FDA Fast Track Designation Highlights New Option in Advanced Liver Cancer

Patients with advanced hepatocellular carcinoma (HCC) often face limited treatment options after standard therapies stop working. In a news release, Abbisko Therapeutics announced that the U.S. Food and Drug Administration (FDA) granted fast track designation to its investigational therapy irpagratinib (ABSK-011) for patients with advanced HCC whose tumors overexpress fibroblast growth factor 19 (FGF19) and who have previously received immune checkpoint inhibitors (ICIs) and multi-targeted kinase inhibitors (mTKIs).

Fast track designation is intended to speed the development and review of therapies that address serious conditions with unmet medical needs. For patients with advanced liver cancer who have already exhausted standard treatments, this designation signals growing momentum behind a new, biomarker-driven approach focused on precision medicine.

Promising Clinical Results Support FDA Decision

The FDA decision was primarily based on positive results from a phase 1 clinical trial that evaluated irpagratinib as a single agent in patients with advanced HCC and FGF19 overexpression. These findings were presented at the 2024 European Society for Medical Oncology (ESMO) Annual Congress.

In this early-phase study, irpagratinib demonstrated meaningful antitumor activity in a population that had already experienced disease progression following ICIs and mTKIs. The objective response rate was 46.7%, meaning nearly half of treated patients experienced tumor shrinkage. The median progression-free survival was 5.5 months, suggesting that the treatment was able to delay disease progression for a notable period in this difficult-to-treat setting.

Equally important for patients, irpagratinib showed a favorable safety and tolerability profile. This is a critical consideration for individuals who may already be managing treatment-related side effects from prior therapies.

Why FGF19 Matters in Liver Cancer

Approximately 30% of patients with HCC have tumors that overexpress FGF19, a signaling protein involved in cell growth and survival. This subgroup of patients is known to have poorer outcomes after first-line targeted and immunotherapy combinations, underscoring the need for more personalized treatment strategies.

Ipragratinib is a highly selective, small-molecule inhibitor of fibroblast growth factor receptor 4 (FGFR4), a key component of the FGF19 signaling pathway. By specifically targeting this pathway, irpagratinib aims to interrupt cancer-promoting signals that drive tumor growth in FGF19-positive disease, rather than broadly affecting multiple pathways.

How the Studies Were Conducted

The phase 1 study supporting the FDA fast track designation enrolled patients with advanced or unresectable HCC whose tumors demonstrated FGF19 overexpression. All participants had previously received ICIs and mTKIs, reflecting a population with limited remaining treatment options.

In addition to monotherapy studies, Abbisko Therapeutics has also evaluated irpagratinib in combination with Tecentriq (atezolizumab), an anti–PD-L1 immunotherapy, in a phase 2 setting. Results from this combination approach were presented at the 2025 ESMO Gastrointestinal Cancers Congress.

The patient population across these studies included both treatment-naive patients and those who had previously received ICIs. All patients had confirmed HCC with FGF19 overexpression, reinforcing the precision-based approach of the therapy. This biomarker-selected design helps ensure that the treatment is tested in patients most likely to benefit.

Additional Findings and What Comes Next

In the combination study of irpagratinib plus Tecentriq, researchers reported objective response rates exceeding 50% and a median progression-free survival of more than seven months. Notably, no new safety signals were observed, suggesting that the combination was generally well tolerated.

Beyond the United States, irpagratinib has already received breakthrough therapy designation from China’s National Medical Products Administration, and a pivotal registration trial is currently underway across more than 30 research centers worldwide.

Experts have highlighted the potential of irpagratinib to become the first truly precision-targeted therapy for liver cancer. With FDA fast track designation now in place, Abbisko Therapeutics plans to accelerate global clinical development with the goal of bringing more precise and effective treatment options to patients with HCC.

References

1. Abbisko Therapeutics' FGFR4 Inhibitor Irpagratinib Granted FDA Fast Track Designation for Advanced HCC Patients with FGF19 Overexpression Previously Treated with ICIs and mTKIs Therapies, by Abbisko Therapeutics Co., Ltd. News release; Feb. 10, 2026.

Editor's note: This article is for informational purposes only and is not a substitute for professional medical advice, as your own experience will be unique. Use this article to guide discussions with your oncologist. Content was generated with AI and reviewed by a human editor.

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