ABBISKO-B (02256) announced that its subsidiary, Shanghai Abbisko Biomedical Technology Co., Ltd., has received Rare Pediatric Disease (RPD) designation from the U.S. Food and Drug Administration (FDA) for its internally developed, highly selective, oral small-molecule FGFR2/3 inhibitor, ABSK061, for the treatment of achondroplasia (ACH). The FDA's RPD designation program was established to encourage the development of innovative therapies for serious rare diseases primarily affecting individuals under 18 years of age by providing financial incentives, thereby accelerating the drug approval process. Based on this RPD designation, if the drug is ultimately approved for this indication, Abbisko will be eligible for a Priority Review Voucher (PRV). This voucher can be used to expedite the FDA's review of another New Drug Application (NDA) or Biologics License Application (BLA), or it can be transferred to another NDA applicant, giving it significant strategic and commercial value. ACH is an autosomal dominant genetic rare disease that leads to severe growth and developmental disorders. Research indicates that the pathogenesis of ACH primarily stems from abnormal activation of FGFR3 caused by a gene mutation, which inhibits the normal ossification process of cartilage. Targeted inhibitors are expected to provide a more precise and effective treatment option for ACH patients. ABSK061 is a highly potent and selective small-molecule FGFR2/3 inhibitor developed by Abbisko. In preclinical studies, it has demonstrated significant target inhibition activity, favorable pharmacokinetic characteristics, and a promising safety profile. Its oral administration offers significant advantages in terms of convenience and treatment adherence, particularly for pediatric patients, positioning ABSK061 as a potentially valuable therapeutic candidate for children and adolescents with ACH. Currently, ABSK061 is undergoing a Phase II clinical trial for ACH patients aged 3 to 12 years.