Group 1 - The FDA has granted orphan drug designation (ODD) to ABSK061, a significant incentive policy aimed at supporting the development and evaluation of innovative therapies for rare diseases [1][3] - ABSK061 is a highly active and selective small molecule FGFR2/3 inhibitor developed by HeYue Pharmaceutical, showing strong target inhibition activity and promising safety characteristics in preclinical studies [1][2] - The drug is currently undergoing a Phase II clinical trial for children aged 3-12 with achondroplasia (ACH), with the first patient dosed in December 2025 and preliminary data expected in the second half of 2026 [1][2] Group 2 - Following the ODD designation, HeYue Pharmaceutical aims to accelerate the global clinical development and registration process for ABSK061, focusing on providing safe and effective innovative treatment options for ACH patients worldwide [2] - ABSK061 is positioned as a next-generation FGFR inhibitor, potentially offering a wider therapeutic window and improved clinical efficacy by reducing inhibition of FGFR1 while maintaining high activity against FGFR2/3 [2]
和誉-B：FGFR2/3抑制剂ABSK061 获FDA授予孤儿药资格认定 用于治疗软骨发育不全