Partial response was confirmed in one patient with FGFR1-mutant esophageal carcinoma and one with FGFR2-mutant cholagiocarcinoma. Pemigatinib had similar PK/PD characteristics to Western population and demonstrated an acceptable safety profile and potential anti-cancer benefit in Chinese patients with FGF/FGFR1-3 altered, advanced, solid tumor.

Patients (≥20 years old) self-administered oral pemigatinib 9, 13.5, or 18 mg QD on intermittent dosing (Part 1) or 13.5 mg QD intermittent or continuous dosing (Part 2). A dosing cycle was 21 days (2 weeks on/1 week off or 21 continuous days)....Clinical responses were observed with five PRs across different tumor types including cholangiocarcinoma with FGFR2 alterations.

FGF/FGFR genomic alterations (GAs)... FGFR2 fusions/rearrangement (RE)..Pts with advanced disease…assigned to cohort A (FGFR2 RE), B (other FGF/FGFR GA), or C (no FGF/FGFR GA). Pts received pemigatinib 13.5 mg QD.... In cohort A, ORR (95% confidence interval) was 40.6% (23.7-59.4) with 1 complete response (Figure); mDOR was 7.5 mo (5.5-7.5), DCR was 87.5% (71.0-96.5), mPFS was 6.9 mo (4.8-9.1), and mOS was 14.7 mo (11.7-not reached).