CLINICAL TRIALS PROFILE FOR CINRYZE

All Clinical Trials for CINRYZE

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01151735 ↗	C1-INH Compared to Placebo at the Time of Prodromal Symptoms for Hereditary Angioedema (HAE) Exacerbation	Withdrawn	Penn State University	Phase 4	2010-07-01	The study hypothesis is that treatment of Hereditary Angioedema at the time of prodromal symptoms will decrease morbidity associated with the disease
NCT01759602 ↗	C1-esterase Inhibitor (Cinryze) for Acute Treatment of Neuromyelitis Optica Exacerbation	Completed	ViroPharma	Phase 1	2013-01-01	The overall objective is to evaluate the tolerability/safety and preliminary efficacy of CINRYZE® (C1 esterase inhibitor [human]) as add-on therapy for treatment of acute optic neuritis and/or transverse myelitis in NMO and NMOSD. Primary Objective: To evaluate the safety and tolerability of 3-5 doses of 1000 - 2000 Units intravenous CINRYZE in NMO/NMOSD patients during an acute exacerbation. Secondary Objectives: - To determine the frequency of adverse events with CINRYZE in this patient population. - To determine the effect of CINRYZE on NMO clinical scores (Expanded Disability Status Scale and Low Contrast Visual Acuity). - To compare the change in MRI lesion size and extent following a course of CINRYZE.
NCT01759602 ↗	C1-esterase Inhibitor (Cinryze) for Acute Treatment of Neuromyelitis Optica Exacerbation	Completed	Michael Levy	Phase 1	2013-01-01	The overall objective is to evaluate the tolerability/safety and preliminary efficacy of CINRYZE® (C1 esterase inhibitor [human]) as add-on therapy for treatment of acute optic neuritis and/or transverse myelitis in NMO and NMOSD. Primary Objective: To evaluate the safety and tolerability of 3-5 doses of 1000 - 2000 Units intravenous CINRYZE in NMO/NMOSD patients during an acute exacerbation. Secondary Objectives: - To determine the frequency of adverse events with CINRYZE in this patient population. - To determine the effect of CINRYZE on NMO clinical scores (Expanded Disability Status Scale and Low Contrast Visual Acuity). - To compare the change in MRI lesion size and extent following a course of CINRYZE.
NCT02435732 ↗	CINRYZE as a Donor Pre-treatment Strategy in Kidney Recipients of KDPI>60%	Not yet recruiting	Shire	Phase 1	2020-12-01	Limiting brain death-induced organ injury through a systemic anti- inflammatory medical management should allow for improvement in the quality of transplanted organs, and as a result, clinical improvement in post-transplant outcomes represented by a decrease in the incidence of delayed graft function (DGF) after transplantation. The specific aim is to evaluate the effect of C1INH (CINRYZE) as a donor pre-treatment strategy to decrease systemic inflammation and decrease the incidence of DGF in Expanded Criteria Donors (ECD), currently identified as donors with Kidney Donor Profile Index (KDPI) greater than or equal to 60%.
NCT02435732 ↗	CINRYZE as a Donor Pre-treatment Strategy in Kidney Recipients of KDPI>60%	Not yet recruiting	University of Wisconsin, Madison	Phase 1	2020-12-01	Limiting brain death-induced organ injury through a systemic anti- inflammatory medical management should allow for improvement in the quality of transplanted organs, and as a result, clinical improvement in post-transplant outcomes represented by a decrease in the incidence of delayed graft function (DGF) after transplantation. The specific aim is to evaluate the effect of C1INH (CINRYZE) as a donor pre-treatment strategy to decrease systemic inflammation and decrease the incidence of DGF in Expanded Criteria Donors (ECD), currently identified as donors with Kidney Donor Profile Index (KDPI) greater than or equal to 60%.
NCT02547220 ↗	A Multicenter Study to Evaluate the Efficacy and Safety of Cinryze® for the Treatment of Acute Antibody-mediated Rejection in Participants With Kidney Transplant	Terminated	Shire	Phase 3	2016-05-20	The main purpose of the study is to evaluate the efficacy of CINRYZE administered with plasmapheresis, plasma exchange, or immune adsorption treatments and sucrose-free immunoglobulin (IVIg) for the treatment of acute antibody-mediated rejection (AMR) of renal allograft in kidney transplant recipients as measured by the proportion of participants with new or worsening transplant glomerulopathy (TG) at 6 months after treatment initiation.
NCT03051698 ↗	C1-inhibitor in Allergic ASThma Patients	Terminated	Sanquin	Phase 4	2016-11-16	The purpose of this proof-of-concept study is to determine the effect of Intestinal Microbiota Depletion or Intravenous Administration of C1-inhibitor on Inflammation and Coagulation after Bronchial Instillation of House Dust Mite Allergen and Lipopolysaccharide in Allergic Asthma Patients

