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Rapamycin

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Rapamycin (sirolimus) is an immunosuppressant used to prevent organ rejection and manage specific autoimmune conditions. It helps by dampening the body’s natural defence system to prevent it from attacking healthy tissue. The medication works by inhibiting the activation of T-cells, which are key components of the immune response.


Ingredient
Availability
In Stock
Delivery Time
Airmail (14-21 days) | EMS trackable (5-9 days)
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Product Sheet

Active Ingredient(s)
Sirolimus
Reference Brand
Rapamune
Manufacturer
Pfizer
Product Form
Tablet, Oral solution
Regulatory Classification
Rx
Primary Category
Transplant Medicine, Immunosuppressant
Product Category
Selective immunosuppressants, mTOR inhibitors
Pharmacological Class
Mammalian Target of Rapamycin (mTOR) Inhibitors
Manufacturer Description
A strong immunosuppressant used to prevent the body from rejecting a kidney transplant.
Mechanism of Action
It works by inhibiting a protein called mTOR, which is key to the activation and growth of T-cells in the immune system. By blocking this process, the medicine prevents the immune system from attacking and rejecting a transplanted organ.
Route of Administration
Oral
Onset Time
Several days to reach stable levels
Duration
More than 24 hours
Contraindications
Hypersensitivity to sirolimus, Severe liver disease, Pre-existing high lung toxicity
Severe Adverse Events
Increased risk of infection, Slow wound healing, Lung inflammation, Increased risk of lymphoma
Common Side Effects
Acne, Mouth ulcers, Joint pain, Nausea
Uncommon Side Effects
High blood pressure, Anaemia, High cholesterol, Urinary tract infections
Drug Interactions
Grapefruit juice, Ketoconazole, Rifampicin, Ciclosporin
Pregnancy Safety Warnings
Should only be used if the benefit outweighs the risk.
Age Restrictions
Adults and children over 13 (under medical supervision)
Storage Guidelines
Tablets: room temperature. Solution: Refrigerate 2-8°C.
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Rapamycin FAQ

Can Rapamycin be used to treat rheumatoid arthritis?

Current UK labeling does not include rheumatoid arthritis as an approved indication. Some small studies suggest potential benefit, but these remain experimental. Use for rheumatoid arthritis would be off-label and should only occur within a clinical trial or specialist-led protocol.

How often should blood levels of sirolimus be checked?

After the initial dose and any subsequent adjustment, trough levels are typically measured 7-10 days later, then every 1-3 months for stable patients. More frequent testing may be required after major changes in therapy or if side effects arise.

Will Rapamycin interact with common antihypertensive drugs?

Most antihypertensives (e.g., ACE inhibitors, beta-blockers) do not have significant pharmacokinetic interactions with sirolimus. However, drugs that affect CYP3A4 (e.g., certain calcium-channel blockers) may alter sirolimus levels and warrant monitoring.

Is it safe to receive a COVID-19 vaccine while on Rapamycin?

Vaccination is generally recommended for immunosuppressed patients, but the immune response may be attenuated. Timing the vaccine at least two weeks after a stable dose adjustment can help optimize efficacy; discuss scheduling with your clinician.

What should I do if I develop a mouth ulcer while taking Rapamycin?

Mouth ulcers are a common side effect. Rinsing with a salt-water solution, using topical anesthetic gels, and maintaining good oral hygiene often provide relief. Persistent or severe ulcers should be reported for possible dose modification.

Can I travel internationally with Rapamycin?

Yes, but keep the medication in its original packaging with a copy of the prescription. Carry a letter from your doctor explaining the need for a controlled medication, and be aware of import regulations of the destination country.

Does Rapamycin affect blood sugar levels?

Sirolimus can cause insulin resistance and elevate blood glucose, potentially unmasking diabetes in susceptible individuals. Routine monitoring of fasting glucose or HbA1c is advisable, especially in patients with a history of diabetes.

Are there any known differences between generic sirolimus tablets and the Rapamycin brand?

Both contain the same active ingredient, sirolimus, and must meet the same bioequivalence standards set by the MHRA. Inactive excipients may differ slightly, which can affect tolerability for some patients.

If ambient temperatures exceed 30 °C, store the tablets in a cool, dry place - for example, a refrigerator (2 °C-8 °C) if instructed by the pharmacist. Avoid freezing and protect the medication from moisture.

