FDA Approves Pemigatinib for Advanced Cholangiocarcinoma

This is the first FDA approval for this patient population.

The FDA approved pemigatinib (Pemazyre) for the treatment of patients with previously treated, advanced cholangiocarcinoma, a rare form of cancer in the bile ducts.

The approval — the first for this patient population, that is traditionally treated with a chemotherapy combination – is for patients with locally advanced or metastatic cholangiocarcinoma who have tumors that have a fusion or other rearrangement of FGRF2.

“This approval demonstrates that while we continue to focus our efforts on addressing the COVID-19 pandemic, the FDA remains committed to the important work of reviewing treatments for patients with cancer and other serious conditions,” 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.

The approval was based on the results of a clinical trial involving 107 patients with locally advanced or metastatic cholangiocarcinoma with FGFR2 fusion or rearrangement who were previously treated. Participants received pemigatinib once daily for 14 days, followed by 7 days off. This 21-day cycle occurred until the disease progressed or the patient experienced intolerable adverse events (AEs).

Patients were scanned every 8 weeks. Overall response rate was 36%, with 2.8% of patients having a complete response and 33% having a partial response. In the 28 patients with a response, 63% (n = 24) had a response that lasted 6 months or longer and 18% (n = 7) had a response of 12 months or longer.

Common AEs occurring in at least 20% of patients were hyperphosphatemia and hypophosphatemia (electrolyte disorders), alopecia, diarrhea, nail toxicity, fatigue, dysgeusia, nausea, constipation, stomatitis (sore or inflammation inside the mouth), dry eye, dry mouth, decreased appetite, vomiting, joint pain, abdominal pain, back pain and dry skin. Ocular (eye) toxicity is also a risk of pemigatinib.

Pemigatinib is a promising option because, at diagnosis, the majority of patients with cholangiocarcinoma have advanced disease.

“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,” Pazdur said.