The product is pemigatinib (Pemazyre, Incyte), an oral kinase inhibitor.
It was approved specifically for use in patients with advanced cholangiocarcinoma who have received prior treatment and who have tumors that have a fusion or other rearrangement of the fibroblast growth factor receptor 2 (FGFR2) gene.
These FGFR2 genetic abnormalities are found in about 9% to 14% of patients with cholangiocarcinoma, notes the FDA.
At diagnosis, a majority of patients with cholangiocarcinoma have advanced disease, meaning that the disease is no longer treatable with surgery, the agency also notes. Until now, a combination of chemotherapy drugs has been the standard initial treatment.
Now the subgroup of patients with FGFR2 tumors have the option of a targeted therapy.
“Although cholangiocarcinoma is considered a rare disease, it has been on the rise over the past three decades,” commented Ghassan Abou-Alfa, MD, Memorial Sloan Kettering Cancer Center, New York City, in a company press release. “It is encouraging to have a new targeted treatment option for patients who historically have had limited options after first-line chemotherapy or surgery, in which relapse rates remain high.”
The accelerated approval was based on the overall response rate (ORR) and duration of response in an open-label clinical trial that involved 107 patients (the FIGHT-202 study).
As a condition of the accelerated approval, the manufacturer will complete and submit the results of a randomized trial demonstrating an improvement in progression-free survival or overall survival, the FDA noted.
Results From Open-Label Clinical Trial
The FIGHT-202 study enrolled 107 patients with locally advanced or metastatic cholangiocarcinoma who had received prior treatment and who had tumors with an FGFR2 fusion or rearrangement.
All patients received pemigatinib once a day for 14 days, followed by 7 days off, in 21-day cycles until the disease progressed or the patient experienced an unreasonable level of side effects. Patients underwent scanning every 8 weeks to assess ORR.
The ORR was 36% (38 of 107 patients), which included 2.8% of patients with a complete response and 33% with partial response.
Among the 38 patients who had a response, 24 patients (63%) had a response that lasted 6 months or longer, and seven patients (18%) had a response that lasted 12 months or longer.
“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,” commented 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.
The most common adverse reactions, which occurred in 20% or more of patients who received pemigatinib, were electrolyte disorders (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. Ocular toxicity was seen rarely, the agency notes.
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