Can Rapamycin be taken with grapefruit juice?

Grapefruit juice inhibits CYP3A4, potentially raising sirolimus concentrations and increasing the risk of toxicity. Patients are advised to avoid large amounts of grapefruit or grapefruit juice while on Rapamycin.

What Is Rapamycin?

Rapamycin, marketed under the name Rapamycin, contains sirolimus as its active ingredient. Sirolimus belongs to a class of drugs known as mTOR inhibitors and is primarily used as an immunosuppressant. In the United Kingdom, Rapamycin is a prescription-only medication (POM) regulated by the Medicines and Healthcare products Regulatory Agency (MHRA). It is supplied as a 1 mg pill.

How Rapamycin Works in the Body

Sirolimus blocks the activity of the mechanistic target of rapamycin (mTOR) pathway, a key regulator of cell growth, proliferation, and survival. By inhibiting mTOR:

  • T-cell activation is reduced, diminishing the immune response that can lead to organ rejection or autoimmune inflammation.
  • Protein synthesis and cytokine production are curtailed, helping to control pathological immune activity.

The drug is absorbed from the gastrointestinal tract, reaches peak concentrations within 1-2 hours, and has a long elimination half-life of approximately 60 hours, allowing once-daily dosing in most patients.

Conditions Treated by Rapamycin

Approved Indications (UK)

  • Prevention of organ transplant rejection - used in combination with other immunosuppressants to maintain graft tolerance.
  • Lymphangioleiomyomatosis (LAM) - a rare lung disease affecting mainly women, where sirolimus slows disease progression.

These indications are supported by MHRA-approved labeling and clinical trial data.

Off-Label and Investigational Applications

Research studies have explored sirolimus for several autoimmune disorders, including systemic lupus erythematosus, systemic sclerosis, and certain vasculitides. Evidence ranges from small open-label trials to case series. Because these uses are not approved by the MHRA, they are considered off-label and should only be undertaken under specialist supervision.

Off-label disclaimer: Off-label use requires medical supervision and individualized risk assessment.

Who Should Use Rapamycin? Contraindications and Cautions

Absolute Contraindications

  • Known hypersensitivity to sirolimus or any excipients in the tablet.
  • Uncontrolled severe infections (e.g., active tuberculosis).
  • Pregnant women - sirolimus is classified as Category D in the UK, indicating evidence of fetal risk. Use is prohibited unless the potential benefit justifies the risk.
  • Breastfeeding - the drug is excreted in milk and may harm the infant.

Relative Contraindications

  • Renal impairment - dosing may need adjustment; severe dysfunction (e.g., CrCl < 30 mL/min) warrants caution.
  • Hepatic impairment - moderate liver disease may increase drug exposure.
  • Elderly patients - increased susceptibility to infections and hematologic effects.
  • Concomitant use of strong CYP3A4 inhibitors (e.g., ketoconazole) or inducers (e.g., rifampicin) may require dose modification.

Special Populations

  • Pregnancy & Lactation: Avoid unless absolutely necessary; discuss risks with a specialist.
  • Pediatric Use: Not routinely approved; occasional off-label use in children with LAM or transplant must be managed by a pediatric transplant team.

Safety Profile: Side Effects and Interactions

Common Side Effects

  • Mouth ulcers - reported in up to 20 % of patients; usually mild and respond to topical therapy.
  • Hyperlipidaemia - elevations in cholesterol and triglycerides; lipid monitoring is advised.
  • Delayed wound healing - may affect surgical recovery; surgeons often pause therapy before major procedures.
  • Mild nausea or diarrhoea - generally transient.

Serious Adverse Events

  • Infections - opportunistic infections (e.g., Pneumocystis jirovecii pneumonia) can be life-threatening; prophylactic antibiotics may be prescribed.
  • Hematologic toxicity - thrombocytopenia, anemia, or leukopenia requiring dose reduction or temporary discontinuation.
  • Renal dysfunction - acute kidney injury, especially when combined with calcineurin inhibitors.
  • Pulmonary toxicity - non-infectious interstitial lung disease, a rare but serious condition.

