ABBISKO-B(02256): Abbisko Therapeutics' Lavengratinib (ABSK061) Granted Orphan Drug Designation by EMA for the Treatment of Achondroplasia

ABBISKO-B(02256) announced that on October 21, 2026, Shanghai Abbisko Therapeutics Co., Ltd. (Abbisko Therapeutics) announced that its independently developed oral selective small molecule FGFR2/3 inhibitor lavengratinib (ABSK061) for the treatment of achondroplasia (Achondroplasia, "ACH") has been granted Orphan Drug Designation ("OD") by the European Medicines Agency ("EMA"). ACH is a rare growth and developmental disorder that may be life-threatening and lead to disability, and currently there are limited treatment options available globally. The granting of EMA Orphan Drug Designation this time 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 leading international regulatory authorities. Previously, lavengratinib for the ACH indication had been granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation ("RPDD") by the U.S. Food and Drug Administration (FDA), and its IND application has been approved by the FDA. Lavengratinib has successively obtained multiple designations granted by U.S. and European regulatory authorities, providing strong momentum for it to address the huge unmet clinical needs in the ACH field and advance the global development process. ACH is an autosomal dominant genetic disease mainly caused by FGFR3 gene mutations, 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 uses Abbisko Therapeutics' unique oral microtablet formulation technology, with each tablet less than 3 mm in diameter, and can be conveniently taken with food or beverages, which is expected to improve the convenience and compliance of long-term medication in pediatric patients with ACH. Currently, Abbisko Therapeutics is actively advancing a Phase II, multicenter, open-label dose-escalation study (ABSK061-202) of lavengratinib in the treatment of children with ACH aged 3 to 12 years. Preliminary data recently announced showed that lavengratinib demonstrated positive growth-promoting signals, with overall safety and tolerability being good. 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 patients with ACH worldwide.