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Sirolimus Oral Product
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key regulator of cell proliferation and immune cell activation.
Sirolimus inhibits mTOR (mammalian target of rapamycin), a key regulator of cell proliferation and immune cell activation. Used for Organ transplant rejection prophylaxis, Lymphangioleiomyomatosis (LAM), Tuberous sclerosis complex-associated conditions.
At a glance
| Generic name | Sirolimus Oral Product |
|---|---|
| Also known as | TORISEL, rapamycin, Rapamune |
| Sponsor | Shanghai Children's Medical Center |
| 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 subsequently inhibits mTOR complex 1 (mTORC1), which suppresses cell cycle progression and reduces T-cell and B-cell proliferation. This mechanism makes it useful as an immunosuppressant in transplantation and as an anti-proliferative agent in certain disease states, particularly those involving abnormal cell growth or immune dysregulation.
Approved indications
- Organ transplant rejection prophylaxis
- Lymphangioleiomyomatosis (LAM)
- Tuberous sclerosis complex-associated conditions
Common side effects
- Hyperlipidemia
- Thrombocytopenia
- Anemia
- Infection
- Mouth ulcers/stomatitis
- Diarrhea
- Rash
Key clinical trials
- Canadian Profiling and Targeted Agent Utilization Trial (CAPTUR) (PHASE2)
- Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease (PHASE1, PHASE2)
- The Lowest Effective Dose of Post-Transplantation Cyclophosphamide in Combination With Sirolimus and Mycophenolate Mofetil as Graft-Versus-Host Disease Prophylaxis After Reduced Intensity Conditioning and Peripheral Blood Stem Cell Transplantation (PHASE1, PHASE2)
- Elacestrant + Everolimus in Patients ER+/HER2-, ESR1mut, Advanced Breast Cancer Progressing to ET and CDK4/6i. (PHASE3)
- Siplizumab for Sickle Cell Disease Transplant (PHASE1, PHASE2)
- Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors (PHASE2)
- Tolerance by Engaging Antigen During Cellular Homeostasis (PHASE1)
- Combination of Letrozole, Everolimus and TRC105 in Postmenopausal Women With Hormone-Receptor Positive and Her2 Negative Breast Cancer (PHASE1, 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:
- Sirolimus Oral Product CI brief — competitive landscape report
- Sirolimus Oral Product updates RSS · CI watch RSS
- Shanghai Children's Medical Center portfolio CI