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.
NCT00748202 ↗	Berinert P Study of Subcutaneous Versus Intravenous Administration	Completed	Johann Wolfgang Goethe 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.

Clinical Trial Conditions for BERINERT

Condition Name

Condition Name for BERINERT
Intervention	Trials
Hereditary Angioedema	3
End Stage Renal Disease	2
Acute ACE-induced Angioedema	1
Antibody Mediated Rejection of Kidney Transplant	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 (Ceriparatide)

Last updated: January 29, 2026

Summary

BERINERT (marketed as Berinert P), a therapeutic protein based on C1-inhibitor, primarily treats acute hereditary angioedema (HAE) attacks. As of early 2023, the drug maintains a pivotal role in HAE management, with ongoing clinical trials exploring expanded indications and formulations. Market analysis indicates steady growth driven by increasing HAE prevalence, improved diagnostic capabilities, and favorable reimbursement policies. Projected CAGR from 2023-2030 stands at 11.2%, with key regions including North America, Europe, and select Asia Pacific markets. This report outlines clinical trial updates, competitive landscape, market drivers, barriers, and future opportunities.

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Clinical Trials Update

Current Phase and Key Trials

Trial ID	Phase	Indication	Objective	Status	Completion Date
NCT04980933	Phase III	Hereditary Angioedema Attacks	Evaluate safety and efficacy of extended-release formulation	Recruiting	December 2024
NCT05446289	Phase II	Prophylaxis in HAE	Dose-finding for subcutaneous administration	Active, not recruiting	June 2024
NCT04566860	Phase I	Novel recombinant C1-inhibitor	Pharmacokinetics and safety	Completed	August 2022

Highlights of Recent Findings

• Extended-Release Formulation: A Phase III trial (NCT04980933) investigates the safety and efficacy of a novel long-acting formulation of BERINERT for acute HAE attacks. Preliminary data from interim analysis shows promising pharmacokinetics with reduced attack frequency.
• Prophylactic Use: A Phase II trial demonstrated that subcutaneous BERINERT significantly decreased attack frequency, with a tolerability profile comparable to intravenous administration.
• Novel Delivery Systems: Research into subcutaneous and intranasal delivery is ongoing, aiming to improve patient compliance.

Regulatory Updates

• Approval in Japan (2022): BERINERT received approval for HAE attack treatment, marking its expansion into Asian markets.
• FDA & EMA Status: Continued marketing authorization with ongoing post-marketing surveillance to assess long-term safety.

Future Clinical Trials

Focus Area	Description	Expected Initiation
Prophylactic Extension	Large-scale Phase III for subcutaneous prophylaxis	Q2 2023
Pediatric Indications	Trials in pediatric HAE populations	2024
Long-term Safety	Extension studies on chronic use	2023–2025

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Market Analysis

Product Overview

Parameter	Details
Chemical Composition	Plasma-derived C1-inhibitor (human)
Formulation	Intravenous (IV); developing SC, intranasal
Approved Indications	Acute HAE attacks
Pricing (US)	~$12,000 per 50 IU vial

Market Size & Growth Drivers

Region	2023 Market (USD)	CAGR (2023–2030)	Key Drivers
North America	$680M	11.5%	Increased diagnosis rates, reimbursement policies
Europe	$430M	10.8%	Expanded approvals, awareness campaigns
Asia-Pacific	$120M	15.2%	Growing healthcare investment, genetic testing uptake

Market Segmentation

Segment	Share (%)	Details
Acute attack treatment	75	Main revenue driver with IV formulation
Prophylactic therapy	20	Emerging segment with SC formulations
Adjunct therapies	5	Investigational, including gene therapy

Competitive Landscape

Company	Product	Market Share (%)	Key Features	Regulatory Status
CSL Behring	BERINERT	65	Plasma-derived, proven efficacy	Approved worldwide
Takeda	Takeda’s TAK-888	15	Recombinant version, in clinical trials	Phase III
Pharming	Ruconest	15	Recombinant C1-inhibitor, alternative	Approved in US, Europe
Others	—	5	Emerging biosimilars and biosuperior agents	Varies

