Biopharmaceutical company InnoCare (HKEX:09969) reported on Wednesday the receipt of the US Food and Drug Administration's (FDA) orphan drug designation (ODD) for its pan-FGFR inhibitor gunagratinib (ICP-192) for the treatment of cholangiocarcinoma, a cancer that begins in the bile duct.

ICP-192 is a highly selective pan-FGFR inhibitor targeting multiple solid tumors with FGFR gene aberrations. Several clinical studies are currently being conducted in China and the United States.

According to the latest clinical data presented at the 2021 American Society of Clinical Oncology (ASCO) annual meeting, about 30 patients had received gunagratinib as of February 2021. Gunagratinib performed well in safety and tolerance and the maximum tolerated dose (MTD) had not been reached.

Among the 12 patients with FGF/FGFR gene aberrations who have completed at least one tumor assessment, the overall response rate (ORR) was 33.3%, including one patient of cholangiocarcinoma with complete response (CR), three patients with partial response (PR) and seven patients having achieved stable disease (SD). The disease control rate (DCR) was 91.7%.

Upon marketing approval, Gunagratinib will qualify for seven-year market exclusivity. The FDA also rewards ODD drugs with comprehensive incentives including tax credit for clinical trial cost, waiver of marketing registration application fee, waiver or reduced annual product fee, and other benefits, such as clinical protocol assistance and qualification for expedited development programs.