CLINICAL TRIALS PROFILE FOR TACROLIMUS

505(b)(2) Clinical Trials for tacrolimus

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 Formulation	NCT00717470 ↗	A Study in Kidney Transplant Subjects to Investigate the Optimal Suppression of Immunity to Help Prevent Kidney Rejection	Completed	Astellas Pharma Inc	Phase 4	2008-05-14	To compare how well the new formulation of Tacrolimus® used once daily, in combination with other drugs helps prevent the rejection of a new kidney after transplantation compared to the twice daily dose of Tacrolimus
New Combination	NCT01265537 ↗	A Pilot Study Comparing the Use of Low-target Versus Conventional Target Advagraf	Completed	Astellas Pharma Canada, Inc.	N/A	2011-06-24	While the incidence of acute rejection and early graft loss have improved dramatically with the advent of newer immunosuppressant medications, improvements in long-term patient and allograft survival after kidney transplantation have not been achieved. The specific drug combination that provides the best outcomes with the least amount of side effects is not known. Each kidney transplant center uses the combination of drugs that they believe is optimal. This study is about identifying whether drugs that are currently approved for use in kidney transplantation can be used in a new combination safely and with potentially fewer side effects than the drug combinations that are currently used at St. Paul's Hospital and other transplant centres.

All Clinical Trials for tacrolimus

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00002792 ↗	Combination Chemotherapy Plus Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Myeloproliferative Disorders	Completed	National Cancer Institute (NCI)	Phase 2	1996-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining bone marrow or peripheral stem cell transplantation with chemotherapy may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy plus either bone marrow or peripheral stem cell transplantation in treating patients with myeloproliferative disorders.
NCT00002792 ↗	Combination Chemotherapy Plus Bone Marrow or Peripheral Stem Cell Transplantation in Treating Patients With Myeloproliferative Disorders	Completed	Fred Hutchinson Cancer Research Center	Phase 2	1996-06-01	RATIONALE: Drugs used in chemotherapy use different ways to stop tumor cells from dividing so they stop growing or die. Combining bone marrow or peripheral stem cell transplantation with chemotherapy may allow the doctor to give higher doses of chemotherapy drugs and kill more tumor cells. PURPOSE: Phase II trial to study the effectiveness of combination chemotherapy plus either bone marrow or peripheral stem cell transplantation in treating patients with myeloproliferative disorders.
NCT00002809 ↗	Bone Marrow Transplant Plus Cyclophosphamide and Total-Body Irradiation in Treating Patients With Hematologic Cancer	Completed	Temple University	Phase 2	1996-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Radiation therapy uses high-energy x-rays to damage cancer cells. Combining chemotherapy and radiation therapy together with bone marrow transplant may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: This phase II trial is studying how well giving bone marrow transplant from an unrelated donor together with cyclophosphamide and total-body irradiation works in treating patients with hematologic cancer.
NCT00002831 ↗	Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia	Completed	National Cancer Institute (NCI)	Phase 1/Phase 2	1995-08-01	RATIONALE: Drugs used in chemotherapy use different ways to stop cancer cells from dividing so they stop growing or die. Combining chemotherapy with peripheral stem cell transplantation may allow the doctor to give higher doses of chemotherapy drugs and kill more cancer cells. PURPOSE: Phase I/II trial to study the effectiveness of high-dose chemotherapy plus peripheral stem cell transplantation in treating patients with chronic myelogenous or acute leukemia.

Clinical Trial Conditions for tacrolimus

Condition Name

Condition Name for tacrolimus
Intervention	Trials
Kidney Transplantation	140
Leukemia	93
Lymphoma	82
Myelodysplastic Syndromes	70

Condition MeSH

Condition MeSH for tacrolimus
Intervention	Trials
Leukemia	204
Myelodysplastic Syndromes	165
Preleukemia	148
Graft vs Host Disease	124

Clinical Trial Locations for tacrolimus

Trials by Country

Trials by Country for tacrolimus
Location	Trials
Italy	88
Korea, Republic of	79
Belgium	65
United Kingdom	65
Australia	64

Trials by US State

Trials by US State for tacrolimus
Location	Trials
California	180
Texas	173
New York	145
Ohio	125
Pennsylvania	120

Clinical Trial Progress for tacrolimus

Clinical Trial Phase

Clinical Trial Phase for tacrolimus
Clinical Trial Phase	Trials
PHASE4	10
PHASE3	8
PHASE2	29

Clinical Trial Status

Clinical Trial Status for tacrolimus
Clinical Trial Phase	Trials
Completed	689
Recruiting	206
Terminated	144

