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Medical Disclaimer: This information is for educational purposes only and is not a substitute for professional medical advice.
Kinase Inhibitor [EPC]
Everolimus is a potent mTOR inhibitor and immunosuppressant used to treat various cancers, including renal cell carcinoma and breast cancer, as well as to prevent organ rejection in transplant recipients.
Name
Everolimus
Raw Name
EVEROLIMUS
Category
Kinase Inhibitor [EPC]
Drug Count
6
Variant Count
82
Last Verified
February 17, 2026
RxCUI
977427, 977431, 977434, 977436, 977438, 977440, 2056895, 2056897, 845507, 845515, 998189, 1119400, 1308428, 1308430, 1308432, 845512, 845518, 998191, 1119402, 1310138, 1310144, 1310147, 2685202, 2685204, 2685206, 2685208
UNII
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About Everolimus
Everolimus is a potent mTOR inhibitor and immunosuppressant used to treat various cancers, including renal cell carcinoma and breast cancer, as well as to prevent organ rejection in transplant recipients.
Detailed information about Everolimus
This page is for informational purposes only and does not replace medical advice. Consult a qualified healthcare professional before using any medication containing Everolimus.
As a therapeutic agent, everolimus serves two primary roles: as an antineoplastic (cancer-fighting) medication and as an immunosuppressant. In the context of oncology, it is used to slow the growth and spread of cancer cells by interfering with the signaling pathways that drive tumor progression. In the context of transplantation, it prevents the patient's immune system from attacking the newly transplanted organ. The drug's versatility stems from its ability to modulate the cell cycle, particularly the transition from the G1 phase to the S phase, thereby inhibiting cellular proliferation across various tissues. According to the FDA-approved labeling, everolimus is a critical component in the management of hormone receptor-positive, HER2-negative breast cancer, neuroendocrine tumors of pancreatic, gastrointestinal, or lung origin, and tuberous sclerosis complex (TSC)-associated tumors such as subependymal giant cell astrocytoma (SEGA).
To understand how everolimus works, one must understand the mTOR pathway. The mTOR protein is a central regulator of cellular metabolism, growth, and survival. It integrates signals from nutrients, energy levels, and growth factors to dictate whether a cell should grow and divide or remain quiescent. In many types of cancer, the mTOR pathway is overactive, leading to uncontrolled cell growth and the formation of new blood vessels (angiogenesis) that feed the tumor.
At the molecular level, everolimus binds with high affinity to an intracellular protein called FK506-binding protein-12 (FKBP-12). This everolimus-FKBP-12 complex then binds to and inhibits the mTOR Complex 1 (mTORC1). By inhibiting mTORC1, everolimus effectively blocks the downstream signaling of two key synthetic pathways: the ribosomal protein S6 kinase (p70S6K) and the eukaryotic initiation factor 4E-binding protein (4E-BP1). The inhibition of these proteins leads to a reduction in the synthesis of proteins involved in the cell cycle, such as cyclin D1, and proteins involved in angiogenesis, such as vascular endothelial growth factor (VEGF). Consequently, the drug induces cell cycle arrest and reduces the blood supply to tumors, effectively 'starving' them of the resources needed for expansion. In transplant patients, this same mechanism prevents the rapid proliferation of T-cells and B-cells, which are the primary drivers of organ rejection.
The pharmacokinetics of everolimus are complex and require careful management by healthcare providers to ensure therapeutic efficacy while minimizing toxicity.
Everolimus is approved for several distinct clinical indications, categorized by the brand name under which it is dispensed:
Off-label uses may include certain types of refractory lymphoma or as part of experimental protocols for anti-aging research (though the latter is not clinically validated or recommended).
Everolimus is primarily available in oral formulations to facilitate long-term outpatient therapy:
> Important: Only your healthcare provider can determine if Everolimus is right for your specific condition. The choice of brand and dosage form depends entirely on the condition being treated and the patient's specific medical profile.
