Orphan Drug Designations

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

paltusotine is referenced in active clinical trials for carcinoid syndrome (designated 2025).

Gene therapy approaches for carcinoid syndrome have been reported in the published literature.

163 publications have been identified in PubMed for carcinoid syndrome. Research spans Case Report / Case Series (53%), Review / Meta-Analysis (18%), and Clinical Trial Publication (10%).

Recent Publications

• Bright MR (2026). [PMID: 41511186](https://pubmed.ncbi.nlm.nih.gov/41511186/). *Postgrad Med J*. [Review / Meta-Analysis]

• Kaessner S (2026). [PMID: 41695584](https://pubmed.ncbi.nlm.nih.gov/41695584/). *J Belg Soc Radiol*. [Case Report / Case Series]

• Fernández Camporro Á (2026). [PMID: 41352585](https://pubmed.ncbi.nlm.nih.gov/41352585/). *Actas Dermosifiliogr*. [Case Report / Case Series]

• Mansolas N (2026). [PMID: 41840662](https://pubmed.ncbi.nlm.nih.gov/41840662/). *J Med Case Rep*. [Case Report / Case Series]

• Mejia H (2026). [PMID: 42272569](https://pubmed.ncbi.nlm.nih.gov/42272569/). *Case Rep Endocrinol*. [Case Report / Case Series]

• Rayson D (2026). [PMID: 41843715](https://pubmed.ncbi.nlm.nih.gov/41843715/). *Neuroendocrinology*. [Epidemiology / Natural History]

• Al Tabaa O (2026). [PMID: 41640080](https://pubmed.ncbi.nlm.nih.gov/41640080/). *The American journal of case reports*. [Epidemiology / Natural History]

• Ammann M (2026). [PMID: 41042466](https://pubmed.ncbi.nlm.nih.gov/41042466/). *Annals of surgical oncology*. [Clinical Trial Publication]

• Recio Ibarz MJ (2026). [PMID: 41822627](https://pubmed.ncbi.nlm.nih.gov/41822627/). *Cureus*. [Case Report / Case Series]

• Sato T (2026). [PMID: 41586345](https://pubmed.ncbi.nlm.nih.gov/41586345/). *Gastro Hep Adv*. [Case Report / Case Series]