SIROLIMUS
Sirolimus is an immunosuppressant used to prevent organ rejection in patients aged 13 and older who have received a kidney transplant. It helps patients by suppressing the immune system's response to the new organ, typically in combination with other medications like corticosteroids and cyclosporine. This medication also treats lymphangioleiomyomatosis, a lung disease involving the abnormal growth of smooth muscle-like cells.
How SIROLIMUS Works
This drug works by binding to the protein FKBP-12 to form a complex that inhibits mTOR, a regulatory kinase responsible for cell growth. This inhibition blocks the activation and proliferation of T-lymphocytes and reduces antibody production, effectively slowing the immune response. In lung tissue, it helps manage the overactive signaling caused by genetic mutations associated with lymphangioleiomyomatosis.
Details
- Status
- Prescription
- First Approved
- 2014-01-08
- Routes
- ORAL
- Dosage Forms
- TABLET, SOLUTION
Companies
SIROLIMUS Approval History
What SIROLIMUS Treats
2 indicationsSIROLIMUS is approved for 2 conditions since its original approval in 2014. These indications span multiple therapeutic areas including oncology, immunology, and more.
- Organ Rejection
- Lymphangioleiomyomatosis
SIROLIMUS Boxed Warning
IMMUNOSUPPRESSION, USE IS NOT RECOMMENDED IN LIVER OR LUNG TRANSPLANT PATIENTS Increased susceptibility to infection and the possible development of lymphoma and other malignancies may result from immunosuppression Increased susceptibility to infection and the possible development of lymphoma may result from immunosuppression. Only physicians experienced in immunosuppressive therapy and management of renal transplant patients should use sirolimus for prophylaxis of organ rejection in patients re...
WARNING: IMMUNOSUPPRESSION, USE IS NOT RECOMMENDED IN LIVER OR LUNG TRANSPLANT PATIENTS Increased susceptibility to infection and the possible development of lymphoma and other malignancies may result from immunosuppression Increased susceptibility to infection and the possible development of lymphoma may result from immunosuppression. Only physicians experienced in immunosuppressive therapy and management of renal transplant patients should use sirolimus for prophylaxis of organ rejection in patients receiving renal transplants. Patients receiving the drug should be managed in facilities equipped and staffed with adequate laboratory and supportive medical resources. The physician responsible for maintenance therapy should have complete information requisite for the follow-up of the patient [ see Warnings and Precautions ( 5.1 ) ]. The safety and efficacy of sirolimus as immunosuppressive therapy have not been established in liver or lung transplant patients, and therefore, such use is not recommended [ see Warnings and Precautions ( 5.2 , 5.3 ) ]. Liver Transplantation – Excess Mortality, Graft Loss, and Hepatic Artery Thrombosis (HAT) The use of sirolimus in combination with tacrolimus was associated with excess mortality and graft loss in a study in de novo liver transplant patients. Many of these patients had evidence of infection at or near the time of death. In this and another study in de novo liver transplant patients, the use of sirolimus in combination with cyclosporine or tacrolimus was associated with an increase in HAT; most cases of HAT occurred within 30 days post-transplantation and most led to graft loss or death [ see Warnings and Precautions ( 5.2 ) ]. Lung Transplantation – Bronchial Anastomotic Dehiscence Cases of bronchial anastomotic dehiscence, most fatal, have been reported in de novo lung transplant patients when sirolimus has been used as part of an immunosuppressive regimen [ see Warnings and Precautions ( 5.3 ) ]. WARNING: IMMUNOSUPPRESS
SIROLIMUS Target & Pathway
ProTarget
A cytokine that promotes Th2 immune responses and IgE production. IL-4 drives allergic inflammation in atopic dermatitis and asthma. Blocking IL-4 (often together with IL-13) reduces the type 2 inflammation underlying these conditions.
SIROLIMUS Competitive Set
ProThree rings of competition based on shared molecular targets and treated indications.
Direct competitors
Same target(s) AND same indication — head-to-head.
MoA expansion candidates
Same target(s), different indications — where else is this mechanism being explored?
Indication competitors
Same indication, different mechanism — what else might this patient receive?
Filters applied: drops same-active-ingredient (505(b)(2) reformulations), route-mismatch (topical vs systemic), and cross-therapeutic-area matches in same-indication rings.
Drugs Similar to SIROLIMUS
3 of 16FDA-approved drugs for similar conditions. Compare mechanisms and indications to understand treatment alternatives.