The dosage of everolimus is highly dependent on the specific condition being treated and must be individualized based on clinical response and tolerability.
Everolimus is FDA-approved for specific conditions in pediatric populations, primarily those related to Tuberous Sclerosis Complex (TSC).
No dosage adjustment is generally required for patients with renal impairment. Everolimus is not significantly cleared by the kidneys, and clinical studies have shown that its pharmacokinetics are not altered by varying degrees of kidney function. However, because everolimus can potentially cause protein in the urine (proteinuria), kidney function should still be monitored.
Everolimus is metabolized by the liver, making dose adjustments critical for patients with liver dysfunction. For oncology patients:
In clinical trials, no overall differences in safety or effectiveness were observed between patients over 65 and younger patients. However, because older adults are more likely to have decreased hepatic or cardiac function, healthcare providers often start at the lower end of the dosing range and monitor more frequently.
If you miss a dose of everolimus, you can take it as soon as you remember, provided it is within 6 hours of the scheduled time. If more than 6 hours have passed, skip the missed dose and take your next dose at the regular time. Do not take two doses at once to make up for a missed one. Frequent missed doses can reduce the effectiveness of the treatment, especially in transplant patients where it may lead to organ rejection.
Experience with everolimus overdose in humans is very limited. Reported cases of accidental ingestion in children have not resulted in severe toxicity at low doses. However, signs of acute overdose may include severe mouth sores, low white blood cell counts (increasing infection risk), or severe gastrointestinal distress. In the event of an overdose, contact a poison control center or seek emergency medical attention immediately. Treatment is generally supportive.
> Important: Follow your healthcare provider's dosing instructions exactly. Do not adjust your dose or stop taking the medication without explicit medical guidance, as this can lead to disease progression or organ transplant failure.
Everolimus is associated with a high frequency of side effects, many of which are manageable with supportive care or dose modifications. The most common include:
> Warning: Stop taking Everolimus and call your doctor immediately if you experience any of these serious symptoms.
Prolonged use of everolimus can lead to chronic metabolic issues. Patients may develop persistent high cholesterol or triglycerides, requiring long-term statin therapy. There is also an increased risk of developing secondary malignancies, such as skin cancer, due to long-term immunosuppression. In pediatric patients, there are concerns regarding the impact on growth and sexual maturation, although long-term data are still being collected. Bone health may also be affected, with some studies suggesting an increased risk of osteopenia over several years of use.
For the brand Zortress (used in transplantation), the FDA has issued several Black Box Warnings:
Report any unusual symptoms or changes in your health to your healthcare provider immediately. Regular blood tests are required to monitor for these side effects throughout your treatment.
Everolimus is a high-alert medication that requires close supervision by a specialist (oncologist or transplant surgeon). It is essential to understand that everolimus can significantly weaken your immune system, making you more likely to get infections or making current infections worse. Patients should avoid contact with individuals who have contagious illnesses and should not receive 'live' vaccines (such as the flu mist, measles, or mumps vaccines) while taking this drug, as the vaccine could cause a serious infection.
As noted in the side effects section, the Zortress brand carries critical FDA Black Box Warnings. These include the risk of Malignancies and Serious Infections, which are inherent to potent immunosuppression. Furthermore, there is a specific warning regarding Kidney Graft Thrombosis, a condition that can cause the loss of a transplanted kidney. Finally, it is explicitly warned that everolimus should not be used in Heart Transplantation due to increased mortality risks observed in clinical studies. For the Afinitor brand used in oncology, there are no Black Box Warnings, but the risks of pneumonitis and infection remain severe.
To ensure safety, your doctor will require frequent blood tests, including:
Everolimus generally does not have a direct effect on the ability to drive or operate machinery. However, because it can cause fatigue, dizziness, or headaches in some patients, you should observe how the medication affects you before performing tasks that require alertness.
There is no direct contraindication between everolimus and moderate alcohol consumption. However, alcohol can contribute to liver stress and may worsen certain side effects like nausea or headaches. It is best to limit alcohol intake and discuss your habits with your doctor, especially if you have pre-existing liver issues.
