和誉医药FGFR2/3高选择性抑制剂ABSK061治疗软骨发育不全的临床申请获CDE批准

此次获批的研究是“一项评价ABSK061在软骨发育不全儿童中的安全性、耐受性、药代动力学和有效性的多中心、非随机、开放性Ⅰ/Ⅱ期研究“。

软骨发育不全（ACH）是导致非匀称性身材矮小的罕见疾病，是人类最常见的侏儒症。根据文献显示，ACH的全球出生患病率约为 4.6/100,000人，估计影响250,000-385,000 人^[1][2] ，并且ACH 人群的平均预期寿命比一般人群更短，对生活质量也会有负面影响^[3]。目前对 ACH 的治疗暂无特效药物或方法。

关于ABSK061

ABSK061是和誉医药独立自主研发并拥有全球知识产权的一款新一代口服、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。

Abbisko Therapeutics Announces IND Clearance from the CDE for ABSK061, a Highly Selective FGFR2/3 Inhibitor, for the Treatment of Achondroplasia

26 March 2025, Shanghai—Abbisko Therapeutics Co., Ltd. (“Abbisko” hereafter) today announced that the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) has cleared the IND application for ABSK061, a highly-selective small molecule inhibitor of FGFR2/3, for the treatment of children with Achondroplasia (“ACH”).

A clinical study of ACH will be conducted under the title “A Phase 1/2, Multicenter, non-randomized, Open-Label Study of ABSK061 to Evaluate Safety, Tolerability, Pharmacokinetics, and Efficacy in Children with ACH”.

Achondroplasia (ACH) is a rare disorder that leads to disproportionate short stature and is the most common form of dwarfism in humans. According to literature, ACH has an estimated incidence rate of approximately 4.6/100,000 live births, affecting over 250,000 to 385,000 individuals worldwide^[1][2]. Patients with ACH are frequently noted to have shorter life expectancy compared to that of the general population^[3], in addition to negative impacts on quality of life. Currently, there are no specific medications or methods for the etiological treatment of ACH in China.

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.

参考文献/References：

1. Stender M, et al. Comprehensive literature review on    the prevalence of comorbid conditions in patients with achondroplasia. Bone.     2022;162:116472.

2. Savarirayan R, et al. International consensus statement on the diagnosis, multidisciplinary management and lifelong care of individuals with achondroplasia[J]. Nat Rev Endocrinol, 2022, 18(3): 173‑189.

3. Wynn J, King TM, Gambello MJ, Waller DK, Hecht JT. Mortality in achondroplasia study: a 42-year follow-up. Am J Med Genet A. 2007;143A(21):2502-2511.