Pemigatinib, A Selective FGFR Inhibitor, Safely Treats Hematologic Malignancies
Data from the phase 1/2, FIGHT-101 trial confirm pemigatinib is safe and tolerable, and demonstrated pharmacologic clinical activity in patients with or without fibroblast growth factor receptor (FGFR) mutant hematologic malignancies.
A total of 128 patients received pemigatinib 1-20 mg once daily intermittently (70 patients had a 2-week on and 1-week off regimen versus 58 patients who had a continuous regimen).
“Overall, 12 partial responses (PR) were achieved, most commonly in cholangiocarcinoma as well as in a broad spectrum of tumors including head and neck, pancreatic, gallbladder, uterine, urothelial carcinoma, recurrent pilocytic astrocytoma, and non-small-cell lung cancer (NSCLC),” explained Vivek Subbiah, MD, MD Anderson Cancer Center, Houston, Texas, and co-researchers.
Results found the median duration of response (DOR) was 7.3 months (95% CI, 3.3-14.5). Further, the overall response rate (ORR) was highest for patients with FGFR fusions and rearrangements (95% CI, 8.7-49.1), followed by those with FGFR mutations (95% CI, 5.0-53.8).
The most common treatment-emergent adverse event (TEAE) was hyperphosphatemia (75%; grade 3 or higher, 2.3%). The most common grade 3 or higher TEAE was fatigue (10.2%). Dose interruption, dose reduction, and TEAE-related treatment discontinuation occurred in 66 (51.6%), 14 (10.9%) and 13 (10.2%) patients, respectively.
“Pemigatinib was associated with a manageable safety profile and pharmacodynamic and clinical activity, with responses seen across tumors and driven by FGFR fusions, rearrangements, and mutations. These results prompted a registrational study in cholangiocarcinoma and phase 2/3 trials in multiple tumor types demonstrating the benefit of precision therapy even in early phase trials,” concluded Dr Subbiah, et al. –Alexa Stoia
