Everolimus is an oral targeted medicine used both as an anti-cancer therapy and as an immunosuppressant to prevent organ transplant rejection. It belongs to a class of medicines called mTOR inhibitors. It is a prescription-only medicine.

• Therapeutic Class: Anti-cancer (oncology) targeted therapy; immunosuppressant (transplant use).

• Pharmacological Class: mTOR (mammalian target of rapamycin) inhibitor.

• Drug Family: Rapamycin (sirolimus) derivatives — macrolide immunosuppressants/antineoplastics.

• ATC Classification: L01EG02 (Everolimus, oncology use) / L04AA18 (Everolimus, immunosuppressant use).

Approved (oncology, Afinitor): Advanced hormone receptor-positive, HER2-negative breast cancer (combined with exemestane); advanced renal cell carcinoma after failure of certain other targeted therapies; progressive neuroendocrine tumours of the gastrointestinal tract, lung, or pancreatic origin; and subependymal giant cell astrocytoma (SEGA) associated with tuberous sclerosis complex.

Approved (transplant, Zortress/Certican): Prevention of organ rejection in adult kidney and liver transplant recipients, used in combination with reduced-dose calcineurin inhibitor and corticosteroids.

Off-label use (only under medical guidance): Sometimes studied in other tumour types or transplant settings under specialist guidance.

●       Oral tablets (Afinitor, oncology): 2.5 mg, 5 mg, 7.5 mg, and 10 mg.

●       Oral tablets for suspension (Afinitor Disperz, oncology, pediatric use): 2 mg, 3 mg, and 5 mg.

●       Oral tablets (Zortress, transplant): 0.25 mg, 0.5 mg, 0.75 mg, and 1 mg.

Everolimus binds to a protein called FKBP-12, and this complex then blocks an enzyme called mTOR (mammalian target of rapamycin). In cancer, mTOR is often overactive and drives tumour cell growth, division, and blood vessel formation (angiogenesis); blocking it helps slow tumour growth. In transplant patients, mTOR blockade also stops immune cells (lymphocytes) from multiplying in response to growth signals, helping prevent organ rejection.

• Standard dose (oncology): 10 mg taken orally once daily, with or without food, continued until disease progression or unacceptable toxicity; dose reductions to 7.5 mg, 5 mg, or lower are used for tolerability.

• Standard dose (transplant): Typically started at a low dose (e.g., around 0.75 mg twice daily) in combination with reduced-dose calcineurin inhibitor and corticosteroids, then adjusted based on blood trough levels — dosing must not be started earlier than 30 days post-liver-transplant.

• Route: Oral, once daily (oncology) or twice daily (transplant, depending on product), with or without food, consistently taken the same way each time.

• Hepatic impairment (oncology dosing): Dose reduced to 7.5 mg daily in mild impairment (Child-Pugh A), or further reduced/adjusted in moderate to severe impairment.

• Missed dose: Take as soon as remembered unless close to the next dose; don't double up.

• Overdose: May worsen known side effects such as mouth sores, infection risk, or metabolic changes; requires medical attention.

• Common: Mouth sores (stomatitis), fatigue, diarrhoea, rash, decreased appetite, and elevated blood sugar/cholesterol.

• Serious: Non-infectious lung inflammation (pneumonitis), serious infections, kidney toxicity, elevated blood sugar (new-onset diabetes risk, particularly in transplant use), impaired wound healing, and increased proteinuria.

• Emergency symptoms: New or worsening shortness of breath or cough, signs of serious infection (fever, chills), significant swelling, or very high blood sugar symptoms (excessive thirst, frequent urination).

• Recovery advice: Mouth sores are one of the most common reasons for dose adjustment and can often be managed with supportive mouth care; agents containing hydrogen peroxide, iodine, or thyme should be avoided for mouth sores as they may worsen them.

• Pregnancy: Not recommended; may cause fetal harm.

• Breastfeeding: Not recommended during treatment.

• Liver disease: Requires dose adjustment based on the degree of impairment (Child-Pugh classification).

• Kidney disease: Used with monitoring, given the transplant-specific risk of increased proteinuria and general kidney effects.

• Elderly: Used with routine monitoring; no specific age-based dose adjustment defined beyond general precautions.

• Children: Afinitor Disperz is approved for SEGA in pediatric patients; Zortress transplant use in children depends on specific regional approval and specialist guidance.

●       Strong CYP3A4 inhibitors (e.g., ketoconazole): substantially increase everolimus levels; concomitant use should generally be avoided.

●       Moderate CYP3A4/PgP inhibitors (e.g., erythromycin, verapamil): also raise everolimus levels; dose reduction is recommended if these must be combined.

●       Strong CYP3A4 inducers (e.g., rifampin): significantly reduce everolimus levels and effectiveness.

●       Calcineurin inhibitors (cyclosporine, tacrolimus, in transplant use): used together at adjusted doses as part of the standard immunosuppressive regimen, with close monitoring of both drug levels.

●       Everolimus trough blood levels (particularly in transplant use).

●       Kidney function tests, including urine protein (proteinuria) monitoring in transplant patients.

●       Blood glucose and lipid levels periodically.

●       Monitoring for new respiratory symptoms suggestive of non-infectious pneumonitis.

Temsirolimus (another mTOR inhibitor, oncology use), Sirolimus (a related mTOR inhibitor, transplant use); other targeted cancer therapies vary by specific tumour type.

In oncology, used in combination with exemestane for certain breast cancers; in transplant use, combined with a reduced-dose calcineurin inhibitor (cyclosporine or tacrolimus) and corticosteroids as part of a standard multi-drug anti-rejection regimen.

●       Targeted Oncology Therapies

●       Organ Transplant / Anti-Rejection Medicines

●       mTOR Inhibitors

1. Breast Cancer Advanced Treatment

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4. Is Air Pollution Silently Destroying Your Lungs and Increasing Cancer Risk?

1. https://www.accessdata.fda.gov/drugsatfda_docs/label/2012/022334s016lbl.pdf

2. https://www.drugs.com/everolimus.html

The information on this page is for educational purposes only and is not a substitute for medical advice. Please consult your doctor to know if this treatment is appropriate for your condition.