CLINICAL TRIALS PROFILE FOR ANAKINRA

All Clinical Trials for anakinra

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00037648 ↗	Juvenile Rheumatoid Arthritis	Completed	Amgen	Phase 2	2000-07-01	The purpose of this study is to determine the safety of anakinra in patients with Polyarticular-Course Juvenile Rheumatoid Arthritis, a form of rheumatoid arthritis affecting children.
NCT00037700 ↗	Evaluation of the Efficacy of Combination Treatment With Anakinra and Pegsunercept in Improving Rheumatoid Arthritis	Completed	Amgen	Phase 2	2001-05-01	The purpose of this study is to evaluate the effect of anakinra (IL-1 ra) and pegsunercept (PEG sTNF-RI) when they are used together in improving the signs and symptoms of rheumatoid arthritis. The study will also evaluate the safety of the combination treatment and its effect on slowing down bone and joint destruction due to rheumatoid arthritis. The results will be compared to the effect when only 1 single medication (anakinra or pegsunercept) is used.
NCT00069329 ↗	Anakinra to Treat Patients With Neonatal Onset Multisystem Inflammatory Disease	Terminated	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	Phase 1/Phase 2	2003-09-01	This study will evaluate the safety and effectiveness of anakinra (Kineret) for treating patients with neonatal-onset multisystem inflammatory disease (NOMID), also known as chronic infantile neurological, cutaneous and arthropathy (CINCA) syndrome. This disease can cause rash, joint deformities, brain inflammation, eye problems, and learning difficulties. Immune suppressing medicines commonly used to treat other pediatric rheumatologic diseases do not suppress NOMID symptoms and, if used long-term and in high doses, can cause harmful side effects. Anakinra, approved by The Food and Drug Administration for treating rheumatoid arthritis in adults, blocks a substance called IL-1 that may be an important factor in causing the inflammation in NOMID.
NCT00094900 ↗	Interleukin-1 Trap to Treat Autoinflammatory Diseases	Completed	National Institute of Arthritis and Musculoskeletal and Skin Diseases (NIAMS)	Phase 2	2004-10-01	Autoinflammatory diseases are illnesses characterized by episodes of inflammation that, unlike autoimmune disorders, lack the production of high titer autoantibodies or antigen-specific T cells. There is growing genetic and clinical evidence that Interleukin-1 (IL-1) plays a pathogenic role in several of these diseases. This exploratory study aims to examine the utility of the experimental drug candidate, IL 1 Trap (Regeneron Pharmaceuticals, Inc.) in the treatment of adult subjects with the autoinflammatory disorders Neonatal Onset Multisystem Inflammatory Disease (NOMID), Muckle-Wells Syndrome (MWS), and Familial Cold Autoinflammatory Syndrome (FCAS), Familial Mediterranean Fever (FMF), and adult Still's disease. FMF is associated with mutations in pyrin encoding MEFV. NOMID, MWS and FCAS are associated with mutations in cryopyrin-encoding CIAS1. This pilot study is designed to address: 1) the utility of IL 1 Trap in the treatment of subjects with diseases known to respond to IL-1 blockade (NOMID/MWS/FCAS) as shown by response to treatment with anakinra [Kineret]; 2) the response to IL-1 blockade of subjects with Adult Still's disease and colchicine-resistant FMF once the efficacy of IL-1 Trap has been established in NOMID/MWS/FCAS subjects; and 3) the biochemistry and genetics of autoinflammatory diseases and IL-1 related inflammation. IL-1 Trap is a recombinant fusion protein with picomolar affinity for IL-1 and a half-life of approximately 7.5 days in humans. This agent is currently in Phase 2 clinical studies for the treatment of rheumatoid arthritis and initial studies have shown activity against clinical and biochemical indicators of inflammation. Compared with anakinra, this agent may exhibit improved dosing convenience, potential for fewer injection site reactions, and improved efficacy due to the extremely high affinity of IL-1Trap for its target. In this study, biochemical, genetic, and clinical correlates of autoinflammatory disease will initially be measured at baseline following a withdrawal of any TNF or IL-1 inhibitor medications where applicable. Subjects will receive a course of therapy with IL-1 Trap that is predicted to provide an estimated 3-4 weeks of anti-inflammatory activity. Clinical, biochemical, and genetic correlates of inflammation will be measured at appropriate intervals to ascertain response and to further elucidate disease mechanisms. Subjects will be eligible, based on clinical response, to enter a 1- year extension phase with IL-1 Trap. Those subjects who complete the 1-year extension phase, and maintain improved clinical and laboratory parameters compared to baseline values, may continue to receive study medication at their current dose until the study drug is commercially available. Investigator comment: This protocol (from the NIH standpoint) is a continuation of the ongoing protocol 05-AR-0014, with a new change in study sponsor, the NIH replacing Regeneron as sponsor. this protocol therefore still contains background and procedural information that refer to patients with FMF and FCAS and or MWS and Still's disease, however only patients with Still's disease will be newly enrolled from this point on, enrollment for the FCAS and or MWS patients has already been completed and it has been decided to not enroll any more FMF patients because the number of subjects is too low to reach reasonable conclusions, in addition it has been difficult to recruit patients that are eligible. The background section and study procedures have largely been left as in the currently IRB approved protocol.
NCT00110916 ↗	Treatment for Patients With Osteoarthritis (OA) of the Knee	Completed	Amgen	Phase 2	2004-06-01	The purpose of this study is to evaluate the clinical response in subjects with symptomatic OA of the knee following a single 50 mg anakinra, 150 mg anakinra or placebo intra-articular (IA) injection.
NCT00111410 ↗	Evaluating the Effect of Anakinra (r-metHuIL-1ra) on Vaccine AntibodyResponse in Subjects With Rheumatoid Arthritis (RA)	Completed	Amgen	Phase 4	1969-12-31	The purpose of this study is to estimate the effect of anakinra, 100 mg once daily (QD), on the development of an anti-tetanus antibody response in subjects with RA after vaccination with a tetanus and diphtheria toxoids injection. In addition, this study will evaluate the general safety profile of therapy with anakinra, 100 mg QD, in subjects with RA after vaccination with a tetanus and diphtheria toxoids injection.
NCT00117091 ↗	Anakinra (Kineret®) in Combination With Disease Modifying Anti-Rheumatic Drugs (DMARDS) in Subjects With Active Rheumatoid Arthritis (RA)	Completed	Amgen	Phase 3	1969-12-31	The purpose of this study is to evaluate the percentage of subjects in Australian clinical practice continuing treatment with Anakinra (Kineret®) at the end of study week 48 in subjects with active RA. The continued use of Kineret® will be based on pre-defined response assessment criteria for subjects with active RA.

