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Abbisko Therapeutics’ Irpagratinib Receives EMA Orphan Drug Status for Liver Cancer
Wednesday, April 01, 2026
Abbisko Therapeutics has received orphan drug designation from the European Medicines Agency for its FGFR4 inhibitor irpagratinib, aimed at treating hepatocellular carcinoma (HCC).
Irpagratinib is an oral, small-molecule therapy designed to selectively target the FGFR4 pathway. The designation is expected to support its clinical development and regulatory progress in Europe, while also offering incentives such as fee reductions, scientific guidance, and a period of market exclusivity after approval.
Primary liver cancer is one of the leading causes of cancer-related deaths worldwide, with HCC accounting for the majority of cases. Although first-line treatment typically involves immune checkpoint inhibitors combined with anti-angiogenic therapies, options for patients who progress beyond this stage remain limited. Around 30% of HCC patients show overexpression of FGF19, a group that often responds poorly to existing therapies. There are currently no approved treatments specifically targeting the FGFR4/FGF19 pathway.
Irpagratinib has shown encouraging safety and anti-tumour activity in earlier studies, both as a single treatment and in combination with other therapies in patients with advanced HCC linked to FGF19 overexpression. Ongoing global trials are evaluating its use in both first-line combination settings and later-line monotherapy. A key registrational study began dosing patients in June 2025 and is being conducted across multiple research centres in China.
The therapy has already received orphan drug and fast track status from the U.S. Food and Drug Administration, as well as breakthrough therapy designation from China’s National Medical Products Administration. These designations are expected to accelerate its overall development and review process.
Irpagratinib is also being studied in combination with atezolizumab, an anti-PD-L1 therapy developed by F. Hoffmann-La Roche. Clinical data presented at a major oncology congress in 2025 indicated promising response rates and progression-free survival in previously treated patients.
No FGFR4-targeting therapy has yet been approved globally, positioning irpagratinib as a potential first-in-class treatment for patients with this specific form of liver cancer.
Source: abbisko.com
