Pemigatinib Approved by FDA as First Targeted Treatment for Patients With Cholangiocarcinoma

The FDA has granted accelerated approval to pemigatinib (Pemazyre, Incyte Corporation) as the first treatment for adults with certain types of previously treated, advanced cholangiocarcinoma.

Cholangiocarcinoma is a rare form of cancer that forms in bile ducts, or slender tubes, that carry the digestive fluid bile from the liver to the gallbladder and small intestine. This approval focuses on patients with cholangiocarcinoma that is locally advanced or metastatic and who have tumors with a fusion or other rearrangement of fibroblast growth factor receptor 2 (FGFR2).

There have been no prior FDA-approved therapies for cholangiocarcinoma before the approval of pemigatinib, according to the FDA, as a combination of chemotherapy drugs has been the standard initial treatment.

Pemigatinib is a tablet that works by blocking FGFR2 in tumor cells to prevent them from growing and spreading. The approval was based on the results of a clinical trial that enrolled 107 patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who had received prior treatment. Patients received pemigatinib once per day for 14 consecutive days, followed by 7 days off in 21-day cycles until the disease progressed or the patient experienced excessive adverse effects (AEs). In addition, patients were scanned every 8 weeks to help measure how many 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 rate and 33% having a partial response. Among the 38 patients who had a response, 63% had a response lasting 6 months or longer and 18% had a response lasting 12 months or longer.

The most common AEs in patients who received pemigatinib were 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.

REFERENCE

FDA approves first targeted treatment for patients with cholangiocarcinoma, a cancer of bile ducts [news release]. Silver Spring, MD; FDA: April 20, 2020. https://www.fda.gov/news-events/press-announcements/fda-approves-first-targeted-treatment-patients-cholangiocarcinoma-cancer-bile-ducts?utm_campaign=041720_PR_FDA%20Approves%20First%20Targeted%20Treatment%20for%20Cholangiocarcinoma&utm_medium=email&utm_source=Eloqua. Published April 20, 2020.