Title: orphan drug
Long Title: IUPAC Gold Book - orphan drug
DOI: 10.1351/goldbook.12699
Status: current

Definition
Pharmaceutical agent that has been approved specifically to treat a rare and commercially unfavourable medical condition.

Related Term
- orphan disease: https://goldbook.iupac.org/terms/view/12698

Source
- PAC, 2013, 85, 1725. 'Glossary of terms used in medicinal chemistry. Part II (IUPAC Recommendations 2013)' on page 1744 (https://doi.org/10.1351/PAC-REC-12-11-23)

Other Outputs
- html: https://goldbook.iupac.org/terms/view/12699/html
- json: https://goldbook.iupac.org/terms/view/12699/json
- xml: https://goldbook.iupac.org/terms/view/12699/xml

Citation: Citation: 'orphan drug' in IUPAC Compendium of Chemical Terminology, 5th ed. International Union of Pure and Applied Chemistry; 2025. Online version 5.0.0, 2025. 10.1351/goldbook.12699
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Disclaimer: The International Union of Pure and Applied Chemistry (IUPAC) is continuously reviewing and, where needed, updating terms in the Compendium of Chemical Terminology (the IUPAC Gold Book). Users of these terms are encouraged to include the version of a term with its use and to check regularly for updates to term definitions that you are using.
Accessed: 2026-06-27T11:02:46+00:00