和誉医药FGFR2/3抑制剂ABSK061治疗儿童软骨发育不全新药临床试验获FDA批准

2026年3月30日，上海和誉生物医药科技有限公司（以下简称“和誉医药”，港交所代码：02256）宣布，其自主研发的高选择性小分子FGFR2/3抑制剂ABSK061治疗软骨发育不全（ACH）儿童患者的新药临床试验申请（IND）已获得美国食品药品监督管理局（FDA）批准。加之近期ABSK061相继获得FDA授予的罕见儿科疾病资格（RPDD）与孤儿药资格（ODD），将助力和誉医药加快推进其海外临床开发进程。

ABSK061目前正在开展用于治疗ACH的II期临床试验，该研究已于2025年12月在中国完成首例患者给药，预计将于2026年下半年公布初步数据。作为ABSK061全球开发战略的重要组成部分，和誉医药计划在美国招募患者参与此项II期研究，以进一步评估ABSK061在治疗ACH患者中的安全性、耐受性及疗效。

ACH是导致儿童严重生长发育障碍的常染色体遗传性罕见病，目前缺乏有效治疗方法。研究表明，ACH的发病机制主要源于成纤维细胞生长因子受体3（FGFR3）基因突变引起的FGFR3异常激活，从而抑制软骨的正常骨化过程¹。因此，针对ACH的分子病因开发靶向抑制剂有望为ACH患者带来精准治疗新希望。

ABSK061是和誉医药自主研发的一款高活性、高选择性小分子FGFR2/3抑制剂，在临床前研究中已显示出显著的靶点抑制活性、良好的药代动力学特征以及安全性优势。其口服给药方式有望极大提升患者，尤其是儿童患者的用药便利性和治疗依从性，在儿童及青少年ACH患者的治疗中展现出良好的应用潜力。

参考文献

1.Savarirayan R, et al. International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022 Mar;18(3):173-189.

关于ABSK061

ABSK061是和誉医药自主研发并拥有全球知识产权的新一代口服、高活性、高选择性小分子FGFR2/3抑制剂，也是全球范围内第一款进入临床的FGFR2/3选择性抑制剂。首代泛FGFR抑制剂已在针对多种携带FGFR2/3变异的肿瘤中展现出临床疗效并在全球范围内逐步获批上市，但安全窗及药效均受限于FGFR1抑制相关副作用。通过降低对FGFR1的抑制以及保持对FGFR2/3的高活性，ABSK061作为第二代FGFR抑制剂有望在临床上取得更好的安全窗及疗效。目前，ABSK061治疗软骨发育不全适应症已获得美国食品药品管理局（FDA）授予的罕见儿科疾病资格（RPDD）和孤儿药资格（ODD），II期临床研究正在顺利推进中。

Abbisko Therapeutics' FGFR2/3 Inhibitor ABSK061 Receives FDA IND Clearance for Achondroplasia in Children

30 March, 2026, Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) announced that the U.S. Food and Drug Administration has cleared the Investigational New Drug (IND) application for ABSK061, a highly selective small-molecule FGFR2/3 inhibitor, for the treatment of children with achondroplasia (ACH). Coupled with the recent Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) granted by the FDA, this will help Abbisko accelerate the overseas clinical development process for ABSK061.

ABSK061 is currently being evaluated in a Phase II clinical trial for ACH, and in December 2025, the study dosed its first patient in China. Preliminary data are expected to be reported in the second half of 2026. As an important part in the global development strategy for ABSK061, Abbisko plans to enroll US patients into the Phase II study to further evaluate safety, tolerability, and efficacy of ABSK061 for the treatment of ACH.

Achondroplasia is a rare autosomal genetic disorder that causes severe growth and developmental impairments. Research has shown that the pathogenesis of ACH is driven by aberrant activation of the fibroblast Growth Factor Receptor 3 (FGFR3) caused by FGFR3 gene mutations, which suppress normal endochondral ossification ¹. Targeted inhibitors offer the potential to deliver more precise and effective treatment options for ACH patients.

ABSK061, independently developed by Abbisko Therapeutics, is a highly potent and selective small-molecule FGFR2/3 inhibitor. It has demonstrated robust target inhibitory activity, favorable pharmacokinetic properties, and a promising safety profile in preclinical studies. Its oral administration offers significant advantages in terms of convenience and treatment compliance-particularly for pediatric patients—and positions ABSK061 as a potentially valuable therapeutic candidate for children and adolescents with ACH.

Reference

1. Savarirayan R, et al. International Consensus Statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia. Nat Rev Endocrinol. 2022 Mar;18(3):173-189.

About ABSK061

ABSK061 is a novel, orally bioavailable, highly potent and selective small molecule inhibitor of FGFR2 and FGFR3 independently discovered and wholly-owned by Abbisko Therapeutics. It is the first FGFR2/3 inhibitor to enter clinical trials globally. First-generation pan-FGFR inhibitors demonstrated clinical efficacy in multiple tumors carrying FGFR2/3 variants and have steadily gained regulatory approval globally. However, the therapeutic window of pan-FGFRs and their clinical efficacy have been limited by side effects associated with FGFR1 inhibition. By reducing FGFR1 activity while maintaining potency against FGFR2 and FGFR3, ABSK061 is expected to achieve a wider therapeutic window with improved clinical efficacy as a new-generation of FGFR inhibitors. ABSK061 for the treatment of achondroplasia has received both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), and its Phase II clinical trial is currently ongoing.

About Abbisko Therapeutics

Founded in April 2016, Abbisko Therapeutics Co., Ltd. (HKEX: 02256.HK), is an oncology-focused biopharmaceutical company based in Shanghai that is dedicated to the discovery and development of innovative medicines to treat unmet medical needs in China and globally. The Company was established by a group of seasoned drug hunters with rich research & development and managerial expertise from top multinational pharmaceutical companies. Since its founding, Abbisko Therapeutics has built an extensive pipeline of innovative programs focused on precision oncology and immuno-oncology.

Please visit www.abbisko.com for more information.

关于和誉

和誉医药（香港联交所代码：02256）成立于2016年，是一家立足中国，着眼全球的创新药研发公司。公司的创始人和管理团队拥有多年顶尖跨国药企的研发和管理经验，并参与了多个临床及上市新药的研发。和誉医药专注于肿瘤新药研发，以小分子肿瘤精准治疗和小分子肿瘤免疫治疗药物为核心，着眼病患及医药市场的需求，秉承国际新药开发的理念和标准，致力于开发新颖及高潜力药物靶点的潜在first-in-class或best-in-class创新药物，用于改善中国及全球病人的生活质量。自成立以来，和誉医药已经建立了丰富的创新产品管线，涵盖肿瘤精准治疗领域以及肿瘤免疫治疗领域。

更多信息，欢迎访问 www.abbisko.com。