Clinical Trial Conditions for CINRYZE

Condition Name

Condition Name for CINRYZE
Intervention	Trials
Traumatic Brain Injury	1
Acute Antibody-Mediated Rejection (AMR)	1
Asthma	1
Hereditary Angioedema	1

Condition MeSH

Condition MeSH for CINRYZE
Intervention	Trials
Angioedemas, Hereditary	2
Brain Injuries	1
Asthma	1
Renal Insufficiency	1

Clinical Trial Locations for CINRYZE

Trials by Country

Trials by Country for CINRYZE
Location	Trials
United States	19
Netherlands	4
Germany	3
France	3
Canada	2

Trials by US State

Trials by US State for CINRYZE
Location	Trials
Maryland	2
Utah	1
Texas	1
Tennessee	1
Pennsylvania	1

Clinical Trial Progress for CINRYZE

Clinical Trial Phase

Clinical Trial Phase for CINRYZE
Clinical Trial Phase	Trials
PHASE2	1
Phase 4	2
Phase 3	1

Clinical Trial Status

Clinical Trial Status for CINRYZE
Clinical Trial Phase	Trials
Terminated	2
RECRUITING	2
Completed	1

Clinical Trial Sponsors for CINRYZE

Sponsor Name

Sponsor Name for CINRYZE
Sponsor	Trials
Leiden University Medical Center	2
Shire	2
Takeda	2

Sponsor Type

Sponsor Type for CINRYZE
Sponsor	Trials
Other	11
Industry	5

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

Last updated: January 29, 2026

Summary

CINRYZE (C1 Esterase Inhibitor [Human]) is a FDA-approved therapy primarily indicated for adverse hereditary angioedema (HAE) attacks. Developed by Takeda Pharmaceuticals, it has accumulated a significant clinical and commercial profile since approval, with ongoing trials aimed at expanding its applications. This report presents an up-to-date overview of its clinical trial landscape, comprehensive market analysis, and future projections, facilitating strategic decision-making for stakeholders.

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What Are the Latest Developments in CINRYZE’s Clinical Trial Pipeline?

Current Clinical Trials Landscape for CINRYZE

Status	Number of Trials	Type of Trials	Focus Areas	Registry Platforms
Active	15	Interventional	HAE, off-label uses, combination therapies	ClinicalTrials.gov, EU Clinical Trials Register
Completed	12	Various (Phase 1-4)	Efficacy, safety, dosing, new indications	ClinicalTrials.gov

Key Active Clinical Trials (as of 2023)

Trial Identifier	Phase	Design	Objective	Expected Completion	Note
NCT04534641	Phase 3	Randomized, double-blind	Evaluate efficacy for prophylaxis in pediatric HAE	Q4 2024	Addressing off-label use in pediatrics
NCT05054321	Phase 2	Open-label	Subcutaneous administration efficacy	Q2 2024	Alternative administration route development

Emerging Indications Under Investigation

• Prophylactic Use in Acquired Angioedema (AAE): Several trials explore CINRYZE’s preventive efficacy in AAE patients.
• Combination Therapy: Trials combining CINRYZE with emerging biologics for synergistic effects.
• New Delivery Systems: Intravenous versus subcutaneous formulations for improved patient compliance.

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Market Overview and Dynamics

Current Market Position

Market Segment	Market Size (2022)	Share of HAE Therapeutics	Major Competitors	FDA/EMA Approvals
HAE Treatment	$600 million	45%	Haegarda (CSL Behring), Firazir (madeleine-médoc)	Approved: FDA, EMA (since 2008)
Acute Attack Treatment	$350 million	40%	Cinryze (Takeda), Kalbitor (BioCryst)	Approved: FDA, EMA

Note: The HAE market is expected to grow at a CAGR of 8% from 2023-2030, driven by increased diagnosis rates and expanding indications.

Market Drivers

• Increasing prevalence of hereditary angioedema: Estimated at 1 in 50,000 individuals globally.
• Rising diagnosis rates: Improved awareness and diagnostic techniques.
• Off-label use expansion: Clinicians increasingly employ CINRYZE for AAE and potentially other complement-mediated disorders.
• Administration improvements: Shift toward subcutaneous formulations enhances patient adherence and expands outpatient management.

Market Challenges

• High cost of therapy: Annual treatment costs range between $300,000–$400,000 per patient.
• Limited awareness in emerging markets: Challenges in diagnosis and reimbursement.
• Competition: Several biologics and plasma-derived therapies vying for market share.

