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Rapamune (sirolimus/rapamycin)
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key signaling protein that controls cell growth and proliferation, thereby suppressing immune cell activation and reducing organ rejection.
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key signaling protein that controls cell growth and proliferation, thereby suppressing immune cell activation and reducing organ rejection. Used for Prophylaxis of organ rejection in patients receiving renal transplants, Lymphangioleiomyomatosis (LAM), Tuberous sclerosis complex (TSC)-associated tumors.
At a glance
| Generic name | Rapamune (sirolimus/rapamycin) |
|---|---|
| Sponsor | University at Buffalo |
| Drug class | mTOR inhibitor |
| Target | mTOR (mammalian target of rapamycin) |
| Modality | Small molecule |
| Therapeutic area | Immunology, Oncology |
| Phase | FDA-approved |
Mechanism of action
Sirolimus binds to FKBP12 and the resulting complex inhibits mTOR kinase, which is essential for T-cell and B-cell proliferation. By blocking mTOR signaling, the drug prevents the progression of lymphocytes from G1 to S phase of the cell cycle, effectively suppressing the immune response. This mechanism makes it useful for preventing organ transplant rejection and treating certain cancers and immunoproliferative disorders.
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
- Headache
- Rash
- Pneumonitis
- Delayed wound healing
Key clinical trials
- A Phase 2, Baseline-Controlled Study Evaluating the Safety and Efficacy of PTX-022 (Sirolimus) Topical Gel 3.9% in the Treatment of Cutaneous Venous Malformations (PHASE2)
- Testing Lutetium Lu 177 Dotatate in Patients With Somatostatin Receptor Positive Advanced Bronchial Neuroendocrine Tumors (PHASE2)
- Comparing Retreatment of 177Lu-DOTATATE PRRT Versus the Usual Treatment in Patients With Metastatic Unresectable Gastroenteropancreatic Neuroendocrine Tumors, NET RETREAT Trial (PHASE2)
- Combination Chemotherapy With or Without Temsirolimus in Treating Patients With Intermediate Risk Rhabdomyosarcoma (PHASE3)
- SELVA: A Phase 3 Study Evaluating QTORIN 3.9% Rapamycin Anhydrous Gel in the Treatment of Microcystic Lymphatic Malformations (PHASE3)
- Risk-Based Therapy in Treating Younger Patients With Newly Diagnosed Liver Cancer (PHASE3)
- Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer (PHASE1, PHASE2)
- A Phase II Study of Sunitinib or Temsirolimus in Patients With Advanced Rare Tumours (PHASE2)
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:
- Rapamune (sirolimus/rapamycin) CI brief — competitive landscape report
- Rapamune (sirolimus/rapamycin) updates RSS · CI watch RSS
- University at Buffalo portfolio CI