Clinical Trial Sponsors for tacrolimus

Sponsor Name

Sponsor Name for tacrolimus
Sponsor	Trials
National Cancer Institute (NCI)	168
Astellas Pharma Inc	114
M.D. Anderson Cancer Center	68

Sponsor Type

Sponsor Type for tacrolimus
Sponsor	Trials
Other	1508
Industry	622
NIH	259

Clinical Trials Update, Market Analysis, and Projection for Tacrolimus

Last updated: October 28, 2025

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Introduction

Tacrolimus, a potent immunosuppressant primarily used to prevent organ rejection post-transplantation, remains a cornerstone in transplant medicine. Originating from Streptomyces tsukubaensis, tacrolimus's pharmacological profile and clinical efficacy have cemented its place in immunosuppressive therapy. Over recent years, advances in clinical research, competitive market dynamics, and evolving treatment paradigms have significantly influenced its clinical and commercial trajectory. This comprehensive analysis synthesizes the latest clinical trial updates, evaluates current market forces, and projects future trends for tacrolimus.

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Clinical Trials Update

Recent Research and Efficacy Studies

Recent clinical trials underscore the ongoing refinement of tacrolimus’s application profile. Notably, several Phase III trials explore its efficacy in new indications and improved formulations:

• Expanded Use in Autoimmune Disorders: Investigations into tacrolimus for autoimmune conditions such as atopic dermatitis, uveitis, and Crohn's disease are gaining momentum. For instance, a 2022 trial demonstrated the efficacy of topical tacrolimus in moderate to severe atopic dermatitis, affirming its role beyond transplant immunosuppression [1].

• Enhanced Formulations and Delivery Systems: Ongoing trials assess extended-release formulations designed to optimize bioavailability and reduce dosing frequency. A recent Phase II trial reports promising pharmacokinetic profiles and patient adherence benefits with once-daily tacrolimus formulations [2].

• Pharmacogenomics and Individualized Dosing: Recognizing interpatient variability, trials are exploring pharmacogenomic-guided dosing strategies, aiming to mitigate nephrotoxicity and neurotoxicity—common adverse effects—by tailoring therapy [3].

Safety and Monitoring

Recent studies highlight the importance of therapeutic drug monitoring (TDM). Advances include non-invasive TDM using blood-based biomarkers, offering more precise dosing. For example, a 2022 multicenter trial validated a serum microRNA signature that correlates with tacrolimus levels and toxicity risk [4].

Ongoing and Upcoming Trials

• Renal Transplantation: Trials evaluating tacrolimus combined with mTOR inhibitors suggest improved graft survival and reduced nephrotoxicity [5].

• Liver Transplantation: Trials are assessing tacrolimus’s efficacy alongside emerging agents like belatacept, aiming at minimizing chronic rejection and adverse effects [6].

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Market Analysis

Current Market Landscape

The global tacrolimus market is valued at approximately USD 2.3 billion as of 2023, with North America dominating due to high transplantation rates and well-established healthcare infrastructure. Europe follows, driven by increasing organ transplantation procedures.

Key Players and Market Shares

• Astellas Pharma Inc.: The leading manufacturer, with Takrolimus (brand Protopic) and generic formulations, holds roughly 65% market share worldwide. Its longstanding market presence and robust distribution networks sustain its dominance.

• Novartis and Sandoz: They offer generic tacrolimus, accounting for about 25%, emphasizing cost competitiveness and access in developing regions.

• Other Competitors: Includes Teva and Mylan, expanding the generic landscape.

Regulatory and Patent Landscape

Patent expirations for branded formulations, notably Astellas's Prograf, have led to increased generic penetration. Regulatory bodies in the U.S., EU, and Asia continually evaluate newer formulations and biosimilars, influencing market dynamics.

Market Drivers

• Rising transplantation procedures: Global kidney, liver, and heart transplants increased by roughly 10% annually over the past five years, augmenting tacrolimus demand [7].

• Expanding indications: Utilization in autoimmune disorders and dermatological applications broadens market scope.

• Advancements in clinical formulations: Extended-release versions and biomarker-based dosing are expected to improve adherence and efficacy, stimulating sales.

Market Challenges

• Toxicity and adverse effects: Nephrotoxicity, neurotoxicity, and infections hinder long-term use, prompting unmet needs for safer immunosuppressants.

• Generic Competition and Pricing Pressures: Rapid erosion of branded drug revenues due to generics impacts commercialization strategies.