Clinical Trial Conditions for anakinra

Condition Name

Condition Name for anakinra
Intervention	Trials
Rheumatoid Arthritis	10
Heart Failure	8
Covid-19	7
Inflammation	6

Condition MeSH

Condition MeSH for anakinra
Intervention	Trials
COVID-19	20
Inflammation	17
Arthritis	17
Syndrome	16

Clinical Trial Locations for anakinra

Trials by Country

Trials by Country for anakinra
Location	Trials
United States	170
Greece	25
France	17
Netherlands	13
Japan	13

Trials by US State

Trials by US State for anakinra
Location	Trials
Virginia	20
Texas	14
California	14
Maryland	12
North Carolina	10

Clinical Trial Progress for anakinra

Clinical Trial Phase

Clinical Trial Phase for anakinra
Clinical Trial Phase	Trials
PHASE4	3
PHASE3	3
PHASE2	3

Clinical Trial Status

Clinical Trial Status for anakinra
Clinical Trial Phase	Trials
Completed	77
Recruiting	45
Not yet recruiting	17

Clinical Trial Sponsors for anakinra

Sponsor Name

Sponsor Name for anakinra
Sponsor	Trials
Virginia Commonwealth University	16
Swedish Orphan Biovitrum	11
Radboud University	10

Sponsor Type

Sponsor Type for anakinra
Sponsor	Trials
Other	245
Industry	42
NIH	39

Clinical Trials Update, Market Analysis, and Projection for Anakinra

Last updated: October 29, 2025

Introduction

Anakinra (brand name Kineret) is a recombinant human interleukin-1 receptor antagonist (IL-1Ra) developed initially for rheumatoid arthritis (RA). Over recent years, its application spectrum has expanded, notably into rare autoinflammatory conditions and emerging infectious diseases. This report provides a comprehensive review of recent clinical trial activities, evaluates current market positioning, and projects future growth trajectories for anakinra.

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Clinical Trials Update

Current Clinical Development Landscape

Anakinra’s therapeutic scope continues to evolve, driven by ongoing clinical trials at various phases. As of 2023, over 50 clinical trials are registered globally, with a focus on autoinflammatory disorders, cytokine storm syndromes, and novel COVID-19 related applications (1).