Drug Interactions

Major Interactions

  • CYP3A4 inhibitors (e.g., itraconazole, clarithromycin) increase sirolimus plasma levels → risk of toxicity.
  • CYP3A4 inducers (e.g., carbamazepine, St John’s wort) decrease levels → loss of efficacy.

Moderate Interactions

  • P-glycoprotein inhibitors (e.g., verapamil) may modestly raise concentrations.
  • Concurrent immunosuppressants (e.g., tacrolimus) require careful therapeutic drug monitoring to avoid additive toxicity.

Interaction guidance: Inform your healthcare provider of all prescription medicines, over-the-counter products, herbal supplements, and vitamins before starting Rapamycin.

Food and Lifestyle Interactions

  • Food: Take the pill consistently with or without food; avoid large variations that could affect absorption.
  • Alcohol: Moderate consumption is permissible, but excessive intake may exacerbate liver toxicity.
  • Driving: Sirolimus does not impair cognition, but severe infection or anemia could affect alertness; assess personal fitness to operate machinery.

Dosing and Administration Guidelines

  • Standard dose: 1 mg taken orally once daily, adjusted to achieve target trough plasma concentrations (typically 5-15 ng/mL for transplant patients).
  • Titration: Dose adjustments are guided by therapeutic drug monitoring (TDM) and clinical response; the 1 mg tablet allows flexible titration in 0.5 mg increments when lower strengths are needed.
  • Special populations:
  • Renal impairment: Start with a reduced dose (e.g., 0.5 mg daily) and monitor levels closely.
  • Hepatic impairment: Consider a 25 % dose reduction.
  • Elderly: Initiate therapy at the lowest effective dose.

Administration tips

  • Swallow the pill whole with a full glass of water.
  • Do not crush or split the tablet unless a healthcare professional advises otherwise.
  • Store at room temperature, protected from moisture and direct sunlight.

Missed dose: Take the missed dose as soon as remembered unless it is close to the time of the next scheduled dose; do not double up.

Overdose: Symptoms may include profound immunosuppression, severe infection, or organ dysfunction. Seek emergency medical care; supportive care and, if needed, temporary cessation of immunosuppressants are the mainstays of treatment.

Discontinuation: Abrupt cessation can precipitate rebound immune activation. Tapering is recommended, especially after long-term use, under physician guidance.

Monitoring and Follow-Up

  • Therapeutic drug monitoring: Trough levels measured 7-10 days after initiation or dose change, then at regular intervals (e.g., monthly).
  • Laboratory tests: CBC, serum creatinine, liver function tests, lipid profile, and fasting glucose every 1-3 months.
  • Clinical assessment: Check for signs of infection, wound healing problems, and pulmonary symptoms at each visit.

Storage and Handling

  • Keep the 1 mg tablets in the original container, tightly closed.
  • Store at 15 °C-30 °C (room temperature) away from excess heat, moisture, and light.
  • Do not use the medication after the expiry date printed on the package.
  • Dispose of unused tablets via a pharmacy take-back program or as directed by local waste-management guidelines.

Medication-Specific Glossary

mTOR (mechanistic Target Of Rapamycin)
A cellular kinase that regulates growth, protein synthesis, and immune cell activation; inhibition suppresses immune responses.
Trough Level
The lowest concentration of a drug in the bloodstream, measured just before the next dose; used to ensure therapeutic efficacy while minimizing toxicity.
Therapeutic Drug Monitoring (TDM)
Laboratory testing of drug concentrations to guide dosing adjustments, essential for drugs with narrow therapeutic windows like sirolimus.
Hyperlipidaemia
Elevated levels of cholesterol or triglycerides in the blood, a common side effect of mTOR inhibition.
Immunosuppression
Deliberate reduction of the immune system’s activity to prevent organ rejection or control autoimmune inflammation.
Toxicity Threshold
The drug concentration above which adverse effects become more likely; for sirolimus, this is generally considered > 15 ng/mL in transplant patients.

Medical Disclaimer

This article provides educational information about Rapamycin and is not a substitute for professional medical advice. Treatment decisions, including use for unapproved indications, must be made under the guidance of a qualified healthcare provider. The content is intended for informational purposes and does not constitute medical recommendations. Always consult a physician before starting, stopping, or changing any medication regimen.

Information for Rapamycin is sourced from established medical standards and updated frequently. It is provided for general guidance and should be confirmed with a healthcare professional before use.
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