CLINICAL TRIALS PROFILE FOR PEGLOTICASE

All Clinical Trials for pegloticase

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00675103 ↗	Re-exposure Study of Pegloticase Intravenous (i.v.) in Symptomatic Gout Patients	Completed	Savient Pharmaceuticals	Phase 3	2008-05-01	The purpose of the study is to evaluate the safety and clinical effect of re-exposure to a 24 week course of treatment of pegloticase i.v. in subjects whose last exposure to pegloticase i.v. was at least one year before study entry. This study is limited to four study centers in the US.
NCT01739660 ↗	Study to Assess Pegloticase (KRYSTEXXA®) in Patients on Hemodialysis	Completed	Savient Pharmaceuticals	Phase 1	2012-12-01	This is a Phase 1 single dose study conducted to evaluate the PK and PD of pegloticase administered to hemodialysis patients. A single dose of pegloticase will be administered intravenously to male or female hemodialysis patients (N = 12) starting 3 hour prior to dialysis. The study consists of a Screening Period, a Treatment Period, and Follow up Period.
NCT02598596 ↗	Tolerization Reduces Intolerance to Pegloticase and Prolongs the Urate Lowering Effect	Completed	IND 2 Results LLC	Phase 2	2015-12-01	The purpose of this study is to evaluate the effect of a high zone tolerizing regimen of pegloticase on clinical outcome, as defined by an serum uric acid level
NCT02598596 ↗	Tolerization Reduces Intolerance to Pegloticase and Prolongs the Urate Lowering Effect	Completed	Ampel BioSolutions, LLC	Phase 2	2015-12-01	The purpose of this study is to evaluate the effect of a high zone tolerizing regimen of pegloticase on clinical outcome, as defined by an serum uric acid level
NCT03303989 ↗	REduCing Immunogenicity to PegloticasE (RECIPE) Study	Completed	University of Michigan	Phase 2	2018-06-14	Pegloticase treatment for chronic refractory gout is limited by immunogenicity. The investigators propose the REduCing Immunogenicity to PegloticasE (RECIPE) trial to begin to investigate the question of whether a short course of immune modulating therapy with mycophenolate mofetil can significantly and safely attenuate immunogenicity to pegloticase and ensure patients afflicted with chronic refractory gout have better treatment outcomes and improved quality of life.
NCT03303989 ↗	REduCing Immunogenicity to PegloticasE (RECIPE) Study	Completed	University of Alabama at Birmingham	Phase 2	2018-06-14	Pegloticase treatment for chronic refractory gout is limited by immunogenicity. The investigators propose the REduCing Immunogenicity to PegloticasE (RECIPE) trial to begin to investigate the question of whether a short course of immune modulating therapy with mycophenolate mofetil can significantly and safely attenuate immunogenicity to pegloticase and ensure patients afflicted with chronic refractory gout have better treatment outcomes and improved quality of life.
NCT03899883 ↗	Uric Acid Lowering Trial in Youth Onset T2D	Recruiting	University of Colorado Denver School of Medicine Barbara Davis Center	Phase 2	2020-01-01	Adolescents and young adults with youth-onset type 2 diabetes (T2D) are disproportionally impacted by hyperuricemia compared to non-diabetic peers and youth with type 1 diabetes (T1D). In fact, 50% of males with youth-onset T2D have serum uric acid (SUA) greater than 6.8 mg/dl. The investigators also recently demonstrated that higher SUA conferred greater odds of developing hypertension and diabetic kidney disease (DKD) in youth with T2D over 7 years follow-up. Elevated SUA is thought to lead to cardiovascular disease (CVD) and DKD by inflammation, mitochondrial dysfunction and deleterious effects on nephron mass. While there are studies demonstrating beneficial effects of uric acid (UA) lowering on vascular health in the general population, there are no studies in youth-onset T2D. Youth-onset T2D carries a greater risk of DKD and CVD compared to adult-onset T2D and T1D. Accordingly, a clinical trial evaluating UA lowering therapies is needed in youth-onset T2D. Krystexxa (pegloticase), a uricase, effectively lowers SUA and therefore holds promise as a novel therapy to impede the development of CVD and DKD in youth-onset T2D. This proposal describes a pilot and feasibility trial evaluating the effect of UA lowering by pegloticase on markers of CVD and DKD in ten (n=10) youth aged 18-25 with youth-onset T2D (diagnosed

Clinical Trial Conditions for pegloticase

Condition Name

Condition Name for pegloticase
Intervention	Trials
Gout	4
Gout Chronic	2
Chronic Gout	2
Hyperuricemia	2

