Mycophenolate mofetil is an immunosuppressant medicine used to help prevent the body's immune system from rejecting a transplanted organ. It belongs to a class of medicines called antimetabolites and is the prodrug form of the same active substance found in mycophenolate sodium. It is a prescription-only medicine.

• Therapeutic Class: Immunosuppressant (systemic).

• Pharmacological Class: Inosine monophosphate dehydrogenase (IMPDH) inhibitor.

• Drug Family: Mycophenolic acid derivatives (antimetabolite immunosuppressants).

• ATC Classification: L04AA06 (Mycophenolic acid).

Approved: Prevention of organ rejection in adult and pediatric kidney, heart, and liver transplant recipients, used in combination with cyclosporine or tacrolimus and corticosteroids.

Commonly used for: Transplant recipients as part of a standard multi-drug immunosuppressive regimen.

Off-label use (only under medical guidance): Used in various autoimmune conditions (e.g., lupus nephritis, certain vasculitides) under specialist guidance, though this is not the core approved indication.

● Oral capsules: 250 mg.

● Oral tablets: 500 mg.

● Oral suspension: for patients unable to take tablets/capsules, particularly children.

● Injection (intravenous): used short-term when oral dosing isn't possible.

Mycophenolate mofetil is converted in the body to its active form, mycophenolic acid, which blocks an enzyme called inosine monophosphate dehydrogenase (IMPDH). Lymphocytes (a type of immune cell central to organ rejection) need this enzyme to produce new DNA building blocks for cell division. By blocking this pathway, mycophenolate mofetil selectively reduces the proliferation of these immune cells, helping prevent them from attacking the transplanted organ.

• Standard dose (kidney transplant, adults): 1 g taken orally or intravenously twice daily (a total daily dose of 2 g).

• Condition-wise dose (heart or liver transplant, adults): 1.5 g taken orally or intravenously twice daily (a total daily dose of 3 g).

• Route: Oral (capsule, tablet, or suspension) or intravenous infusion; oral dosing is generally on an empty stomach, though some flexibility exists based on tolerability.

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

• Overdose: May increase the risk of infection or low blood counts; requires medical attention.

• Common: Diarrhoea, nausea, vomiting, and low blood counts (particularly white blood cells).

• Serious: Increased risk of infections (including opportunistic infections) and certain cancers (including lymphoma and skin cancers) due to immune suppression, and significant birth defects/pregnancy loss risk if used during pregnancy.

• Emergency symptoms: Signs of serious infection (fever, chills), unusual bruising or bleeding, or severe, persistent diarrhoea.

• Recovery advice: Regular blood count monitoring helps catch low white cell counts early, allowing dose adjustment before infection risk becomes significant.

• Pregnancy: Contraindicated except in specific, carefully considered circumstances; effective contraception is essential, as detailed in the boxed warning above.

• Breastfeeding: Not recommended during treatment.

• Liver disease: Used with routine monitoring; no formal dose adjustment established for hepatic impairment.

• Kidney disease: Used with caution in patients with severe chronic kidney graft dysfunction; dose adjustment may be needed.

• Elderly: Used with caution, given a potentially higher risk of certain side effects, including infection, in this age group.

• Children: Approved for pediatric kidney transplant recipients, with body-surface-area-based dosing.

● Antacids containing magnesium and aluminum: reduce mycophenolate mofetil absorption; dosing should be separated.

● Cholestyramine and other bile acid-binding drugs: can reduce mycophenolic acid absorption, potentially reducing effectiveness.

● Azathioprine: not typically combined, as both are antimetabolite immunosuppressants with overlapping toxicity (particularly on blood counts).

● Live vaccines: should generally be avoided during treatment due to the immunosuppressive effect.

● Complete blood counts regularly, particularly in the first months of treatment.

● Kidney function and liver function tests periodically.

● Pregnancy testing before starting in patients who could become pregnant, given the boxed warning.

● Monitoring for signs of infection throughout treatment.

Mycophenolate sodium (a related enteric-coated formulation of the same active substance), Azathioprine (an older antimetabolite immunosuppressant).

Used specifically in combination with cyclosporine or tacrolimus and corticosteroids as part of the standard triple-immunosuppression regimen for transplant rejection prevention; not typically combined with azathioprine.

● Organ Transplant / Anti-Rejection Medicines

● Antimetabolite Immunosuppressants

● Transplant Supportive Care

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1. https://www.accessdata.fda.gov/drugsatfda_docs/label

2. https://www.drugs.com/dosage/cellcept.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.