WASHINGTON — Pemigatinib (Pemazyre) is now FDA-approved as the first targeted treatment for adults with previously treated, locally advanced or metastatic cholangiocarcinoma that has a fusion or rearrangement of the fibroblast growth factor receptor 2 gene (FGFR2).
According to the FDA, the majority of patients with this rare cancer of the bile ducts have advanced disease upon diagnosis, and surgery is no longer an option. “For these patients, until today, there have been no FDA-approved therapies; a combination of chemotherapy drugs has been the standard initial treatment,” the FDA explained.
With this new approval, pemigatinib — which received Priority Review, Breakthrough Therapy, and Orphan Drug designations — will be available to the 9%-14% of patients with cholangiocarcinoma whose tumors have FGFR2 fusions.
“With [pemigatinib], we considered the observed efficacy results to be clinically meaningful and the overall risk to benefit assessment for patients with tumors harboring FGFR2 gene fusions and other rearrangements to be favorable, particularly when we considered that these patients have no other good options following first line treatment with chemotherapy,” said Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, in a statement.
This approval was based on results from a trial that analyzed pemigatinib’s efficacy in 107 patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who had received prior chemotherapy treatment. “During the clinical trial, patients received [pemigatinib] once a day for 14 consecutive days, followed by 7 days off, in 21-day cycles until the disease progressed or the patient experienced an unreasonable level of side effects,” the FDA wrote. “To assess how well [pemigatinib] was working during the trial, patients were scanned every eight weeks. The trial used established criteria to measure how many patients experienced a complete or partial shrinkage of their tumors during treatment. The overall response rate was 36%, with 2.8% of patients having a complete response and 33% having a partial response. Among the 38 patients who had a response, 24 patients (63%) had a response lasting 6 months or longer and 7 patients (18%) had a response lasting 12 months or longer.”
Common side effects — which occurred in 20% or more of patients receiving the drug — include hyperphosphatemia and hypophosphatemia, alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis, dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain, and dry skin, though the agency warned that ocular toxicity is also a risk when taking pemigatinib.
Pemigatinib is manufactured by Incyte Corporation.
John McKenna, Associate Editor, BreakingMED™
Cat ID: 120
Topic ID: 78,120,730,120,935,192,725