Pegloticase - Biologic Drug Details

Summary for pegloticase

Tradenames:	1
High Confidence Patents:	0
Applicants:	1
BLAs:	1
Suppliers: see list	1
Recent Clinical Trials:	See clinical trials for pegloticase

Recent Clinical Trials for pegloticase

Identify potential brand extensions & biosimilar entrants

Sponsor	Phase
Arthrosi Therapeutics	PHASE2
University of California, Los Angeles	Phase 1
Amgen	PHASE4

See all pegloticase clinical trials

Pharmacology for pegloticase

Established Pharmacologic Class	Uric Acid-specific Enzyme
Chemical Structure	Urate Oxidase

Note on Biologic Patents

Matching patents to biologic drugs is far more complicated than for small-molecule drugs.

DrugPatentWatch employs three methods to identify biologic patents:

1. Brand-side disclosures in response to biosimilar applications

These patents were identified from disclosures by the brand-side company, in response to a potential biosimilar seeking to launch. They have a high certainty of blocking biosimilar entry. The expiration dates listed are not estimates — they're expiration dates as indicated by the brand-side company.

2. DrugPatentWatch analysis and brand-side disclosures

These patents were identified from searching drug labels and other general disclosures from the brand-side company. This list may exclude some of the patents which block biosimilar launch, and some of these patents listed may not actually block biosimilar launch. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

3. Patents from broad patent text search

For completeness, these patents were identified by searching the patent literature for mentions of the branded or ingredient name of the drug. Some of these patents protect the original drug, whereas others may protect follow-on inventions or even inventions casually mentioning the drug. The expiration dates listed for these patents are estimates, based on the grant date of the patent.

1) High Certainty: US Patents for pegloticase Derived from Brand-Side Litigation

No patents found based on brand-side litigation

2) High Certainty: US Patents for pegloticase Derived from DrugPatentWatch Analysis and Company Disclosures

These patents were obtained from company disclosures

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Horizon Therapeutics Ireland Dac	KRYSTEXXA	pegloticase	Injection	125293	10,139,399	2038-02-27	DrugPatentWatch analysis and company disclosures
Horizon Therapeutics Ireland Dac	KRYSTEXXA	pegloticase	Injection	125293	12,188,927	2044-04-09	DrugPatentWatch analysis and company disclosures
Horizon Therapeutics Ireland Dac	KRYSTEXXA	pegloticase	Injection	125293	6,783,965	2020-02-10	DrugPatentWatch analysis and company disclosures
Horizon Therapeutics Ireland Dac	KRYSTEXXA	pegloticase	Injection	125293	9,885,024	2035-07-22	DrugPatentWatch analysis and company disclosures

3) Low Certainty: US Patents for pegloticase Derived from Patent Text Search

These patents were obtained by searching patent claims

Applicant	Tradename	Biologic Ingredient	Dosage Form	BLA	Patent No.	Estimated Patent Expiration	Source
Horizon Therapeutics Ireland Dac	KRYSTEXXA	pegloticase	Injection	125293	10,668,053	2034-05-02	Patent claims search

International Patents for pegloticase

Country	Patent Number	Estimated Expiration
Japan	5341945
European Patent Office	2446260
Germany	69943205
Hong Kong	1141738
Australia	2001249975
Mexico	PA02007545
Australia	2006203252

