Hereditary Angioedema Therapeutics Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Drug Class (C1 Esterase Inhibitor, Selective Bradykinin B2 Receptor Antagonist, Kallikrein Inhibitor, Other Drug Classes), By Distribution Channel (Hospital Pharmacy, Retail Pharmacy, Other Distribution Channels), By Application (Prophylaxis, On-demand), By Region and Competition, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 4.41 Billion
CAGR (2026-2031)	8.91%
Fastest Growing Segment	Prophylaxis
Largest Market	North America
Market Size (2031)	USD 7.36 Billion

Market Overview

The Global Hereditary Angioedema Therapeutics Market will grow from USD 4.41 Billion in 2025 to USD 7.36 Billion by 2031 at a 8.91% CAGR. Hereditary Angioedema (HAE) therapeutics encompass specialized pharmaceutical interventions designed to manage C1-inhibitor deficiency, categorized into acute therapies for sudden attacks and prophylactic agents to reduce swelling frequency. The market is primarily driven by the increasing identification of previously undiagnosed patients and the rising demand for oral preventative regimens that offer improved convenience over injectable alternatives. Additionally, favorable reimbursement frameworks in developed nations continue to facilitate broader patient access to these essential biologic and small-molecule drugs.

However, the market faces a significant hurdle regarding substantial diagnostic delays, which often leave the addressable patient population untreated for prolonged periods. Misdiagnosis remains a critical barrier, as symptoms are frequently confused with common allergic reactions, limiting the effective reach of available therapies. Illustrating the current accessibility landscape, according to HAE International, in 2024, 78% of its member organizations reported the availability of at least one hereditary angioedema medication. This statistic highlights that while therapeutic reach is expanding, gaps in consistent access persist as challenges to full market potential.

Key Market Drivers

The paradigm shift from on-demand treatment to long-term prophylaxis, supported by the growing adoption of patient-friendly oral administration routes, stands as a foremost market driver. Patients and physicians are increasingly favoring preventative regimens that reduce attack frequency and administration burden compared to traditional injectable acute therapies, significantly improving quality of life. This transition is commercially evident and rapidly reshaping revenue streams within the sector. According to BioCryst Pharmaceuticals, November 2024, in the 'Third Quarter 2024 Financial Results', ORLADEYO net revenue reached $116.3 million, a 35.7% increase year-over-year, underscoring the strong patient demand for oral prophylactic options over legacy treatments.

Simultaneously, the market is propelled by a robust clinical pipeline of next-generation biologics and gene therapies designed to offer durable, potentially curative outcomes. Innovations such as in vivo CRISPR-based editing are advancing through clinical stages with promising efficacy profiles that could redefine standard care by eliminating the need for chronic dosing. Highlighting this progress, according to Intellia Therapeutics, October 2024, in the 'Phase 2 Study of NTLA-2002', a single 50 mg dose resulted in an 81% mean monthly attack rate reduction compared to placebo during weeks 5 through 16. Furthermore, the commercial sector remains vigorous; according to Pharming Group N.V., in 2024, total third-quarter revenues increased by 12% to US$74.8 million, reflecting the sustained economic momentum across the global therapeutic landscape.

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Key Market Challenges

The primary hurdle obstructing the growth of the Global Hereditary Angioedema Therapeutics Market is the persistent issue of diagnostic delays and misdiagnosis. Because HAE symptoms closely mimic common histaminergic allergic reactions, frontline healthcare providers frequently fail to identify the condition accurately, often prescribing ineffective antihistamines or corticosteroids instead of necessary targeted therapies. This confusion at the point of care severely restricts the conversion of the potential patient population into active consumers of HAE therapeutics. Consequently, pharmaceutical companies face a significant barrier where the addressable market remains artificially suppressed, as a large volume of patients are unable to access or purchase these specialized biologic and small-molecule drugs simply because they have not been correctly identified.

The magnitude of this gap is underscored by recent data highlighting how many patients remain invisible to the healthcare system. According to HAE International, in 2024, the diagnosis rate for the condition was reported to be only 53%. This statistic indicates that nearly half of the potential patient base remains undiagnosed and, by extension, unreachable for therapeutic intervention. Until these diagnostic bottlenecks are resolved, the market will continue to operate well below its full revenue potential, unable to capitalize on the demand from a significant segment of the population that requires these life-altering prophylactic and acute treatments.

Key Market Trends

The commercialization of RNA-targeted therapies represents a precise evolution in prophylactic care, moving beyond protein replacement to the upstream inhibition of prekallikrein synthesis. Unlike traditional C1-esterase inhibitors, these antisense oligonucleotides silence the specific mRNA responsible for the disease pathway, offering a novel mechanism for patients unresponsive to standard treatments or those seeking less frequent dosing intervals. This therapeutic class effectively reduces attack frequency significantly, validating the potential of RNA interference to redefine standard management protocols. According to Ionis Pharmaceuticals, May 2024, in the 'Phase 3 OASIS-HAE Study', patients treated with the investigational RNA-targeted medicine donidalorsen achieved an 87% reduction in mean monthly attack rates compared to placebo, highlighting the robust efficacy of this emerging modality.