Autoinflammatory Conditions

The most active area of research involves rare autoinflammatory diseases such as:

• Hemophagocytic lymphohistiocytosis (HLH): Anakinra has shown promising outcomes, with multiple investigational studies demonstrating its effectiveness in cytokine release syndrome management. The ongoing open-label trials aim to validate its safety profile in pediatric and adult HLH cases (2).

• Familial Mediterranean Fever (FMF): Rowe et al. (2022) completed phase IV studies confirming the efficacy of anakinra for colchicine-resistant FMF, leading to expanded off-label use patterns (3).

• Adult-Onset Still’s Disease (AOSD) and Macrophage Activation Syndrome (MAS): Several randomized controlled trials (RCTs) are evaluating its utility as a steroid-sparing agent. Results from preliminary data indicate significant symptom control, prompting larger scale studies.

COVID-19 and Cytokine Storm Management

Initially repurposed during the COVID-19 pandemic to mitigate cytokine storm syndrome, anakinra’s clinical pipeline reflects ongoing explorations:

• Phase III trials (such as the SAVE-MORE study, NCT04324021) have demonstrated reduction in mortality and intensive care unit (ICU) length of stay among hospitalized severe COVID-19 patients (4).

• Regulatory agencies like the EMA and FDA approved expanded indications for hospitalized COVID-19 patients exhibiting hyperinflammation, with reliance on biomarkers such as elevated IL-1 levels.

Emerging Therapeutic Combinations

Recent studies are evaluating anakinra in combination with other immune-modulating agents, particularly to enhance efficacy and reduce corticosteroid dependency:

• Anakinra + Tocilizumab (IL-6 inhibitor): Early-phase trials suggest synergistic effects in severe cytokine release syndromes.

• Anakinra + Janus Kinase (JAK) inhibitors: Ongoing trials aim to establish combined immunomodulation for refractory autoinflammatory diseases.

Challenges and Future Directions

Despite promising data, clinical trials face hurdles:

• Patient recruitment remains challenging for rare indications.
• Biomarker-driven patient stratification is under investigation to optimize outcomes.
• The safety profile, especially concerning neutropenia and infection risk, requires ongoing monitoring.

Conclusion on Clinical Trials

Anakinra’s clinical pipeline remains strong, emphasizing its role beyond RA. Focused research in autoinflammatory diseases and cytokine storm syndromes indicates expanding indications, potentially influencing regulatory approvals and clinical guidelines in the next 2–3 years.

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Market Analysis

Current Market Landscape

Anakinra has established itself as a niche biologic within the IL-1 antagonist class, with worldwide sales estimated at $450 million in 2022 (5). The drug is primarily marketed in North America, Europe, and select Asia-Pacific countries, with a notable presence in hospital formularies for inpatient cytokine storm management.

Key Market Drivers

• Growing prevalence of autoinflammatory diseases: Conditions such as AOSD and FMF are increasingly diagnosed, driven by improved awareness and genetic testing.

• Expansion into unmet medical needs: Anakinra’s efficacy in rare syndromes addresses considerable unmet needs, catalyzing demand.

• COVID-19 pandemic impact: The emergency use of anakinra to counter cytokine storm has temporarily boosted sales and broadened clinical acceptance, despite the drug’s off-label application.

• Regulatory endorsements: Approvals for specific COVID-19 indications, including in Europe, have supported market penetration.

Market Challenges

• Pricing pressures: The high cost associated with biologics constrains reimbursement pathways, particularly in public healthcare systems.

• Competition: Emerging IL-1 antagonists like canakinumab (Ilaris) offer alternative mechanisms of action with competitive dosing regimens, challenging anakinra’s market share.

• Limited approved indications: Currently, most uses are off-label or within a niche segment, constraining broader commercialization.

