Incyte's Pemazyre becomes FDA's first and only approved unresectable, advanced cholangiocarcinoma treatment

The FDA has approved the United States’ first and only treatment for previously treated, unresectable locally advanced or metastatic cholangiocarcinoma, a rare form of bile duct cancer, with the marketing authorisation of Incyte’s Pemazyre (pemigatinib).

The kinase inhibitor is approved specifically in adult cholangiocarcinoma patients with a fibroblast growth factor receptor 2 (FGFR2) fusion or other rearrangement as detected by an FDA-approved test.

Incyte scored the approval via the FDA’s Breakthrough Therapy and Orphan Drug designation pathways, sealing the decision after the drug demonstrated a 36% overall response as a monotherapy, meeting its main goal in clinical trials. Furthermore, the drug also met its secondary endpoint, displaying duration of response of 9.1 months.

Incyte CEO Hervé Hoppenot commented on the decision: “Our research into FGFR2 fusions or rearrangements in cholangiocarcinoma and the development of Pemazyre as the first targeted treatment option demonstrates our commitment to translating scientific discovery into solutions that can positively impact patients’ lives. We’re proud to bring Pemazyre to patients and will make this new treatment available immediately.”

Matt Fellows