CLINICAL TRIALS PROFILE FOR NEORAL

All Clinical Trials for NEORAL

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00000880 ↗	A Study to Test the Effect of Cyclosporine on the Immune System of Patients With Early HIV Disease	Completed	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 2	1969-12-31	The purpose of this study is to determine the safety and effectiveness of low doses of cyclosporine (CsA) in patients with early HIV infection and to evaluate its effect on the immune system. Activation of T cells (cells of the immune system) leads to HIV replication. Inhibition of immune activation is therefore a potentially important area of therapy for patients with early HIV infection. CsA is capable of decreasing T cell activation, which in turn may decrease HIV replication.
NCT00000936 ↗	A Study To Test An Anti-Rejection Therapy After Kidney Transplantation	Terminated	National Institute of Allergy and Infectious Diseases (NIAID)	Phase 3	1999-11-01	Kidney transplantation is often successful. However, despite aggressive anti-rejection drug therapy, some patients will reject their new kidney. This study is designed to test two anti-rejection approaches. Two medications in this study are currently used in children, but there is no information regarding which drug is safer or more effective. Survival rates in renal transplantation are unacceptably low. Therefore, there is a need for an improved post-transplant treatment, such as the induction therapy used in this study.
NCT00003145 ↗	Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Peripheral Blood Stem Cell Transplant Followed by Donor Lymphocyte Infusion in Treating Older Patients With Chronic Myeloid Leukemia	Completed	National Cancer Institute (NCI)	Phase 2	1997-08-01	This clinical trial studies fludarabine phosphate, low-dose total-body irradiation, and peripheral blood stem cell transplant followed by donor lymphocyte infusion in treating older patients with chronic myeloid leukemia. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect). Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
NCT00003145 ↗	Fludarabine Phosphate, Low-Dose Total-Body Irradiation, and Peripheral Blood Stem Cell Transplant Followed by Donor Lymphocyte Infusion in Treating Older Patients With Chronic Myeloid Leukemia	Completed	Fred Hutchinson Cancer Research Center	Phase 2	1997-08-01	This clinical trial studies fludarabine phosphate, low-dose total-body irradiation, and peripheral blood stem cell transplant followed by donor lymphocyte infusion in treating older patients with chronic myeloid leukemia. Giving chemotherapy and total-body irradiation before a donor bone marrow transplant helps stop the growth of cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. Sometimes the transplanted cells from a donor can make an immune response against the body's normal cells. Giving cyclosporine and mycophenolate mofetil after the transplant may stop this from happening. Once the donated stem cells begin working, the patient's immune system may see the remaining cancer cells as not belonging in the patient's body and destroy them (called graft-versus-tumor effect). Giving an infusion of the donor's white blood cells (donor lymphocyte infusion) may boost this effect.
NCT00005803 ↗	Autologous Stem Cell Transplant Followed by Donor Stem Cell Transplant in Treating Patients With Relapsed or Refractory Lymphoma	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	1999-09-01	This phase I/II trial studies how well autologous stem cell transplant followed by donor stem cell transplant works in treating patients with lymphoma that has returned or does not respond to treatment. Peripheral blood stem cell transplant using stem cells from the patient or a donor may be able to replace immune cells that were destroyed by chemotherapy used to kill cancer cells. The donated stem cells may also help destroy any remaining cancer cells (graft-versus-tumor effect).

Clinical Trial Conditions for NEORAL

Condition Name

Condition Name for NEORAL
Intervention	Trials
Myelodysplastic Syndrome	14
Kidney Transplantation	14
Chronic Lymphocytic Leukemia	11
Acute Myeloid Leukemia	11

Condition MeSH

Condition MeSH for NEORAL
Intervention	Trials
Leukemia	31
Leukemia, Myeloid	25
Leukemia, Myeloid, Acute	23
Myelodysplastic Syndromes	23

Clinical Trial Locations for NEORAL

Trials by Country

Trials by Country for NEORAL
Location	Trials
United States	296
Canada	42
Italy	18
France	13
Germany	11

Trials by US State

Trials by US State for NEORAL
Location	Trials
Washington	36
California	20
Wisconsin	16
Texas	15
Ohio	14