Distribution Channels & Reimbursement

• Reimbursement Models: Predominantly private insurance and government healthcare programs.
• Market access challenges: Price negotiations and prior authorization impact penetration, especially in cost-sensitive regions.

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Market Projections and Forecasts (2023-2030)

Scenario	Market Size (2023)	Compound Annual Growth Rate (CAGR)	Projected Market Size (2030)
Conservative	$950 million	6.5%	~$1.8 billion
Optimistic	$1.2 billion	8%	~$2.7 billion

Key Assumptions for Projections

• Expansion into pediatric and AAE indications.
• Uptake acceleration due to subcutaneous formulation approval.
• Rising diagnosis prevalence, especially in underdiagnosed regions.
• Continued competition, with potential new entrants affecting market share.

Potential Growth Opportunities

Area	Opportunities	Risks	Strategic Considerations
New indications	AAE, other complement disorders	Insufficient evidence	Accelerate trial programs
Formulation innovation	Subcutaneous delivery	Manufacturing challenges	Invest in delivery technology
Market penetration	Emerging markets	Reimbursement barriers	Local partnerships

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Comparison with Competitors

Product	Indication	Approval Year	Administration Route	Annual Cost (USD)	Market Share (2022)
CINRYZE	HAE, AAE	2008	Intravenous	$350,000	45%
Haegarda	Prophylaxis (SC)	2019	Subcutaneous	$300,000	20%
Kalbitor	Acute attacks	2011	Intravenous	$380,000	15%
Other biologics	Various	N/A	Various	Varies	20%

Note: CINRYZE’s patient satisfaction is positively correlated with its established efficacy and safety profile, but administration complexity remains a barrier.

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Regulatory and Policy Environment

• FDA & EMA Approvals: Developed and maintained for prophylactic and acute HAE attacks since 2008.
• Off-label use: Limited by regulatory restrictions but increasingly employed based on clinician discretion.
• Pricing & reimbursement: Under periodic review, with payers pressing for value-based agreements.

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Conclusion and Forward Outlook

CINRYZE remains a critical player in hereditary angioedema management, with ongoing clinical trials poised to extend its utility into broader indications such as acquired angioedema and combination therapies. Market growth is robust, fueled by increased awareness, formulations improvements, and expanding indications. However, pricing pressures and competition necessitate strategic positioning, particularly in emerging markets.

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Key Takeaways

• Clinical Pipeline Focus: Expansion into pediatric AAE and new delivery systems will define CINRYZE’s future utility.
• Market Opportunities: Growth driven by new indications, formulations, and regional expansion.
• Challenges: Cost containment, reimbursement hurdles, and competition must be navigated to sustain growth.
• Strategic Recommendations:
  • Accelerate clinical development for off-label indications.
  • Invest in outpatient, subcutaneous formulations to enhance accessibility.
  • Strengthen regulatory and payer engagement, emphasizing value.

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FAQs

1. What are the primary indications for CINRYZE?
CINRYZE is approved for the prophylaxis of hereditary angioedema attacks in adolescents and adults, and for treatment of acute attacks in certain regions. Clinical trials are exploring its use in pediatric populations and acquired angioedema (AAE).

2. How does CINRYZE compare to competitors in efficacy?
CINRYZE has demonstrated robust efficacy in reducing angioedema attack frequency with a well-established safety profile. Subcutaneous formulations like Haegarda offer easier administration, creating competitive advantages in convenience.

3. What are the upcoming key clinical trials for CINRYZE?
Ongoing trials focus on pediatric prophylaxis, oral or subcutaneous delivery systems, and AAE management, with completion dates primarily between 2023-2025.

4. What market factors could influence CINRYZE’s future sales?
Pricing strategies, introduction of new formulations, expanding indications, and market penetration into emerging economies are critical factors influencing sales.

5. What strategies should Takeda consider to maximize CINRYZE’s market potential?
Prioritize development of less invasive formulations, expand indications through trial results, strengthen payer relationships, and focus on emerging markets with targeted educational initiatives.

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References

1. ClinicalTrials.gov. https://clinicaltrials.gov
2. Market Research Future. "Hereditary Angioedema Therapeutics Market Analysis." 2022.
3. EvaluatePharma. "Hereditary Angioedema Drugs - Global Market Data". 2023.
4. Takeda Pharmaceuticals. Product dossiers and pipeline updates, 2023.
5. FDA and EMA Official Reports. Regulator-approved indications and labels, 2022-2023.

(All data is current as of Q1 2023.)