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	119
Germany	20
France	15
Canada	14
Spain	12

Trials by US State

Trials by US State for Myfortic
Location	Trials
California	13
Pennsylvania	13
New York	8
Florida	7
Wisconsin	7

Clinical Trial Progress for Myfortic

Clinical Trial Phase

Clinical Trial Phase for Myfortic
Clinical Trial Phase	Trials
Phase 4	55
Phase 3	21
Phase 2/Phase 3	1

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
Sidney Kimmel Cancer Center at Thomas Jefferson University	5

Sponsor Type

Sponsor Type for Myfortic
Sponsor	Trials
Other	113
Industry	85
NIH	11

Myfortic: Clinical Trials, Market Analysis, and Projections

Introduction

Myfortic, also known as enteric-coated mycophenolate sodium (EC-MPS), is a crucial immunosuppressant drug used primarily in the prevention of acute rejection in organ transplant patients. This article delves into the clinical trials, market analysis, and future projections for Myfortic.

Clinical Trials Overview

De Novo and Conversion Trials in Renal Transplant Patients

In clinical trials involving renal transplant patients, Myfortic has been compared to mycophenolate mofetil (MMF), another commonly used immunosuppressant. In the de novo trial, patients were administered either Myfortic 1.44 grams per day or MMF 2 grams per day, in combination with cyclosporine and corticosteroids, for 12 months. The results showed similar efficacy and safety profiles between the two treatments[1].

In the conversion trial, patients who were at least 6 months post-transplant and receiving MMF were randomized to either Myfortic 1.44 grams per day or MMF 2 grams per day for 12 months. Again, the outcomes indicated that Myfortic was therapeutically similar to MMF with a comparable safety profile[1].

Heart Transplant Trials

A single-blind, multicenter trial involving de novo heart transplant recipients compared EC-MPS (Myfortic) 1,080 mg twice daily to MMF 1,500 mg twice daily. The study found that the incidence of biopsy-proven and treated acute rejection, graft loss, or death was similar for both treatments at 6 and 12 months. The overall safety and tolerability of EC-MPS were also comparable to MMF[4].

Market Analysis

Current Market Size and Growth

The transplant immunosuppressant drugs market, which includes Myfortic, is significant and growing. As of 2024, the market size was estimated to be USD 6.22 billion and is projected to reach USD 10.86 billion by 2035, with a Compound Annual Growth Rate (CAGR) of 5.2% between 2025 and 2035[2].

Regional Market Dynamics

North America is currently the largest market for transplant immunosuppressant drugs, while the Asia Pacific region is expected to be the fastest-growing market. This growth is driven by increasing healthcare expenditures, a rising number of transplant procedures, and advancements in immunosuppressive therapies[2].

Key Market Drivers

The growth in the mycophenolate mofetil market, which includes Myfortic, is driven by several factors:

• Increasing Need in Healthcare: The growing demand for effective immunosuppressants in the pharmaceutical and healthcare sectors.
• Advancements in Personalized Medicine: Developments in personalized medicine and gene therapy are paving the way for more tailored and effective immunosuppressant treatments.
• Expansion into Autoimmune Diseases: The increasing focus on treating autoimmune diseases is also expanding the market for immunosuppressant drugs[2][5].

Market Projections

Mycophenolate Mofetil Market

The global mycophenolate mofetil market, which includes Myfortic, was valued at USD 1487.9 million in 2021 and is expected to reach USD 2735.54 million by 2031, exhibiting a CAGR of 6.28% during this period. This growth is attributed to the increasing adoption of mycophenolate mofetil in various transplant procedures and its expanding use in treating autoimmune diseases[5].

Future Opportunities

Key opportunities in the transplant immunosuppressant drugs market include:

• Development of Novel Drugs: The development of new immunosuppressive drugs with fewer side effects and better efficacy.
• Personalized Medicine: Advances in personalized medicine and gene therapy that allow for tailored immunosuppressant treatments.
• Autoimmune Disease Treatments: The increasing focus on treating autoimmune diseases, which expands the market for immunosuppressant drugs[2].

Competitive Landscape

Top Companies

The mycophenolate mofetil market is competitive, with several key players including:

• Par Pharmaceutical
• West Ward Pharmaceuticals
• Mylan
• Jubilant Cadista
• Teva
• Alkem Laboratories
• Strides Pharma
• Genentech
• Accord Healthcare
• Sandoz
• Akorn
• Passauer Pharma GmbH[5].

Regulatory and Safety Considerations

Safety Profile

Clinical trials have consistently shown that Myfortic has a comparable safety profile to MMF. The drug's enteric coating delays the release of mycophenolic acid, which can reduce gastrointestinal side effects. However, as with any immunosuppressant, there are risks associated with increased susceptibility to infections and other adverse effects[1][4].

Regulatory Landscape

The regulatory landscape for immunosuppressant drugs is stringent, with ongoing monitoring and evaluation by regulatory bodies such as the U.S. Food and Drug Administration (FDA). Compliance with regulatory requirements is crucial for the approval and continued use of these drugs.

Conclusion

Myfortic, as an enteric-coated mycophenolate sodium, has established itself as a viable and effective immunosuppressant in the prevention of acute rejection in transplant patients. With a strong clinical trial background and a growing market, Myfortic is poised to continue playing a significant role in the transplant immunosuppressant drugs market.

Key Takeaways

• Clinical Efficacy: Myfortic has shown similar efficacy and safety profiles to MMF in various clinical trials.
• Market Growth: The transplant immunosuppressant drugs market is expected to grow significantly, reaching USD 10.86 billion by 2035.
• Regional Dynamics: North America is the largest market, while the Asia Pacific region is the fastest-growing.
• Market Drivers: Increasing healthcare needs, advancements in personalized medicine, and expanding use in autoimmune diseases drive market growth.
• Competitive Landscape: Several key players are active in the mycophenolate mofetil market.

FAQs

What is the expected market size of the transplant immunosuppressant drugs market by 2035?

The transplant immunosuppressant drugs market is expected to reach USD 10.86 billion by 2035[2].

How does Myfortic compare to MMF in clinical trials?

Myfortic has been shown to have similar efficacy and safety profiles to MMF in various clinical trials involving renal and heart transplant patients[1][4].

What are the key drivers of the mycophenolate mofetil market?

The key drivers include the growing need in the pharmaceutical and healthcare sectors, advancements in personalized medicine, and the increasing focus on treating autoimmune diseases[2][5].

Which regions are expected to see significant growth in the transplant immunosuppressant drugs market?

North America is currently the largest market, while the Asia Pacific region is expected to be the fastest-growing[2].

What are the potential future opportunities for Myfortic and similar immunosuppressants?

Future opportunities include the development of novel drugs with fewer side effects, advances in personalized medicine, and the expanding use in treating autoimmune diseases[2].