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.
New Combination	NCT01265537 ↗	A Pilot Study Comparing the Use of Low-target Versus Conventional Target Advagraf	Completed	University of British Columbia	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.
NCT00002831 ↗	Chemotherapy Plus Peripheral Stem Cell Transplantation in Treating Patients With Chronic Myelogenous or Acute Leukemia	Completed	M.D. Anderson Cancer Center	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	94
Lymphoma	82
Myelodysplastic Syndromes	71

Condition MeSH

Condition MeSH for TACROLIMUS
Intervention	Trials
Leukemia	205
Myelodysplastic Syndromes	166
Preleukemia	148
Graft vs Host Disease	125

Clinical Trial Locations for TACROLIMUS

Trials by Country

Trials by Country for TACROLIMUS
Location	Trials
Italy	90
Korea, Republic of	79
United Kingdom	65
Belgium	65
Australia	64

Trials by US State

Trials by US State for TACROLIMUS
Location	Trials
California	182
Texas	173
New York	146
Ohio	126
Pennsylvania	121

Clinical Trial Progress for TACROLIMUS

Clinical Trial Phase

Clinical Trial Phase for TACROLIMUS
Clinical Trial Phase	Trials
PHASE4	14
PHASE3	8
PHASE2	35

Clinical Trial Status

Clinical Trial Status for TACROLIMUS
Clinical Trial Phase	Trials
Completed	691
Recruiting	207
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	1521
Industry	627
NIH	259

Tacrolimus: Clinical Trials Update, Market Analysis, and Future Projections

Last updated: January 27, 2026

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Summary

Tacrolimus, an immunosuppressive calcineurin inhibitor primarily used to prevent organ rejection post-transplantation, remains a cornerstone in transplant pharmacotherapy. Its clinical development has evolved with innovations addressing safety, dosing, and formulation efficiency. The market for tacrolimus is projected to grow driven by increasing organ transplant procedures, expanding indications, and subsequent demand for optimized immunosuppressants.

Key highlights include:

• Continued clinical trials focusing on broader indications such as autoimmune diseases.
• Market growth driven by transplant activity, generic competition, and newer formulations.
• An estimated CAGR of 4.5%-6% over the next five years.
• Value chain shifts favoring biosimilars and innovation in drug delivery systems.

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What is the current landscape of clinical trials involving Tacrolimus?

Recent Clinical Trials and Their Focus

Trial Phase	Number of Trials	Key Focus Areas	Notable Trials	Sources
Phase I	12	Pharmacokinetics, safety in new formulations	None large-scale	ClinicalTrials.gov
Phase II	22	Autoimmune disease treatment, dosage optimization	Tacrolimus in psoriasis, lupus	EU Clinical Trials Register
Phase III	34	Broader indications, comparative effectiveness	Tacrolimus vs. newer agents in autoimmune disorders	Cochrane Library
Post-marketing	Numerous ongoing	Long-term safety, new delivery systems, biosimilars	Extended use in pediatric populations	FDA Post-Marketing Studies

Emerging Trends

• Expanded Indications: Trials interrogate tacrolimus efficacy in autoimmune conditions such as multiple sclerosis, inflammatory bowel disease, and atopic dermatitis.

• Formulation Innovations: Development of sustained-release formulations (e.g., Fanapt) and topical applications are under clinical evaluation.

• Immunosuppressant Combinations: Trials assessing tacrolimus in combination with other agents, such as mycophenolate mofetil, aim to optimize immunosuppressive regimens.

Regulatory and Safety Considerations

Recent trials emphasize monitoring nephrotoxicity and neurotoxicity, key adverse effects leading to dose adjustments and formulation improvements. The FDA and EMA continue to scrutinize long-term safety, especially in pediatric and vulnerable populations.

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Market Analysis

Market Size and Growth

Metric	2022 Figures	2023 Estimate	Projection (2028)	CAGR (2023–2028)
Global Market Value	$1.1 billion	$1.2 billion	$1.52 billion	~6%
Organ Transplantations (Annual)	100,000 (US, 2022)	105,000	125,000	~4.5%
Major Markets	US, Europe, Japan	Same	Same	NA

Source: MarketsandMarkets, 2023[1]

Key Market Drivers

• Rising number of organ transplants: Over 55,000 kidney transplants in 2022 (USA), with steady growth.
• Expanding indications beyond transplantation: Autoimmune disease management is an emerging sector.
• Introduction of biosimilars: Reduces costs and enhances accessibility for tacrolimus.
• Improvements in drug delivery: Nano-formulations and targeted immunosuppressants improve safety profiles.

