Are Sirolimus and Rapamycin the same drug?
Yes, sirolimus and rapamycin refer to the same compound. Sirolimus is the name used for FDA-approved medications, while rapamycin was the original name given by researchers after its discovery. Clinically, this drug is widely used to prevent organ transplant rejection and is known for its role in inhibiting the mTOR (mechanistic target of rapamycin) pathway, which is essential in cell growth and immune regulation.
What Is Sirolimus (Rapamycin)?
Sirolimus is a macrolide compound classified as an immunosuppressant and mTOR inhibitor. Chemically, it is a natural macrocyclic lactone produced by the bacterium Streptomyces hygroscopicus. Its primary medical use is to suppress the immune system, especially in patients who have received organ transplants, to prevent rejection.
Brief History
- Discovery: Sirolimus was first isolated in 1972 from soil samples collected on Easter Island (Rapa Nui), which inspired the original name “rapamycin”.
- Development: Initially investigated as an antifungal agent, it was later found to have potent immunosuppressive and antiproliferative effects, leading to its use in medicine.
- Brand Names: Sirolimus is marketed under several brand names, most notably Rapamune. Other formulations include Fyarro (for certain cancers) and Hyftor (for topical use).
Why Are There Two Names?
While sirolimus and rapamycin are chemically identical, they are known by different names depending on the context. Understanding why this single compound has two names helps clarify how it is referenced in scientific research versus clinical practice. The distinction arises from naming conventions used in drug development and regulation.
- Rapamycin: This is the original name, derived from “Rapa Nui,” the native name for Easter Island, where the compound was discovered. It is commonly used in scientific literature and research contexts.
- Sirolimus: This is the International Nonproprietary Name (INN), adopted for regulatory and clinical use, including FDA approval. The INN system ensures each drug has a unique, globally recognized generic name for prescribing and labeling. “Sirolimus” is the term found on prescriptions, drug labels, and in clinical guidelines.
Both names refer to the exact same active compound. The difference is purely in naming conventions, not in the drug’s chemical structure or effects.
Summary
Sirolimus and rapamycin are two names for the same drug, a groundbreaking immunosuppressant and mTOR inhibitor with a unique history and important role in modern medicine.
References
- European Medicines Agency. Rapamune. European Medicines Agency. Published November 27, 2008. Updated February 4, 2025. Accessed July 10, 2025. https://www.ema.europa.eu/en/medicines/human/EPAR/rapamune
- Kuerec, A. H., & Maier, A. B. 2023. Why Is Rapamycin Not a Rapalog?. Gerontology, 69(6), 657–659. https://doi.org/10.1159/000528985
- National Center for Biotechnology Information. PubChem Compound Summary for CID 5284616, Sirolimus. Accessed July 11, 2025 at https://pubchem.ncbi.nlm.nih.gov/compound/Sirolimus
- Seto B. 2012. Rapamycin and mTOR: a serendipitous discovery and implications for breast cancer. Clinical and translational medicine, 1(1), 29. https://doi.org/10.1186/2001-1326-1-29
- Sirolimus tablet [package insert]. Updated September 2023. Dr. Reddy's Laboratories Limited. Accessed on July 10, 2025 at https://dailymed.nlm.nih.gov/dailymed/drugInfo.cfm?setid=2e6130e5-fa94-dcf8-605c-817bf396e93d
- World Health Organization. Guidance on International Nonproprietary Names (INN). World Health Organization. Accessed July 10, 2025. https://www.who.int/teams/health-product-and-policy-standards/inn/guidance-on-inn
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