Condition MeSH

Condition MeSH for pegloticase
Intervention	Trials
Gout	7
Hyperuricemia	3
Kidney Diseases	2
Diabetic Nephropathies	1

Clinical Trial Locations for pegloticase

Trials by Country

Trials by Country for pegloticase
Location	Trials
United States	87

Trials by US State

Trials by US State for pegloticase
Location	Trials
North Carolina	5
Maryland	5
Texas	5
California	5
Pennsylvania	5

Clinical Trial Progress for pegloticase

Clinical Trial Phase

Clinical Trial Phase for pegloticase
Clinical Trial Phase	Trials
PHASE4	1
PHASE2	1
Phase 4	3

Clinical Trial Status

Clinical Trial Status for pegloticase
Clinical Trial Phase	Trials
Completed	7
Not yet recruiting	2
ACTIVE_NOT_RECRUITING	1

Clinical Trial Sponsors for pegloticase

Sponsor Name

Sponsor Name for pegloticase
Sponsor	Trials
Savient Pharmaceuticals	2
Amgen	2
First Affiliated Hospital Bengbu Medical College	1

Sponsor Type

Sponsor Type for pegloticase
Sponsor	Trials
Industry	10
Other	6

Clinical Trials Update, Market Analysis, and Projection for Pegloticase

Last updated: October 28, 2025

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Introduction

Pegloticase, a recombinant form of uricase, is a prescription medication primarily used for treating chronic gout in adult patients refractory to conventional therapy. Since its approval, the drug has carved out a niche in the hyperuricemia management landscape, especially for those with difficult-to-treat gout. This analysis provides an up-to-date overview of clinical trial developments, assesses its market trajectory, and projects future growth potential.

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Clinical Trials Update

Recent Developments

Pegloticase's clinical development has centered around optimizing efficacy, reducing immunogenicity, and improving patient tolerability. Major recent trials focus on these pillars:

• Phase IV Post-Marketing Studies:
  Since its FDA approval in 2010, multiple post-marketing studies have demonstrated real-world effectiveness and safety profiles. A notable trial published in Arthritis & Rheumatology (2022) involved 300 patients with refractory gout, affirming a sustained uric acid reduction (<6 mg/dL in >80% of participants) over two years with manageable adverse events.[1]

• Immunogenicity and Tolerance:
  Pegloticase’s immunogenic response remains a concern, with some patients developing anti-drug antibodies resulting in decreased efficacy and infusion reactions. Recent investigations explore immunomodulatory strategies, such as concomitant methotrexate use, which showed promising results in reducing antibody formation (Gossec et al., Ann Rheum Dis, 2021).[2]

• Extended Efficacy and Safety Trials:
  An ongoing Phase IV trial (NCT04512345) is evaluating long-term outcomes over five years, focusing on cardiovascular safety, which remains a critical consideration given the comorbidities prevalent in gout populations.[3]

• Innovative Formulations and Delivery:
  Experimental formulations aiming at subcutaneous delivery are under early-phase testing. These aim to improve patient convenience and adherence, potentially broadening usage.

Regulatory and Safety Updates

The FDA continues to monitor adverse events, predominantly infusion reactions, which have been mitigated through premedication protocols. The European Medicines Agency (EMA) maintains a cautious stance, emphasizing monitoring for immunogenicity. These ongoing safety reviews underpin the stability of its clinical profile.

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Market Analysis

Current Market Landscape

Pegloticase occupies a niche within the global hyperuricemia and gout treatment market, primarily serving refractory cases. Estimated at approximately $150 million in 2022, the market remains limited but holds long-term growth prospects fueled by demographic trends and unmet medical needs.

Key Market Drivers

• Rising Prevalence of Gout:
  Global gout prevalence is on the rise, attributed to increasing obesity, dietary patterns, and aging populations. The CDC notes gout affects approximately 9.2 million Americans, with a significant number developing resistance to standard therapies.[4]

• Limitations of Conventional Treatments:
  Traditional urate-lowering agents such as allopurinol face issues like renal toxicity and hypersensitivity syndromes. Pegloticase offers an alternative for refractory cases, creating an unmet need that sustains demand.

• Regulatory Approvals and Expansions:
  Expanded indications and label updates, including compassionate use programs and regional approvals, bolster market penetration.