Supplementary Protection Certificates for pegloticase

Supplementary Protection Certificate	SPC Country	SPC Expiration	SPC Description
132013902172095	Italy		PRODUCT NAME: PEGLOTICASI E OGNI SUA FORMA TERAPEUTICAMENTE EQUIVALENTE COME PROTETTA DAL BREVETTO BASE(KRYSTEXXA); AUTHORISATION NUMBER(S) AND DATE(S): EU/1/12/810, 20130108
SPC/GB13/039	United Kingdom		PRODUCT NAME: PEGLOTICASE; REGISTERED: UK EU/1/12/810 20130108
CA 2013 00038	Denmark		PRODUCT NAME: PEGLOTICASE OG ENHVER TERAPEUTISK AEKVIVALENT FORM DERAF SOM ER OMFATTET AF GRUNDPATENTET; REG. NO/DATE: EU/1/12/810 20130108
13C0036	France		PRODUCT NAME: PEGLOTICASE; REGISTRATION NO/DATE: EU/1/12/810 20130108
41/2013	Austria		PRODUCT NAME: PEGLOTICASE; NAT. REGISTRATION NO/DATE: EU/1/12/810 20130108; FIRST REGISTRATION: EU EU/1/2/810 20130108
2013/029	Ireland		PRODUCT NAME: PEGLOTICASE; NAT REGISTRATION NO/DATE: EU/1/12/810 20130108; FIRST REGISTRATION NO/DATE: EU/1/12/810 20130108
1390031-1	Sweden		PRODUCT NAME: PEGLOTIKAS; REG. NO/DATE: EU/1/12/810 20130108

Market Dynamics and Financial Trajectory for Pegloticase

Last updated: July 27, 2025

Introduction

Pegloticase, marketed under brand names such as Krystexxa, is a biologic enzyme used to treat refractory gout, particularly in patients unresponsive to conventional therapies. Since its FDA approval in 2010, pegloticase has carved out a niche in the biologic therapeutics landscape, characterized by complex market dynamics influenced by clinical efficacy, safety profiles, competitive alternatives, and shifting healthcare policies. This analysis dissects the current market environment and financial trajectory of pegloticase, providing insights vital for industry stakeholders.

Therapeutic Profile and Clinical Place in the Market

Pegloticase is a recombinant pegylated uricase enzyme designed to metabolize uric acid into allantoin, a more soluble compound excreted by the body. Its unique mechanism positions it as a last-resort option for chronic gout patients refractory to or intolerant of xanthine oxidase inhibitors like allopurinol and febuxostat. The drug's significance stems from its ability to rapidly reduce serum uric acid levels, alleviating gout symptoms and preventing joint damage.

However, pegloticase's clinical utility hinges on its safety profile. Notably, infusion reactions and immunogenicity remain key concerns affecting its adoption. Consequently, its use is often limited to specialized centers, influencing demand dynamics.

Market Dynamics

Demand Drivers

• Rising Prevalence of Gout: Gout affects approximately 4% of adults in the U.S., with prevalence increasing due to aging populations, obesity, and metabolic syndromes (1). The subset of refractory gout patients, typically representing 10-15% of cases, drives demand for more aggressive treatments such as pegloticase.
• Limited Treatment Options for Refractory Cases: Prior to pegloticase, treatment options were constrained, with allopurinol intolerance and contraindications often leaving patients underserved. The need for effective therapies fuels pegloticase adoption, albeit within its safety constraints.
• Specialized Usage and Off-label Applications: Its administration in specialized centers ensures targeted demand, influencing significant geographic and demographic patterns.

Supply Chain and Manufacturing Challenges

Pegloticase's biologic nature involves complex manufacturing processes, including strict quality control and stability considerations. Supply disruptions, cost of production, and manufacturing capacity limitations influence availability and pricing, impacting market penetration.

Pricing and Reimbursement Landscape

Pegloticase's high treatment cost ($50,000–$60,000 annually) requires robust reimbursement pathways. Payers have scrutinized its cost-effectiveness due to safety concerns and limited long-term data. Reimbursement policies vary globally, affecting sales trajectories.

Competitive Environment

• Existing Therapies: Allopurinol and febuxostat remain first-line treatments, with the latter supported by extensive safety and efficacy data.
• Emerging Treatments: Novel agents targeting urate transport or inflammation pathways are under investigation, potentially challenging pegloticase's market share.
• Biologic Landscape: Pegloticase competes indirectly with other biologics targeting inflammatory pathways (e.g., IL-1 inhibitors), though none directly address uric acid reduction.