Simultaneously, the development of extended half-life biologics continues to command significant market share, maintaining relevance despite the growing influx of oral alternatives. While oral regimens offer convenience, monoclonal antibodies that target plasma kallikrein provide sustained, high-efficacy control for patients prioritizing maximum attack prevention over administration route. This segment is expanding through formulations that allow for less frequent subcutaneous dosing, ensuring long-term adherence and stability for the most severe cases. The economic resilience of this category is substantial; according to Takeda Pharmaceutical Company Limited, October 2024, in the 'Consolidated Financial Results for the First Half of Fiscal Year 2024', revenue for the subcutaneous prophylactic treatment Takhzyro grew to 95.3 billion JPY, demonstrating persistent demand for established injectable biologics amidst a diversifying competitive landscape.

Segmental Insights

The Prophylaxis segment currently represents the fastest-growing category in the Global Hereditary Angioedema Therapeutics Market due to a fundamental shift toward long-term disease management. This expansion is primarily driven by the introduction of convenient oral and subcutaneous therapies that significantly reduce the treatment burden compared to traditional acute care. Recent approvals by regulatory authorities such as the US FDA have validated these preventive options, encouraging physicians to prescribe long-term solutions more frequently. Consequently, the ability to minimize attack frequency and improve patient quality of life is fueling the sustained adoption of prophylactic treatments globally.

Regional Insights

North America leads the Global Hereditary Angioedema Therapeutics Market, supported by a highly developed healthcare infrastructure that drives high diagnostic rates compared to other global regions. This dominance is reinforced by the concentration of key biopharmaceutical firms and substantial investment in clinical research. Furthermore, the region benefits from established reimbursement models and efficient regulatory processes under the U.S. Food and Drug Administration (FDA), which accelerate the commercialization of novel therapies. Consequently, widespread patient access to both prophylactic and acute treatments secures North America’s position as the primary market contributor.

Recent Developments

• In June 2025, CSL Behring received approval from the U.S. Food and Drug Administration (FDA) for garadacimab-gxii, a novel prophylactic treatment for hereditary angioedema (HAE). The regulatory body authorized this first-in-class monoclonal antibody, which inhibits activated factor XII (FXIIa), for use in adult and pediatric patients aged 12 years and older. The approval was based on the pivotal Phase 3 VANGUARD trial, where the therapy demonstrated a substantial reduction in the frequency of attacks compared to placebo. This milestone introduced a new once-monthly subcutaneous injection option, offering patients a mechanism that targets the HAE cascade at its initiation point.
• In November 2024, Ionis Pharmaceuticals announced that the U.S. FDA accepted its New Drug Application for donidalorsen, an investigational RNA-targeted medicine designed for hereditary angioedema prophylaxis. The submission sought approval for preventing attacks in adult and pediatric patients aged 12 years and older. Supporting data from the Phase 3 OASIS-HAE and OASISplus studies, along with long-term extension results, demonstrated that the treatment achieved significant and sustained reductions in attack rates. The agency established a target action date for August 2025, positioning the company to potentially introduce a new therapeutic class that reduces the production of prekallikrein to control the disease.
• In October 2024, Intellia Therapeutics presented positive data from the Phase 2 study of NTLA-2002 at the American College of Allergy, Asthma & Immunology Annual Scientific Meeting. The biotechnology company reported that its in vivo CRISPR-based gene editing therapy met all primary and secondary endpoints in patients with hereditary angioedema. Results showed that a single infusion led to a profound and durable reduction in monthly attack rates, with the majority of patients remaining attack-free during the observation period. These findings validated the decision to advance the 50 mg dose into a global Phase 3 trial to evaluate its potential as a one-time functional cure.
• In September 2024, Pharvaris presented long-term data for deucrictibant, its oral bradykinin B2 receptor antagonist, at the Bradykinin Symposium in Berlin. The clinical-stage company shared findings from the open-label extension of its CHAPTER-1 study, which evaluated the drug's efficacy in preventing hereditary angioedema attacks. The analysis revealed that patients maintained a high level of disease control, experiencing a reduction in attack frequency of over 90 percent compared to baseline after one year of treatment. This evidence supported the continued development of the formulations to provide an effective oral alternative to injectable therapies for both on-demand and prophylactic use.

Key Market Players

• Takeda Pharmaceutical Company Limited
• CSL Behring LLC
• BioCryst Pharmaceuticals, Inc.
• Pharming Group N.V.
• Ionis Pharmaceuticals, Inc.
• KalVista Pharmaceuticals, Inc.
• Intellia Therapeutics, Inc.
• Astria Therapeutics, Inc.
• Alnylam Pharmaceuticals, Inc.
• Novartis AG

By Drug Class	By Distribution Channel	By Application	By Region
C1 Esterase Inhibitor Selective Bradykinin B2 Receptor Antagonist Kallikrein Inhibitor Other Drug Classes	Hospital Pharmacy Retail Pharmacy Other Distribution Channels	Prophylaxis On-demand	North America Europe Asia Pacific South America Middle East & Africa

Report Scope:

In this report, the Global Hereditary Angioedema Therapeutics Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• Hereditary Angioedema Therapeutics Market, By Drug Class:

• C1 Esterase Inhibitor
• Selective Bradykinin B2 Receptor Antagonist
• Kallikrein Inhibitor
• Other Drug Classes

• Hereditary Angioedema Therapeutics Market, By Distribution Channel:

• Hospital Pharmacy
• Retail Pharmacy
• Other Distribution Channels

• Hereditary Angioedema Therapeutics Market, By Application:

• Prophylaxis
• On-demand

• Hereditary Angioedema Therapeutics Market, By Region:

• North America
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE