The United States hereditary angioedema therapeutics market has witnessed a substantial transformation in recent years, fueled by advancements in genetic therapies, improved diagnostic awareness, and novel biologic drug launches. Valued at USD 5.18 billion in 2024, the market is projected to expand at an impressive CAGR of 13.10% between 2025 and 2034, ultimately reaching USD 17.74 billion by 2034.

The significant increase in diagnosis rates and the rise in patient access to innovative treatments have revolutionized the management of hereditary angioedema (HAE)—a rare genetic disorder characterized by unpredictable episodes of severe swelling in various parts of the body. The introduction of prophylactic therapies and targeted biologics has drastically improved patient quality of life, leading to sustained market growth across the country.

Overview of the United States Hereditary Angioedema Therapeutics Market

Hereditary angioedema (HAE) is primarily caused by a deficiency or dysfunction of the C1-esterase inhibitor (C1-INH), a protein crucial for regulating inflammation and vascular permeability. The U.S. has been at the forefront of therapeutic innovation for HAE, with ongoing research focusing on long-term prophylactic therapies and oral formulations.

The market landscape is being shaped by pharmaceutical giants like Takeda Pharmaceutical Company Limited, BioCryst Pharmaceuticals, and CSL Behring, all of which are investing heavily in R&D to introduce advanced treatment options that are more effective and patient-friendly.

United States Hereditary Angioedema Therapeutics Market Size and Share Analysis

The U.S. hereditary angioedema therapeutics market accounted for a significant share of the global market in 2024, owing to its robust healthcare infrastructure and growing adoption of biologic therapies.

• Market Value (2024): USD 5.18 Billion

• Forecast Value (2034): USD 17.74 Billion

• CAGR (2025–2034): 13.10%

The United States leads globally in both prophylactic and on-demand HAE therapies, thanks to early regulatory approvals, patient awareness programs, and active participation in clinical trials. With innovative therapies in the pipeline, the country is expected to maintain its dominance throughout the forecast period.

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Market Breakup by Drug Type

• C1-Esterase Inhibitor (C1-INH):
  This segment holds a substantial market share due to the widespread adoption of plasma-derived and recombinant C1-INH products. These therapies are used both for on-demand and preventive treatment.

• Bradykinin B2 Receptor Antagonist:
  Drugs targeting the bradykinin pathway have gained popularity for their effectiveness in acute attacks. They help reduce swelling by inhibiting bradykinin activity.

• Kallikrein Inhibitor:
  The kallikrein inhibitor segment is expanding rapidly with the introduction of subcutaneous and oral formulations, offering improved convenience and long-term management benefits.

• Others:
  Includes emerging biologics and next-generation gene therapies currently in the research and development phase.

Market Breakup by Route of Administration

• Parenteral:
  Parenteral formulations continue to dominate due to their rapid onset of action, critical for acute attacks. Intravenous and subcutaneous routes are commonly used.

• Oral:
  The oral route is gaining traction as pharmaceutical companies develop user-friendly formulations that ensure consistent dosing and enhanced patient adherence.

Market Dynamics and Key Trends

Rising Diagnosis and Awareness

Increased disease awareness among clinicians and patients, along with advanced genetic testing, has significantly improved HAE diagnosis rates across the U.S. Early detection allows for effective disease management and drives treatment uptake.

Shift Toward Prophylactic Therapies

There is a growing shift from on-demand to prophylactic treatment regimens. Long-term prevention reduces attack frequency and severity, improving patients’ daily functioning and mental well-being.

Technological Advancements and Innovation

Advancements in biologic and RNA-based therapeutics are redefining treatment paradigms. Companies like Ionis Pharmaceuticals and Arrowhead Pharmaceuticals are pioneering novel approaches targeting disease pathways at the genetic level.

Favorable Regulatory Landscape

The U.S. Food and Drug Administration (FDA) continues to support innovation through orphan drug designations, priority reviews, and expedited approvals, accelerating the market expansion.

Growth Opportunities in the U.S. Hereditary Angioedema Therapeutics Market

• R&D in Gene Therapy and RNA-based Treatments:
  The focus on disease-modifying therapies, including gene editing and RNA interference (RNAi), presents a major opportunity for long-term cures.

