SHANGHAI, March 31, 2026 /PRNewswire/ — 31 March 2026, Abbisko Therapeutics Co., Ltd. (“Abbisko Therapeutics” hereafter, HKEX code: 02256) announced 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). Irpagratinib is currently being evaluated in multiple clinical studies across different regions globally. The ODD granted by the EMA is expected to strongly support the product’s clinical development, regulatory filings, and commercialization in Europe.
Orphan Drug Designation is a key incentive established by the EMA to facilitate the development and authorisation of medicines for rare diseases. The ODD granted to irpagratinib not only reflects regulatory recognition of its potential clinical value and need, but also provides access to a range of incentives, including eligibility for protocol assistance (PA), regulatory fee reductions, and 10 years of market exclusivity after marketing authorisation—thereby providing a strong foundation for future registration and commercialization.
Primary liver cancer ranks as the third leading cause of cancer-related mortality worldwide, with HCC accounting for approximately 75%–85% of cases. Currently, immune checkpoint inhibitors (ICIs) combined with anti-angiogenic therapy have become the standard first-line treatment for advanced HCC. However, effective second-line and later-line options remain limited. Additionally, approximately 30% of HCC patients exhibit FGF19 overexpression, and this subgroup tends to derive limited benefit from first-line targeted immunotherapy combinations. As there are no approved therapies targeting the FGFR4/FGF19 signaling pathway, significant unmet medical needs remain for patients with FGF19 overexpression under the current standard of care.
Irpagratinib is a highly selective, orally administered small-molecule FGFR4 inhibitor independently developed by Abbisko Therapeutics. In prior clinical studies, irpagratinib has demonstrated favorable safety and tolerability along with antitumor activity both as a monotherapy and in combination regimens in patients with FGF19-overexpressing advanced HCC. Currently, 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. Among these, the pivotal registrational study of irpagratinib monotherapy administered the first patient dose in June 2025 and currently involves more than 50 clinical research centers across China, and is progressing smoothly.
In addition to the EMA ODD, 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 China’s National Medical Products Administration (NMPA). Leveraging the expedited review advantages provided by these regulatory designations, Abbisko Therapeutics will continue to advance the global clinical development and regulatory filings of irpagratinib, with the goal of bringing this innovative therapy to patients with hepatocellular carcinoma worldwide as early as possible and providing a new safe and effective precision treatment option for this challenging disease.
About Irpagratinib (ABSK-011)
Irpagratinib is a highly-selective FGFR4 small molecule inhibitor designed to target overexpression of the FGF19 signaling pathway. Several epidemiological studies indicate that approximately 30% of HCC patients worldwide exhibit FGF19 overexpression. Development of targeted therapies against FGFR4 represent an innovative and novel approach to the treatment of HCC.
To date, no FGFR4 inhibitor has been granted regulatory approval globally. According to Frost & Sullivan, irpagratinib is expected to become the first breakthrough treatment for the treatment of HCC patients with FGF19 overexpression.
In addition to monotherapy, Abbisko Therapeutics is exploring irpagratinib in combination with atezolizumab, an anti-PD-L1 antibody manufactured by F. Hoffmann-La Roche and Roche (China), in a Phase II study. At the the 2025 ESMO GI Congress, Abbisko presented clinical data showing that the combination of irpagratinib and atezolizumab achieved an objective response rate (ORR) exceeding 50% and a median progression-free survival (mPFS) of more than 7 months in FGF19-overexpressing HCC patients previously treated with immune checkpoint inhibitors.
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SOURCE Abbisko
