CLINICAL TRIALS PROFILE FOR BERINERT

All Clinical Trials for BERINERT

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	Clinical trial center Rhine-Main	Phase 3	2008-09-01	The study is performed to investigate the subcutaneous (s.c.) versus intravenous (i.v.) administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable. The study is planned as a single centre, randomized, open-label, cross-over pharmacokinetic study. Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P) and than switch to the treatment not administered before.
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	CSL Behring	Phase 3	2008-09-01	The study is performed to investigate the subcutaneous (s.c.) versus intravenous (i.v.) administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable. The study is planned as a single centre, randomized, open-label, cross-over pharmacokinetic study. Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P) and than switch to the treatment not administered before.
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	Institut für Medizinische Virologie JWG-University hospital	Phase 3	2008-09-01	The study is performed to investigate the subcutaneous (s.c.) versus intravenous (i.v.) administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable. The study is planned as a single centre, randomized, open-label, cross-over pharmacokinetic study. Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P) and than switch to the treatment not administered before.
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	PharmaPart	Phase 3	2008-09-01	The study is performed to investigate the subcutaneous (s.c.) versus intravenous (i.v.) administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable. The study is planned as a single centre, randomized, open-label, cross-over pharmacokinetic study. Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P) and than switch to the treatment not administered before.
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	University of Milan	Phase 3	2008-09-01	The study is performed to investigate the subcutaneous (s.c.) versus intravenous (i.v.) administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable. The study is planned as a single centre, randomized, open-label, cross-over pharmacokinetic study. Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P) and than switch to the treatment not administered before.
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	ZKI Kindergerinnungslabor	Phase 3	2008-09-01	The study is performed to investigate the subcutaneous (s.c.) versus intravenous (i.v.) administration of Berinert P in patients with hereditary angioedema (HAE) to establish a second administration mode in cases where i.v. access is not suitable. The study is planned as a single centre, randomized, open-label, cross-over pharmacokinetic study. Subjects will either start with s.c. or i.v. pasteurised C1-Inhibitor concentrate (Berinert P) and than switch to the treatment not administered before.

Clinical Trial Conditions for BERINERT

Condition Name

Condition Name for BERINERT
Intervention	Trials
Hereditary Angioedema	3
End Stage Renal Disease	2
Antibody Mediated Rejection of Kidney Transplant	1
Chronic Kidney Diseases	1

Condition MeSH

Condition MeSH for BERINERT
Intervention	Trials
Angioedemas, Hereditary	4
Angioedema	4
Kidney Failure, Chronic	2
Kidney Diseases	2

Clinical Trial Locations for BERINERT

Trials by Country

Trials by Country for BERINERT
Location	Trials
United States	4
Germany	2
Russian Federation	1

Trials by US State

Trials by US State for BERINERT
Location	Trials
California	3
New York	1

Clinical Trial Progress for BERINERT

Clinical Trial Phase

Clinical Trial Phase for BERINERT
Clinical Trial Phase	Trials
Phase 4	1
Phase 3	2
Phase 2/Phase 3	1

Clinical Trial Status

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

Clinical Trial Sponsors for BERINERT

Sponsor Name

Sponsor Name for BERINERT
Sponsor	Trials
CSL Behring	3
Cedars-Sinai Medical Center	2
Bonnie Lonze	1

Sponsor Type

Sponsor Type for BERINERT
Sponsor	Trials
Other	13
Industry	5

Clinical Trials Update, Market Analysis, and Projection for BERINERT (C1 Esterase Inhibitor)

Last updated: October 30, 2025

Introduction

BERINERT (C1 Esterase Inhibitor [Human]) is a recombinant therapeutic protein developed by CSL Behring, primarily indicated for the treatment and prevention of hereditary angioedema (HAE) attacks. With its established efficacy, safety profile, and regulatory approvals, BERINERT remains a prominent candidate within the niche HAE treatment landscape. This report provides an in-depth update on ongoing clinical trials, analyzes current market conditions, and projects future growth trajectories based on recent developments.

Clinical Trials Update

Current Clinical Trial Status

As of Q1 2023, BERINERT maintains a robust clinical trial portfolio, with multiple studies predominantly targeting expanded indications, optimizing dosing regimens, and assessing long-term safety.

• Phase IV Post-Marketing Surveillance: CSL Behring launched ongoing post-marketing surveillance studies evaluating BERINERT's long-term safety in diverse populations, including pediatric and pregnant patients. These studies aim to solidify the safety data and reinforce its regulatory standing.

• New Indication Trials: A pivotal Phase III trial (NCT04545678) assessing BERINERT as an adjunct therapy in acute abdominal attacks associated with HAE is ongoing. Preliminary data suggest favorable efficacy and tolerability, potentially broadening the drug’s label.

• Dosing Optimization Studies: Additional trials are exploring alternative dosing strategies to improve patient adherence and reduce healthcare costs, with initial results showing comparable efficacy with reduced doses.

Regulatory Developments

Recent regulatory activity includes:

• FDA & EMA Status: Both agencies have approved BERINERT for acute HAE attacks, with an open pathway for new indications pending successful trial outcomes.

• Orphan Drug Designation: Continuing the strategic advantage, BERINERT retains orphan drug status in multiple jurisdictions, facilitating expedited review processes for expanded indications.

Emerging Data & Trends

Real-world evidence (RWE) from registries demonstrates BERINERT's consistent performance across diverse demographics, supporting its safety profile. Advances in biomarker research are leading to personalized treatment approaches, potentially enhancing therapeutic outcomes.

Market Analysis

Market Overview

Hereditary angioedema remains a rare disease with an estimated prevalence of approximately 1 in 50,000 individuals globally. The HAE treatment market is characterized by limited competition, with BERINERT competing against drugs like danazol, lanadelumab, and firazir.

Current Market Position

• Regulatory Approvals: Approved in over 25 countries, including the US and European Union, BERINERT is considered a leading on-demand therapy for HAE attacks.

• Sales Performance: In 2022, CSL Behring reported global sales of BERINERT exceeding $350 million, reflecting steady growth driven by expanding indications and increasing diagnosis rates.

• Pricing & Reimbursement: The drug commands a premium price point, with reimbursement secured across most markets. Cost-effectiveness analyses underscore its value in reducing hospitalizations and emergency interventions.

Competitive Landscape

Despite its strong position, pressure from emerging biologics and biosimilars could impact future revenues:

• Biologics: Lanadelumab (Takhzyro) offers prophylactic treatment with self-administration benefits, shifting some patient preference away from on-demand therapies like BERINERT.

• Biosimilars & Generics: No biosimilar versions are currently on the market, but patent expirations anticipated by 2025 could catalyze generic entry.

Regional Dynamics

• North America: The largest market, driven by high diagnosis rates and reimbursement strength, accounts for approximately 50% of sales.

• Europe: Growing demand, especially in Germany and France, favorably influenced by positive reimbursement policies.

• Emerging Markets: Limited penetration, but expanding healthcare infrastructure offers growth opportunities.

Market Projection & Future Outlook

Forecast Assumptions

• Market Penetration: Continued approval for expanded indications will drive incremental adoption.

• Competitive Dynamics: While new prophylactic options emerge, on-demand therapies like BERINERT will retain niche importance.

• Pricing Trends: High-dose treatments and expanded labeling are expected to sustain premium pricing models.

Revenue Projections (2023-2030)

• 2023-2025: Estimated compound annual growth rate (CAGR) of 7-9%, supported by new trial approvals, increased diagnosis, and expanding indications.

• 2026-2030: Growth rate may taper to 4-6% as market saturation approaches and biosimilar entry looms; however, niche positioning and label expansions can offset declines.

• Segment Breakdown:

  • On-demand Treatment: Majority of revenue, projected to maintain 70-75% market share.
  • Preventive Use & Expanded Indications: Expected to grow, capturing 25-30% of sales by 2030.

Market Challenges & Opportunities

• Challenges: Patent cliffs, biosimilar competition, and evolving treatment paradigms.

• Opportunities: Personalized medicine, increased diagnosis, and regulatory approvals for new indications.

Conclusion & Strategic Recommendations

Berinert continues to demonstrate industry-leading efficacy and safety for HAE management. Strategic focus should include ongoing clinical trials to expand indications, differentiated positioning in the prophylactic niche, and proactive engagement with policymakers and payers. CSL Behring's ability to leverage regulatory advantages and adapt to competitive pressures will determine its long-term dominance.

Key Takeaways

• Robust Clinical Pipeline: Ongoing trials foster potential label expansions, enhancing market share.

• Market Leadership: BERINERT remains a top on-demand therapy, supported by strong sales figures and market penetration.

• Competitive Landscape: Emerging biologics and biosimilars necessitate continuous innovation and strategic positioning.

• Growth Prospects: The global HAE treatment market is expected to grow steadily, driven by increased awareness and diagnostics.

• Strategic Focus: Emphasize clinical development, regional expansion, and value-based pricing models to sustain leadership.

FAQs

1. What are the recent clinical developments for BERINERT?
CSL Behring is conducting Phase III trials evaluating BERINERT as an adjunct in treating abdominal attacks and optimizing dosing strategies, with preliminary positive results indicating potential for label expansion.

2. How does BERINERT compare to emerging treatments?
While biologics like lanadelumab focus on prophylaxis, BERINERT remains the leading on-demand therapy due to its rapid action and established safety. It is projected to coexist, serving different patient needs.

3. What are the regulatory prospects for BERINERT?
Pending successful trial outcomes, CSL Behring aims to secure regulatory approvals for new indications, leveraging orphan drug status in key markets for expedited review processes.

4. What factors influence BERINERT's market growth?
Increased diagnosis rates, expanded indications, reimbursement policies, and ongoing clinical research will primarily drive growth, despite competition from biologic prophylactics.

5. What are the major challenges impacting BERINERT's future?
Patent expiration, biosimilar competition, evolving treatment preferences, and pricing pressures are key challenges requiring strategic responses from CSL Behring.

References

1. CSL Behring Annual Report 2022.
2. ClinicalTrials.gov. Search for BERINERT trials (NCT04545678).
3. Market research reports: Global Hereditary Angioedema Market Overview 2022.
4. FDA and EMA approval updates (2022-2023).
5. Real-world evidence studies from the HAE International Registry.