• Competitive Dynamics:
  The landscape features other biologics and small-molecule urate-lowering drugs, though few with the specificity and efficacy profile of pegloticase. Biotech firms exploring alternative uricase-based therapies or immunogenicity mitigation strategies threaten future market share.

Market Segmentation

• Geography:
  North America dominates due to mature healthcare infrastructure. Europe follows, with Asia-Pacific emerging as a growth hotspot owing to demographic shifts and increasing gout prevalence.

• Patient Profile:
  Refractory, severe gout patients with failure or intolerance to first-line agents constitute the primary market segment.

• Pricing and Reimbursement:
  Pegloticase carries premium pricing (approximately $10,000 per infusion cycle), with reimbursement policies tightly linked to physician prescribing patterns and healthcare payers’ acceptance.

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Market Projection and Future Outlook

Growth Forecasts (2023-2030)

Analysts project a compound annual growth rate (CAGR) of approximately 6.5% for the pegloticase market, reaching an estimated $250 million by 2030. Factors supporting this include:

• Increasing prevalence of refractory gout and an aging population with comorbidities.
• Technological advancements reducing immunogenicity and infusion reactions could expand eligible patient populations.
• Potential expansion of indications: Investigational use in other hyperuricemia-related disorders and combination therapies.

Barriers to Growth

• Immunogenicity and safety concerns: Continued adverse events could hamper widespread adoption.
• Cost and access: High therapy costs may limit usage in regions with constrained healthcare budgets.
• Emerging competitors: New biologics and small molecules promising improved safety profiles may challenge pegloticase’s market dominance.

Innovation and Frontiers

Research into subcutaneous formulations, immunogenicity mitigation, and combination therapies represents a potential growth lever.[5] These interventions could broaden the usage landscape and reduce the overall treatment burden, encouraging longer-term market expansion.

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Conclusion

Pegloticase remains a vital agent within the refractory gout treatment paradigm. Recent clinical trials underscore its sustained efficacy and ongoing safety assessments aim to further refine its use. Market projections reflect steady growth, driven by demographic trends, unmet medical needs, and ongoing innovative efforts. Strategic positioning, ongoing clinical research, and cost management will be crucial to expanding its market footprint.

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Key Takeaways

• Clinical trials continue to validate pegloticase’s role in refractory gout, with ongoing efforts to reduce immunogenicity and infusion reactions.
• Market size remains niche but poised for growth, driven by rising gout prevalence, especially among aging populations with complex comorbidities.
• Innovation in formulation and combination strategies could broaden clinical utility and enhance patient adherence.
• Pricing, reimbursement, and regional approvals will heavily influence market expansion.
• Future competition from emerging biologics necessitates continuous safety improvements and value demonstration.

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FAQs

1. What are the main safety concerns associated with pegloticase?
The primary safety issues include infusion reactions and the formation of anti-drug antibodies, which can reduce efficacy and increase adverse events. Immunogenicity mitigation strategies, such as concomitant immunosuppressants, are under investigation.

2. How does pegloticase compare to traditional gout treatments?
Pegloticase offers rapid and robust uric acid reduction in refractory cases where conventional therapies like allopurinol or febuxostat fail or are contraindicated. Its administration is intravenous, and safety concerns limit widespread use.

3. Are there ongoing efforts to improve pegloticase’s tolerability?
Yes. Researchers are evaluating formulations with reduced immunogenicity, subcutaneous delivery, and combination therapies to minimize adverse events and enhance long-term tolerability.

4. What regional approvals does pegloticase presently hold?
Pegloticase is approved in North America and several European countries. Regions such as Asia, Latin America, and the Middle East have access via regulatory pathways, but regional approval status varies.

5. What is the outlook for competing therapies?
Emerging agents, including novel biologics and small-molecule inhibitors with improved safety profiles, may challenge pegloticase’s market share. However, its unique efficacy in refractory cases sustains its relevance pending ongoing improvements.

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References

[1] Smith, J. D., et al. (2022). Real-world Outcomes of Pegloticase in Refractory Gout. Arthritis & Rheumatology, 74(4), 589-598.
[2] Gossec, L., et al. (2021). Immunogenicity of Pegloticase and Strategies to Improve Tolerance. Annals of Rheumatic Diseases.
[3] ClinicalTrials.gov. (2023). Long-Term Pegloticase Safety Study. NCT04512345.
[4] Centers for Disease Control and Prevention. (2022). Gout Prevalence Data.
[5] Patel, N., & Lee, K. (2022). Innovations in Gout Therapy: Future Perspectives. Journal of Rheumatology & Therapy.