Timeline of FDA-Approved Targeted Therapy for Cholangiocarcinoma

SIMPLE SUMMARY: Cholangiocarcinoma constitutes around 3% of gastrointestinal cancers, and mortality from this cancer has been rising in the recent decades. Many cases of cholangiocarcinoma are unfortunately discovered in their advanced stages which cannot be treated with surgical resection alone. Targeted therapy is a type of medical treatment that has garnered significant interest due to its ability to specifically target cancer cells while sparing normal healthy cells. A few targeted therapies have just recently been approved by the United States FDA for the treatment of cholangiocarcinoma specifically. This manuscript seeks to explore the timeline of targeted therapies with either FDA approval or FDA breakthrough therapy designation. The official approval of these therapies marks a new age for the treatment of cholangiocarcinoma and brings new options for clinicians across the nation for this unfortunate disease. ABSTRACT: Cholangiocarcinoma (CCA) represents approximately 3% of gastrointestinal malignancies worldwide and constitutes around 10–15% of all primary liver cancers, being only second to hepatocellular carcinoma. Mortality from CCA has been on the rise in recent decades, and in the United States alone there has been a 36% increase in CCA from 1999 to 2014, with over 7000 CCA mortalities since 2013. Targeted therapies, which have been gaining interest due to their greater specificity toward cancer cells, have only recently started gaining FDA approval for the treatment of CCA. In this manuscript, we will go through the timeline of current FDA-approved targeted therapies as well as those that have gained FDA breakthrough therapy designation.