Dr. Verstovsek on Pemigatinib in FGFR1-Rearranged Myeloid Neoplasms

Srdan Verstovsek, MD, PhD, United Energy Resources, Inc., professor of medicine, hematologist-oncologist, Department of Leukemia, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center, discusses the FDA approval of pemigatinib (Pemazyre), an FGFR1 inhibitor, in patients with myeloid neoplasms with an FGFR1 rearrangement.

On August 26th, 2022, the FDA approved pemigatinib in patients with relapsed/refractory myeloid neoplasms with an FGFR1 rearrangement. The regulatory decision was based on findings from the phase 2 FIGHT-203 trial (NCT03011372), in which the agent demonstrated a complete cytogenic response rate of 79%, with 78% of patients with chronic phase in the marrow achieving a complete response.

Myeloid neoplasms with FGFR1 rearrangement are a rare condition, and the exact number of patients with this disease is unknown, but more of this population will hopefully be identified now that pemigatinib is a treatment option, Verstovsek says. Pemigatinib has transformed the treatment landscape in this population, where previous therapies, such as chemotherapy and supportive care, did not provide meaningful benefit, Verstovsek explains.

Now, with an agent that targets FGFR1, the driver of these myeloid neoplasms, this disease can be eliminated in the majority of patients, Verstovsek notes. These unprecedented findings pave the way for a better understanding of this disease, so patients can live longer with a higher quality of life, Verstovsek concludes.