Last reviewed 2026-05-26 · How we verify

Tacrolimus/Sirolimus

Tacrolimus/Sirolimus is a Calcineurin inhibitor and mTOR inhibitor combination Small molecule drug developed by University of Miami. It is currently FDA-approved for Organ transplant rejection prevention (renal, cardiac, hepatic transplantation).

Tacrolimus and sirolimus are immunosuppressive agents that inhibit T-cell activation and proliferation through different pathways to prevent organ rejection.

Tacrolimus is a small molecule inhibitor of the FK506-binding protein 1A, used as an immunosuppressive treatment in various conditions, including Type 1 Diabetes, Islet Transplantation, End Stage Renal Failure With Renal Transplant, End-Stage Renal Disease, and Kidney Failure. It is often used in combination with other immunosuppressive agents, such as Everolimus, for maintenance treatment.

At a glance

Mechanism of action

Tacrolimus inhibits calcineurin, blocking T-cell receptor signaling and IL-2 production, while sirolimus inhibits mTOR, preventing T-cell proliferation. Together, they provide synergistic immunosuppression with potentially reduced nephrotoxicity compared to tacrolimus monotherapy, making the combination useful in transplant recipients.

Approved indications

• Organ transplant rejection prevention (renal, cardiac, hepatic transplantation)

Common side effects

• Nephrotoxicity
• Hyperglycemia
• Hypertension
• Hyperlipidemia
• Infections
• Tremor
• Headache

Key clinical trials

• Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia (PHASE2)
• 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)
• Allo HSCT for High Risk Hemoglobinopathies (PHASE2)
• Advancing Transplantation Outcomes in Children (PHASE2)
• HLA-Identical Sibling Renal Transplant Tolerance (NA)
• Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies (PHASE1, PHASE2)

Primary sources

Every claim on this page is sourced from regulatory or scientific primary sources. See our editorial policy for full methodology.

Competitive intelligence

For the full competitive landscape — auto-detected comparators, recent regulatory actions across the set, upcoming PDUFA, patent timeline, sponsor landscape:

• Tacrolimus/Sirolimus CI brief — competitive landscape report
• Tacrolimus/Sirolimus updates RSS · CI watch RSS
• University of Miami portfolio CI

Frequently asked questions about Tacrolimus/Sirolimus

Related

• Drug class: All Calcineurin inhibitor and mTOR inhibitor combination drugs
• Target: All drugs targeting Calcineurin (tacrolimus) and mTOR (sirolimus)
• Manufacturer: University of Miami — full pipeline
• Therapeutic area: All drugs in Immunology / Transplantation
• Indication: Drugs for Organ transplant rejection prevention (renal, cardiac, hepatic transplantation)

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing