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, 2020-2030F

Market Overview

Global Hereditary Angioedema Therapeutics Market was valued at USD 4.36 Billion in 2024 and is expected to reach USD 7.28 Billion in the forecast period with a CAGR of 8.89% through 2030. The global hereditary angioedema therapeutics market is experiencing significant expansion, driven by increasing awareness, improved diagnostic capabilities, and the introduction of novel treatment options. Advances in genetic research have enabled early and accurate diagnosis, allowing for timely intervention and better disease management. Pharmaceutical companies are focusing on developing targeted therapies, including monoclonal antibodies and small-molecule inhibitors, to provide long-term prophylaxis and acute treatment solutions. The growing prevalence of hereditary angioedema, coupled with rising healthcare expenditure and patient advocacy efforts, is further supporting market growth. Furthermore, regulatory approvals for innovative therapies and the emergence of subcutaneous and oral treatment options are enhancing patient adherence and convenience, creating a favorable outlook for the industry.

A strong pipeline of novel drugs and increased investment in research and development are shaping the competitive landscape. Biopharmaceutical companies are exploring next-generation therapies that offer enhanced efficacy, fewer side effects, and improved administration methods. The shift from intravenous to subcutaneous and oral treatments is transforming the standard of care, reducing the burden on patients and healthcare providers. Collaborations and strategic partnerships between pharmaceutical firms, research institutions, and patient organizations are accelerating drug development and market penetration. The expansion of prophylactic treatments is expected to drive long-term market sustainability, as more patients seek preventive measures rather than relying solely on acute treatments.

Challenges such as high treatment costs, limited awareness in certain populations, and reimbursement hurdles remain key concerns. Despite the availability of advanced therapies, access barriers in some healthcare systems hinder optimal patient outcomes. Competition among market players is intensifying, with companies striving to differentiate their products through improved safety profiles and cost-effectiveness. The need for continuous innovation to address unmet clinical needs, including therapies for pediatric patients and milder disease forms, presents ongoing opportunities for industry players. As technological advancements continue to shape drug development and patient care, the hereditary angioedema therapeutics market is poised for sustained growth, driven by scientific progress and evolving treatment paradigms.

Key Market Drivers

Advancements in Research and Development (R&D)

Advancements in Research and Development (R&D) have been pivotal in boosting the Global Hereditary Angioedema Therapeutics Market. Hereditary Angioedema is a rare genetic disorder characterized by debilitating and unpredictable swelling episodes that can be life-threatening. In recent years, the relentless pursuit of scientific knowledge and innovative therapies has transformed the landscape of HAE treatment.

One of the most significant drivers of market growth has been the breakthrough discoveries in understanding the underlying mechanisms of HAE. Researchers have delved deep into the genetic and biochemical aspects of the disease, leading to a more precise understanding of how it develops and progresses. This increased knowledge has paved the way for the development of targeted therapies that are specifically designed to address the root causes of HAE.

Monoclonal antibodies, in particular, have emerged as a revolutionary approach in HAE therapeutics. These monoclonal antibodies, such as lanadelumab and ecallantide, have been engineered to specifically inhibit the activity of proteins like plasma kallikrein, which play a critical role in HAE attacks. This precision in drug design has proven highly effective in preventing and managing HAE episodes, providing patients with newfound hope and relief from the burden of their condition.

Furthermore, the collaboration between pharmaceutical companies, academic institutions, and research organizations has facilitated the rapid translation of scientific discoveries into tangible treatments. This synergy has accelerated the development and approval of innovative therapies, ensuring that they reach the patients who need them in a timely manner.

The advancements in R&D have not only led to the discovery of novel treatments but have also expanded the understanding of HAE subtypes and disease variations. This knowledge has paved the way for personalized medicine approaches, tailoring treatments to suit individual patients' needs and genetic profiles.

In terms of recent statistics, the U.S. Food and Drug Administration (FDA) approved lanadelumab in August 2018 for prophylactic treatment to prevent HAE attacks in patients aged 12 years and older. Additionally, berotralstat received FDA approval in December 2020 as the first oral therapy for preventing HAE attacks in patients aged 12 years and older. These approvals signify significant milestones in the availability of effective treatments for HAE patients.

