United States Hereditary Angioedema Therapeutics Market By Treatment, By Route of Administration, By End-use, Region and Competition, Forecast and Opportunities, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 883.56 Million
CAGR (2026-2031)	4.28%
Fastest Growing Segment	Prophylaxis
Largest Market	Midwest
Market Size (2031)	USD 1136.17 Million

Market Overview

The United States Hereditary Angioedema Therapeutics Market will grow from USD 883.56 Million in 2025 to USD 1136.17 Million by 2031 at a 4.28% CAGR. Hereditary Angioedema therapeutics encompass specialized pharmaceutical agents, such as C1-esterase inhibitors and bradykinin receptor antagonists, designed to manage the recurrent, severe swelling attacks associated with this rare genetic disorder. The market is primarily propelled by the paradigm shift from acute on-demand treatment to long-term prophylactic care, which ensures consistent medication usage and improved patient outcomes. Additionally, enhanced diagnostic screening is successfully identifying previously undiagnosed patients, thereby expanding the addressable market volume. According to the US Hereditary Angioedema Association, in 2025, a landmark study utilizing real-world claims data identified a prevalence of approximately 2.67 cases per 100,000 individuals in the United States, indicating a patient population significantly larger than historic estimates.

Despite these positive developments, a significant impediment to market scalability involves the substantial financial burden of lifelong biologic therapy and the resulting complex reimbursement landscape. High annual per-patient costs frequently trigger stringent payer utilization management tactics, such as step-therapy protocols or prior authorization delays, which can severely hinder patient access to these essential life-saving treatments.

Key Market Drivers

The Shift Toward Long-Term Prophylactic Treatment Regimens is fundamentally reshaping the market as patients and providers increasingly prioritize the prevention of attacks over acute management. This transition is driven by the development of advanced therapies that offer sustained disease control and reduced treatment burden, moving away from reactive acute care. A major milestone in this shift occurred with the introduction of RNA-targeted therapies, which promise extended dosing intervals and improved compliance. According to Ionis Pharmaceuticals, in August 2025, the U.S. FDA approved Dawnzera (donidalorsen), the first RNA-targeted prophylactic therapy for hereditary angioedema in patients aged 12 and older. This regulatory approval highlights the expanding arsenal of long-acting preventive solutions designed to significantly minimize attack frequency and enhance overall patient outcomes.

Concurrently, the Adoption of Novel Oral and Non-Invasive Therapies is accelerating, addressing the significant unmet need for needle-free administration in both acute and prophylactic settings. Patients are actively seeking alternatives to injectable biologics to alleviate the logistical and physical burden associated with lifelong therapy. According to KalVista Pharmaceuticals, in July 2025, the company received U.S. regulatory approval for Ekterly (sebetralstat), the first oral on-demand treatment for acute attacks. The growing market preference for convenient options is further evidenced by the financial performance of established oral agents. According to BioCryst Pharmaceuticals, in January 2025, preliminary full-year 2024 net revenue for the oral prophylactic Orladeyo reached $437 million, representing a 34% increase year-over-year. These developments signal a robust trend toward user-friendly, non-invasive treatment modalities that empower patients with greater independence.

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Key Market Challenges

The substantial financial burden of lifelong biologic therapy and the resulting complex reimbursement landscape significantly impede the commercial scalability of the United States Hereditary Angioedema Therapeutics Market. Because these specialized prophylactic agents command high annual per-patient costs, insurers frequently implement stringent utilization management tactics to control expenditures. These measures, including rigorous step-therapy protocols and prolonged prior authorization processes, create administrative bottlenecks that delay or deny patient access to prescribed regimens. Consequently, this disruption in continuity of care prevents the consistent medication usage necessary for prophylaxis, leading to preventable breakthrough attacks and limiting the real-world uptake of advanced therapeutics. This friction between clinical necessity and economic containment restricts the accessible patient base and stifles revenue growth for manufacturers. According to the World Allergy Organization, in 2025, an industry survey highlighted that 48% of treating physicians reported that fewer than half of their patients achieved optimal disease control, explicitly identifying reimbursement restrictions and high treatment costs as the principal obstacles to effective management.

Key Market Trends

The Emergence of Curative Gene Editing and RNA Interference Therapies is propelling the market toward one-time functional cures, fundamentally advancing beyond the scope of chronic disease management. While RNA-targeted prophylactics have improved dosing intervals, in vivo CRISPR-based gene editing candidates are now demonstrating the ability to permanently inactivate the kallikrein B1 gene, offering patients the prospect of total freedom from lifelong therapy. This transformative potential was highlighted by recent clinical milestones in gene insertion technologies that promise to eliminate the heavy treatment burden associated with standard-of-care regimens. According to Intellia Therapeutics, in November 2025, in a press release regarding the Phase 1/2 study of lonvoguran ziclumeran, 97% of patients treated with a single 50 mg dose remained completely attack-free and free from long-term prophylaxis through the data cutoff.

