The Hereditary Angioedema (HAE) market was valued at nearly USD 3 billion in 2023 and is poised for robust expansion, largely fueled by the approval and uptake of novel therapeutic options. HAE is a rare inherited disorder marked by repeated, unpredictable episodes of severe swelling affecting the face, extremities, gastrointestinal system, and upper airway. Available treatment options include C1-esterase inhibitor (C1-INH) replacement therapies such as BERINERT and RUCONEST, kallikrein inhibitors like ecallantide, and bradykinin B2 receptor antagonists including FIRAZYR. Preventive therapies such as CINRYZE, HAEGARDA, and lanadelumab are also widely used. The United States dominates the global HAE market, accounting for close to 90% of total revenue, while women represent approximately 65% of the diagnosed patient population.
The HAE development pipeline remains highly active, with several promising candidates including navenibart (STAR-0215), donidalorsen, NTLA-2002, and sebetralstat. Significant developments in 2024 included the FDA’s acceptance of Ionis Pharmaceuticals’ NDA for donidalorsen, KalVista Pharmaceuticals’ NDA filing for sebetralstat, and Astria Therapeutics’ strategic collaboration with Ypsomed for STAR-0215. Notably, positive Phase III OASIS-HAE trial outcomes for donidalorsen demonstrated more than a 90% reduction in attack frequency, highlighting strong potential for future market growth.
DelveInsight’s publication, “Hereditary Angioedema Market Insights, Epidemiology, and Market Forecast – 2034,” offers an in-depth evaluation of the HAE landscape. The report analyzes historical and forecasted epidemiology alongside market trends across the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan. It examines existing treatment paradigms, emerging therapies, individual drug contributions, and market size projections from 2020 through 2034 across the seven major markets. The report also discusses current treatment algorithms, key growth drivers, market limitations, and unmet clinical needs, enabling stakeholders to identify opportunities and assess long-term market potential.
To explore detailed insights on the Hereditary Angioedema market outlook, therapy adoption, treatment landscape, and epidemiological trends, click here; Hereditary Angioedema Market Forecast
Key Highlights from the Hereditary Angioedema Market Report
- The global HAE market was estimated at approximately USD 3 billion in 2023 and is expected to register a strong CAGR during the forecast period (2020–2034).
- In September 2025, following FDA approval of Dawnzera (donidalorsen) for HAE, Piper Sandler maintained an Overweight rating on Ionis Pharmaceuticals and increased its price target to USD 65, citing the company’s commercial execution and growth prospects in the competitive HAE market.
- In August 2025, Ionis Pharmaceuticals (NASDAQ: IONS) received FDA approval for Dawnzera (donidalorsen) to prevent HAE attacks in patients aged 12 years and older. Dawnzera became the first RNA-targeted therapy approved for HAE, designed to suppress plasma prekallikrein, a central driver of inflammatory attacks.
- In July 2025, KalVista Pharmaceuticals announced FDA approval of EKTERLY® (sebetralstat), the first oral, on-demand therapy for acute HAE attacks in patients aged 12 years and above, offering rapid symptom relief and marking the first major on-demand innovation in more than a decade.
- In June 2025, BioCryst Pharmaceuticals (NASDAQ: BCRX) reported new long-term efficacy and safety data for ORLADEYO® (berotralstat), used as a prophylactic therapy for HAE across all age groups.
- In March 2025, KalVista Pharmaceuticals (NASDAQ: KALV) completed patient enrollment for the KONFIDENT-KID open-label trial evaluating sebetralstat in pediatric HAE patients aged 2–11 years.
- In February 2025, Astria Therapeutics initiated the Phase III ALPHA-ORBIT study, a randomized, placebo-controlled trial assessing navenibart in adults and adolescents with Type I or Type II HAE.
- In January 2025, Intellia Therapeutics dosed the first patient in its global Phase III HAELO trial evaluating NTLA-2002, a CRISPR-based investigational therapy, in adults with HAE.
- In December 2024, Astria Therapeutics (NASDAQ: ATXS) announced positive final Phase 1b/2 ALPHA-STAR data for navenibart, demonstrating a 90–95% reduction in monthly HAE attacks over six months, with favorable safety and tolerability, supporting quarterly or biannual dosing.
- In November 2024, Ionis Pharmaceuticals confirmed FDA acceptance of its NDA for donidalorsen for the prevention of HAE attacks in adults and adolescents.
- In August 2024, Astria Therapeutics entered a partnership with Ypsomed to develop an autoinjector for STAR-0215, improving ease of administration and patient convenience.
- In June 2024, KalVista Pharmaceuticals submitted an NDA to the FDA for sebetralstat as an oral treatment for acute HAE attacks in patients aged 12 years and older.
- In May 2024, Ionis Pharmaceuticals released positive Phase III OASIS-HAE and OASISplus trial results showing sustained reductions exceeding 90% in monthly attack rates with donidalorsen, independent of dosing interval.
- The U.S. remains the largest HAE market, valued at nearly USD 2 billion in 2023, with continued growth expected.
- Among EU4 and the UK, France represented the largest market, valued at approximately USD 100 million in 2023.
- Females account for nearly 65% of all diagnosed HAE cases.
- Long-term prevention of HAE Types I and II currently relies on three main drug classes: C1-INH therapies, 17-alpha-alkylated androgens, and antifibrinolytics.
- In 2023, the diagnosed HAE population in the U.S. was estimated at around 7,000 patients, with Type I HAE comprising the majority of cases.
- Approximately 1,000 diagnosed patients fell within the 17–65 age group in 2023.
