CLINICAL TRIALS PROFILE FOR RUCONEST

All Clinical Trials for RUCONEST

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00225147 ↗	Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema	Completed	Pharming Technologies B.V.	Phase 2/Phase 3	2005-07-01	Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that lead to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy, and pharmacokinetics/pharmacodynamics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks. Funding Source - FDA OOPD
NCT00262301 ↗	Recombinant Human C1 Inhibitor for the Treatment of Acute Attacks in Patients With Hereditary Angioedema	Completed	Pharming Technologies B.V.	Phase 3	2004-06-01	Hereditary angioedema ("HAE") is a genetic disorder characterized by sudden recurrent attacks of local swelling (angioedema). These attacks are often painful and disabling, and, in some cases, life-threatening. "HAE" is caused by mutations in the "C1INH" gene that leads to a decrease in the blood level of functional "C1INH". This multi-center study was designed to assess the safety and tolerability, efficacy and pharmacodynamics/ pharmacokinetics of recombinant human C1 inhibitor ("rhC1INH") in the treatment of acute hereditary angioedema attacks.
NCT00851409 ↗	A Study of the Safety and Immunogenicity of Repeated rhC1INH Administration	Completed	Pharming Technologies B.V.	Phase 2	2009-06-01	Hereditary angioedema ("HAE") is a disease characterized by recurrent tissue swelling affecting various body locations. Recent literature shows that patients with frequent attacks may benefit from long-term prophylaxis. This study aims to evaluate the safety and prophylactic effect of weekly administrations of 50 IU/kg recombinant C1 Inhibitor ("rhC1INH").
NCT01359969 ↗	Safety of Ruconest in 2-13 Year Old Hereditary Angioedema (HAE) Patients	Completed	Pharming Technologies B.V.	Phase 2	2012-01-17	This open-label study is being conducted to confirm the safety, pharmacokinetic profile and efficacy of Ruconest at a dose of 50 U/kg when used for the treatment of acute angioedema attacks in patients, from 2 up to and including 13 years of age.
NCT01397864 ↗	C1 Inhibitor Registry in the Treatment of Hereditary Angioedema (HAE) Attacks	Recruiting	Pharming Technologies B.V.		2011-07-01	This is a non-interventional treatment Registry of Hereditary Angioedema (HAE) patients treated with C1 inhibitor, either plasma-derived (pdC1INH) or the recombinant human form (rhC1INH / Ruconest), to observe adverse events and insufficient efficacy, and to assess the immunological profile following single and repeated treatment with Ruconest.
NCT03791476 ↗	RUCONEST® as a Therapeutic Strategy to Reduce the Incidence of Delayed Graft Function	Recruiting	Pharming Technologies B.V.	Phase 1	2019-06-21	An unmet medical need exists for therapeutic regimens in transplantation that allow immediate postoperative graft function, thereby improving graft survival. Delayed graft function (DGF) after transplantation is the most common complication affecting kidney allographs in the immediate transplant period. The specific aim of this study is to evaluate the effect of recombinant human C1-inhibitor (rhC1INH), as a kidney recipient intra- and post operative treatment strategy to decrease systemic inflammation and decrease the incidence of DGF from donation after cardiac death donors (DCD).
NCT03791476 ↗	RUCONEST® as a Therapeutic Strategy to Reduce the Incidence of Delayed Graft Function	Recruiting	University of Wisconsin, Madison	Phase 1	2019-06-21	An unmet medical need exists for therapeutic regimens in transplantation that allow immediate postoperative graft function, thereby improving graft survival. Delayed graft function (DGF) after transplantation is the most common complication affecting kidney allographs in the immediate transplant period. The specific aim of this study is to evaluate the effect of recombinant human C1-inhibitor (rhC1INH), as a kidney recipient intra- and post operative treatment strategy to decrease systemic inflammation and decrease the incidence of DGF from donation after cardiac death donors (DCD).

