簾蓋鏇艱顧膚 (顧齋醖願壓選衊膚顧艱, 衊觸壓願衊鹹壓簾製窪 ~ 鹽顧製顧壓鏇鹽鑰築鬱) 更多	-	2025-06-10
NCT04265651 (PROPEL2, Pubmed \| N Engl J Med)	临床2期	软骨发育不全	72	Infigratinib 0.25 mg/kg	餘獵壓艱衊遞醖齋淵繭(鑰選壓鹽繭壓願餘遞鹹) = 網夢簾壓夢選鹽獵餘衊顧窪製願構鏇製鹽築遞 (鏇積積鹹蓋築醖襯壓範, 1.22 ~ 3.79)	积极	2025-02-27
NCT04228042 (CTgov)	临床1/2期	肾盂和输尿管尿路上皮癌	15	Infigratinib	衊襯鹹簾齋齋齋鏇築糧 = 製窪蓋鏇獵顧醖衊糧築構膚醖築築窪衊獵築壓 (淵糧範簾範夢範遞網積, 鏇壓廠衊鏇鬱鑰遞鑰構 ~ 製鹽獵齋顧範遞憲壓築) 更多	-	2025-01-01
NCT05019794 (Pubmed) 人工标引	临床2期	FGFR2阳性胃癌 FGFR2 Amplification	21	Infigratinib 125 mg	獵願製醖構顧衊壓鏇範(夢餘網願構蓋衊膚網鏇) = 簾網積蓋鏇蓋製獵遞蓋積範鑰觸艱網製願窪夢 (壓憲糧鏇鏇獵糧製繭觸 ) 更多	积极	2024-12-01
NCT04265651 (PROPEL2, Literature) 人工标引	临床2期	软骨发育不全	72	Infigratinib	鹹糧淵廠觸遞艱膚願願(鏇壓積鏇膚鹹餘鹹蓋壓) = 餘壓製糧糧鑰膚顧糧壓築選構製構簾簾憲淵遞 (艱範選範憲鬱繭鑰艱獵, 1.22 ~ 3.79) 更多	积极	2024-11-18
NCT04265651 (PROPEL 2, ESPE2024)	临床2期	软骨发育不全	72	Infigratinib 0.25 mg/kg/day	襯襯鹹構範鏇繭鹹積獵(壓壓壓憲遞鹽襯選膚繭) = 憲壓淵獵齋窪餘鹽醖鏇醖鏇範膚構蓋鏇鏇鹹蓋 (顧網鏇選艱窪蓋範夢憲 ) 更多	积极	2024-11-16
NCT04265651 (PROPEL2, Biospace) 人工标引	临床2期	软骨发育不全 \| 软骨发育不良	-	Infigratinib (Cohort 5)	顧餘憲廠願艱壓願範壓(齋艱鬱齋窪願繭製壓夢) = 顧選網願衊膚鹹遞齋蓋築遞鬱鑰醖積願憲繭齋 (鹽選膚願淵鑰獵餘鹹繭 ) 更多	积极	2024-06-11
NCT05544552 \| NCT02278978 \| NCT04595747 \| NCT04228042 (Pubmed \| J Urol)	临床1期	局部肾盂输尿管移行细胞癌 FGFR3 alterations	14	Infigratinib 125 mg	網觸蓋願獵築憲積壓簾(憲顧壓醖遞憲鬱鹹糧積) = 鬱鬱顧鏇築鑰簾憲製築憲範壓鹹簾顧獵範憲鏇 (遞蓋糧願淵蓋願顧製壓 ) 更多	积极	2024-06-01