Orphan Drug Designations

The following drugs have received orphan drug designation from the FDA for biliary tract cancer. Orphan designation reflects regulatory interest and does not indicate approval for treatment.

Gene therapy approaches for biliary tract cancer have been reported in the published literature.

469 publications have been identified in PubMed for biliary tract cancer. Research spans Review / Meta-Analysis (31%), Clinical Trial Publication (21%), and Epidemiology / Natural History (15%).

Recent Publications

• Neureiter D (2026). [PMID: 41287511](https://pubmed.ncbi.nlm.nih.gov/41287511/). *Expert review of anticancer therapy*. [Review / Meta-Analysis]

• Mangiavillano B (2026). [PMID: 40695478](https://pubmed.ncbi.nlm.nih.gov/40695478/). *Endoscopy*. [Epidemiology / Natural History]

• Zhu Y (2026). [PMID: 41912087](https://pubmed.ncbi.nlm.nih.gov/41912087/). *J Hepatol*. [Basic Science / Preclinical]

• Erul E (2026). [PMID: 42087549](https://pubmed.ncbi.nlm.nih.gov/42087549/). *Expert Rev Mol Diagn*. [Review / Meta-Analysis]

• Kosaka H (2026). [PMID: 41966637](https://pubmed.ncbi.nlm.nih.gov/41966637/). *J Gastroenterol*. [Epidemiology / Natural History]

• Xu W (2026). [PMID: 41932868](https://pubmed.ncbi.nlm.nih.gov/41932868/). *Cell Death Dis*. [Basic Science / Preclinical]

• Eaton DJ (2026). [PMID: 41512568](https://pubmed.ncbi.nlm.nih.gov/41512568/). *Clin Oncol (R Coll Radiol)*. [Clinical Trial Publication]

• Takaoka S (2026). [PMID: 42059478](https://pubmed.ncbi.nlm.nih.gov/42059478/). *Jpn J Clin Oncol*. [Epidemiology / Natural History]

• Lang H (2026). [PMID: 41537967](https://pubmed.ncbi.nlm.nih.gov/41537967/). *Chirurgie (Heidelb)*. [Review / Meta-Analysis]

• Yoon J (2026). [PMID: 41297674](https://pubmed.ncbi.nlm.nih.gov/41297674/). *J Hepatol*. [Clinical Trial Publication]