Do not stop taking everolimus abruptly. In oncology, stopping the drug may lead to rapid tumor regrowth (rebound effect). In transplant patients, stopping the drug can lead to acute organ rejection within days. If the drug must be stopped due to toxicity (like severe pneumonitis), your doctor will provide a specific plan to manage the transition to another therapy.
> Important: Discuss all your medical conditions, including any history of hepatitis, diabetes, or lung disease, with your healthcare provider before starting Everolimus.
Everolimus should never be used with certain drugs that profoundly affect the CYP3A4 enzyme system, as this can lead to dangerously high or low levels of the drug.
Everolimus does not typically interfere with the chemical reactions of lab tests, but it significantly alters the results of many tests. It can cause false elevations in blood glucose readings and lipid panels. It may also lead to decreased hemoglobin and platelet counts, which are not 'errors' in the test but actual physiological effects of the drug.
For each major interaction, the management strategy usually involves either avoiding the combination, adjusting the everolimus dose, or increasing the frequency of blood level monitoring (TDM).
> Important: Tell your doctor about ALL medications, supplements, and herbal products you are taking, including over-the-counter vitamins and minerals.
There are several scenarios where everolimus must NEVER be used due to the risk of severe harm or death:
These are conditions where the drug may be used, but only with extreme caution and a careful risk-benefit analysis by the physician:
Patients who have had an allergic reaction to Sirolimus (Rapamune) or Temsirolimus (Torisel) are highly likely to be allergic to everolimus as well. This is because these drugs share a very similar chemical structure (macrolide lactone). If you have ever had a reaction to any 'limus' drug, you must inform your doctor before taking everolimus.
> Important: Your healthcare provider will evaluate your complete medical history, including any past allergic reactions and current infection status, before prescribing Everolimus.
Everolimus is classified by the FDA as having potential for embryo-fetal toxicity (formerly Category D). Based on animal studies and its mechanism of action, everolimus can cause fetal harm when administered to a pregnant woman. It can cross the placenta and interfere with the development of the fetus. Women of childbearing potential must use highly effective contraception during treatment and for at least 8 weeks after the last dose. Male patients with female partners should also use effective contraception for 4 weeks after stopping the drug, as everolimus may be present in sperm and could affect fetal development.
It is not known whether everolimus is excreted in human milk. However, because many drugs are excreted in milk and because of the potential for serious adverse reactions in nursing infants (such as immune suppression and growth issues), women are advised NOT to breastfeed during treatment and for at least 2 weeks after the final dose.
Everolimus is specifically approved for children with Tuberous Sclerosis Complex (TSC) who have SEGA or partial-onset seizures. For these children, it has been shown to be effective in shrinking brain tumors and reducing seizure frequency. However, its safety and efficacy in general pediatric oncology or for preventing organ rejection in children have not been established. Long-term effects on growth and pubertal development are still being monitored in ongoing clinical registries.
Clinical studies of everolimus included a significant number of patients aged 65 and over. While no overall differences in effectiveness were noted compared to younger adults, the incidence of certain side effects—such as anemia, fatigue, and peripheral edema—was higher in the elderly population. Dose selection for an elderly patient should be cautious, usually starting at the low end of the dosing range, reflecting the greater frequency of decreased hepatic, renal, or cardiac function.
Everolimus is not cleared by the kidneys; therefore, no dose adjustment is necessary for patients with renal impairment. It is not removed by hemodialysis. However, everolimus can cause or worsen proteinuria (protein in the urine). If a patient's urinary protein-to-creatinine ratio becomes significantly elevated, the dose may need to be reduced or temporarily held.
This is a critical area for everolimus safety. Because the liver is the primary site of metabolism, any decrease in liver function leads to higher drug levels in the blood.
Frequent blood level monitoring (TDM) is essential in all patients with liver disease.
