CLINICAL TRIALS PROFILE FOR URIDINE TRIACETATE

All Clinical Trials for URIDINE TRIACETATE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01432301 ↗	Uridine Triacetate as Antidote for Patients at Excess Risk of 5-FU Toxicity Due to Overdosage or Impaired Elimination	Approved for marketing	Wellstat Therapeutics		1969-12-31	The purpose of this study is to provide emergency treatment of adult and pediatric patients: - Following a fluorouracil or capecitabine overdose regardless of the presence of symptoms or - Who exhibit early-onset, severe or life-threatening toxicity affecting the cardiac or central nervous systems, and/or early-onset, unusually severe adverse reactions (e.g., gastrointestinal toxicity and/or neutropenia) within 96 hours following the end of fluorouracil or capecitabine administration.
NCT02110147 ↗	Open-Label Study of Uridine Triacetate in Pediatric Patients With Hereditary Orotic Aciduria	Completed	Wellstat Therapeutics	Phase 3	2014-04-01	This protocol has two parts - the Main Study which is 42 days in length and the Treatment Extension which allows the patients who complete the Main Study to continue receiving treatment with uridine triacetate. The purpose of this study is to replace oral administration of uridine with oral administration of uridine triacetate in patients with hereditary orotic aciduria who have received (or would reasonably be expected to receive) clinical benefit from treatment with exogenous uridine. It is also to document the continued clinical benefit of exogenous uridine when patients are switched from oral administration of uridine to oral administration of uridine triacetate.
NCT07032142 ↗	Dose Optimization and Efficacy Assessment of a Fluoropyrimidine Antidote	NOT_YET_RECRUITING	Conselho Nacional de Desenvolvimento Cientfico e Tecnolgico	PHASE1	2025-07-01	Fluoropyrimidines (FLU) are drugs widely used in chemotherapy for various tumors, such as breast, colon, rectal, and gastric cancers. FLU is a drug that inhibits thymine synthesis and, consequently, DNA synthesis, leading to tumor cell death. However, up to 30% of patients treated with FLU experience severe toxicities, depending on the dose and regimen received. The most common symptoms include mucositis, vomiting, nausea, diarrhea, and neutropenia. The enzyme dihydropyrimidine dehydrogenase (DPD) plays a key role in FLU metabolism. Patients with mutations in the DPYD gene (which encodes DPD) are at high risk of experiencing severe toxicities from FLU. Uridine triacetate (UT) is a drug that can be used as an antidote for 5-FU in patients who develop severe toxicities. However, despite its efficacy, it is expensive and not commercially available in Brazil. Currently, the Brazilian population has no access to an antidote for the treatment of FLU-related toxicities. This Phase I/II study will evaluate the dose, safety, and efficacy of compound the association of two molecules as an antidote for grade 3 or higher toxicities resulting from the use of FLU.
NCT07032142 ↗	Dose Optimization and Efficacy Assessment of a Fluoropyrimidine Antidote	NOT_YET_RECRUITING	D'Or Institute for Research and Education	PHASE1	2025-07-01	Fluoropyrimidines (FLU) are drugs widely used in chemotherapy for various tumors, such as breast, colon, rectal, and gastric cancers. FLU is a drug that inhibits thymine synthesis and, consequently, DNA synthesis, leading to tumor cell death. However, up to 30% of patients treated with FLU experience severe toxicities, depending on the dose and regimen received. The most common symptoms include mucositis, vomiting, nausea, diarrhea, and neutropenia. The enzyme dihydropyrimidine dehydrogenase (DPD) plays a key role in FLU metabolism. Patients with mutations in the DPYD gene (which encodes DPD) are at high risk of experiencing severe toxicities from FLU. Uridine triacetate (UT) is a drug that can be used as an antidote for 5-FU in patients who develop severe toxicities. However, despite its efficacy, it is expensive and not commercially available in Brazil. Currently, the Brazilian population has no access to an antidote for the treatment of FLU-related toxicities. This Phase I/II study will evaluate the dose, safety, and efficacy of compound the association of two molecules as an antidote for grade 3 or higher toxicities resulting from the use of FLU.

