Chinese biopharmaceutical company Abbisko Therapeutics Co Ltd (HKEX:02256) announced on Tuesday that its independently developed, highly selective, oral small-molecule FGFR4 inhibitor irpagratinib (ABSK-011) has been granted Orphan Drug Designation (ODD) by the European Medicines Agency (EMA) for the treatment of hepatocellular carcinoma (HCC).

The ODD granted by the EMA is expected to strongly support the product's clinical development, regulatory filings, and commercialisation in Europe.

Abisko says that irpagratinib is a highly selective, orally administered small-molecule FGFR4 inhibitor that it has developed independently. In prior clinical studies, irpagratinib has demonstrated favourable safety and tolerability along with anti-tumour activity both as a monotherapy and in combination regimens in patients with FGF19-overexpressing advanced HCC.

Multiple clinical trials of irpagratinib are underway globally in patients with FGF19-overexpressing advanced HCC, including studies evaluating irpagratinib in combination with different targeted immunotherapies in the first-line setting, as well as monotherapy in the second-line and later-line settings.