FDA has approved the selective fibroblast growth factor receptor (FGFR) inhibitor Pemazyre (pemigatinib) to treat adults with relapsed or refractory myeloid/lymphoid neoplasms (MLNs) with FGFR1 rearrangement.
The drug, developed by Incyte (Nasdaq:INCY), first won approval for adults with previously treated, unresectable locally advanced or metastatic cholangiocarcinoma.
The latest indication, MLNs with FGFR1 rearrangement, covers an extremely rare and aggressive form of blood cancer.
A 2019 article in Frontiers in Oncology noted that there had been approximately 100 cases of the cancer reported to date.
“The approval of Pemazyre represents an important treatment advancement for people living with MLNs with FGFR1 rearrangement who currently have limited treatment options,” said Hervé Hoppenot, Incyte CEO, in a news release. “These are complex hematologic malignancies with a range of presentations, and this approval highlights Incyte’s continued leadership and commitment to advancing care for patients with rare blood cancers.”
FDA based its approval on data from the Phase 2 FIGHT-203 study, an open-label, single-arm trial.
The trial tested the drug in 28 patients with relapsed or refractory MLNs with FGFR1 rearrangement.
Recipients of Pemazyre in the study had a “high rate of complete response,” noted Dr. Srdan Verstovsek, principal investigator of the FIGHT-203 study. Verstovsek noted that the the results of the study were clinically meaningful, including “complete cytogenetic response in patients with chronic phase disease and the high rate of complete cytogenetic response in patients with blast phase disease.”
Existing first-line therapies lack such specific responses, Verstovsek concluded.
Patients in the FIGHT-203 study received 13.5 mg of pemigatinib daily for 21-day cycles.
Incyte notes that the drug is the only FGFR inhibitor that is FDA approved for multiple indications.
The drug has also been the focus of Phase 2 studies in adenosquamous carcinoma, bladder cancer, colorectal cancer; endometrial cancer, glioblastoma, lymphoma; non-small cell lung cancer, solid tumors and urogenital cancer.
Filed Under: Oncology