ABBISKO-B(02256): FGFR2/3 inhibitor ABSK061 has been granted orphan drug designation by the FDA for the treatment of achondroplasia.

ABBISKO-B(02256) announces that its subsidiary Shanghai Heyu Biomedical Technology Co., Ltd. (Heyu Medicine) has obtained orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for its independently developed highly selective oral small molecule FGFR2/3 inhibitor ABSK061, which is used to treat achondroplasia (ACH). This milestone comes after ABSK061 recently received rare pediatric disease (RPD) designation from the FDA, marking another key milestone in its global development process. ODD is expected to provide strong support for the clinical development, registration, and commercialization process of the product in the United States. Orphan drug designation is an important incentive policy established by the FDA to support the development and evaluation of innovative therapies for rare diseases. The ODD designation for ABSK061 not only reflects the FDA's recognition of its potential clinical value and demand, but also brings several potential benefits, including tax credits for qualified clinical trial expenses, exemption from NDA/BLA fees, and potential eligibility for seven years of market exclusivity after approval. ACH is a rare autosomal genetic disease that causes severe growth and developmental disorders. Research has shown that the pathogenesis of ACH is mainly due to abnormal activation of the FGFR3 gene mutation, which inhibits the normal ossification process of cartilage. Targeted inhibitors are expected to provide more precise and effective treatment options for ACH patients. ABSK061 is a highly active, highly selective small molecule FGFR2/3 inhibitor developed independently by Heyu Medicine. Preclinical studies have shown that it has potent target inhibitory activity, good pharmacokinetic characteristics, and promising safety features. The oral administration route is expected to significantly improve the convenience and compliance of treatment, especially for pediatric patients, highlighting its potential as a treatment option for children and adolescents with ACH. ABSK061 is currently being evaluated in a Phase II clinical trial for ACH children aged 3-12. The study dosed the first patient in China in December 2025, and preliminary data is expected to be released in the second half of 2026. After obtaining RPD and ODD designation from the FDA, Heyu Medicine will continue to accelerate the global clinical development and registration process of ABSK061, aiming to provide safe and effective innovative treatment options for ACH patients worldwide. ABSK061 is a novel oral small molecule FGFR2/3 inhibitor with good bioavailability, high activity, and high selectivity developed and fully owned by Heyu Medicine. First-generation pan-FGFR inhibitors have shown clinical efficacy in a variety of tumors harboring FGFR2/3 mutations and have gradually received regulatory approvals globally. However, the therapeutic window and clinical efficacy of pan-FGFR inhibitors are limited by the related side effects of inhibiting FGFR1. By reducing inhibition of FGFR1 and maintaining high activity on FGFR2/3, ABSK061, as a new generation FGFR inhibitor, is expected to achieve a wider therapeutic window and improve clinical efficacy. ABSK061 has been granted RPD and ODD designation by the FDA for the treatment of achondroplasia, and the Phase II clinical trial is currently ongoing.