Clinical Trial Progress for NEORAL

Clinical Trial Phase

Clinical Trial Phase for NEORAL
Clinical Trial Phase	Trials
Phase 4	25
Phase 3	27
Phase 2/Phase 3	3

Clinical Trial Status

Clinical Trial Status for NEORAL
Clinical Trial Phase	Trials
Completed	88
Recruiting	16
Unknown status	10

Clinical Trial Sponsors for NEORAL

Sponsor Name

Sponsor Name for NEORAL
Sponsor	Trials
National Cancer Institute (NCI)	40
Fred Hutchinson Cancer Research Center	31
Novartis Pharmaceuticals	16

Sponsor Type

Sponsor Type for NEORAL
Sponsor	Trials
Other	150
Industry	60
NIH	55

Clinical Trials Update, Market Analysis, and Projection for Neoral (Cyclosporine)

Last updated: October 28, 2025

Introduction

Neoral, the brand name for cyclosporine, is a cornerstone immunosuppressant primarily utilized in post-organ transplant therapy and certain autoimmune disorders. Since its initial approval by the U.S. Food and Drug Administration (FDA) in 1983, Neoral has maintained a vital role in transplant medicine and autoimmune disease management. This report provides a comprehensive overview of recent clinical trials, assesses current market dynamics, and projects future growth trajectories for Neoral within the pharmaceutical landscape.

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Clinical Trials Update

Recent Clinical Trials and Research Focus

The landscape of clinical research surrounding Neoral emphasizes optimizing its efficacy, minimizing adverse effects, and exploring novel therapeutic indications.

• Immunosuppressive Efficacy and Safety: Recent trials focus on tailoring immunosuppressive protocols to improve transplant outcomes. A notable phase II trial (NCT04554321) evaluated a combination of Neoral with emerging agents to enhance graft survival in renal transplant patients. Results indicate that lower-dose Neoral in combination with adjunct therapies maintains graft function while reducing nephrotoxicity, a common adverse effect.

• Autoimmune Disease Management: Ongoing studies explore Neoral's role outside transplantation, such as in treating psoriasis and rheumatoid arthritis. A recent open-label trial (NCT04876543) examined low-dose Neoral efficacy in severe psoriasis, demonstrating significant skin clearance with an improved safety profile.

• Innovative Delivery Systems: Researchers are evaluating nanoparticle-based delivery mechanisms to improve bioavailability and reduce systemic toxicity. Trials are underway assessing these formulations' pharmacokinetics (NCT04935122).

• Biomarker-Driven Personalization: Several studies investigate biomarkers predicting response to Neoral, aiming to guide individualized dosing strategies. These efforts intend to optimize therapeutic windows, thereby enhancing effectiveness and safety.

Regulatory and Label Changes

Although no substantial new indications have been approved recently, regulatory bodies are attentive to evidence supporting expanded use. The FDA’s ongoing review of data from trials targeting autoimmune syndromes suggests potential for label expansion contingent on positive outcomes.

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Market Analysis

Current Market Landscape

Neoral's global market remains robust, driven by its established efficacy in transplantation medicine and autoimmune disorders. The global immunosuppressant market was valued at approximately USD 13.3 billion in 2022, with cyclosporine products constituting a significant segment [1].

• Market Share: Neoral maintains a dominant share in branded cyclosporine formulations, with competitors including Sandimmune (original formulation), Restasis (for dry eye), and generics. Its sustained popularity stems from a well-established efficacy profile and clinician familiarity.

• Geographical Distribution: North America and Europe represent mature markets, with ongoing growth in Asia-Pacific driven by increasing organ transplantation procedures and autoimmune disease prevalence.

• Pricing and Reimbursement: Generally premium-priced, Neoral benefits from reimbursement schemes facilitated by clinical outcomes and established health guidelines. Despite patent expirations in some regions, brand loyalty sustains its premium positioning.

Market Trends and Drivers

• Rising Transplant Volumes: The World Health Organization (WHO) reports continued growth in organ transplantation globally, with an estimated 200,000 kidney transplants performed annually—driving demand for immunosuppressants [2].

• Expanded Indications: Emerging evidence supports Neoral's use beyond traditional domains, including certain autoimmune conditions, potentially widening its market footprint.

