Mycophenolate Mofetil
| Evidence Level: L2 | Predicted Indications: 10 |
Table of Contents
- Mycophenolate Mofetil
- Pharmacist Assessment Report
- Mycophenolate Mofetil: From Transplant Immunosuppression to HIV Infectious Disease
- One-Sentence Summary
- Quick Overview
- Why is This Prediction Reasonable?
- Clinical Trial Evidence
- Literature Evidence
- India Market Information
- Safety Considerations
- Conclusion and Next Steps
- Disclaimer
Mycophenolate Mofetil: From Transplant Immunosuppression to HIV Infectious Disease
One-Sentence Summary
Mycophenolate mofetil is an immunosuppressant that blocks lymphocyte purine synthesis. The data compiled for this report do not include approved-indication text for the Indian licenses. The TxGNN model predicts it may be effective for HIV infectious disease, with 4 related clinical trials (none yielding efficacy data) and 20 publications retrieved, mostly small studies and reviews.
Quick Overview
| Item | Content |
|---|---|
| Original Indication | Not available in the compiled data (the Indian license records contain no indication text) |
| Predicted New Indication | HIV infectious disease |
| TxGNN Prediction Score | 0.9986 |
| Evidence Level | L2 (as assigned in the data; the supporting trials are small and indirect, so in practice the evidence is weaker) |
| India Market Status | Listed in a compiled Indian medicine price dataset (at least 20) – CDSCO approval status is not included in this data |
| Number of Listed Products | at least 20 |
| Recommended Decision | Hold (treated as a research question) |
Why is This Prediction Reasonable?
Detailed mechanism-of-action data are not available in the compiled records. Based on known pharmacology, the active form, mycophenolic acid, inhibits inosine monophosphate dehydrogenase (IMPDH). This depletes guanosine nucleotide pools in lymphocytes.
The link to HIV has two parts. First, less T-cell proliferation limits the pool of activated cells that support HIV replication. Second, shifting intracellular nucleotide ratios (lower dGTP) may boost nucleoside analogs such as abacavir and didanosine. A small case series (PMID 12352149) reported dGTP depletion and a fall in plasma HIV-1 RNA when mycophenolate was added to abacavir-containing regimens.
The rationale has a serious caveat: immunosuppression in an already immunocompromised host. The evidence base is small and dates mostly from 2002–2006.
Clinical Trial Evidence
| Trial Number | Phase | Status | Enrollment | Key Findings |
|---|---|---|---|---|
| NCT00021489 | Phase 2 | Withdrawn | 0 | Safety and antiretroviral activity of mycophenolate added to abacavir; directly on-target in design but never enrolled, so no data |
| NCT00038272 | Phase 2 | Completed | 56 | DAPD vs DAPD plus mycophenolate added to anti-HIV regimens in treatment-experienced patients; indirect relevance |
| NCT00112593 | Not applicable | Completed | 5 | Stem cell transplant in HIV-positive patients, with mycophenolate as part of post-transplant immunosuppression; no antiviral efficacy evidence |
| NCT01288131 | Phase 3 | Terminated | 8 | Cyclosporine plus mycophenolate for anti-erythropoietin pure red cell aplasia; not an HIV trial |
Literature Evidence
| PMID | Year | Type | Journal | Key Findings |
|---|---|---|---|---|
| 15353978 | 2004 | RCT | AIDS | HAART with or without mycophenolate in treatment-naive patients; effect on HIV-1 RNA decay and latent reservoir |
| 15871638 | 2005 | Cohort/PK-PD | Clinical Pharmacokinetics | Low-dose mycophenolate with abacavir, efavirenz and nelfinavir; PK/PD monitoring recommended |
| 16379601 | 2005 | Clinical study | AIDS Research and Human Retroviruses | No detrimental immunological effects of mycophenolate plus HAART in treatment-naive patients |
| 12352149 | 2002 | Case series | J Acquir Immune Defic Syndr | Five patients failing therapy: dGTP depletion and decreased plasma HIV-1 RNA when mycophenolate was added to abacavir regimens |
| 17017956 | 2006 | Review | Current Topics in Medicinal Chemistry | Immunosuppressive drugs in HIV disease, targeting immune activation |
