Abbisko Therapeutics has announced that its investigational drug irpagratinib (ABSK-011), a highly selective oral FGFR4 inhibitor, has been granted Orphan Drug Designation (ODD) by the European Medicines Agency for the treatment of Hepatocellular Carcinoma (HCC), the most common form of primary liver cancer.

The designation is expected to support the drug’s clinical development, regulatory approval process and eventual commercialisation across Europe. Orphan Drug Designation is a key regulatory incentive aimed at encouraging the development of treatments for rare diseases by offering benefits such as protocol assistance, reduced regulatory fees and up to 10 years of market exclusivity following approval.

Hepatocellular carcinoma accounts for nearly 75–85 percent of primary liver cancer cases and remains one of the leading causes of cancer-related deaths globally. While first-line treatment for advanced HCC has evolved to include immune checkpoint inhibitors combined with anti-angiogenic therapies, treatment options for second-line and later-stage patients remain limited.

A significant unmet need exists among patients with FGF19 overexpression, estimated to represent around 30 percent of HCC cases. This subgroup typically shows limited response to current therapies, and there are no approved treatments specifically targeting the FGFR4/FGF19 signalling pathway.

Irpagratinib, developed independently by Abbisko Therapeutics, has shown encouraging results in earlier clinical studies, demonstrating favourable safety, tolerability and anti-tumour activity both as a standalone therapy and in combination with other treatments. The drug is currently being evaluated in multiple global clinical trials, including a pivotal registrational study that began dosing patients in June 2025 and spans more than 50 research centres in China.

In addition to the EMA designation, irpagratinib has also received Orphan Drug Designation 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.

Abbisko Therapeutics stated that it will continue to leverage these regulatory advantages to accelerate the global development and approval of irpagratinib, with the aim of providing a targeted and effective treatment option for patients with advanced liver cancer.