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21 October 2026, Abbisko Therapeutics Co., Ltd. ("Abbisko Therapeutics" hereafter, HKEX code: 02256.HK) today announced that its proprietary selective orally available small-molecule FGFR2/3 inhibitor, lavengratinib (ABSK061), has been granted Orphan Designation (OD) by the European Medicines Agency (EMA) for the treatment of achondroplasia (ACH).

Achondroplasia is a rare, potentially life-threatening and debilitating growth disorder with limited treatment options currently available worldwide. The granting of the EMA Orphan Designation marks another important milestone in the international development of lavengratinib and further underscores its innovative potential and clinical value recognized by a leading international regulatory authority. Previously, lavengratinib for the treatment of ACH received both 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 cleared by the FDA. The successive designations granted by regulatory authorities in the U.S. and Europe provide robust momentum for lavengratinib to address the substantial unmet medical needs in ACH and advance its global development.

Achondroplasia is an autosomal dominant disorder primarily caused by mutations in the fibroblast growth factor receptor 3 (FGFR3) gene, which can lead to severe systemic skeletal developmental and ossification abnormalities and associated complications. Lavengratinib is a novel, orally available, highly potent and selective small-molecule inhibitor of FGFR2 and FGFR3 independently discovered by Abbisko Therapeutics. It leverages the company's unique oral mini-tablet formulation technology, with each tablet being less than 3 mm in diameter and allowing convenient administration with food and drink, potentially improving the convenience and adherence of long-term treatment for children with ACH.

Abbisko Therapeutics is currently advancing a Phase II, multicenter, open-label, dose-escalation study (ABSK061-202) of lavengratinib in children aged 3-12 years with ACH. Recent preliminary data demonstrated positive signals for growth promotion, with favorable overall safety and tolerability.

Abbisko Therapeutics will continue to advance the global clinical development of lavengratinib for ACH, further evaluate its safety and efficacy, and explore its potential to provide a new treatment option for children with ACH worldwide.

About Lavengratinib (ABSK061)

Lavengratinib is a novel, orally bioavailable, highly potent and selective small molecule inhibitor of FGFR2 and FGFR3 independently discovered and wholly-owned by Abbisko Therapeutics. It is the first FGFR2/3 inhibitor to enter clinical trials globally. First-generation pan-FGFR inhibitors demonstrated clinical efficacy in multiple tumors carrying FGFR2/3 variants and have steadily gained regulatory approval globally. However, the therapeutic window of pan-FGFRs and their clinical efficacy have been limited by side effects associated with FGFR1 inhibition. By reducing FGFR1 activity while maintaining potency against FGFR2 and FGFR3, lavengratinib is expected to achieve a wider therapeutic window with improved clinical efficacy as a new-generation of FGFR inhibitors. lavengratinib for the treatment of achondroplasia has received both Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA), as well as ODD by the European Medicines Agency (EMA), and its Phase II clinical trial is currently ongoing.

About Abbisko Therapeutics

Founded in 2016, Abbisko Therapeutics Co., Ltd. (HKEX: 02256) is a pharmaceutical company committed to the research, discovery, and development of innovative and differentiated medicines designed to address unmet medical needs in China and globally.

Adhering to international concepts and standards for drug development, Abbisko primarily centers on small molecules, emphasizing precision oncology and immuno-oncology, with a growing expansion into both non-oncology therapeutic areas and other modalities. Its self-developed Class 1 innovative drug, pimicotinib, has received approval from the China NMPA, while dozens of other candidates are currently in clinical development stages.

Abbisko is committed to creating first-in-class and best-in-class innovative drugs. Through an unwavering pursuit of scientific innovation, the company strives to provide transformative therapies with breakthrough efficacy for patients worldwide.

Please visit www.abbisko.com for more information.