Clinical Trial Registry
227 trials| Trial | Sponsor ID | Phase | Status | Title |
|---|---|---|---|---|
| NCT05896839 | NCI-2023-04306 NCI-2023-04306, ETCTN10614 | Ph 1, Ph 2 | recruiting | Immunotherapy in Combination With Prednisone and Sirolimus for Kidney Transplant Recipients With Unresectable or Metastatic Skin Cancer |
| NCT02042326 MAV-RAPA | PHRCN10-PR-DEVAUCHELLE 2011-000321-69 | Ph 2 | recruiting | Prospective Evaluation of the Efficacy of Sirolimus (Rapamune®) in the Treatment of Severe Arteriovenous Malformations |
| NCT06876142 | 10001811 001811-C | Ph 1, Ph 2 | suspended | Combination Therapy (Mirdametinib and Sirolimus) for RAS Mutated Relapsed Refractory Multiple Myeloma |
| NCT02629120 | 160032 16-I-0032 | Ph 1, Ph 2 | active not recruiting | High Dose Peripheral Blood Stem Cell Transplantation With Post Transplant Cyclophosphamide for Patients With Chronic Granulomatous Disease |
| NCT07581704 | 202601163 | Ph 1 | not yet recruiting | Sirolimus Pre-conditioning on T Cell Activity and T-cell Engaging Bispecific Antibody Efficacy in Multiple Myeloma |
| NCT05088356 | IRB-60439 NCI-2021-12228 | Ph 1 | active not recruiting | Reduced Intensity Allogeneic HCT in Advanced Hematologic Malignancies w/T-Cell Depleted Graft |
| NCT07582172 | 25663 NCI-2026-03208, 25663 | Ph 2 | not yet recruiting | Total Marrow and Lymphoid Irradiation in Combination With Fludarabine and Melphalan as Conditioning for Allogeneic Peripheral Blood Stem Cell Hematopoietic Cell Transplant in Older Patients With Refractory and Relapsed Acute Myeloid Leukemia and High-risk Myelodysplastic Syndrome |
| NCT05463133 | 10000977 000977-I | Ph 1, Ph 2 | recruiting | Allogeneic Hematopoietic Stem Cell Transplantation for Chronic Granulomatous Disease (CGD) With an Alemtuzumab, Busulfan and TBI-based Conditioning Regimen Combined With Cytokine (IL-6, +/- IFN-gamma) Antagonists |
| NCT04888741 MoTD | RG_19-116 | Ph 2 | recruiting | Methods of T Cell Depletion Trial (MoTD) |
| NCT07113743 | 10000186 000186-I | Ph 1, Ph 2 | enrolling by invitation | Part B- G1X-CGD (Lentiviral Vector Transduced CD34+ Cells) in Patients With X-Linked Chronic Granulomatous Disease |
| NCT03214354 Sickle-AID | TRU-17-001 | Ph 2 | recruiting | Nonmyeloablative Stem Cell Transplant in Children With Sickle Cell Disease and a Major ABO-Incompatible Matched Sibling Donor |
| NCT01231412 results posted | 2448.00 NCI-2010-02035, 2448.00 | Ph 3 | completed | Graft-Versus-Host Disease Prophylaxis in Treating Patients With Hematologic Malignancies Undergoing Unrelated Donor Peripheral Blood Stem Cell Transplant |
| NCT06145282 results posted | 10001635 001635-H | Ph 1, Ph 2 | active not recruiting | Non-myeloablative Haploidentical HCT Study for Patients With Sickle Cell Disease, Including Compromised Organ Function |
| NCT05327023 | 10000489 000489-C | Ph 1, Ph 2 | recruiting | Donor Lymphocyte Infusion After Allogeneic Hematopoietic Cell Transplantation for High-Risk Hematologic Malignancies |
| NCT07162038 | 10001785 001785-C | Ph 1 | recruiting | Phase I Trial Integrating HLA-Haploidentical Anti-CD19 CAR-T Cells With Post-Transplantation Cyclophosphamide-Based HLA-Haploidentical Hematopoietic Cell Transplantation |
| NCT04959175 | 10000359 000359-C | Ph 1, Ph 2 | recruiting | Phase I/II Study to Reduce Post-transplantation Cyclophosphamide Dosing for Older or Unfit Patients Undergoing Bone Marrow Transplantation for Hematologic Malignancies |
| NCT05436418 | 10000613 000613-C | Ph 1, Ph 2 | recruiting | 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 |
| NCT00977691 results posted | 090225 09-H-0225 | Ph 1, Ph 2 | active not recruiting | Haploidentical PBMC Transplant for Severe Congenital Anemias |
