CLINICAL TRIALS PROFILE FOR MYFORTIC

505(b)(2) Clinical Trials for MYFORTIC

This table shows clinical trials for potential 505(b)(2) applications. See the next table for all clinical trials

Trial Type	Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
New Formulation	NCT00374803 ↗	Study of Myfortic in Combination With Tacrolimus and Thymoglobulin in Early Corticosteroid Withdrawal	Completed	Novartis	Phase 4	2006-04-01	To determine the safety and efficacy of a new formulation of Myfortic in combination with tacrolimus and thymoglobulin.
New Formulation	NCT00374803 ↗	Study of Myfortic in Combination With Tacrolimus and Thymoglobulin in Early Corticosteroid Withdrawal	Completed	University of Cincinnati	Phase 4	2006-04-01	To determine the safety and efficacy of a new formulation of Myfortic in combination with tacrolimus and thymoglobulin.
New Combination	NCT03249831 ↗	A Blood Stem Cell Transplant for Sickle Cell Disease	Recruiting	California Institute for Regenerative Medicine (CIRM)	Phase 1	2019-01-04	Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.
New Combination	NCT03249831 ↗	A Blood Stem Cell Transplant for Sickle Cell Disease	Recruiting	City of Hope Medical Center	Phase 1	2019-01-04	Blood stem cells can produce red blood cells (which carry oxygen), white blood cells of the immune system (which fight infections) and platelets (which help the blood clot). Patients with sickle cell disease produce abnormal red blood cells. A blood stem cell transplant from a donor is a treatment option for patients with severe sickle cell disease. The donor can be healthy or have the sickle cell trait. The blood stem cell transplant will be given to the patient as an intravenous infusion (IV). The donor blood stem cells will then make normal red blood cells - as well as other types of blood cells - in the patient. When blood cells from two people co-exist in the patient, this is called mixed chimerism. Most children are successfully treated with blood stem cells from a sibling (brother/sister) who completely shares their tissue type (full-matched donor). However, transplant is not an option for patients who (1) have serious medical problems, and/or (2) do not have a full-matched donor. Most patients will have a relative who shares half of their tissue type (e.g. parent, child, and brother/sister) and can be a donor (half-matched or haploidentical donor). Adult patients with severe sickle cell disease were successfully treated with a half-matched transplant in a clinical study. Researchers would like to make half-matched transplant an option for more patients by (1) improving transplant success and (2) reducing transplanted-related complications. This research transplant is being tested in this Pilot study for the first time. It is different from a standard transplant because: 1. Half-matched related donors will be used, and 2. A new combination of drugs (chemotherapy) that does not completely wipe out the bone marrow cells (non-myeloablative treatment) will be used to prepare the patient for transplant, and 3. Most of the donor CD4+ T cells (a type of immune cells) will be removed (depleted) before giving the blood stem cell transplant to the patient to improve transplant outcomes. It is hoped that the research transplant: 1. Will reverse sickle cell disease and improve patient quality of life, 2. Will reduce side effects and help the patient recover faster from the transplant, 3. Help the patient keep the transplant longer and 4. Reduce serious transplant-related complications.

All Clinical Trials for MYFORTIC

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00101738 ↗	Freedom Study: Myfortic in Kidney Transplant Patients	Completed	Novartis Pharmaceuticals	Phase 3	2003-03-01	The primary objective of the study is to evaluate that 3 immunosuppressant regimens will have comparable kidney function results in kidney transplant patients.
NCT00149968 ↗	Measurement of Patient Reported Gastrointestinal (GI) and Health-related Quality of Life (HRQL) Outcomes in Renal Transplant Recipients (MyLife)	Completed	Novartis	Phase 4	2005-04-01	The purpose of this study is to assess whether a switch from mycophenolate mofetil (MMF) to enteric-coated mycophenolate sodium (EC-MPS) results in improved GI- and/or health-related quality of life outcomes, and to determine the proportion of renal transplant recipients who are experiencing any GI complaints under MMF-based immunosuppressive treatment.
NCT00154310 ↗	Efficacy and Safety of Everolimus With Enteric-Coated Mycophenolate Sodium (EC-MPS) in a Cyclosporine Microemulsion-free Regimen Compared to Standard Therapy in de Novo Renal Transplant Patients	Completed	Novartis	Phase 4	2005-06-01	The purpose of this study is to assess whether a calcineurin inhibitor (CNI)-free regimen with enteric-coated mycophenolate sodium (EC-MPS) and everolimus is as safe and well-tolerated as the standard regimen containing enteric-coated mycophenolate sodium (EC-MPS) and cyclosporine microemulsion, but results in better renal function.
NCT00167492 ↗	Enteric Coated Myfortic for Liver Transplant Recipients	Withdrawn	Novartis	Phase 4	2005-09-01	The purpose of this study is to replace the mycophenolate mofetil (Cellcept) which is our usual therapy after liver transplantation with sodium mycophenolic acid (Myfortic®) and to find out the effect this change may have on the development of side effects such as relief of gastrointestinal (stomach) problems. In the past we have had to stop Cellcept (our current drug) because of these side effects. We will also try to see if improved usage of this drug (Myfortic®) will allow us to use lower doses of other medications that lower your immune system. We will do some special tests on your blood to see if the amount of the drug is related with its effect on the immune system and side effects. Both Cellcept and Myfortic® are FDA approved medications although Myfortic® is not approved for use after liver transplantation. Myfortic® is really the same active drug as Cellcept® (Mycophenolic acid) but has been coated to prevent breakdown of the drug in the stomach and is made to lower the known gastrointestinal effects of Cellcept such as diarrhea, abdominal pain and nausea.
NCT00167492 ↗	Enteric Coated Myfortic for Liver Transplant Recipients	Withdrawn	The University of Texas Health Science Center, Houston	Phase 4	2005-09-01	The purpose of this study is to replace the mycophenolate mofetil (Cellcept) which is our usual therapy after liver transplantation with sodium mycophenolic acid (Myfortic®) and to find out the effect this change may have on the development of side effects such as relief of gastrointestinal (stomach) problems. In the past we have had to stop Cellcept (our current drug) because of these side effects. We will also try to see if improved usage of this drug (Myfortic®) will allow us to use lower doses of other medications that lower your immune system. We will do some special tests on your blood to see if the amount of the drug is related with its effect on the immune system and side effects. Both Cellcept and Myfortic® are FDA approved medications although Myfortic® is not approved for use after liver transplantation. Myfortic® is really the same active drug as Cellcept® (Mycophenolic acid) but has been coated to prevent breakdown of the drug in the stomach and is made to lower the known gastrointestinal effects of Cellcept such as diarrhea, abdominal pain and nausea.

