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SHANGHAI, March 31, 2026 /PRNewswire/ — On 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.
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Reference |
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[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.
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