Abbisko-B (02256) has issued an announcement. On October 21, 2026, Shanghai Abbisko Biopharmaceutical Technology Co., Ltd. (Abbisko Therapeutics) announced that its independently developed oral selective small molecule FGFR2/3 inhibitor lavengratinib (ABSK061) for the treatment of achondroplasia (ACH) has been granted Orphan Drug Designation (OD) by the European Medicines Agency (EMA).

ACH is a rare growth and developmental disorder that can be life-threatening and lead to disability. Currently, treatment options available globally are limited. The granting of this EMA orphan drug designation marks another important milestone in the international development process of lavengratinib, and further demonstrates that its innovative potential and clinical value have been recognized by a leading international regulatory authority.

Previously, lavengratinib for the treatment of ACH had already received Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) from the U.S. Food and Drug Administration (FDA), and its IND application has been approved by the FDA. The successive qualifications granted to lavengratinib by both U.S. and European regulatory authorities provide strong momentum for addressing the significant unmet clinical needs in the ACH field and advancing its global development process.

ACH is an autosomal dominant genetic disease mainly caused by mutations in the FGFR3 gene, which can lead to severe systemic skeletal development and ossification disorders as well as related complications. Lavengratinib is a novel oral, highly active, selective small molecule FGFR2 and FGFR3 inhibitor independently developed by Abbisko Therapeutics. It utilizes Abbisko's unique oral mini-tablet formulation technology, with each tablet less than 3 mm in diameter, and can be conveniently taken with food or drink, which is expected to improve the convenience and compliance of long-term medication for pediatric ACH patients.

Currently, Abbisko Therapeutics is actively advancing a Phase II, multicenter, open-label dose-escalation study (ABSK061-202) of lavengratinib for the treatment of ACH in children aged 3 to 12 years. Recently published preliminary data show that lavengratinib demonstrates positive growth-promoting signals, with an overall favorable safety and tolerability profile. Abbisko Therapeutics will continue to advance the global clinical development of lavengratinib for the treatment of ACH, further evaluate its safety and efficacy, and explore its potential to provide a new treatment option for pediatric ACH patients worldwide.