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Neoral/Sirolimus
Sirolimus inhibits mTOR kinase to suppress T-cell proliferation and immune activation, while Neoral (cyclosporine) blocks calcineurin to prevent T-cell activation.
Sirolimus inhibits mTOR kinase to suppress T-cell proliferation and immune activation, while Neoral (cyclosporine) blocks calcineurin to prevent T-cell activation. Used for Organ transplant rejection prevention (renal, cardiac, hepatic transplantation).
At a glance
| Generic name | Neoral/Sirolimus |
|---|---|
| Sponsor | University of Miami |
| Drug class | Calcineurin inhibitor + mTOR inhibitor combination |
| Target | Calcineurin (cyclosporine); mTOR kinase (sirolimus) |
| Modality | Small molecule |
| Therapeutic area | Immunology / Transplantation |
| Phase | FDA-approved |
Mechanism of action
This combination pairs two immunosuppressive agents with complementary mechanisms: cyclosporine inhibits calcineurin-dependent IL-2 production and T-cell activation, while sirolimus (rapamycin) blocks mTOR signaling downstream to prevent cell cycle progression. Together they provide synergistic immunosuppression for transplant rejection prevention and autoimmune conditions.
Approved indications
- Organ transplant rejection prevention (renal, cardiac, hepatic)
- Autoimmune and inflammatory conditions
Common side effects
- Nephrotoxicity
- Hypertension
- Hyperglycemia
- Hyperlipidemia
- Infections
- Gingival hyperplasia
- Tremor
Key clinical trials
- A Study to Evaluate Axatilimab Versus Best Available Therapy in Participants With Chronic Graft Versus Host Disease After at Least 2 Prior Lines of Systemic Therapy (PHASE3)
- A Study to Evaluate Axatilimab Versus Best Available Therapy in Pediatric Participants With Chronic Graft-Versus-Host Disease After at Least 2 Prior Lines of Systemic Therapy (AGAVE-256) (PHASE2)
- Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS) (PHASE2)
- Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia (PHASE2)
- Effects of Telitacicept vs Cyclophosphamide on Lupus Related Interstitial Lung Disease (PHASE4)
- Co-infusion of Treg-enriched Donor Lymphocytes With CD3-depleted Hematopoietic Stem Cell Graft to Prevent Graft-versus Host Disease After Allogeneic Hematopoietic Stem Cell Transplantation Among Children With Hematologic Malignancies (PHASE2, PHASE3)
- TUMORAPA 1: Efficacy of Rapamycin in Secondary Prevention of Skin Cancers in Kidney Transplant Recipients (PHASE3)
- Optimizing PTCy Dose and Timing (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:
- Neoral/Sirolimus CI brief — competitive landscape report
- Neoral/Sirolimus updates RSS · CI watch RSS
- University of Miami portfolio CI