Rapamycin

Sirolimus

Evidence & Status

  • FDA Approved
  • Strong Human Evidence
  • Strong

FDA Approval Scope

Sirolimus is an established prescription medicine with product- and indication-specific labeling that includes renal-transplant rejection prophylaxis and, for applicable products, lymphangioleiomyomatosis; no longevity indication is established.

At a Glance

Sirolimus (rapamycin) is an established prescription medicine with product-specific transplant and rare-disease uses. Mouse lifespan findings are preclinical; human longevity benefit and a longevity dose remain unestablished.

Plain English

Sirolimus, also called rapamycin, is a prescription drug that suppresses mTOR signaling through an FKBP12 complex. Doctors use product-specific formulations for approved indications such as transplant care and, for applicable products, LAM. Researchers study its aging biology, but mouse lifespan findings are not proof that it extends human life, and no longevity dosing plan is established.

Overview

Rapamycin is the common name for sirolimus, an established prescription drug distinct from everolimus, temsirolimus, and other rapalogs. Sirolimus binds FKBP12, and the complex inhibits mTOR signaling, particularly mTORC1; this does not imply identical inhibition of every mTOR complex in every condition. Approved oral sirolimus use is product- and indication-specific. Longevity use remains investigational, with no established longevity dose.

Research Summary

Sirolimus has established human medical use in approved product- and indication-specific contexts, including renal-transplant rejection prophylaxis and applicable rare-disease labeling. In genetically heterogeneous mice, rapamycin fed late in life extended lifespan; that is animal preclinical evidence and does not establish human longevity efficacy. Human sirolimus safety and therapeutic monitoring must remain label-specific. The strong exact-drug rating reflects established approved medicine, while longevity evidence remains preliminary and investigational.

Research Areas

  • mTORC1 signaling
  • Organ transplantation
  • Lymphangioleiomyomatosis
  • Immunosuppression
  • Aging biology research

Safety & Risks

Serious Risks

  • Serious and opportunistic infections: including Pneumocystis jirovecii pneumonia (PCP), cytomegalovirus (CMV), and invasive fungal infections — prophylaxis required in transplant settings per FDA labeling
  • Increased risk of lymphoma and non-melanoma skin cancers with prolonged immunosuppression
  • Hepatic artery thrombosis: reported in liver transplant recipients; use in this population is not recommended per FDA labeling
  • Interstitial lung disease and non-infectious pneumonitis: reported with use; may require dose reduction or discontinuation
  • Angioedema: risk is increased when used with ACE inhibitors or angiotensin receptor blockers
  • Impaired male fertility: reduced testosterone, altered spermatogenesis, and azoospermia reported in clinical settings
  • Prescription-only immunosuppressant: should never be used without physician supervision, therapeutic drug monitoring, and appropriate infection prophylaxis

Reported Side Effects

  • Increased susceptibility to bacterial, viral, and fungal infections
  • Impaired wound healing
  • Hyperlipidemia (elevated triglycerides and total cholesterol)
  • Thrombocytopenia (low platelet count)
  • Anemia
  • Oral ulcers / mouth sores
  • Acne-like skin rash
  • Peripheral edema
  • Hypertension
  • Hyperglycemia
United States
Sirolimus is an FDA-approved prescription medicine with product-specific labeling that includes renal-transplant rejection prophylaxis and, for applicable products, lymphangioleiomyomatosis. No longevity indication is established.
United Kingdom
International regulatory status is not represented by the selected sources in this record.
Australia
International regulatory status is not represented by the selected sources in this record.
Canada
International regulatory status is not represented by the selected sources in this record.

Citations & Sources