| NCT03077542 results posted | 170069 17-H-0069 | Ph 1, Ph 2 | active not recruiting | Nonmyeloablative Haploidentical Peripheral Blood Mobilized Hematopoietic Precursor Cell Transplantation for Sickle Cell Disease |
| NCT07542314 ENHANCE | SRP-9001-402 | Ph 4 | not yet recruiting | Study to Evaluate the Safety and Effectiveness of ELEVIDYS in Participants With Duchenne Muscular Dystrophy Treated in a Post-Marketing Setting |
| NCT06261060 | 2023-0918 NCI-2024-01333 | Ph 2 | recruiting | Low-Dose Sirolimus to Increase Hematopoietic Function in Patients With RUNX1 Familial Platelet Disorder |
| NCT04177004 | 19214 NCI-2019-06454, 19214 | Ph 1 | suspended | Human Lysozyme Goat Milk for the Prevention of Graft Versus Host Disease in Patients With Blood Cancer Undergoing a Donor Stem Cell Transplant |
| NCT07052929 | 2957-CL-0101 2025-523213-29 | Ph 1, Ph 2 | recruiting | Study of ASP2957 in Male Participants With X-linked Myotubular Myopathy Who Need Ventilators |
| NCT03192397 results posted | I 44417 NCI-2017-01069, I 44417 | Ph 1, Ph 2 | active not recruiting | Chemotherapy, Total Body Irradiation, and Post-Transplant Cyclophosphamide in Reducing Rates of Graft Versus Host Disease in Patients With Hematologic Malignancies Undergoing Donor Stem Cell Transplant |
| NCT00811915 EPARGNE | 2007/125/HP | Ph 3 | terminated | Study to Compare the Safety and Efficacy of Sirolimus (Rapamune) to Tacrolimus (Advagraf) Associated to Mycophenolate Mofetil (CellCept) Between 12 and 36 Months After Kidney Transplantation |
| NCT00792948 results posted | NCI-2009-00800 NCI-2009-00800, S0805 | Ph 2 | active not recruiting | Combination Chemotherapy With or Without Donor Stem Cell Transplant in Treating Patients With Acute Lymphoblastic Leukemia |
| NCT06084780 TRANSCAPE | CASE7Z23 | Ph 2 | not yet recruiting | Intestinal & Multivisceral Transplantation for Unresectable Mucinous Carcinoma Peritonei (TRANSCAPE) |
| NCT06325709 | 10001580 001580-I | Ph 1, Ph 2 | recruiting | Base Editing for Mutation Repair in Hematopoietic Stem & Progenitor Cells for X-Linked Chronic Granulomatous Disease |
| NCT02891603 results posted | MCC-18783 5R01HL133823-02 | Ph 1, Ph 2 | completed | A Phase I/II GVHD Prevention Trial Combining Pacritinib With Sirolimus-Based Immune Suppression |
| NCT03605927 | MCC-19305 | Ph 1 | completed | CD40-L Blockade for Prevention of Acute Graft-Versus-Host Disease |
| NCT04411654 | J3Z-MC-OJAB (PRV-GD2-101) | Ph 1, Ph 2 | active not recruiting | Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE) |
| NCT06843811 | 24-022433 | Ph 2 | enrolling by invitation | Sirolimus for Leigh Syndrome |
| NCT04339101 results posted | 19576 NCI-2020-01722, 19576 | Ph 2 | completed | Itacitinib, Tacrolimus, and Sirolimus for the Prevention of GVHD in Patients With Acute Leukemia, Myelodysplastic Syndrome, or Myelofibrosis Undergoing Reduced Intensity Conditioning Donor Stem Cell Transplantation |
| NCT06959771 | 10002385 002385-I | Ph 1, Ph 2 | recruiting | Base Editing Hematopoietic Stem Cell and T Cell Gene Therapy for CD40L-HyperIgM Syndrome: Single Patient Study |
| NCT07270549 GAIN-CTNNB1 | 0120-536/2025-2711 2025-522719-40-00 | Ph 1, Ph 2 | recruiting | Gene Replacement Therapy for Treatment of Paediatric Patients With CTNNB1 Neurodevelopmental Syndrome |
| NCT04141020 | 20190857 1R01NS111119-01A1 | Ph 2 | recruiting | Effect of Sirolimus on Molecular Alterations in Cerebral Aneurysms |
| NCT04127578 | J3Z-MC-OJAA formerly PRV-PD101 | Ph 1, Ph 2 | active not recruiting | Phase 1/2a Clinical Trial of PR001 (LY3884961) in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL) |