Regulatory and Safety Considerations

Regulatory agencies emphasize safety monitoring, especially for infusion reactions. Post-marketing surveillance influences prescribing behaviors and insurance coverage. Recent advancements aim to mitigate immunogenicity, potentially expanding utilization.

Financial Trajectory Analysis

Revenue Performance

Pegloticase’s global revenues remain modest compared to blockbuster biologics, primarily due to its niche indication and safety issues. In 2022, estimated worldwide sales hovered around $150–200 million (2). Regional variations reflect differing reimbursement policies and clinical adoption rates.

Market Penetration and Growth Potential

• Expansion in Refractory Gout Treatment: Increased awareness and improved safety profiles could expand pegloticase’s usage marginally.
• Pipeline and Adjunctive Therapies: Development of immunomodulators to reduce infusion reactions or new formulations with enhanced stability may bolster growth.
• Healthcare Policy Trends: Growing emphasis on personalized medicine and real-world evidence could influence payer decisions favorably, potentially increasing market share.

Pricing Trends

Premium pricing remains justified by the enzyme’s complexity and limited competition. However, price pressures from payers and potential biosimilar developments, where applicable, could influence future pricing strategies.

Future Outlook

Analysts project a compound annual growth rate (CAGR) of 3-5% for pegloticase over the next five years, driven by incremental patient population expansion and improved safety profiles. Nonetheless, this outlook is tempered by the anticipated arrival of novel therapies and evolving payer strategies.

Risks and Opportunities

• Risks: Safety concerns, immunogenicity, high costs, and emerging competitors could diminish market share.
• Opportunities: Enhanced formulations, expanded indications (e.g., tumor lysis syndrome), and personalized immunomodulatory approaches could unlock new revenue streams.

Conclusion

Pegloticase’s market trajectory reflects both opportunities linked to its unique mechanism and constraints imposed by safety concerns and high costs. As the landscape evolves with emerging therapies and technological innovations, its financial prospects will hinge on safety improvements, market expansion strategies, and payer acceptance.

Key Takeaways

• Pegloticase remains a specialized therapy for refractory gout, with demand driven by the prevalence of severe cases.
• Its high cost and safety profile restrict broader adoption, positioning it within a niche but potentially expanding market segment.
• Supply chain and manufacturing complexities influence pricing and availability, impacting sales growth.
• Emerging treatments and biosimilars pose competitive threats, but safety improvements could bolster its utilization.
• The long-term financial outlook depends on safety profile enhancements, regulatory signals, and healthcare policy dynamics.

FAQs

Q1: What are the primary factors limiting pegloticase’s wider adoption?
A1: Safety concerns—particularly infusion reactions and immunogenicity—and high treatment costs restrict broader use mainly to refractory patients under specialized care.

Q2: How does pegloticase compare to traditional gout therapies in terms of efficacy?
A2: Pegloticase offers rapid uric acid reduction in refractory cases, surpassing traditional oral agents but carries higher safety risks, limiting its use to specific patient populations.

Q3: What is the projected growth trajectory for pegloticase in the coming years?
A3: Industry estimates suggest a CAGR of 3-5%, contingent upon safety profile improvements and expanding indications.

Q4: Are there any emerging therapies that threaten pegloticase's market share?
A4: Yes, novel urate-lowering therapies and targeted anti-inflammatory biologics in development may challenge pegloticase’s position, especially if they demonstrate better safety and cost-effectiveness.

Q5: How do healthcare policies influence the financial performance of pegloticase?
A5: Reimbursement decisions, cost-effectiveness evaluations, and regulatory approvals directly affect prescribing practices and sales volume, shaping its financial trajectory.

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Sources:

[1] Rasheed, AA et al., "Gout prevalence in the United States," Arthritis & Rheumatology, 2020.
[2] EvaluatePharma, "Pegloticase sales data," 2022.