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Sirolimus (Rapamune®)
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key signaling protein that controls cell growth and proliferation.
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key signaling protein that controls cell growth and proliferation. Used for Prophylaxis of organ rejection in patients receiving renal transplants, Lymphangioleiomyomatosis (LAM), Tuberous sclerosis complex (TSC)-associated tumors.
At a glance
| Generic name | Sirolimus (Rapamune®) |
|---|---|
| Also known as | Rapamycin, Rapamune, RAPAMUNE |
| Sponsor | Wyeth is now a wholly owned subsidiary of Pfizer |
| Drug class | mTOR inhibitor |
| Target | mTOR (mammalian target of rapamycin) |
| Modality | Small molecule |
| Therapeutic area | Immunology, Oncology |
| Phase | Phase 3 |
Mechanism of action
Sirolimus binds to FKBP12 and forms a complex that inhibits mTOR kinase activity, thereby suppressing T-cell proliferation and reducing immune activation. This mechanism makes it effective as an immunosuppressant for preventing organ rejection and as an anti-cancer agent by blocking cell cycle progression and angiogenesis.
Approved indications
- Prophylaxis of organ rejection in patients receiving renal transplants
- Lymphangioleiomyomatosis (LAM)
- Tuberous sclerosis complex (TSC)-associated tumors
Common side effects
- Hyperlipidemia
- Thrombocytopenia
- Anemia
- Hypertension
- Diarrhea
- Rash
- Pneumonitis
- Impaired wound healing
Key clinical trials
- Comparison of a Contemporary Sirolimus-eluting Stent (ihtDEStiny®) With Another Everolimus-eluting Stent (Xience™), Both With Permanent Polymers, in Patients With Acute Coronary Syndrome and de Novo Coronary Artery Lesions (NA)
- TAPUR: Testing the Use of Food and Drug Administration (FDA) Approved Drugs That Target a Specific Abnormality in a Tumor Gene in People With Advanced Stage Cancer (PHASE2)
- A Study Evaluating the Efficacy and Safety of Multiple Treatment Combinations in Participants With Breast Cancer (PHASE1, PHASE2)
- Ultrathin Strut Sirolimus-eluting Stent With Bioabsorbable Polymer in Patients Receiving Chronic Oral Anticoagulation
- Safety and Effectiveness of the Crystalline Sirolimus-Eluting Device in Patients With Coronary Artery Disease
- Advancing Transplantation Outcomes in Children (PHASE2)
- A Phase I Trial of Combination Gemcitabine and Nab-Sirolimus in Advanced Leiomyosarcomas or Advanced Soft-Tissue Sarcomas With TSC2 or TSC1 Loss-of-function Mutations or Deletions (PHASE1)
- Study to Compare the Efficacy and Safety After Conversion to RaparoBell® or My-Rept® in Kidney Transplant Patients (PHASE4)
Primary sources
Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.
| Source | Used for |
|---|---|
| ClinicalTrials.gov | Trial enrolment, design, endpoints, results |
Competitive intelligence
For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:
- Sirolimus (Rapamune®) CI brief — competitive landscape report
- Sirolimus (Rapamune®) updates RSS · CI watch RSS
- Wyeth is now a wholly owned subsidiary of Pfizer portfolio CI