Abbisko Therapeutics Secures Breakthrough Therapy Designation for Irpagratinib in HCC Treatment
Monday, May 26, 2025
Abbisko Therapeutics has received Breakthrough Therapy Designation (BTD) approval from China’s Centre for Drug Evaluation (CDE), part of the National Medical Products Administration (NMPA), for its novel small molecule FGFR4 inhibitor, irpagratinib (ABSK011).
Irpagratinib is an oral, highly selective FGFR4 inhibitor developed to block abnormal FGF19 signalling, which is observed in roughly 30% of HCC cases globally. FGFR4 inhibitors represent a new approach in the treatment landscape for liver cancer. Currently, no FGFR4-targeted therapy has been approved worldwide.
Irpagratinib is the first targeted therapy to use molecular biomarkers for the personalised treatment of HCC. The CDE grants BTD status to innovative drugs or modified therapies that aim to treat serious or life-threatening conditions, especially where there are limited or no existing treatment options. This recognition is based on early clinical data from Phase I trials showing potential clinical benefit.
The designation is for the treatment of hepatocellular carcinoma (HCC) in patients with FGF19 overexpression.
Patients with advanced or inoperable HCC often face limited treatment choices, particularly after therapies based on immune checkpoint inhibitors (ICIs) and multi-target kinase inhibitors (mTKIs). Among this group, those with FGF19 overexpression tend to have a poorer prognosis.
The BTD status is expected to accelerate regulatory review, potentially allowing quicker access to the therapy.
Source: abbisko.com