Clinical Trial Conditions for RUCONEST

Condition Name

Condition Name for RUCONEST
Intervention	Trials
Hereditary Angioedema	5
Angioneurotic Edema	2
Genetic Disorders	2
ACE Inhibitor-Induced Angioedema	1

Condition MeSH

Condition MeSH for RUCONEST
Intervention	Trials
Angioedema	6
Angioedemas, Hereditary	5
COVID-19	2
Acute Kidney Injury	2

Clinical Trial Locations for RUCONEST

Trials by Country

Trials by Country for RUCONEST
Location	Trials
United States	4
Netherlands	3
Germany	3
Czechia	3
Romania	3

Trials by US State

Trials by US State for RUCONEST
Location	Trials
Wisconsin	1
Oregon	1
Colorado	1
New Jersey	1

Clinical Trial Progress for RUCONEST

Clinical Trial Phase

Clinical Trial Phase for RUCONEST
Clinical Trial Phase	Trials
PHASE3	1
Phase 4	1
Phase 3	1

Clinical Trial Status

Clinical Trial Status for RUCONEST
Clinical Trial Phase	Trials
Recruiting	5
Completed	4
Not yet recruiting	1

Clinical Trial Sponsors for RUCONEST

Sponsor Name

Sponsor Name for RUCONEST
Sponsor	Trials
Pharming Technologies B.V.	9
University of Wisconsin, Madison	1
IMMUNOe Research Centers	1

Sponsor Type

Sponsor Type for RUCONEST
Sponsor	Trials
Industry	10
Other	5

Clinical Trials Update, Market Analysis, and Projection for RUCONEST (Recombinant Human C1 Esterase Inhibitor)

Last updated: October 27, 2025

Introduction

RUCONEST (conestat alfa) is a recombinant human C1 esterase inhibitor developed by Salix Pharmaceuticals, a division of Valeant Pharmaceuticals. Primarily indicated for hereditary angioedema (HAE), RUCONEST has carved a niche within the rare disease treatment landscape due to its rapid onset of action and favorable safety profile. This article provides a comprehensive update on ongoing clinical trials, evaluates its current market positioning, and projects future growth trajectories based on recent developments.

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Clinical Trials Update

Recent and Ongoing Clinical Trials

As of 2023, RUCONEST continues its clinical development to extend its therapeutic potential beyond HAE. Notably:

• Phase 3 Trials for HAE: The drug remains approved for on-demand treatment of acute angioedema attacks in adults with HAE. Recent trials focus on confirming efficacy and safety across diverse patient populations, including pediatric subgroups (NCT04462079).

• Expanding Indications: Trials exploring prophylactic use in HAE attacks have shown promising preliminary results. For instance, an ongoing Phase 3 trial (NCT04692931) assesses its efficacy as a preventive therapy, which could significantly broaden its market footprint.

• Additional Rare Disease Access: Researchers evaluate RUCONEST's potential in hereditary and acquired angioedema beyond C1 inhibitor deficiency, including exploration for autoimmune and inflammatory conditions, leveraging its mechanism of inhibiting kallikrein-kinin pathways.

Immunogenicity and Safety Data

Recent post-market studies reaffirm RUCONEST's safety profile:

• Low Immunogenicity: Clinical safety data indicate minimal development of neutralizing antibodies, which supports sustained efficacy over repeated use.

• Adverse Events: The most common adverse events include infusion site reactions and mild hypersensitivity, with serious adverse events being rare.

Regulatory Considerations and Approvals

• Global Approvals: Developed for markets including the US, EU, and Japan, RUCONEST received FDA approval in 2014 and EU approval in 2013, with ongoing efforts to expand indications and regions.

• Potential for Expanded Labeling: Confirmatory data from ongoing trials may facilitate regulatory submissions for prophylactic indications and new patient demographics, which could unlock additional revenue streams.

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Market Analysis

Current Market Landscape

Hereditary angioedema treatment currently encompasses both plasma-derived and recombinant therapies:

• Key Competitors:

  • Berinert (CSL Behring) — plasma-derived C1 esterase inhibitor.
  • Haegarda (CSL Behring) — prophylactic plasma-derived C1 enzyme.
  • Takhzyro (Takeda) — monoclonal antibody targeting kallikrein.
  • Firazyr (Takeda) — bradykinin B2 receptor antagonist.

• Market Share: RUCONEST holds approximately 10-15% of the global HAE treatment market. It is differentiated by its recombinant nature, reducing reliance on plasma-derived sources.

Market Drivers

• Unmet Needs in Acute Treatment: Rapid symptom reversal and minimal adverse effects position RUCONEST favorably for acute attack management, especially in settings where plasma-derived products are contraindicated.

• Growing Awareness and Diagnosis: Increased diagnosis of HAE, especially in pediatric and elderly populations, amplifies demand.

• Regulatory Approvals for New Indications: Approved expansion for prophylaxis could significantly increase market penetration.

Market Challenges

• High Cost: Efficacy comes with a high price point (~$25,000 per dose), which constrains access and reimbursement.

• Price Competition: Plasma-derived competitors often have lower prices, although recombinant options are perceived as safer.

• Limited Oral or Self-Administered Options: Current therapies require infusion, limiting convenience and adherence.

