April 20, 2020
1 min read

FDA approves first targeted therapy for cholangiocarcinoma

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The FDA granted accelerated approval to pemigatinib for treatment of certain adults with previously treated unresectable cholangiocarcinoma.

The approval applies to use of pemigatinib (Pemazyre, Incyte) by patients with locally advanced or metastatic cholangiocarcinoma whose tumors have a fusion or other rearrangement of fibroblast growth factor receptor 2 (FGFR2).

Pemigatinib — which blocks FGFR2 in tumor cells to prevent them from growing or spreading — is the first targeted treatment for cholangiocarcinoma, a rare form of cancer that forms in the bile ducts.

Most patients with cholangiocarcinoma present with advanced disease at diagnosis, and standard treatment typically consists of a combination of chemotherapy agents. An estimated 9% to 14% of patients have FGFR2 fusions.

Richard Pazdur, MD
Richard Pazdur

“With Pemazyre, 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,” 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, said in a press release.

The FDA based the approval on results of a trial that included 107 patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusions or rearrangements who had received prior treatment.

Trial participants received pemigatinib once daily in a 14-days-on, 7-days-off schedule. Treatment continued until disease progression or unacceptable toxicity.

Researchers reported a 36% overall response rate, with 2.8% of patients achieving complete response and 33% achieving partial response.

Among the 38 patients who achieved response, 24 (63%) had responses that lasted at least 6 months and seven (18%) had responses that lasted at least 12 months.

The most common adverse events reported among pemigatinib-treated patients included 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.

The FDA previously granted priority review, breakthrough therapy and orphan drug designations to pemigatinib for this indication.