In 2020, there were no FDA-approved drugs available for biliary tract cancer (BTC). Fast forward to 2025, and BTC has received 5 FDA approvals within 3 years and several designations in the NCCN Guidelines for BTCs. Capecitabine remains the standard adjuvant therapy, and a feasibility trial of a neoadjuvant chemotherapy combination has paved the way for ongoing studies in this space. The establishment of 2 chemotherapy regimens as standard of care in the second-line setting, along with 2 positive first-line trials of chemoimmunotherapy, has resulted in their inclusion in the NCCN Guidelines for the management of systemic disease. Several targeted agents, such as those directed at IDH1, BRAF V600E, FGFR2, and HER2, are also now recommended in the subsequent-line setting.
Disclosures: Dr. Javle has disclosed receiving consulting fees from AbbVie Inc., Array BioPharma Inc., Astellas Pharma US, Inc., AstraZeneca Pharmaceuticals LP, Bayer Healthcare, BeiGene, Ltd., Biocartis, Boehringer Ingelheim GmbH, Bristol Myers Squibb, Celgene Corporation, Daiichi Sankyo, Inc., GSK plc, Halozyme Therapeutics, Inc., Helsinn Therapeutics (US), Inc., Incyte Corporation, Ipsen, and Janssen Pharmaceutica Products, LP; and receiving grant/research support from AbbVie Inc., Agios, Inc., Array BioPharma Inc., Astellas Pharma US, Inc., AstraZeneca Pharmaceuticals LP, Bayer Healthcare, BeiGene, Ltd., Biocartis, Boehringer Ingelheim GmbH, Bristol Myers Squibb, Celgene Corporation, Daiichi Sankyo, Inc., Eli Lilly and Company, EMD Serono Inc., GSK plc, Halozyme Therapeutics, Inc., Helsinn Therapeutics (US), Inc., Incyte Corporation, Ipsen, Janssen Pharmaceutica Products, LP, Meclun, Merck Sharp & Dohme, Novartis Pharmaceuticals Corporation, OncoSil Medical Ltd, QED Therapeutics, Inc., Servier, Taiho Pharmaceuticals Co., Ltd, and TransThera.