This phase I/II trial evaluated the safety, activity, and efficacy of pemigatinib, a selective FGFR 1–3 inhibitor, in patients with refractory advanced malignancies, with or without FGF and FGFR gene alterations. Pemigatinib demonstrated a favorable safety profile and pharmacologic/clinical activity in a variety of solid tumor types, including cholangiocarcinoma, urothelial carcinoma, recurrent pilocytic astrocytoma and head and neck, pancreatic, gallbladder, uterine, and non–small cell lung cancer.
Pemigatinib was previously approved for advanced FGFR2-rearranged cholangiocarcinoma. These results suggest the potential benefits of pemigatinib in other cancers with FGFR rearrangements and/or mutations.