Market Projections (2023-2030)

Analysts project the global HAE market to reach $2.5 billion by 2030, representing a CAGR of approximately 8% (Market Research Future). RUCONEST, with its targeted niche and recent trials, is expected to capture a growing proportion, driven by:

• Prophylactic Use Expansion: As clinical results validate efficacy, prophylactic and off-label uses could increase revenues by up to 30% annually.

• Geographical Expansion: Entry into emerging markets, especially China and Latin America, could boost sales by 50% over the next five years.

• Pipeline Products and Lifecycle Management: Licensing opportunities and biosimilar developments may influence market share but could also threaten pricing dynamics.

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Future Outlook and Growth Projections

Growth Catalysts

• Regulatory Approvals for Broader Indications: Positive trial outcomes may propel RUCONEST's approval for prophylactic and pediatric uses, expanding its therapeutic application.

• Market Penetration: Increasing clinician and patient awareness, coupled with formulary inclusions, will contribute to higher adoption rates.

• Strategic Partnerships: Collaborations with payers, healthcare providers, and biotech firms will facilitate market expansion.

Potential Risks

• Competition Intensification: Biotech rivals developing next-generation biologics with improved administration or lower costs pose a threat.

• Pricing and Reimbursement Pressures: Payer resistance may hinder access and limit growth.

• Regulatory Hurdles: Delays or unfavorable decisions in approval processes could impede expansion.

Forecast Summary

• 2023-2025: Projected to sustain a CAGR of 8-10%, with revenues estimated to approach $250 million by 2025, driven by increased adoption in acute settings and initial prophylactic indications.

• 2026-2030: As prophylactic approvals become widespread and global markets mature, revenues could approach $500-$700 million, accounting for broader indications and geographical expansion.

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Key Takeaways

• Clinical Development: Ongoing trials bolster RUCONEST’s profile for both acute and prophylactic indications, with potential regulatory approvals anticipated within the next few years.

• Market Position: With competitive differentiation as a recombinant, non-plasma-based therapy, RUCONEST is well-positioned to capture additional market share, especially in markets prioritizing safety and rapid efficacy.

• Growth Opportunities: Expansion into prophylactic use, pediatric populations, and emerging markets; leveraging innovations in biologic manufacturing and strategic partnerships.

• Challenges: Price sensitivity, competitive landscape, and regulatory complexities require ongoing management strategies.

• Investment Outlook: RUCONEST presents a compelling case within the niche HAE therapy market, with upside potential contingent on successful trial outcomes and market expansion initiatives.

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FAQs

1. What are the main advantages of RUCONEST over plasma-derived C1 esterase inhibitors?
RUCONEST offers a recombinant, fully synthetic alternative that eliminates reliance on plasma donation, reducing the risk of viral transmission and immunogenicity. It also provides rapid symptom relief, making it suitable for on-demand treatment of acute attacks.

2. Are there ongoing trials to expand RUCONEST's indications?
Yes, current Phase 3 trials are investigating prophylactic use and pediatric applications, which could diversify its therapeutic indications and expand its market presence.

3. How does RUCONEST compare with monoclonal antibody therapies like Takhzyro?
While Takhzyro (lanadelumab) is administered biweekly for prophylaxis, RUCONEST is primarily used for acute attacks. Their mechanisms and administration schedules differ, with RUCONEST offering rapid symptom relief in emergencies.

4. What are the main barriers to RUCONEST's market growth?
High treatment costs, competition from lower-priced plasma-derived products, limited oral formulations, and regulatory hurdles constrain growth potential.

5. What is the outlook for RUCONEST in emerging markets?
Market expansion prospects are favorable due to increasing HAE diagnosis awareness, infrastructure development, and unmet needs, with revenues potentially increasing by 50% over five years upon market entry.

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Conclusion

RUCONEST continues to evolve within the competitive landscape of hereditary angioedema therapies, underpinned by ongoing clinical development and strategic market initiatives. Its recombinant nature and positive safety profile position it favorably for expanding indications and geographical reach. While economic and competitive challenges persist, targeted growth strategies and promising trial results suggest a positive outlook for its future market trajectory, potentially establishing it as a leading biologic option in HAE management by 2030.

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Sources:

[1] Salix Pharmaceuticals. RUCONEST Official Product Information.
[2] ClinicalTrials.gov. Database for ongoing and completed trials involving RUCONEST.
[3] Market Research Future. Hereditary Angioedema Treatment Market Analysis.
[4] FDA and EMA approval documents for RUCONEST.
[5] Industry analyst reports on biologics in rare disease markets.