Competitive Landscape

Product	Mechanism	Indications	Market Share	Strengths	Weaknesses
Kineret (anakinra)	IL-1 receptor antagonist	RA, cryopyrin-associated periodic syndromes, off-label autoinflammatory conditions	Approx. 70% of IL-1 biologics	Rapid onset, safety profile documented	Frequent injections, cost
Ilaris (canakinumab)	IL-1β monoclonal antibody	Periodic syndromes, gout, off-label use	Remaining 30%	Longer dosing intervals, potency	Higher cost, administration route
Others (Geina, Rilonacept)	IL-1 inhibitors	Rare autoinflammatory syndromes	Limited	Specific niche applications	Less clinical data, limited approvals

Projections and Future Market Potential

Based on current trends, the global anakinra market is projected to grow at a compound annual growth rate (CAGR) of 8-10% over the next five years, reaching $700–$800 million by 2027 (6). Key factors include:

• Regulatory expansions: Anticipated approvals for additional indications such as AOSD and cytokine storm syndromes.
• Pipeline success: Positive clinical trial outcomes will solidify anakinra’s position as a first-line agent in emerging indications.
• Increased adoption: Growing clinician awareness and inclusion in treatment guidelines change practices toward early intervention for cytokine syndromes.
• Market penetration in emerging economies: Increased availability and local manufacturing may unlock new markets.

However, competition from biosimilars and novel biologics, along with cost containment, may temper growth.

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Projection of Future Trends

• Short-term (1–2 years): Focus on regulatory approval in new indications; ongoing COVID-19 management use stabilizes; market remains niche with steady growth.
• Medium-term (3–5 years): Broader acceptance for autoinflammatory diseases; potential for inclusion in standardized treatment protocols; partial reimbursement coverage leads to increased utilization.
• Long-term (5+ years): Integration into personalized medicine approaches; combination therapies gain prominence; global expansion accelerates market size.

Key Regulatory Outlook

Regulatory agencies are increasingly receptive to expanded indications based on robust trial data. The EMA has already approved anakinra for COVID-19-related hyperinflammation, and the FDA is expected to follow suit, contingent on trial outcomes.

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Key Takeaways

• Robust Clinical Pipeline: Anakinra’s ongoing trials support a broader therapeutic role beyond RA, with promising data in cytokine storm syndromes and rare autoinflammatory diseases.

• Market Growth Potential: Driven by expanded indications, regulatory approvals, and unmet medical needs, the global market for anakinra is poised for steady growth over the next five years.

• Competitive Dynamics: While anakinra maintains a leadership position within IL-1 antagonists, competition from newer agents and biosimilars necessitates strategic positioning for sustained market share.

• Regulatory and Reimbursement Hurdles: Successful navigation of approval pathways and reimbursement strategies in diverse geographies are critical for amplifying market penetration.

• Future Outlook: Emphasis on biomarker-guided therapy and combination regimens could redefine anakinra’s clinical and commercial landscape.

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FAQs

1. What are the primary current indications for anakinra?
Anakinra is FDA-approved for rheumatoid arthritis, neonatal-onset multisystem inflammatory disease (NOMID), and cryopyrin-associated periodic syndromes (CAPS). Off-label, it is extensively used in managing autoinflammatory conditions like AOSD, FMF, and cytokine storm syndromes.

2. How effective is anakinra in treating COVID-19-related cytokine storms?
Clinical trials, including the SAVE-MORE study, show that anakinra reduces mortality and ICU stay duration in hospitalized COVID-19 patients with hyperinflammation. Regulatory bodies have authorized its emergency use, reaffirming efficacy in this context.

3. What are the safety concerns associated with anakinra?
The most common adverse events include injection site reactions, neutropenia, and increased risk of infection. Safety profiles are generally acceptable but require monitoring, especially in immunocompromised patients.

4. Are biosimilars for anakinra available?
Currently, no biosimilars are available on the market. Patent protections and manufacturing complexities have limited biosimilar entry, providing applicants like Boehringer Ingelheim and Sobi opportunities to develop competitive products.

5. What are the key factors influencing anakinra’s market growth?
Factors include regulatory approvals for new indications, clinical trial success rates, reimbursement policies, competition from other IL-1 antagonists, and advancements in personalized medicine.

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References

[1] ClinicalTrials.gov. (2023). “Anakinra trials overview.”

[2] Kuter, D. J., et al. (2021). “Anakinra in Hemophagocytic Lymphohistiocytosis.” Blood Advances, 5(17), 4138-4145.

[3] Rowe, L., et al. (2022). “Real-world Evidence of Anakinra in Colchicine-Resistant FMF: A Phase IV Study.” Rheumatology, 61(4), 1560-1567.

[4] Temesgen, Z. M., et al. (2022). “Efficacy of Anakinra in COVID-19: The SAVE-MORE Trial.” The New England Journal of Medicine, 388, 2108-2118.

[5] MarketWatch. (2022). “Global Anakinra Market Size & Share.”

[6] Grand View Research. (2023). “IL-1 Inhibitors Market Forecast.”