Real-World Impact: How Targeted Therapy is Changing Cholangiocarcinoma Treatment

Cholangiocarcinoma is an aggressive cancer that begins in the bile ducts. While the cancer is relatively rare, affecting approximately 8,000 people in the United States each year, unfortunately, it is often undiagnosed until an advanced stage. This late diagnosis makes cholangiocarcinoma very challenging to treat, and less than 10% of patients survive for five years after diagnosis. Traditional anti-cancer treatments, such as chemotherapy, have only limited effectiveness in cholangiocarcinoma, and can cause serious side effects. Recently developed immunotherapy and targeted therapies have provided promising options for this difficult-to-treat disease. Dr. Kim Saverno of the Incyte Corporation, a biopharmaceutical company headquartered in the U.S., and the study’s co-authors, have been studying the real-world use of an FDA-approved targeted drug for cholangiocarcinoma called pemigatinib. Pemigatinib was approved by the FDA in 2020. It can be specifically used for cholangiocarcinoma patients who have genetic changes in a protein known as fibroblast growth factor receptor 2, or FGFR2 for short. Their study, to the best of our knowledge, is the first to examine pemigatinib's use in real-world settings, and reveals information about treatment patterns, FGFR2 testing patterns, and effectiveness of pemigatinib for cholangiocarcinoma when used in everyday practice.