> Important: Special populations require individualized medical assessment and more frequent monitoring to ensure the drug is used safely and effectively.
Everolimus is an inhibitor of the mammalian target of rapamycin (mTOR), a serine-threonine kinase that is a downstream component of the PI3K/AKT pathway. Specifically, it binds to the intracellular protein FKBP-12, creating a complex that inhibits the mTOR Complex 1 (mTORC1). This inhibition blocks the translation of mRNAs that code for proteins involved in the G1 to S phase transition of the cell cycle. By doing so, it stops the proliferation of tumor cells and vascular endothelial cells. In the immune system, it prevents the cytokine-driven expansion of T-lymphocytes, which is why it is effective in preventing transplant rejection.
The pharmacodynamic effect of everolimus is dose-dependent. Higher doses lead to greater inhibition of p70 S6 kinase, a marker of mTOR activity. The onset of action for immunosuppression is immediate upon reaching steady-state levels (usually within 4-6 days), while the antineoplastic effects (tumor shrinkage) may take weeks or months to become visible on imaging scans. There is no evidence of tolerance development; the drug remains effective as long as the mTOR pathway remains the primary driver of the disease.
| Parameter | Value |
|---|---|
| Bioavailability | ~15-20% (Afinitor) |
| Protein Binding | 92% (primarily to albumin) |
| Half-life | ~30 hours |
| Tmax | 1 to 2 hours |
| Metabolism | Hepatic (CYP3A4 and P-gp) |
| Excretion | Fecal 80%, Renal 5% |
Everolimus is classified as a Kinase Inhibitor and an mTOR Inhibitor Immunosuppressant. It is closely related to sirolimus (Rapamune) and temsirolimus (Torisel). Within the oncology space, it is often grouped with other targeted therapies that interfere with signal transduction pathways rather than traditional cytotoxic chemotherapies.
Medications containing this ingredient
Common questions about Everolimus
Everolimus is a versatile medication used primarily in two medical fields: oncology and transplant medicine. In oncology, it is FDA-approved to treat advanced renal cell carcinoma, certain types of breast cancer, and various neuroendocrine tumors of the pancreas, lungs, and gastrointestinal tract. It is also used for patients with Tuberous Sclerosis Complex (TSC) to treat non-cancerous brain and kidney tumors and certain types of seizures. In transplant medicine, under the brand name Zortress, it is used to prevent the body from rejecting a newly transplanted kidney or liver. Because it targets the mTOR pathway, it is effective at both slowing cancer cell growth and suppressing the immune system's response to foreign tissue. Your doctor will determine the specific use based on your diagnosis and previous treatment history.
The most common side effect of everolimus is stomatitis, which refers to painful inflammation and sores in the mouth and throat, occurring in up to 70% of patients. Other very common side effects include increased susceptibility to infections (like the common cold or urinary tract infections), persistent fatigue, and skin rashes or itchiness. Patients also frequently experience metabolic changes, such as significant increases in blood sugar (hyperglycemia) and blood fats like cholesterol and triglycerides. Gastrointestinal issues, including diarrhea and nausea, are also frequently reported during the first few weeks of therapy. Because many of these side effects can be serious, regular blood tests and medical check-ups are a mandatory part of taking this medication.
There is no known direct, dangerous interaction between everolimus and alcohol that would require absolute avoidance. However, healthcare providers generally recommend limiting alcohol consumption while on this medication for several reasons. First, everolimus is processed by the liver, and alcohol can add additional stress to this organ, potentially increasing the risk of liver-related side effects. Second, alcohol can worsen common everolimus side effects like headaches, dizziness, and nausea. Finally, alcohol can affect blood sugar and lipid levels, which everolimus already tends to elevate. If you choose to drink, it is important to do so in moderation and only after discussing it with your medical team to ensure it is safe for your specific health status.