Clinical Trial Conditions for MYFORTIC

Condition Name

Condition Name for MYFORTIC
Intervention	Trials
Kidney Transplantation	21
Renal Transplantation	11
Liver Transplantation	6
Immunosuppression	6

Condition MeSH

Condition MeSH for MYFORTIC
Intervention	Trials
Kidney Failure, Chronic	9
Renal Insufficiency	8
Lymphoma, Non-Hodgkin	6
Lymphoma	6

Clinical Trial Locations for MYFORTIC

Trials by Country

Trials by Country for MYFORTIC
Location	Trials
United States	123
Germany	20
France	15
Canada	14
Spain	12

Trials by US State

Trials by US State for MYFORTIC
Location	Trials
California	14
Pennsylvania	13
New York	9
Florida	8
Wisconsin	7

Clinical Trial Progress for MYFORTIC

Clinical Trial Phase

Clinical Trial Phase for MYFORTIC
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	55
Phase 3	21

Clinical Trial Status

Clinical Trial Status for MYFORTIC
Clinical Trial Phase	Trials
Completed	64
Terminated	20
Unknown status	16

Clinical Trial Sponsors for MYFORTIC

Sponsor Name

Sponsor Name for MYFORTIC
Sponsor	Trials
Novartis Pharmaceuticals	35
Novartis	28
National Institute of Allergy and Infectious Diseases (NIAID)	5

Sponsor Type

Sponsor Type for MYFORTIC
Sponsor	Trials
Other	113
Industry	85
NIH	12

Clinical Trials Update, Market Analysis, and Projection for MYFORTIC (Mycophenolic Acid)

Last updated: October 29, 2025

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Introduction

MYFORTIC (mycophenolic acid delayed-release tablets) is a prescription immunosuppressant primarily indicated for preventing organ rejection in adult and pediatric kidney transplant recipients. Since its approval by the U.S. Food and Drug Administration (FDA) in 2009, MYFORTIC has established a niche within the immunosuppressive therapy landscape. This analysis provides a comprehensive overview of recent clinical trial updates, current market dynamics, and future projections for MYFORTIC, emphasizing its position amid evolving transplant medicine and immunosuppressive protocols.

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Clinical Trials Update

Ongoing Trials and Recent Data

While MYFORTIC currently maintains approval status for kidney transplant rejection prophylaxis, recent clinical investigations focus on optimizing its efficacy, safety, and expanding indications. The most pertinent trials include:

• Pediatric and Adolescent Studies: Trials exploring dose adjustments and safety profiles in pediatric populations are ongoing, emphasizing the drug's safety in younger demographics.

• Comparison with Other Immunosuppressants: Recent phase IV studies compare MYFORTIC directly with other agents like mycophenolate mofetil (CellCept) in terms of efficacy, tolerability, and adverse event profiles, aiming to establish comparative advantages.

• Extended Indications: Trials evaluating MYFORTIC’s potential in lupus nephritis and other autoimmune conditions are under preliminary stages, although none have yet advanced to pivotal phases.

Clinical Trial Outcomes

• Efficacy: Studies reaffirm MYFORTIC's effectiveness in reducing acute rejection episodes post-transplantation, matching or exceeding that of mycophenolate mofetil with comparable safety profiles.

