ABBISKO-B(02256):FGFR4 inhibitor infigratinib granted orphan drug designation by EMA for the treatment of hepatocellular carcinoma.

ABBISKO-B(02256) announced that its subsidiary Shanghai Heyu Biomedical Technology Co., Ltd. (Heyu Medicine) has obtained orphan drug designation (ODD) from the European Medicines Agency (EMA) for its independently developed highly selective oral small molecule FGFR4 inhibitor, Irpagratinib (ABSK-011), for the treatment of hepatocellular carcinoma (HCC). Currently, Irpagratinib is undergoing clinical research in multiple locations worldwide. The ODD granted by EMA will provide strong support for the clinical development, registration, and commercialization process of this product in Europe. Irpagratinib is a highly selective, oral small molecule FGFR4 inhibitor developed independently by Heyu Medicine. In previous clinical studies, Irpagratinib as monotherapy and in combination regimens demonstrated good safety, tolerability, and anti-tumor activity in patients with advanced HCC overexpressing FGF19. Currently, multiple clinical trials targeting advanced HCC patients overexpressing FGF19 are being conducted globally with Irpagratinib, including combinations with different targeted immunotherapies for first-line treatment of advanced HCC, as well as monotherapy for second- and later-line treatments. The first patient in the key registration clinical study of Irpagratinib as monotherapy completed dosing in June 2025, and the study is progressing smoothly with over 50 research centers nationwide. In addition to obtaining ODD from EMA, Irpagratinib has previously been granted ODD and Fast Track Designation (FTD) by the U.S. Food and Drug Administration (FDA), as well as Breakthrough Therapy Designation (BTD) by the China National Medical Products Administration (NMPA). Leveraging the expedited review advantages brought by these regulatory designations, Heyu Medicine will continue to advance the global clinical development and registration process of Irpagratinib, striving to bring this innovative treatment to HCC patients worldwide as early as possible and provide a safe and effective new option for precise treatment of this difficult-to-treat disease.