Everolimus is not considered safe for use during pregnancy and is known to cause fetal harm. Based on its mechanism of action as an mTOR inhibitor, it can interfere with the essential cellular processes required for a fetus to develop normally. Animal studies have shown that it can lead to embryo-fetal death and structural abnormalities. Therefore, women of childbearing age must have a negative pregnancy test before starting the drug and must use highly effective contraception during treatment and for at least 8 weeks after the final dose. Men taking everolimus should also use contraception if they have a female partner of childbearing age. If you become pregnant while taking this medication, you must notify your healthcare provider immediately.
The time it takes for everolimus to work depends on the condition being treated. For transplant patients, the drug begins suppressing the immune system within days of reaching a steady concentration in the blood, which usually takes about 4 to 6 days of consistent dosing. In oncology, the 'work' of the drug is often to stabilize the disease rather than provide an immediate cure. It may take 2 to 4 months of continuous therapy before a follow-up CT scan or MRI shows that a tumor has shrunk or stopped growing. Some patients may notice an improvement in symptoms, such as reduced pain or better breathing, within the first few weeks. It is crucial to continue taking the medication as prescribed, even if you do not feel an immediate difference.
You should never stop taking everolimus suddenly without first consulting your healthcare provider. For transplant recipients, stopping the medication abruptly can lead to acute organ rejection, which is a medical emergency and can result in the loss of the transplanted organ. For cancer patients, stopping the drug can cause the cancer to begin growing or spreading again rapidly. If you are experiencing severe side effects, such as difficulty breathing or a severe rash, your doctor may instruct you to pause the medication, but this must be done under strict medical supervision. Usually, if the drug needs to be discontinued, your doctor will have a plan to transition you to an alternative therapy to ensure your condition remains managed.
If you miss a dose of everolimus, the action you should take depends on how much time has passed since your scheduled dose. If it has been less than 6 hours since you were supposed to take it, take the missed dose immediately and then resume your normal schedule the next day. However, if more than 6 hours have passed, you should skip the missed dose entirely and simply take your next dose at the usual time. Never take two doses at once to 'catch up,' as this can significantly increase the risk of toxic side effects. To help prevent missed doses, many patients find it helpful to use a pillbox or set a daily alarm on their phone, as consistent blood levels are vital for the drug's success.
Weight gain is not a typical direct side effect of everolimus; in fact, weight loss and decreased appetite are more commonly reported in oncology clinical trials. However, everolimus can cause peripheral edema, which is swelling in the arms, hands, legs, or feet due to fluid retention. This fluid buildup can sometimes look like weight gain or cause the number on the scale to increase. Additionally, because everolimus is often taken in combination with corticosteroids (like prednisone) in transplant patients, those patients may experience weight gain due to the steroid component of their regimen. If you notice rapid weight gain or significant swelling, you should contact your doctor, as it may indicate a need to adjust your medications or check your kidney and heart function.
Everolimus has a high potential for drug interactions because it is processed by the CYP3A4 enzyme system in the liver. Many common medications, including certain antibiotics (like clarithromycin), antifungals (like ketoconazole), and blood pressure medicines (like diltiazem), can significantly increase or decrease the levels of everolimus in your blood. Taking it with ACE inhibitors can also increase the risk of a dangerous allergic reaction called angioedema. Because of these risks, it is essential that you provide your doctor and pharmacist with a complete list of every medication, supplement, and herbal product (especially St. John's Wort) that you are taking. Your doctor will then monitor your blood levels and adjust your doses accordingly to prevent dangerous interactions.
Yes, everolimus is available as a generic medication. The FDA approved the first generic versions of Afinitor (the oncology brand) in late 2019 and 2020. Generic versions of Zortress (the transplant brand) are also available. Generic medications are required to have the same active ingredient, strength, dosage form, and route of administration as the brand-name drug, and they must prove 'bioequivalence' to the original. This means the generic version should work the same way in your body as the brand-name version. Using generic everolimus can significantly reduce the cost of treatment for many patients. You should discuss with your doctor or pharmacist whether the generic version is appropriate for your specific treatment plan.