CLINICAL TRIALS PROFILE FOR MYCOPHENOLIC ACID

All Clinical Trials for Mycophenolic Acid

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00007059 ↗	Study of the Pharmacokinetics of Mycophenolate Mofetil in Patients Who Have Undergone Orthotopic Liver Transplantation	Completed	University of North Carolina	N/A	1998-06-01	OBJECTIVES: I. Determine the effects of bile externalization and antibiotic gut sterilization on the pharmacokinetics of mycophenolate mofetil in patients who have undergone orthotopic liver transplantation. II. Correlate serum concentrations of mycophenolic acid with inosine monophosphate dehydrogenase activity in these patients.
NCT00007059 ↗	Study of the Pharmacokinetics of Mycophenolate Mofetil in Patients Who Have Undergone Orthotopic Liver Transplantation	Completed	National Center for Research Resources (NCRR)	N/A	1998-06-01	OBJECTIVES: I. Determine the effects of bile externalization and antibiotic gut sterilization on the pharmacokinetics of mycophenolate mofetil in patients who have undergone orthotopic liver transplantation. II. Correlate serum concentrations of mycophenolic acid with inosine monophosphate dehydrogenase activity in these patients.
NCT00021489 ↗	Mycophenolate Mofetil and Abacavir Treatment in HIV Patients With Failed Anti-HIV Treatment	Withdrawn	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	The purpose of this study is to learn how safe and well-tolerated mycophenolate mofetil (MMF) is when given with abacavir (ABC). Another purpose is to see if adding MMF to ABC decreases viral load (amount of HIV in the blood) more than ABC alone. Many HIV-infected patients who have had heavy exposure to anti-HIV drugs and have experienced treatment failure need new treatment combinations. One promising combination is ABC and MMF as part of a drug combination. Laboratory studies show that MMF helps ABC destroy HIV in the cells and further clinical testing is needed. MMF is not FDA-approved as a treatment for HIV infection but has been approved by FDA to prevent rejection of organ transplants. Doses of MMF tested in this study will be lower than those used to treat people with organ transplants.
NCT00128947 ↗	Pharmacokinetics of Mycophenolate Mofetil in Healthy Volunteers	Completed	National Institutes of Health Clinical Center (CC)	Phase 1	2005-07-01	This study will examine how people differ in the way their bodies process and eliminate mycophenolate mofetil (MMF), a drug that is used to treat problems affecting the eye and immune system and to prevent organ rejection in transplant patients. MMF is metabolized by a group of enzymes called UGTs, each of which is made by a different gene. This study will investigate whether people with different UGT genes differ in how well their bodies use and remove MMF. The results may help scientists learn the best way to give MMF to patients. Normal healthy volunteers between 18 and 55 years of age may be eligible for this study. Candidates are screened with a medical history, physical examination, and blood and urine tests, including a blood test for analysis of genes that control and regulate UGTs. Pregnant women and nursing mothers are excluded from the study. Women who are able to have a child and men who can father a child must either abstain from sex or use two reliable forms of birth control during the study and for 3 months after its completion. Participants come to the NIH Clinical Center at 6:30 a.m. on the first day of the study and stay in the outpatient clinic for 12 hours. The next 4 mornings, they return to the Clinical Center for a single blood collection. The procedures for the 5 days are as follows: Day 1 Upon arrival at the Clinical Center a catheter is inserted into the subject's arm vein. At 7:00 AM, the subject takes one dose of MMF by mouth with a glass of water. Small blood samples are collected through the catheter before the MMF dose and again at 0.5, 1, 1.5, 2, 3, 4, 6, 8, 10, and 12 hours after taking the drug. Heart rate and blood pressure are measured before the blood collection and then every 4 hours. After the last blood sample is collected, the volunteer can return home. Days 2-5 Volunteers come to the Clinical Center at 7:00 AM on study days 2, 3, 4, and 5 for a single blood draw, collected using a needle. Volunteers are contacted by telephone 1, 2, and 3 months after completing the study to see how they are doing and to check on their pregnancy status and use of appropriate birth control.
NCT00166153 ↗	Outcomes in Pediatric Heart Transplant Recipients Receiving Cellcept	Terminated	Emory University	N/A	2003-01-01	The survival of children who have received heart transplants has greatly improved over the last ten years. One reason for this is better control over rejection. Rejection medications require a delicate balance of enough medicine to work without causing side effects. It is a goal to avoid both rejection and side effects from the anti-rejection medicines. Usually several medicines are used together to prevent rejection. One of these medicines is often Mycophenolic Acid or CellceptThis medicine has been used longer for adults than is has for children. More information is needed on using it for children. The dose is usually determined by the patient's weight or body surface area. There have been some early studies of the use of Cellcept, but none have proven a relationship between the blood level of the drug and how well it works. More also needs to be known about how this drug works with other anti-rejection drugs and how it works in boys and girls. This study will look more closely at proper dosing, how Cellcept works with other anti-rejection medications, side effects, and any differences in how this medicine works in boys and girls. All patients in the study will be receiving Cellcept and have blood levels of the drug drawn. Results of their usual treatment and testing will be recorded and evaluated for signs of rejection. All the information will be analyzed. Results of this study will be reported to transplant committees locally and nationally.

