25 April 2024, Shanghai – Abbisko Therapeutics Co., Ltd. (referred to as “Abbisko Therapeutics” hereafter) announced that its innovative FGFR4 inhibitor, irpagratinib, has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA) for the treatment of Hepatocellular Carcinoma (HCC).

The FDA grants ODD to investigational drugs and biologics that are intended for the treatment of rare diseases that affect fewer than 200,000 people in the U.S.. Receiving ODD provides drug developers with various benefits designed to support the development of novel drugs and biologics, including marketing exclusivity for seven years upon FDA approval, tax credits for qualified clinical trial expenditures, and exemptions from FDA application and user fees.

Irpagratinib is a highly selective FGFR4 small molecule inhibitor intended for the treatment of advanced solid tumors that present with abnormalities in the FGF19/FGFR4 signaling pathway (e.g., ligand FGF19 amplification/overexpression, FGFR4 mutation/amplification/fusion), including advanced HCC, cholangiocarcinoma, breast cancer, among others. The FGFR4 signaling pathway is a recognized and promising target for the treatment of HCC. Clinical data with irpagratinib have demonstrated improved potency and anti-tumor efficacy compared to competitors, among other favorable therapeutic properties. 

At the 2023 European Society of Medical Oncology (ESMO) conference, Abbisko Therapeutics presented updated clinical data for irpagratinib demonstrating a 40.7% objective response rate (ORR) in patients with previously treated hepatocellular carcinoma with aberrant FGF19 expression. According to Frost & Sullivan, irpagratinib is a novel and potentially first-in-class FGFR4 inhibitor for the treatment of HCC with FGF19/FGFR4 over-amplification.

Based on epidemiological records from the International Agency for Research on Cancer (IARC), a specialized cancer agency of the World Health Organization (WHO), there were 42,284 new cases of primary liver and 31,078 attributable deaths in the United States in 2020, with rates highest among American Indian or Alaska Native individuals [1]. HCC accounts for 85%-90% of primary liver cancers. The ratio of incidence rate to fatality rate is high, which seriously threatens human life and health.

Reference:

1. World Health Organisation: Globocan 2020 – United States of America Factsheet. Available at: https://gco.iarc.fr/today/data/factsheets/populations/840-united-states-of-america-fact-sheets.pdf