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.

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.