Shanghai-based biotechnology company Abbisko Therapeutics Co., Ltd (HKG: 2256) has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for its novel small-molecule FGFR4 inhibitor, irpagratinib. The designation is in recognition of the drug’s potential in treating hepatocellular carcinoma (HCC).
Irpagratinib, an FGFR4 inhibitor originally discovered and developed by Abbisko, holds global intellectual property rights. It addresses the aberrant activation of FGF19-FGFR4 signaling, which is found in about 30% of HCC patients and is a key driver of tumor growth. The compound has shown high potency, excellent selectivity, and favorable physical-chemical properties in pre-clinical studies.
Clinical data has demonstrated that irpagratinib exhibits impressive initial efficacy and safety in patients with HCC who have FGF19 overexpression. Notably, the overall response rate (ORR) in the twice-daily dosing cohort reached 40.7%, outperforming several other FGFR4 inhibitors currently in clinical development globally.- Flcube.com
