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.