• Safety Profile: Recent data highlight a favorable safety profile, with lower incidences of gastrointestinal disturbances and leukopenia compared to MMF, which supports its role as an alternative in immunosuppressive regimens.

• Adverse Events and Monitoring: Ongoing surveillance aims to better delineate long-term safety, especially regarding infection rates and malignancy risks, consistent with immunosuppressant class effects.

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Market Analysis

Current Market Landscape

MYFORTIC operates within the immunosuppressant segment, dominated by major players such as CellCept (mycophenolate mofetil), tacrolimus, and cyclosporine. According to IQVIA data, the global immunosuppressant market valued at approximately USD 13 billion in 2022, with mycophenolate derivatives accounting for a significant share, estimated at around USD 2.8 billion [1].

MYFORTIC's market share remains modest due to:

• Competitive Pricing: Generics and established brand rivals provide lower-cost alternatives, limiting premium pricing for MYFORTIC.
• Physician Preference: Physicians tend to favor proven agents like MMF, especially given extensive long-term data.
• Regulatory Factors: Differing approval statuses across regions influence global penetration.

Market Drivers and Challenges

• Drivers:

  • Increasing organ transplantation rates globally, notably in Asia-Pacific and Latin America.
  • A growing preference for delayed-release formulations due to improved tolerability.
  • Evolving transplant protocols favoring personalized immunosuppression regimens.

• Challenges:

  • Competition from generics and new immunosuppressants, including belatacept.
  • Regulatory hurdles in expanding indications.
  • Safety concerns surrounding long-term immunosuppression, impacting prescribing habits toward newer agents with better safety profiles.

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Market Projection and Growth Outlook

Forecast Period (2023-2030)

The immunosuppressant drug market is expected to grow at a compound annual growth rate (CAGR) of approximately 4.5-6%, driven by increasing transplantation procedures and demographic shifts.

MYFORTIC’s specific outlook hinges on several factors:

• Market Penetration: Currently holding an estimated 2-3% of the mycophenolate segment, with potential to increase through targeted clinical data supporting broader indications and improved safety profiles.
• Geographical Expansion: Emerging markets, particularly in Asia-Pacific, present growth opportunities, given the rising transplant rates and unmet needs in autoimmune conditions.
• Strategic Initiatives: Incorporation into guidelines as a preferred or alternative immunosuppressant could expand prescriptions.

By 2030, MYFORTIC is projected to capture a larger fraction of the immunosuppressant market, reaching an estimated USD 350-500 million globally, assuming successful clinical trial outcomes and regulatory advancements.

Key Factors Enhancing Growth

• Clinical Validation: Data demonstrating superior tolerability could favor MYFORTIC’s adoption.
• Formulation Innovations: Development of pediatric-friendly or other specialized formulations.
• Regulatory Approvals: Expanding indications like autoimmune diseases could significantly boost revenues.

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Conclusion

MYFORTIC remains a vital player within the transplant immunosuppressant arena. Although its current market share is limited by intense competition and entrenched prescribing habits, ongoing clinical trials and the expanding transplant landscape position MYFORTIC favorably for future growth. Its safety profile, especially regarding gastrointestinal tolerability, coupled with strategic expansions into autoimmune indications, could unlock new demand asymptotes.

The landscape underscores a cautious but optimistic outlook, emphasizing the importance of continuous clinical validation, regulatory engagement, and market expansion strategies.

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Key Takeaways

• MYFORTIC's clinical trials reinforce its non-inferiority to competitors like MMF, with potential safety advantages.
• Market share remains constrained but is poised to grow as transplant indications expand and physicians seek tailored immunosuppressive options.
• Future growth depends on successful integration into clinical guidelines, regulatory approvals for new indications, and strategic geographic expansion.
• The global immunosuppressant market's growth will likely uplift MYFORTIC's revenues, especially in emerging regions.
• Continued surveillance of long-term safety and comparative effectiveness will be critical for sustained market success.

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FAQs

1. Is MYFORTIC superior to mycophenolate mofetil (MMF)?
Clinical trials show comparable efficacy with some evidence of improved gastrointestinal tolerability. However, superiority remains unestablished; choice often depends on physician preference and patient response.

2. What are the main adverse effects associated with MYFORTIC?
Common adverse effects include gastrointestinal disturbances, leukopenia, and increased infection risk — typical of mycophenolate-based therapies.

3. Are there ongoing efforts to expand MYFORTIC's approved indications?
Yes, clinical research is exploring its safety and efficacy in autoimmune diseases such as lupus nephritis, but these indications are not yet approved.

4. How does MYFORTIC’s market penetration compare regionally?
It is more established in North America and Europe, with emerging markets showing increasing adoption due to rising transplantation rates.

5. What is the outlook for MYFORTIC’s growth over the next decade?
With strategic updates, expanded indications, and favorable clinical data, MYFORTIC could see a significant increase in market share, potentially reaching USD 350-500 million globally by 2030.

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References

[1] IQVIA. "Global Immunosuppressants Market Report, 2022."