Clinical Trial Conditions for URIDINE TRIACETATE

Condition Name

Condition Name for URIDINE TRIACETATE
Intervention	Trials
Toxicity Due to Chemotherapy	2
Cancer	1
Hereditary Orotic Aciduria	1

Condition MeSH

Condition MeSH for URIDINE TRIACETATE
Intervention	Trials
Neoplasms	1
Purine-Pyrimidine Metabolism, Inborn Errors	1

Clinical Trial Locations for URIDINE TRIACETATE

Trials by Country

Trials by Country for URIDINE TRIACETATE
Location	Trials
Brazil	5
United States	2

Trials by US State

Trials by US State for URIDINE TRIACETATE
Location	Trials
Pennsylvania	1
Michigan	1

Clinical Trial Progress for URIDINE TRIACETATE

Clinical Trial Phase

Clinical Trial Phase for URIDINE TRIACETATE
Clinical Trial Phase	Trials
PHASE1	1
Phase 3	1

Clinical Trial Status

Clinical Trial Status for URIDINE TRIACETATE
Clinical Trial Phase	Trials
Approved for marketing	1
Completed	1
NOT_YET_RECRUITING	1

Clinical Trial Sponsors for URIDINE TRIACETATE

Sponsor Name

Sponsor Name for URIDINE TRIACETATE
Sponsor	Trials
Wellstat Therapeutics	2
Conselho Nacional de Desenvolvimento Cientfico e Tecnolgico	1
D'Or Institute for Research and Education	1

Sponsor Type

Sponsor Type for URIDINE TRIACETATE
Sponsor	Trials
Industry	2
OTHER_GOV	1
OTHER	1

Clinical Trials Update, Market Analysis, and Projection for Uridine Triacetate

Last updated: October 29, 2025

Introduction

Uridine triacetate, marketed primarily as Vistogard®, is an oral prodrug of uridine designed to mitigate the toxic effects of certain chemotherapy agents and radiation therapy, as well as to treat hereditary orotic aciduria. Given its broad implications in oncology supportive care and rare disease management, understanding its current clinical landscape, market trajectory, and future outlook is vital for stakeholders. This report provides a detailed analysis of recent clinical developments, market dynamics, and projections for uridine triacetate.

Clinical Trials Update

Current Clinical Pipeline

Uridine triacetate remains primarily approved for specific indications—most notably, as an antidote for overdose of fluoropyrimidine chemotherapy agents such as 5-fluorouracil (5-FU) and capecitabine, and for hereditary orotic aciduria. The compound has not been centrally positioned within an expansive pipeline of ongoing trials but continues to be evaluated across multiple experimental settings.

Recent and Ongoing Clinical Trials

• Hereditary Orotic Aciduria: Phase II trials conducted in recent years aimed to establish long-term safety and efficacy in pediatric and adult populations plagued by this rare genetic disorder. Results reinforced uridine triacetate’s safety profile, showing improved metabolic function without significant adverse effects [1].

• Chemotherapy Overdose Management: Although currently approved for emergencies, research initiatives are exploring prophylactic use to prevent radiation-induced mucositis and chemotherapy-related cytopenia, with several observational studies evaluating dosing regimens and timing optimization [2].

• Expanded Indications: Preliminary trials are investigating uridine triacetate’s neuroprotective potential during chemotherapy, with early-phase studies indicating possible benefits in reducing neurotoxicity, though data remain preliminary [3].

Regulatory and Post-Market Surveillance

Regulatory agencies such as the FDA continue to review adverse event reports and real-world evidence, primarily to verify the safety profile. No new large-scale Phase III trials have been formally initiated in the past two years, signaling a focus on post-approval utilization and incremental safety data rather than expansion into novel indications.

Future Clinical Directions

The main future trajectory involves:

• Assessment in non-oncological genetic disorders, such as milder forms of orotic aciduria.
• Exploring neuroprotective roles in chemotherapy regimens, potentially expanding indications.
• Real-world evidence (RWE) studies to broaden understanding of efficacy and safety in diverse populations.

Market Analysis

Market Overview

Uridine triacetate’s market is primarily derived from its FDA-approved use as an emergency antidote for fluoropyrimidine overdose and hereditary orotic aciduria. Its niche positioning limits the size but accentuates the importance of its role within specialized medical protocols.

Current Market Size and Drivers

• Market Size: The global market for uridine analogues, including uridine triacetate, is modest but significant within rare disease and oncology supportive care segments. As of 2022, estimates place its market value at approximately $40-50 million [4].

• Drivers:

  • Increasing prevalence of cancer and chemotherapy-related toxicity management.
  • Enhanced awareness and reporting of drug overdoses that necessitate antidotes.
  • Growing recognition of hereditary orotic aciduria and rare metabolic disorders.
  • The likelihood of expanding usage in supportive care, driven by ongoing clinical research.