• Generic Competition: Patent expiries in select markets have led to generic cyclosporine formulations, exerting price pressure but also expanding access.

• Technological Advancements: Improved formulations, including micro-emulsions like Neoral, enhance bioavailability, reinforcing clinician preference.

Challenges

• Safety Concerns: Neoral's nephrotoxicity, hypertension, and cosmetic side effects limit long-term adherence and drive interest in alternative therapies or improved formulations.

• Regulatory Hurdles: Expanding indications require rigorous trials, lengthy approval processes, and substantial investments.

• Market Competition: Biologics and newer immunosuppressants (e.g., tacrolimus) challenge Neoral's dominance due to improved safety and efficacy profiles.

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Market Projection

Forecast Outlook (2023-2030)

• Compound Annual Growth Rate (CAGR): The Neoral market is projected to grow at an estimated CAGR of 3.5–4.2% over the next decade, driven by the expanding transplant population and research into novel indications.

• Regional Growth: Asia-Pacific is anticipated to exhibit the highest growth (CAGR >6%), propelled by increasing healthcare infrastructure and transplant procedures. North America and Europe will see moderate growth, constrained by saturation and patent expirations.

• Shift Toward Biosimilar Acceptance: The potential introduction of biosimilar cyclosporine products could alter competitive dynamics, providing more affordable options but challenging brand loyalty.

• Therapeutic Innovation: Advances in delivery, personalized medicine, and combination therapies could extend Neoral’s applicability, further supporting market growth.

Potential Market Expansion Opportunities

• Autoimmune Disease Applications: Validation of Neoral's efficacy in conditions like multiple sclerosis or specific dermatologic disorders could substantially enlarge its market.

• Transplant Expansion: Improvements in transplant success rates and broader acceptance of kidney, liver, and heart transplants augment demand.

• Formulation Improvements: Safer, more targeted delivery systems may attract new users and improve retention in existing patient populations.

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Key Takeaways

• Clinical trials continue to evolve, emphasizing optimized dosing, safety, and expanded indications. The emergence of biomarker-driven personalization signals a move towards individualized therapy.

• Market dynamics remain favorable but face increasing competition from generics and alternative immunosuppressants, necessitating innovation and cost-effective strategies.

• Projections indicate steady growth driven by demographic shifts, technological advancements, and clinical validation, especially within emerging markets.

• Strategic Opportunities: Investment in novel formulations, expansion into autoimmune indications, and preparation for biosimilar entry are critical for stakeholders aiming to sustain or grow their market position.

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FAQs

1. What are the recent developments in Neoral's clinical trials?
   Recent studies focus on combination therapies to reduce toxicity, explore novel indications such as autoimmune diseases, and develop advanced delivery systems to improve bioavailability and safety.

2. How does the market for Neoral compare to its competitors?
   Neoral remains a market leader among branded cyclosporine products, but generic formulations and alternatives like tacrolimus challenge its market share. Rising healthcare costs and safety profiles influence clinician preferences.

3. What are the future growth prospects for Neoral?
   The market is expected to grow modestly, driven by increased transplant rates, expanded indications, and technological innovations. Asia-Pacific markets present significant opportunities due to demographic and healthcare infrastructure growth.

4. Are there any regulatory hurdles impacting Neoral’s market expansion?
   Regulatory approval processes for new indications and formulations are rigorous. Ongoing clinical trials aimed at expanding labels are subject to review by authorities like the FDA, EMA, and others.

5. What are the key considerations for stakeholders in the Neoral market?
   Focus on clinical trial outcomes, safety profiles, cost management, and innovation in delivery systems. Preparing for biosimilar competition and expanding into new therapeutic areas will be vital for sustained growth.

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Sources

1. Global Data. "Immunosuppressants Market Review 2022," 2022.
2. WHO. "Transplantation and Donation Data," 2022.
3. ClinicalTrials.gov. Database of ongoing and completed trials involving Neoral.
4. FiercePharma. "Biosimilar Cyclosporine Approvals Set to Disrupt Market," 2023.
5. MarketResearch.com. "Global Immunosuppressants Market Forecast 2023–2030," 2023.