- Leading HAE market players include Ionis Pharmaceuticals, Astria Therapeutics, KalVista Pharmaceuticals, Intellia Therapeutics, BioMarin Pharmaceutical, CSL Behring, Pharvaris Netherlands B.V., Takeda, and Shire.
- Key pipeline therapies include donidalorsen, navenibart (STAR-0215), KVD900, NTLA-2002, BMN 331, garadacimab, TAK-743, CSL312, lanadelumab, and other investigational agents.
- Gender-based epidemiological analysis across the 7MM shows a higher prevalence among females.
- Increasing disease awareness, improved diagnostics, and the emergence of innovative multi-stage pipeline therapies are expected to significantly reshape the HAE treatment landscape in the coming years.
Hereditary Angioedema Overview
Hereditary Angioedema (HAE) is an uncommon inherited condition characterized by recurrent, severe swelling episodes involving the face, limbs, gastrointestinal tract, and airway, which may become life-threatening without timely intervention. The disease is caused by a deficiency or functional impairment of C1-esterase inhibitor, resulting in excessive bradykinin production and increased vascular permeability. HAE is categorized into Type I, Type II, and normal C1-INH variants. Women constitute the majority of affected individuals, and attacks may be triggered by stress, trauma, or hormonal fluctuations. Treatment strategies include on-demand agents such as C1-INH concentrates, kallikrein inhibitors, and bradykinin receptor antagonists, along with preventive therapies like lanadelumab and HAEGARDA. Emerging modalities, including RNA-based treatments, monoclonal antibodies, and gene-editing approaches, aim to deliver more durable and convenient disease control. Despite progress, challenges remain in achieving global access and long-term disease modification.
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Hereditary Angioedema Epidemiology
In 2023, the United States recorded approximately 7,000 diagnosed HAE cases, with around 5,500 attributed to Type I disease. Nearly 1,000 diagnosed patients were identified within the 17 to under-65 age bracket.
Epidemiology Segmentation Covered in the Report:
- Overall prevalence of Hereditary Angioedema
- Severity-based prevalence of HAE
- Gender-specific prevalence patterns
- Diagnosed episodic and chronic HAE cases
Download the report to understand the factors influencing HAE epidemiology trends: Hereditary Angioedema Epidemiology Forecast
Hereditary Angioedema Drug Uptake and Pipeline Highlights
Donidalorsen (Ionis Pharmaceuticals):
Donidalorsen (IONIS-PKK-LRx) is an investigational ligand-conjugated antisense therapy developed by Ionis Pharmaceuticals to suppress plasma prekallikrein production, a key driver of HAE attacks. By reducing PKK levels, the therapy aims to effectively prevent acute episodes. In the Phase III OASIS-HAE study, subcutaneous administration every four weeks reduced monthly attack rates by 81% compared with placebo, while eight-week dosing achieved a 55% reduction.
Navenibart (Astria Therapeutics):
Navenibart (STAR-0215) is a long-acting monoclonal antibody targeting plasma kallikrein, designed for sustained HAE prevention with dosing every three or six months. Data from the ALPHA-STAR Phase Ib/II trial demonstrated a 90–96% reduction in attack frequency, along with favorable safety and tolerability. Based on these outcomes, Astria plans to initiate Phase III trials in early 2025, with top-line results anticipated by late 2026.
Key Hereditary Angioedema Therapies and Companies
- Donidalorsen (IONIS-PKK-LRx) – Ionis Pharmaceuticals
- Navenibart (STAR-0215) – Astria Therapeutics
- KVD900 – KalVista Pharmaceuticals
- NTLA-2002 – Intellia Therapeutics
- BMN 331 – BioMarin Pharmaceutical
- Garadacimab – CSL Behring
- PHA121 – Pharvaris Netherlands B.V.
- TAK-743 – Takeda
- CSL312 – CSL Behring
- Lanadelumab – Shire
Discover therapies poised to capture significant HAE market share: Hereditary Angioedema Medication and Companies
Hereditary Angioedema Market Dynamics
Hereditary Angioedema Market Drivers:
- Increasing disease awareness among patients and clinicians
- Expanding availability of effective on-demand and preventive therapies
- Strong pipeline innovation with long-acting and convenient treatment options
- High clinical burden due to life-threatening attacks
- Regulatory incentives and orphan drug designations
- Strong dominance of the U.S. market supported by reimbursement frameworks
Hereditary Angioedema Market Barriers:
- High cost of advanced therapies limiting accessibility
- Frequent misdiagnosis or delayed diagnosis
- Small patient population typical of rare diseases
- Administration burden associated with injectable therapies
- Uneven awareness and access across global regions
To know more about Hereditary Angioedema companies working in the treatment market, visit @ Hereditary Angioedema Clinical Trials and Therapeutic Assessment
Scope of the Hereditary Angioedema Market Report
- Study Period: 2020–2034
- Geographic Coverage: United States, EU5 (Germany, France, Italy, Spain, UK), and Japan
- Key Companies: Ionis Pharmaceuticals, Astria Therapeutics, KalVista Pharmaceuticals, Intellia Therapeutics, BioMarin Pharmaceutical, CSL Behring, Pharvaris Netherlands B.V., Takeda, Shire, and others
- Key Therapies: Donidalorsen, Navenibart, KVD900, NTLA-2002, BMN 331, Garadacimab, CSL312, Lanadelumab, and others
- Analysis Includes: Market dynamics, competitive intelligence, unmet needs, market access, reimbursement, and strategic assessments
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