| NCT05357482 | 10000539 000539-H | Ph 1, Ph 2 | active not recruiting | Addition of JSP191 (C-kit Antibody) to Nonmyeloablative Hematopoietic Cell Transplantation for Sickle Cell Disease and Beta-Thalassemia |
| NCT03970096 | RG1005364 9749, NCI-2019-03188 | Ph 2 | recruiting | Graft Versus Host Disease-Reduction Strategies for Donor Blood Stem Cell Transplant Patients With Acute Leukemia or Myelodysplastic Syndrome (MDS) |
| NCT06752694 | RG1124040 NCI-2024-06524, 20575 | Ph 2 | recruiting | Ruxolitinib Based GVHD Prophylaxis Regimen Before, During, and After Hematopoietic Cell Transplantation in Older Adult Patients With Acquired Aplastic Anemia |
| NCT06872333 | 2024LS140 | Ph 2 | recruiting | Allo HSCT for High Risk Hemoglobinopathies |
| NCT05386914 | 2091042 | Ph 1 | active not recruiting | This Study Consists of Two Study Parts Conducted Under a Single IRB. Part I: Short Term ApoE-dependent Cerebral Blood Flow Response to Sirolimus in Cognitively Normal Adults Part II: Short Term ApoE-dependent Cerebral Blood Flow and Lung Perfusion Response to Sirolimus in Cognitively Normal Adults |
| NCT04861064 | 00106369 | Ph 2 | recruiting | Weekly Sirolimus Therapy |
| NCT03933904 results posted | 832465 | Ph 2 | active not recruiting | Sirolimus in Previously Treated Idiopathic Multicentric Castleman Disease |
| NCT06055608 ADVANTage | DAIT CTOT-41 | Ph 2 | recruiting | Advancing Transplantation Outcomes in Children |
| NCT02574728 AflacST1502 | IRB00082488 | Ph 2 | active not recruiting | Sirolimus in Combination With Metronomic Chemotherapy in Children With Recurrent and/or Refractory Solid and CNS Tumors |
| NCT04469530 AflacST1903 | STUDY00000113 | Ph 2 | recruiting | Sirolimus in Combination With Metronomic Chemotherapy in Children With High-Risk Solid Tumors |
| NCT05170828 | PRESERVE | Ph 1 | withdrawn | Cryopreserved MMUD BM With PTCy for Hematologic Malignancies |
| NCT07426484 | 25-743 | Ph 4 | not yet recruiting | Cosibelimab for CSCC in Patients With Kidney Transplant or Hematologic Malignancy |
| NCT01203722 results posted | J1055 NA_00039823, P01CA015396 | Ph 1, Ph 2 | completed | Reduced Intensity, Partially HLA Mismatched BMT to Treat Hematologic Malignancies |
Showing 50 of 227 trials
Active Pipeline
Ongoing clinical trials by development phase
Key Completed Trials
Completed studies with published results, ranked by significance
Trial Timeline
Full development history with FDA approval milestones
Understanding FDA Approval Types
| Count | Type | What it means |
|---|---|---|
| - | ORIG | Original approval - drug first enters market |
| - | SUPPL - Efficacy | New indication (new disease/condition approved) |
| - | SUPPL - Labeling | Label text changes (warnings, dosing updates) |
| - | SUPPL - Manufacturing | Production changes (new facility) |
| - | SUPPL - Chemistry | Formulation changes (new dosage strength) |
Green lines in the timeline show ORIG and Efficacy approvals - the clinically meaningful milestones.
SIROLIMUS FDA Label Details
Indications & Usage
SIROLIMUS is indicated for the treatment of Organ Rejection; Lymphangioleiomyomatosis.
WARNING: IMMUNOSUPPRESSION, USE IS NOT RECOMMENDED IN LIVER OR LUNG TRANSPLANT PATIENTS Increased susceptibility to infection and the possible development of lymphoma and other malignancies may result from immunosuppression Increased susceptibility to infection and the possible development of lympho...
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Full clinical data, patents, trials, and competitive landscape for sirolimus.
Data Sources
Data sourced from official FDA and NIH databases. Click links to verify on original sources.