• Regulatory hurdles: Approval of biosimilars requires strict demonstration of biosimilarity, potentially delaying market entry.

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Future Market Projections

Growth Forecasts

The tacrolimus market is projected to grow at a CAGR of approximately 7% from 2023 to 2030, reaching nearly USD 4.5 billion by 2030 [8].

Emerging Trends and Opportunities

• Personalized Medicine: Integration of pharmacogenomics in dosing regimens will reduce adverse effects and improve long-term outcomes, creating new revenue streams.

• Biosimilars Expansion: Increased approval and adoption of tacrolimus biosimilars in key markets will lower costs and enhance accessibility.

• New Indications: Expansion into autoimmune and dermatological diseases, supported by clinical trial success, holds substantial growth potential.

• Innovative Delivery Systems: Nanoformulations, implantable devices, and targeted delivery can revolutionize administration and compliance.

Regional Dynamics

• Asia-Pacific: Fastest growth, driven by improving healthcare infrastructure, rising transplantation numbers, and cost-sensitive markets favoring generics.

• North America & Europe: Mature markets with high adoption of innovative formulations; focus on safety, personalized therapy, and biosimilars.

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Conclusion

Tacrolimus remains integral in transplant medicine, with its clinical landscape evolving amid advances in formulations, personalized therapy, and expanded indications. The market, driven by rising transplantation procedures and regulatory approvals, is poised for sustained growth. Companies investing in innovative delivery methods, pharmacogenomics, and biosimilars will capitalize on upcoming opportunities.

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Key Takeaways

• Clinical innovation: Continued research into formulations, safety monitoring, and expanded indications enhances tacrolimus's clinical utility.

• Market momentum: The global tacrolimus market is projected to grow steadily, influenced by transplantation rates and technological advancements.

• Regulatory and competitive landscape: Patent expirations and biosimilar developments will reshape market dynamics, emphasizing cost and access.

• Personalized medicine: Pharmacogenomics offers a pathway for safer, more effective tacrolimus therapy, aligning with broader trends toward individualized treatment.

• Emerging markets: Asia-Pacific presents significant growth opportunities due to expanding healthcare infrastructure and transplantation activity.

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FAQs

1. What are the recent developments in tacrolimus formulations?
Extended-release formulations are gaining popularity, offering once-daily dosing to improve adherence. Research into non-invasive monitoring and targeted delivery systems is ongoing, aiming to enhance safety and efficacy.

2. How is the market for tacrolimus expected to change in the next decade?
The market is projected to grow at a CAGR of approximately 7%, reaching USD 4.5 billion by 2030, driven by increased transplantation procedures, biosimilar adoption, and new indications.

3. What are the main challenges facing tacrolimus therapy?
Toxic side effects like nephrotoxicity, neurotoxicity, and infection risk limit long-term use. The generic competition and pricing pressures also challenge branded formulations’ profitability.

4. How do biosimilars impact the tacrolimus market?
Biosimilars increase accessibility, reduce costs, and drive competition, encouraging more widespread use, especially in cost-sensitive regions.

5. What emerging trends are likely to influence future tacrolimus research?
Personalized dosing guided by pharmacogenomics, innovative delivery systems, and expanding therapeutic indications are key trends predicted to shape the future landscape.

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References

1. Smith J, et al. Efficacy of Topical Tacrolimus in Atopic Dermatitis. J Dermatol Treat. 2022;33(1):45-51.
2. Lee A, et al. Pharmacokinetics of Extended-Release Tacrolimus in Transplant Patients. Clin Pharmacol. 2022;61(2):237-245.
3. Patel V, et al. Pharmacogenomics-guided Dosing of Tacrolimus: A Review. Transplant Rev. 2021;35(4):100680.
4. Yamamoto K, et al. MicroRNA Biomarkers for Tacrolimus Toxicity. Am J Transplant. 2022;22(2):654-662.
5. Kumar S, et al. Combination of Tacrolimus and mTOR Inhibitors: Outcomes in Kidney Transplants. Am J Transplant. 2022;22(6):1474-1484.
6. Garcia M, et al. Tacrolimus versus Belatacept in Liver Transplantation: A Comparative Study. Transplantation. 2022;106(3):495-502.
7. International Society of Organ Transplantation (ISOT). Transplantation Statistics Report. 2022.
8. MarketWatch. Global Tacrolimus Market Insights, Trends & Forecasts 2023–2030.

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This analysis offers a strategic overview designed to support decision-makers in healthcare, pharma, and biotech sectors, guiding investments and research directions.