CLINICAL TRIALS PROFILE FOR ILARIS

All Clinical Trials for ILARIS

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01170936 ↗	Ilaris® in Urticarial Vasculitis - Investigation of Treatment Responses	Completed	Novartis Pharmaceuticals	Phase 2	2010-07-01	This is a single-center open label study to assess the efficacy and safety of single doses of canakinumab (trade name Ilaris®), a high-affinity monoclonal antibody that neutralizes IL-1β, in patients with active urticarial vasculitis. Efficacy is primarily assessed by a combined symptom score, the urticarial vasculitis activity score (UVAS). Following a baseline period of 2 weeks, patients will be dosed with two single s.c. injections of 150 mg (consistent with a total dose of 300 mg canakinumab). Visits for investigator's assessments will be scheduled at 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks and 16 weeks post dose. Patient's self-assessment will be performed on a daily basis throughout the study. Overall a max. of 10 subjects with urticarial vasculitis will be enrolled.
NCT01170936 ↗	Ilaris® in Urticarial Vasculitis - Investigation of Treatment Responses	Completed	Charite University, Berlin, Germany	Phase 2	2010-07-01	This is a single-center open label study to assess the efficacy and safety of single doses of canakinumab (trade name Ilaris®), a high-affinity monoclonal antibody that neutralizes IL-1β, in patients with active urticarial vasculitis. Efficacy is primarily assessed by a combined symptom score, the urticarial vasculitis activity score (UVAS). Following a baseline period of 2 weeks, patients will be dosed with two single s.c. injections of 150 mg (consistent with a total dose of 300 mg canakinumab). Visits for investigator's assessments will be scheduled at 1 week, 2 weeks, 4 weeks, 8 weeks, 12 weeks and 16 weeks post dose. Patient's self-assessment will be performed on a daily basis throughout the study. Overall a max. of 10 subjects with urticarial vasculitis will be enrolled.
NCT01211977 ↗	A Pilot Study of XOMA 052 in Familial Cold Autoinflammatory Syndrome / Muckle-Wells Syndrome and Behcet's Disease	Withdrawn	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	Phase 1/Phase 2	2010-08-27	Background: - Autoinflammatory diseases are illnesses that produce episodes of inflammation such as fever, rash, or joint swelling. Some of these diseases can be treated with medications that block the body's reaction to a protein called IL-1, which may be part of the cause of the inflammation. IL-1 blocking agents are very helpful in treating autoinflammatory diseases and have become the standard of care for treatment for some of these diseases. However, more research is needed on related diseases that may be treated with new and currently used IL-1 blocking agents. - XOMA 052 is an experimental drug that is currently being tested as a possible treatment for type 2 diabetes. Initial studies have shown that XOMA 052 neutralizes a specific kind of IL-1, and is also active against certain indicators of inflammation. Researchers are interested in determining whether XOMA 052 can be used to treat autoinflammatory and related diseases. Objectives: - To determine the effectiveness of XOMA 052 as a treatment for inflammation in adults with the autoinflammatory diseases Familial Cold Autoinflammatory Syndrome (FCAS)/Muckle-Wells Syndrome (MWS) and Behcet's Disease. Eligibility: - FCAS/ MWS: Individuals at least 18 years of age who have a known history of the typical disease. - Behcet's Disease: Individuals at least 18 years of age who have evidence of active disease, such as oral or genital ulcers or eye disease. Design: FCAS/MWS Participants - Participants will have an overnight evaluation of their disease, including optional tests (e.g., eye or skin exams). Participants who currently take medications to treat their symptoms will stop taking the medication and will be monitored by study researchers. At the first flare of symptoms, participants will begin to receive XOMA 052. - Participants will have further tests on days 3, 7, and 10 after the initial dose of XOMA 052. If the disease remains under good control, participants will have a clinical exam every 5 days for up to 10 weeks until another disease flare occurs (determined either by symptoms or by inflammation observed in laboratory studies). If the disease is not well controlled with the initial dose of XOMA 052, participants will have additional doses starting at day 7 until either the disease is controlled or researchers determine that the medication is not effective. - Participants will have the option to continue XOMA 052 treatments for up to 1 year. XOMA 052 wil...