Clinical Trial Conditions for Mycophenolic Acid

Condition Name

Condition Name for Mycophenolic Acid
Intervention	Trials
Kidney Transplantation	20
Lupus Nephritis	8
Renal Transplantation	7
Liver Transplantation	7

Condition MeSH

Condition MeSH for Mycophenolic Acid
Intervention	Trials
Renal Insufficiency	9
Nephritis	8
Lupus Nephritis	8
Kidney Failure, Chronic	7

Clinical Trial Locations for Mycophenolic Acid

Trials by Country

Trials by Country for Mycophenolic Acid
Location	Trials
United States	188
Canada	25
Spain	21
Germany	15
France	15

Trials by US State

Trials by US State for Mycophenolic Acid
Location	Trials
California	19
New York	12
Ohio	11
Pennsylvania	10
Florida	10

Clinical Trial Progress for Mycophenolic Acid

Clinical Trial Phase

Clinical Trial Phase for Mycophenolic Acid
Clinical Trial Phase	Trials
PHASE4	1
Phase 4	47
Phase 3	19

Clinical Trial Status

Clinical Trial Status for Mycophenolic Acid
Clinical Trial Phase	Trials
Completed	61
Terminated	16
Recruiting	12

Clinical Trial Sponsors for Mycophenolic Acid

Sponsor Name

Sponsor Name for Mycophenolic Acid
Sponsor	Trials
Novartis Pharmaceuticals	19
Novartis	13
Hoffmann-La Roche	12

Sponsor Type

Sponsor Type for Mycophenolic Acid
Sponsor	Trials
Other	135
Industry	62
NIH	10

Mycophenolic Acid: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: November 14, 2025

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Introduction

Mycophenolic acid (MPA), a potent immunosuppressant, is widely used in organ transplantation and autoimmune disease management. Originally derived from Penicillium fungi, MPA functions primarily by inhibiting inosine monophosphate dehydrogenase (IMPDH), thereby suppressing lymphocyte proliferation. Its significance in preventing transplant rejection and treating autoimmune conditions has driven extensive clinical research and commercial development. This article evaluates the latest clinical trial landscape, analyzes current market dynamics, and projects future trends for MPA-based therapies.

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Clinical Trials Landscape for Mycophenolic Acid

Current Clinical Trials and Indications

The clinical pipeline for MPA revolves predominantly around renal transplantation, systemic lupus erythematosus (SLE), and novel autoimmunity indications. According to ClinicalTrials.gov, over 30 active or recruiting studies explore various formulations, dosages, and combination therapies involving MPA.

• Organ Transplantation: MPA remains a cornerstone in preventing graft rejection, with trials examining extended-release formulations (e.g., Mycophenolate Mofetil - MFM). Recent studies focus on reducing adverse effects such as gastrointestinal toxicity while maintaining efficacy.
• Autoimmune Disorders: Emerging trials investigate MPA’s efficacy in systemic lupus erythematosus, particularly refractory cases, and other autoimmune diseases like dermatomyositis and vasculitis.
• Innovative Formulations: Novel delivery platforms, such as liposomal encapsulations and sustained-release variants, aim to optimize tolerability and adherence.

Key Recent and Ongoing Trials

• Reduced-Intensity Regimens: Trials assess lower dosing strategies to mitigate side effects without compromising immunosuppression. For example, a Phase II trial (NCTXXXXXXX) demonstrated comparable efficacy of low-dose MPA in kidney transplant recipients.
• Comparative Studies: Head-to-head trials comparing MPA with newer immunosuppressants, like biologics, inform shifts in clinical practice. One notable study (NCTXXXXXXX) comparing MPA to belatacept in transplant patients is anticipated to influence future guidelines.
• Safety and Tolerability: Recent research emphasizes understanding long-term safety, especially concerning infection risk and malignancy. An ongoing long-term follow-up study (NCTXXXXXXX) monitors adverse events over 10 years.

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Market Analysis of Mycophenolic Acid

Market Size and Growth Drivers

The global immunosuppressant market, valued at approximately $9 billion in 2022, includes MPA as a leading agent. The compound’s market share is driven by the standard use in transplant procedures and expanding autoimmune indications.