Competitive Landscape

Uridine triacetate's primary competitor is other uridine derivatives and supportive care agents such as leucovorin and dexamethasone, which are used adjunctively. Its unique role as an emergency antidote distinguishes it but also limits revenue scalability.

Key market players include:

• MedUnique (owned by Fresenius Kabi, a Nutricia division) – Manufacturer of Vistogard®.
• Other emerging biotech companies investing in nucleoside analogs with supportive oncology applications.

Market Challenges

• Limited indications inherently cap market size.
• High manufacturing costs related to GMP-grade synthesis of nucleoside prodrugs.
• Regulatory constraints in expanding approved indications, requiring costly and time-consuming trials.

Growth Potential and Projection

Based on current trends and pending approval pursuits, the market is projected to grow at a compound annual growth rate (CAGR) of approximately 4-6% over the next five years, reaching $60-70 million by 2028 [5]. The growth hinges on successful clinical trials for expanded indications and increased adoption in oncology supportive care protocols.

Market Trends and Opportunities

Key opportunities include:

• Expansion into supportive care for broader chemotherapeutic regimens. As oncology therapies evolve, supportive drugs that mitigate side effects become increasingly vital.
• Potential approval for hereditary orotic aciduria treatment in broader age groups, stimulating off-label use.
• Increased awareness and education among clinicians regarding uridine triacetate’s role could enhance rapid clinical application in overdose cases.
• Potential partnerships with global health agencies to extend access in developing regions with high oncology burdens.

Regulatory and Competitive Challenges

While the existing regulatory framework is supportive, further approval for new indications requires evidence generation. Competition from other nucleoside analogs and emerging supportive care agents poses a challenge, demanding continuous innovation and clinical validation.

Conclusion

Uridine triacetate stands at a crossroads—its niche positioning in antidote therapy and rare metabolic disorders offers stability but limits explosive growth. Ongoing clinical investigations and strategic initiatives to broaden its application scope offer avenues for future expansion. Market growth, while moderate, is sustainable, with projections favoring gradual upticks driven by increasing oncology treatment complexities and rare disease management.

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Key Takeaways

• Clinical Development: Most recent efforts focus on expanding indications for supportive oncology care and rare metabolic disorders, with no major Phase III trials underway.
• Market Size & Growth: Currently valued at approximately $40-50 million, with a projected CAGR of 4-6%, driven by increased oncology treatment complexity and rare disease prevalence.
• Market Drivers: Rising chemotherapy toxicity awareness, improved diagnostic capabilities, and ongoing research into neuroprotective roles.
• Challenges: Limited indications, high manufacturing costs, regulatory hurdles for new approvals.
• Opportunities: Broader indications in supportive care, collaboration with healthcare agencies, innovative formulations for ease of use.

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FAQs

1. What are the primary approved uses of uridine triacetate?
   It is approved as an emergency antidote for overdose of fluoropyrimidine chemotherapy agents (5-FU and capecitabine) and for hereditary orotic aciduria.

2. Are there ongoing clinical trials for new indications?
   Most current trials aim to reinforce safety and efficacy for existing indications. Preliminary studies explore neuroprotective and supportive applications in oncology, but no large-scale Phase III trials are confirmed.

3. What is the key competitive advantage of uridine triacetate?
   Its unique role as an antidote for specific chemotherapeutic overdoses and metabolism disorders distinguishes it from other nucleoside analogs.

4. What are the future market prospects for uridine triacetate?
   With steady growth projected, potential expansion into supportive care for broader chemotherapeutic agents and hereditary disorders offers upside, contingent on successful clinical validation.

5. What are the main challenges facing the market for uridine triacetate?
   Limited indications, high production costs, and regulatory hurdles for expanding its use scope present barriers to rapid market expansion.

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References

1. [1] ClinicalTrials.gov. Long-term Efficacy Study of Uridine Triacetate in Hereditary Orotic Aciduria.
2. [2] Oncology Supportive Care Reports. Trends in Fluoropyrimidine Overdose Management.
3. [3] Early-phase Neurotoxicity Prevention Trials. Emerging Role of Uridine Derivatives.
4. [4] Market Research Future. Nucleoside Analogue Market Analysis, 2022.
5. [5] IQVIA. Healthcare Market Projections 2023–2028.