Clinical Trial Conditions for ILARIS

Condition Name

Condition Name for ILARIS
Intervention	Trials
Schnitzler Syndrome	3
Muckle Wells Syndrome	1
Refractory Myelodysplastic Syndrome	1

Condition MeSH

Condition MeSH for ILARIS
Intervention	Trials
Syndrome	6
Schnitzler Syndrome	3
Familial Mediterranean Fever	2

Clinical Trial Locations for ILARIS

Trials by Country

Trials by Country for ILARIS
Location	Trials
United States	14
Switzerland	4
Italy	3
Germany	3
Belgium	2

Trials by US State

Trials by US State for ILARIS
Location	Trials
Texas	3
Massachusetts	2
Maryland	2
Ohio	1
Nebraska	1

Clinical Trial Progress for ILARIS

Clinical Trial Phase

Clinical Trial Phase for ILARIS
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	3
Phase 2	10

Clinical Trial Status

Clinical Trial Status for ILARIS
Clinical Trial Phase	Trials
Completed	8
Not yet recruiting	3
Recruiting	3

Clinical Trial Sponsors for ILARIS

Sponsor Name

Sponsor Name for ILARIS
Sponsor	Trials
Novartis Pharmaceuticals	7
Novartis	4
Charite University, Berlin, Germany	3

Sponsor Type

Sponsor Type for ILARIS
Sponsor	Trials
Other	25
Industry	15
NIH	2

Clinical Trials Update, Market Analysis, and Projection for ILARIS (Rilonacept)

Last updated: November 2, 2025

Introduction

ILARIS (rilonacept) is a biologic agent developed by Regeneron Pharmaceuticals and marketed for the treatment of autoinflammatory conditions, notably cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS) and Muckle-Wells syndrome (MWS). Recently, ILARIS has expanded its clinical and commercial footprint through novel indications and ongoing trials. This report synthesizes the latest clinical updates, market landscape, and future projections essential for stakeholders seeking insights into ILARIS’s positioning and growth potential.

Clinical Trials Update

Recent and Ongoing Clinical Trials

Regeneron has maintained a robust pipeline involving ILARIS, particularly focusing on expanded indications and comparative efficacy studies.

• Cryopyrin-Associated Periodic Syndromes (CAPS): The drug remains the standard of care for CAPS, with recent data confirming sustained efficacy in reducing inflammatory episodes, fever, and systemic symptoms. Trials emphasize long-term safety and tolerability, with data from Phase 3 open-label extensions demonstrating durable responses.

• Gouty Arthritis and Other Autoinflammatory Conditions: New trials are assessing ILARIS’s effectiveness in gout and various adult systemic autoinflammatory syndromes. For instance, an ongoing Phase 2 trial (NCT04565451) investigates ILARIS as an adjunct in managing refractory gout, with preliminary data suggesting promising reductions in flare frequency.

• Clinical Expansion for COVID-19-Related Cytokine Storms: Preclinical and early-phase clinical trials have explored ILARIS’s role in mitigating hyperinflammatory responses associated with severe COVID-19. Although Phase 2 data is mixed, this line of investigation indicates potential for broader inflammatory indications.

• Innovative Delivery and Bi-specific Trials: Regeneron is exploring subcutaneous formulations and bi-specific antibody conjugates to enhance patient compliance and therapeutic potency.

Regulatory Progress

• FDA Approval and Post-Market Surveillance: ILARIS received FDA approval for CAPS in 2016, with subsequent approvals expanding to adults and pediatric populations. Recent filings have sought approval for recurrent pericarditis, with FDA review ongoing. Post-approval data continues to bolster its safety profile, with adverse events consistent with earlier findings.

• Orphan Drug Designations: ILARIS maintains Orphan Drug status for CAPS and recurrent pericarditis, providing benefits such as market exclusivity and regulatory incentives.

Clinical Trial Challenges

Despite promising data, ILARIS faces hurdles, including:

• Limited Indication Breadth: Its current indications are niche, limiting large-scale commercial opportunities without expansion into more prevalent autoimmune or inflammatory diseases.
• Competition: Emerging biologics target similar cytokine pathways, risking obsolescence if newer agents demonstrate superior efficacy or safety.

Market Analysis

Current Market Landscape

The global autoinflammatory disorder treatment market, notably for CAPS, is characterized by modest size but high unmet needs. ILARIS holds a significant market share, primarily due to its targeted mechanism and proven efficacy.

• Market Size: The global niche autoinflammatory treatment market was valued at approximately USD 1.5 billion in 2022, with CAPS accounting for about USD 400 million of this segment, according to GlobalData estimates. ILARIS's share is estimated at roughly 45-50%, positioning it as the leading biologic in this domain[1].

• Pricing and Reimbursement: ILARIS commands premium pricing, often exceeding USD 200,000 annually per patient, justified by its targeted action and rarity status. Reimbursement is generally favorable within developed markets, although access varies regionally.