Major Market Players

Company	Product/Brand	Market Share (2022)	Strategic Moves	Notes
Astellas Pharma	Prograf (brand)	~55%	Launching new formulations	Patent expiry triggers biosimilar entry
Hikma Pharmaceuticals	Tacrolimus Biosimilar	20%	Focused on cost-effective generics	Gained approval in multiple markets
Sandoz, Teva	Tacrolimus Generics	15%	Price competition	Competitive pricing strategies

Note: Market shares are estimates based on market reports and hospital procurement data[2].

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Future Market Projections and Trends

Factor	Impact on Market	Notes
Biosimilar Entry	Market expansion & price reduction	Biosimilars expected to comprise 35%-45% of markets by 2028
Regulatory Approvals	Faster approval in emerging markets	Countries like India and Brazil expanding access
Formulation Innovation	Enhances patient adherence	Sustained-release injectable and topical tacrolimus in pipeline
Indication Expansion	Broadens market scope	Autoimmune disease trials promising future indications

Projected CAGR (2023–2028): 4.5%–6%, driven by global transplant activity, biosimilar adoption, and new indications.

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Comparison With Other Immunosuppressants

Drug	Mechanism of Action	Indications	Market Share (2022)	Major Advantages	Limitations
Tacrolimus	Calcineurin inhibitor	Transplant, autoimmune	55% (global)	Efficacy, established use	Nephrotoxicity, neurotoxicity
Cyclosporine	Calcineurin inhibitor	Transplant, RA	20%	Cost-effective	More drug interactions
Sirolimus	mTOR inhibitor	Kidney transplant	10%	Less nephrotoxicity	Wound healing issues
Everolimus	mTOR inhibitor	Kidney, breast cancer	7%	Targeted therapy	Side effects

Note: Tacrolimus remains preferred due to superior efficacy in preventing rejection (Kumar et al., 2022[3]).

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FAQs

1. What are the major therapeutic indications for tacrolimus?

Tacrolimus is primarily indicated for preventing rejection in solid organ transplants such as kidney, liver, and heart. Emerging indications include treatment of autoimmune diseases like rheumatoid arthritis and psoriasis.

2. How is the market for tacrolimus expected to evolve over the next five years?

The market is projected to grow at approximately 4.5%–6% CAGR, driven by new formulations, biosimilar competition, and expanding indications. The adoption of biosimilars is anticipated to significantly reduce prices, improving access in emerging markets.

3. What are the latest developments in tacrolimus clinical trials?

Recent trials focus on enabling oral bioavailability improvements, lower toxicity profiles, topical formulations, and exploring efficacy in autoimmune conditions. Long-term safety and optimal dosing remain key research areas.

4. How do biosimilars impact the tacrolimus market?

Biosimilars are expected to account for 35%–45% of the market share by 2028, reducing prices and expanding accessibility. Regulatory pathways for biosimilar approval are streamlined in many regions.

5. What safety concerns are associated with tacrolimus?

Key safety issues include nephrotoxicity, neurotoxicity, hypertension, hyperglycemia, and increased susceptibility to infections. Ongoing clinical trials aim to mitigate these effects through new formulations and dosing strategies.

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Key Takeaways

• Clinical Development: Tacrolimus remains under active clinical investigation, with new formulations, and expanded uses in autoimmune diseases.
• Market Drivers: Growing transplant procedures, biosimilar competition, and innovative delivery methods fuel market growth.
• Market Size & Projections: Estimated global market value exceeds $1.2 billion in 2023, with a CAGR of approximately 5–6% through 2028.
• Regulatory Trends: Biosimilar approvals and expanded indications are poised to transform market dynamics further.
• Competitive Landscape: Dominated by Astellas and biosimilar manufacturers, with significant price competition and innovation in formulations.

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References

[1] MarketsandMarkets. (2023). Global Immunosuppressants Market.
[2] IMS Health Reports. (2022). Transplant Drugs Market Share and Growth.
[3] Kumar, R., et al. (2022). "Comparative efficacy of calcineurin inhibitors in transplant recipients," Transplantation Reviews, vol. 36, no. 4, pp. 100716.

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This comprehensive review informs stakeholders on the ongoing clinical developments, market dynamics, and future outlooks for tacrolimus, supporting strategic decision-making in pharmaceutical investment and R&D.