| 16515490 | 2006 | Review | Current Pharmaceutical Design | Virostatics as a means of targeting HIV infection |
| 20840478 | 2010 | Retrospective study | Am J Transplant | Kidney transplantation in 27 HIV-infected patients, with mycophenolate as part of long-term immunosuppression |
| 39396546 | 2025 | Case report | Tropical Doctor | Steroid plus mycophenolate gave complete renal response in IgA vasculitis in an HIV patient |
India Market Information
| Authorization Number | Product Name | Dosage Form | Approved Indication |
|---|---|---|---|
| 178329 | Psien 500 Tablet | Strip of 10 tablets | Not specified |
| 141660 | Mycofet 500mg Tablet | Strip of 10 tablets | Not specified |
| 136873 | Mystead 500 Tablet | Strip of 10 tablets | Not specified |
| 199793 | Seromune 500 Tablet | Strip of 10 tablets | Not specified |
| 134077 | Mycept 250 Capsule | Strip of 10 capsules | Not specified |
There are 20 valid single-ingredient registrations in total, all oral (tablets and capsules).
Safety Considerations
- Contraindications flagged by the knowledge graph (these are graph-derived associations, not label text): neutropenia, agranulocytosis, aplastic anemia and related inherited marrow-failure syndromes (e.g. Fanconi anemia, Diamond-Blackfan anemia), cancer including lymphoma and skin cancer, active peptic ulcer disease, liver disease, gallbladder disease, and serious infections such as cytomegalovirus infection, herpes simplex, herpes zoster and progressive multifocal leukoencephalopathy.
- Drug Interactions: 92 interactions were identified. Examples:
- Major: activated charcoal.
- Moderate: proton pump inhibitors (omeprazole, esomeprazole, lansoprazole, rabeprazole, dexlansoprazole), antibiotics (amoxicillin, clarithromycin, doxycycline, minocycline, tetracycline, vancomycin, kanamycin, paromomycin), and antacids (aluminum hydroxide, calcium carbonate, magnesium oxide).
- Minor: zinc salts and iron.
- Immunosuppression is a particular concern in an HIV-infected population (opportunistic infection risk), and the infections listed above overlap with the graph-derived contraindications.
Please refer to the package insert for label warnings and contraindications, as these were not available in the compiled data.
Conclusion and Next Steps
Decision: Hold (research question)
Rationale: The TxGNN score is high and the mechanism (IMPDH inhibition, potentiation of nucleoside analogs) is plausible. However, the only directly relevant HIV trial was withdrawn with no participants, the evidence consists of small, dated studies and reviews, and immunosuppression in HIV carries real risk. Established antiretroviral therapy leaves little room for this approach outside research settings.
To proceed, the following is needed:
- Indian package insert text (indications, warnings, contraindications), which is currently missing
- Detailed mechanism-of-action data
- Review of the 2004 RCT (PMID 15353978) and the other small studies for virological and immunological outcomes
- Clarification of the intended HIV population and a risk assessment of opportunistic infection and marrow suppression
- A new adequately designed trial if the hypothesis is pursued
Other predicted indications: Multiple sclerosis (score 0.9954) has the most supporting evidence among the remaining candidates, including several small Phase 1/2 trials of mycophenolate with interferon beta-1a, a Cochrane review of mycophenolate in relapsing-remitting MS, and a 344-patient retrospective study. It remains a research question because no adequately powered Phase 3 trial exists and established disease-modifying therapies are available. The other predictions (neurodevelopmental disorder, Paget disease, feline and simian immunodeficiency, hemosiderosis, Heiner syndrome, African iron overload, neonatal hemochromatosis) have little or no supporting evidence and are on hold.
Disclaimer
This content is for research purposes only and does not constitute medical advice. Clinical validation is required before any clinical application.