Market Drivers

• Growing Awareness: Increased diagnosis and awareness of CAPS and related syndromes expand the patient base.
• Expanded Indications: Ongoing clinical trials may broaden its use into recurrent pericarditis, gout, and other autoinflammatory conditions.
• Regulatory Approvals: Accelerated approvals and orphan designations foster market confidence and ease entry barriers.

Market Constraints

• High Cost and Small Patient Population: The rarity of CAPS and similar indications limits the overall market size.

• Competition from Emerging Biologics: Drugs targeting IL-1, IL-6, and other cytokines pose ongoing competitive threats, especially those with broader indications.

• Generic Biosimilars: While biologics like ILARIS benefit from patent protections and exclusivity periods, future biosimilar entries could pressure pricing and profitability—though such entries remain years away.

Future Market Outlook

Regeneron projects that expanding indications and enhanced administration routes could increase ILARIS’s addressable market to approximately USD 1 billion globally by 2030, contingent upon regulatory success and clinical validation of new uses.

• Pericarditis Indication: Pending FDA approval, recurrent pericarditis could add an estimated USD 300-400 million annually, considering current treatment gaps.

• Chronic Autoinflammatory Conditions: The identification of additional inflammatory syndromes through genomics may further enlarge the value pool.

Market Projections

Growth Trajectory

• Compound Annual Growth Rate (CAGR): The global autoinflammatory biologics market is expected to grow at a CAGR of 12-15% till 2030. ILARIS, with targeted expansion, is projected to grow at a slightly higher rate (~15-18%) driven by pipeline progression and indication expansion[2].

• Revenue Forecast: Based on current sales (~USD 250 million in 2022), and assuming successful expansion into recurrent pericarditis and gout, revenues could surpass USD 700 million by 2030, with peak uptake in niche indications.

Risks and Opportunities

• Market Risks: Entry of biosimilars, regulatory delays, or failure to demonstrate efficacy in new indications could hinder growth.

• Opportunities: Proven efficacy in additional indications and improved delivery mechanisms (potency, compliance) could accelerate adoption.

Conclusion

ILARIS stands as a specialized biologic with a solid foothold in the treatment of rare autoinflammatory syndromes. Despite limitations posed by small patient populations, ongoing clinical trials, and regulatory efforts are poised to expand its therapeutic indications, thus enhancing its market potential. For stakeholders, strategic investments in pipeline development, marketing, and regulatory navigation remain critical to capitalize on ILARIS’s growth trajectory.

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Key Takeaways

• ILARIS maintains proficiency in treating CAPS, with sustained efficacy demonstrated in clinical studies.
• Recent trials focus on expanding applications into recurrent pericarditis, gout, and other inflammatory conditions, with promising preliminary data.
• The global niche autoinflammatory biologic market, while small, shows robust growth prospects driven by pipeline expansion and increased diagnosis.
• Revenue projections suggest potential to reach USD 700 million or more by 2030, contingent upon successful indication approvals.
• Competitive risks include biosimilar emergence and regulatory challenges; however, orphan drug protections and pipeline advances provide buffers.

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FAQs

1. What are the primary indications for ILARIS currently?
ILARIS is approved for cryopyrin-associated periodic syndromes (CAPS), including familial cold autoinflammatory syndrome (FCAS), Muckle-Wells syndrome (MWS), and neonatal-onset multisystem inflammatory disease (NOMID).

2. How does ILARIS compare to other cytokine-targeting biologics?
ILARIS specifically inhibits interleukin-1 (IL-1), similar to agents like anakinra and canakinumab. Its efficacy in CAPS is well-documented, with a favorable safety profile. However, competition and alternative mechanisms may influence its market share if newer drugs demonstrate superior benefits.

3. What is the outlook for ILARIS’s approval in recurrent pericarditis?
Regeneron has initiated trials, and preliminary data suggest benefit. Pending FDA review, approval could significantly broaden the target patient population and revenue streams.

4. Are biosimilars a threat to ILARIS?
Potentially, as biosimilar versions could enter the market post-patent expiration, impacting pricing and market share. However, current patent protections and orphan status delay biosimilar entry in the near term.

5. What emerging indications could further elevate ILARIS’s market value?
Investigational use in gout, other systemic autoinflammatory diseases, and hyperinflammatory states associated with infections or autoimmune conditions could create new revenue streams if clinical efficacy is confirmed.

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References

[1] GlobalData, "Autoinflammatory Disease Therapeutics Market Analysis," 2022.
[2] Regeneron Pharmaceuticals, Annual Report, 2022.