• Expansion of Patient Assistance Programs:
  Pharmaceutical companies are introducing financial aid and access initiatives, ensuring that high-cost therapies reach a wider patient base.

• Strategic Collaborations and Licensing Agreements:
  Partnerships between biotech firms and large pharmaceutical companies are facilitating faster product development and commercialization.

• Rising Healthcare Expenditure and Specialty Care:
  The increasing investment in rare disease management and specialized healthcare centers further boosts market accessibility.

Challenges Facing the United States Hereditary Angioedema Therapeutics Market

Despite its strong growth trajectory, the market faces some notable challenges:

• High Cost of Treatment: HAE therapies, especially biologics, remain expensive, limiting access for uninsured or underinsured patients.

• Limited Disease Awareness in Rural Areas: Although awareness is rising, delayed diagnosis in remote regions continues to be an issue.

• Dependence on Imported Raw Materials: Manufacturing complexities and supply chain dependencies can cause production bottlenecks.

• Adverse Effects and Long-term Safety: Continuous monitoring of biologic drugs is necessary to manage safety and efficacy concerns.

Competitive Landscape and Key Players

The U.S. hereditary angioedema therapeutics market is highly competitive, dominated by multinational corporations and emerging biotech innovators focusing on next-generation therapeutics.

Key Players Include:

• Takeda Pharmaceutical Company Limited – Leader in C1-INH products and prophylactic therapies.

• BioCryst Pharmaceuticals, Inc. – Known for developing the first oral kallikrein inhibitor, Orladeyo®.

• Sanofi – Expanding its rare disease portfolio through strategic acquisitions and biologics.

• CSL Behring LLC. – Focused on plasma-derived therapies with a global manufacturing presence.

• Pharming Group N.V. – Offers recombinant C1-INH therapy for acute treatment.

• Cipla, Inc. – Strengthening presence through partnerships and generics in rare disease segments.

• Ionis Pharmaceuticals Inc. and Arrowhead Pharmaceuticals, Inc. – Pioneers in RNAi and antisense technologies.

• Attune Pharmaceuticals, Inc., Adverum Biotechnologies, Inc., and Pharvaris B.V. – Developing innovative oral and gene-based treatment options.

Recent Developments

• BioCryst’s Orladeyo® continued to show robust growth in the U.S. market, marking a significant milestone for oral prophylaxis.

• Takeda launched new C1-INH formulations aimed at enhancing ease of administration.

• Pharvaris announced positive Phase II clinical trial results for its oral bradykinin B2 receptor antagonist.

• Ionis Pharmaceuticals is progressing its investigational antisense therapy for long-term disease control.

Future Outlook

The future of the United States hereditary angioedema therapeutics market looks promising, driven by a strong research pipeline, growing patient advocacy, and advancements in genetic medicine. By 2034, innovations in gene editing and next-generation biologics could potentially offer curative therapies, transforming HAE management from lifelong symptom control to permanent resolution.

Frequently Asked Questions (FAQs)

1. What is the current size of the United States hereditary angioedema therapeutics market?
The U.S. hereditary angioedema therapeutics market was valued at USD 5.18 billion in 2024 and is expected to reach USD 17.74 billion by 2034, growing at a CAGR of 13.10%.

2. What factors are driving the United States hereditary angioedema therapeutics market?
Key drivers include advancements in biologic therapies, growing awareness of genetic disorders, favorable FDA approvals, and increased patient diagnosis rates.

3. Which drug type dominates the hereditary angioedema therapeutics market in the U.S.?
C1-esterase inhibitors currently dominate the market, followed by kallikrein inhibitors and bradykinin B2 receptor antagonists.

4. What are the major challenges in the U.S. hereditary angioedema market?
High treatment costs, limited rural awareness, and long-term safety monitoring of biologics are major challenges.

5. Who are the leading companies operating in this market?
Leading companies include Takeda, BioCryst Pharmaceuticals, CSL Behring, Sanofi, and Pharming Group N.V.