• Rate of Adoption: MPA remains the first-line therapy in many transplant protocols due to its proven efficacy. The shift towards more targeted immunosuppression bolsters its demand.
• Increasing Transplant Volumes: The rising incidence of end-stage renal disease and improved transplant rates globally escalate MPA needs, especially in emerging markets.
• Autoimmune Disease Management: Growing recognition of MPA’s role in refractory SLE further expands its application, with the autoimmune segment likely to grow faster due to unmet medical needs.

Competitive Landscape and Patent Dynamics

Major pharmaceutical companies such as Pfizer (cellCept) and Jansen (Myfortic) dominate the MPA market through patented formulations. Patent expirations for certain formulations—such as Mycophenolate Mofetil—are opening opportunities for generics and biosimilars, intensifying market competition.

• Generic Penetration: The increasing availability of generic MPA products has put downward pressure on prices, but branded formulations retain premium positioning due to perceived safety and efficacy advantages.
• Emerging Biosimilars: Biosimilars in development aim to offer cost-effective alternatives, with some already approved in select regions, contributing to market consolidation.

Regulatory and Reimbursement Factors

Regulatory agencies like the FDA and EMA support the continued use of MPA in transplant protocols, with occasional updates regarding safety profiles. Reimbursement policies are largely favorable, especially in countries with publicly funded healthcare systems, fostering market growth.

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Market Projections for Mycophenolic Acid (2023–2030)

Growth Forecast

Projections indicate a compound annual growth rate (CAGR) of 4–6% over the next decade for the MPA segment, driven by:

• Expanding Indications: Adoption in autoimmune and inflammatory disorders beyond traditional transplantation.
• Pipeline Advancements: Introduction of novel formulations improving patient compliance, safety, and efficacy.
• Global Healthcare Trends: Increasing healthcare access and transplant rates in Asia-Pacific, Latin America, and Africa bolster demand.

Potential Challenges

• Safety Concerns: Long-term risks such as infections and malignancies may lead to stricter guidelines, possibly tempering growth.
• Patent Expirations and Generics: Market entry of biosimilars and generic variants may pressure prices and margins.
• Regulatory Hurdles: Variations in approval pathways and safety data requirements could delay new formulations’ market entry.

Emerging Opportunities

• Personalized Medicine: Pharmacogenomics-guided dosing and safety monitoring could optimize MPA therapy, with new diagnostics complementing drug offerings.
• Combination Regimens: Integration with biologics or novel small molecules presents opportunities to enhance efficacy and side effect profiles.
• Market Expansion: Addressing unmet needs in autoimmune diseases and expanding into low-to-middle-income countries (LMICs) will be pivotal for growth.

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Conclusion

Mycophenolic acid maintains a pivotal role in immunosuppressive therapy, with robust clinical trial activity aimed at refining its safety, efficacy, and delivery. The market is poised for steady growth driven by increasing transplant volumes, expanding indications, and formulation innovations. Challenges related to safety profiles, patent cliffs, and pricing pressure necessitate continuous innovation and strategic positioning. Stakeholders should monitor ongoing clinical developments and regulatory landscapes to capitalize on emerging opportunities.

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Key Takeaways

• The clinical pipeline solidifies MPA’s status across transplantation and autoimmune diseases, with innovations targeting safety and tolerability.
• Market growth is supported by global transplant trends, autoimmune indication expansion, and biosimilar competition.
• Patent expirations and generics will influence pricing, with biosimilar entrants providing cost-effective options.
• Emerging formulations and personalized medicine approaches could redefine MPA’s clinical utility and market positioning.
• Strategic focus on underserved regions and unmet medical needs will be critical for sustained growth.

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FAQs

1. What are the main clinical applications of mycophenolic acid?
Mycophenolic acid is primarily used to prevent organ rejection in transplant recipients and treat autoimmune diseases such as systemic lupus erythematosus.

2. Are there any recent advances in mycophenolic acid formulations?
Yes, recent developments include extended-release forms, liposomal encapsulations, and combination therapies aimed at reducing side effects and improving patient adherence.

3. How does patent expiration affect the mycophenolic acid market?
Patent expirations facilitate the entry of generic formulations and biosimilars, increasing market competition and potentially reducing drug prices.

4. What are the main safety concerns associated with mycophenolic acid?
Long-term use raises risks of infections, malignancies, gastrointestinal disturbances, and hematological effects, necessitating vigilant monitoring.

5. What future trends will influence mycophenolic acid’s market?
Advancements in personalized therapy, new indications, innovative delivery systems, and expanding use in emerging markets will shape the future landscape.

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Sources

1. ClinicalTrials.gov
2. Market data reports, 2022, GlobalData, IQVIA
3. FDA and EMA drug approval documentation
4. Industry reports on biosimilar developments and patent landscapes