FDA Approves Targeted Treatment for Patients with Cholangiocarcinoma

The U.S. Food and Drug Administration granted accelerated approval to Pemazyre (pemigatinib) for previously treated, advanced cholangiocarcinoma with an FGFR2 fusion or rearrangement. This is the first treatment approved for these patients.

Approval was based on clinical trial results of 107 previously treated patients with locally advanced or metastatic cholangiocarcinoma with an FGFR2 fusion or rearrangement who received pemigatinib once a day for 14 consecutive days, followed by 7 days off, in 21-day cycles. Treatment continued until disease progression or unreasonable side effects were noted.

The overall response rate was 36%. Complete response and partial response was recorded in 2.8% and 33%, respectively. In the patients who had a response, 63% had a response ≥ 6 months and 18% had a response lasting ≥12 months. 

Source: U.S. Food and Drug Administration