The Expansion of Therapeutic Labels for Pediatric Patient Populations is simultaneously broadening the addressable market by providing younger patients with access to advanced, less invasive prophylactic options. Historically, children have been limited to on-demand acute treatments or burdensome injectable prophylaxis, but recent regulatory successes are validating oral alternatives specifically for this demographic. This trend addresses a critical gap in care, as early intervention is essential to prevent the psychosocial impact of unpredictable attacks during formative years. According to BioCryst Pharmaceuticals, in December 2025, in a press release announcing the FDA approval of Orladeyo oral pellets, clinical data from the APeX-P trial showed that the therapy reduced the average monthly attack rate in children aged 2 to 11 years from 1.5 at baseline to 0.3.

Segmental Insights

The Prophylaxis segment currently represents the fastest-growing category within the United States Hereditary Angioedema Therapeutics Market. This rapid expansion is primarily driven by a strategic shift in clinical management towards long-term disease prevention rather than acute on-demand treatment. The United States Food and Drug Administration has recently approved novel oral and subcutaneous therapies that significantly improve patient adherence by reducing the administration burden associated with older intravenous options. Consequently, the increased availability of these convenient preventative options has encouraged broader adoption among healthcare providers, thereby fueling the sustained development of the prophylactic sector.

Regional Insights

The Midwest dominates the United States Hereditary Angioedema Therapeutics Market, primarily driven by a high density of specialized treatment centers and a consolidated patient population seeking expert care. This regional leadership is reinforced by the strategic presence of major pharmaceutical manufacturers and research hubs, which facilitates the rapid development and distribution of therapeutic solutions. Furthermore, the accessibility of clinical experts and robust healthcare infrastructure attracts patients for both long-term management and clinical trials. These factors collectively establish the Midwest as the central hub for HAE therapeutic innovation and commercial availability.

Recent Developments

• In August 2025, Ionis Pharmaceuticals announced that the U.S. FDA approved Dawnzera (donidalorsen) for the prophylactic treatment of hereditary angioedema in patients aged 12 years and older. This regulatory milestone established the drug as the first RNA-targeted medicine authorized for this specific genetic condition. The approval was supported by compelling efficacy data from the Phase 3 OASIS-HAE trial, which demonstrated that the therapy significantly reduced the mean monthly rate of swelling attacks compared to placebo. The company prepared for an immediate commercial launch to provide a new, sustained preventative option for the HAE patient community.
• In July 2025, KalVista Pharmaceuticals obtained U.S. FDA approval for Ekterly (sebetralstat), a novel oral plasma kallikrein inhibitor. This approval marked a significant advancement as the first oral, on-demand treatment for acute hereditary angioedema attacks in adults and pediatric patients aged 12 and older. The decision was backed by results from the Phase 3 KONFIDENT trial, which demonstrated that the drug achieved significantly faster symptom relief and attack resolution compared to placebo. Management emphasized that this portable therapy addresses a critical unmet need by providing a needle-free alternative for managing sudden and unpredictable swelling events.
• In June 2025, CSL Behring received approval from the U.S. Food and Drug Administration for its novel prophylactic treatment, Andembry (garadacimab-gxii). This monoclonal antibody became the first therapy in the United States to target activated Factor XII, treating the condition by inhibiting the cascade of events that lead to swelling attacks at their source. The regulatory clearance covered adult and pediatric patients aged 12 years and older. The company announced that the product would be launched immediately, offering a new mechanism of action for patients seeking effective, long-term prevention of debilitating hereditary angioedema episodes.
• In October 2024, Intellia Therapeutics announced positive results from a Phase 2 study of NTLA-2002, an investigational in vivo CRISPR gene editing therapy designed for Hereditary Angioedema. The clinical data indicated that a single 50 mg dose resulted in a mean monthly attack rate reduction of approximately 81% compared to placebo during the observation period. The company highlighted that the majority of patients in this dosage arm remained completely attack-free following the one-time infusion. Based on these promising findings, the company selected the 50 mg dose for its global pivotal Phase 3 study to further evaluate the therapy's potential as a functional cure.

Key Market Players

• BioCryst Pharmaceuticals, Inc.
• Ionis Pharmaceuticals, Inc.
• Seqirus Pty Ltd.
• Attune Pharmaceuticals, Inc.
• Adverum Biotechnologies, Inc.
• KalVista Pharmaceuticals, Inc.
• Takeda Pharmaceutical Company Limited
• Pharming Healthcare N.V.
• Sanofi AG
• Cipla Ltd.

By Region

Northeast Midwest South West

Report Scope:

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

• United States Hereditary Angioedema Therapeutics Market, By Region:

• Northeast
• Midwest
• South
• West