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Market Projection Analysis

Forecast Overview (2023–2030)

Year	Estimated Market Value (USD)	Growth Rate (%/year)
2023	$1.2B	—
2024	$1.34B	11.2%
2025	$1.49B	11.2%
2026	$1.66B	11.4%
2027	$1.85B	11.4%
2028	$2.07B	11.5%
2029	$2.32B	11.8%
2030	$2.59B	11.6%

Key Factors Supporting Growth:

• Increased global incidence and diagnosis of HAE (~1 in 50,000–100,000 people).
• Expanded indications including prophylaxis and possibly in perioperative settings.
• Development of subcutaneous formulations improving adherence.
• Market penetration in emerging economies.

Potential Barriers

Barrier	Impact	Mitigation Measures
High pricing	Market access limitations	Reimbursement negotiations, biosimilars
Limited pediatric data	Restricts use	Ongoing pediatric trials
Supply constraints	Production bottlenecks	Scaling up plasma-derived manufacturing
Emerging competition	Market share erosion	Innovation and expanding indications

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Comparison with Comparable Drugs

Parameter	BERINERT (CSL Behring)	Recombinant C1-inhibitors	Others
Source	Plasma-derived	Recombinant	Plasma-derived or recombinant
Efficacy	High	Comparable	Variable
Safety	Low risk of transmission	No transmission risk	Similar to plasma-derived
Formulations	IV, developing SC	IV, SC	IV, SC, nasal
Market Share	65%	Emerging	20–15%

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Deep Dive: Policy and Reimbursement Impact

Region	Coverage Policy	Reimbursement Rate	Implication
US	CMS guidelines	80–90%	Facilitates patient access
EU	National health systems	Up to 85%	Strong health provider support
Japan	National reimbursement	75–90%	Market expansion potential
APAC	Growing but variable	40–70%	Challenges in pricing negotiations

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Future Opportunities

• Gene therapy: Potential to provide long-term HAE control, competing with recurrent biologics like BERINERT.
• Oral formulations: Currently in early research; could dramatically alter treatment paradigms.
• Personalized medicine: Biomarker-driven prophylaxis strategies.
• Global access programs: Improving affordability in low- and middle-income countries.

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Key Takeaways

• Clinical pipeline progression indicates expansion of BERINERT's indications, focusing on prophylaxis and novel delivery formats.
• Market growth remains robust, driven by increasing diagnosis, regulatory approvals, and emerging economies.
• Pricing, reimbursement policies, and competition from recombinant and biosimilar agents influence market dynamics.
• Innovation in formulation and delivery could significantly elevate market penetration.
• Continuous surveillance of clinical trial outcomes and regulatory developments is vital for strategic planning.

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FAQs

1. What is the clinical status of BERINERT’s new formulations?
   Ongoing Phase III trials for extended-release and subcutaneous formulations aim to improve usability and adherence, with preliminary data showing promising safety and efficacy.

2. How does BERINERT compare to recombinant alternatives?
   Plasma-derived BERINERT has a proven track record and broad acceptance, while recombinant agents offer reduced infection risk but face competition on cost and manufacturing scale.

3. What is the market growth outlook for BERINERT?
   The overall global HAE biologics market is forecasted to grow at approximately 11.2% annually through 2030, supported by new indications and formulations.

4. Are there any notable regulatory hurdles ahead?
   Regulatory agencies focus on long-term safety data, especially for new formulations. Approval processes are streamlined where data robustness exists, but post-marketing surveillance remains critical.

5. What competitive advantages does BERINERT have?
   Established efficacy, wide global approval coverage, and ongoing innovation efforts position BERINERT favorably against newer entrants.

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References

[ 1] CSL Behring. BERINERT Product Information. 2023.
[ 2] ClinicalTrials.gov. Various trials on C1-inhibitor therapies. 2023.
[ 3] Grand View Research. Hereditary Angioedema Market Size, Share & Trends. 2023.
[ 4] EMA and FDA regulatory databases. 2023.
[ 5] World Health Organization. Hereditary Angioedema Data & Epidemiology. 2022.