Moreover, the European Medicines Agency (EMA) authorized garadacimab in February 2025 for the routine prevention of recurrent HAE attacks in individuals aged 12 years and older. This approval further expands the therapeutic options available to HAE patients in the European Union. These recent regulatory approvals underscore the progress made in developing effective therapies for HAE, offering patients improved options for managing their condition and enhancing their quality of life.

Emergence of Monoclonal Antibodies

The global hereditary angioedema therapeutics market has been significantly transformed by the advent of monoclonal antibodies, particularly lanadelumab. Lanadelumab, marketed as Takhzyro, is a fully human monoclonal antibody that targets plasma kallikrein, a key enzyme in the pathogenesis of HAE. By inhibiting plasma kallikrein, lanadelumab effectively reduces the frequency and severity of HAE attacks. The U.S. Food and Drug Administration (FDA) approved Takhzyro in August 2018 for prophylaxis to prevent HAE attacks in patients aged 12 years and older. 

Hereditary angioedema is a rare genetic disorder with an estimated prevalence of approximately 1 in 50,000 individuals worldwide. It is characterized by recurrent episodes of severe swelling in various parts of the body, including the skin, gastrointestinal tract, and airways. These episodes can be debilitating and, in cases involving the airway, potentially life-threatening. Traditional treatment options required frequent administrations, often leading to challenges with patient compliance and quality of life.

The introduction of lanadelumab has addressed these challenges by offering a more convenient dosing regimen. Administered subcutaneously, the recommended starting dose is 300 mg every two weeks. For patients who achieve stable disease control, a dosing interval of 300 mg every four weeks may be considered after six months of therapy. This less frequent dosing schedule not only enhances patient adherence but also significantly improves quality of life by reducing the treatment burden.

The efficacy of lanadelumab has been demonstrated in clinical trials, showing a substantial reduction in the number of HAE attacks. Its targeted mechanism of action and favorable safety profile have made it a preferred option among healthcare providers and patients. The success of lanadelumab has also spurred further research and development in the field of monoclonal antibodies for HAE, encouraging pharmaceutical companies to invest in innovative therapies that offer improved outcomes and patient experiences.

Increased Disease Awareness

Increased disease awareness has played a significant role in boosting the Global Hereditary Angioedema Therapeutics Market. Hereditary Angioedema is a rare genetic disorder characterized by recurrent and often debilitating swelling episodes that can affect various parts of the body, including the skin, gastrointestinal tract, and airways. Historically, HAE has been underdiagnosed or misdiagnosed due to its rarity and the lack of awareness among healthcare professionals. However, in recent years, there has been a noticeable shift in the perception and recognition of this condition.

One of the key drivers behind the growing HAE therapeutics market is the heightened awareness of HAE among healthcare providers. Medical professionals, including allergists, immunologists, and emergency room physicians, are now better equipped to identify and diagnose HAE accurately. This improvement in diagnostic accuracy has led to more patients receiving the appropriate medical attention and treatment, thus expanding the patient pool in need of HAE therapeutics.

In addition to healthcare professionals, patient advocacy groups and organizations have played a crucial role in increasing awareness about Hereditary Angioedema. Groups such as the HAE Association and HAEi (Hereditary Angioedema International) have been actively involved in raising awareness, providing support to patients and their families, and advocating for better access to treatment options. Their efforts have not only empowered patients to seek proper medical care but have also influenced policy changes and increased funding for HAE research and development.

The rise of the internet and social media platforms has further contributed to disease awareness. Patients and their families are now able to connect with one another, share their experiences, and access valuable information about HAE. This online community has become a powerful force in educating the public and dispelling myths surrounding the condition.

Download Free Sample Report

Key Market Challenges

High Treatment Costs

The High treatment costs have emerged as a significant obstacle hindering the growth and accessibility of the Global Hereditary Angioedema Therapeutics Market. HAE is a rare genetic disorder characterized by recurrent and potentially life-threatening swelling episodes. While advancements in therapeutics have improved the quality of life for HAE patients, the exorbitant cost of these treatments poses a considerable challenge. The prohibitively high cost of treatment makes these life-saving therapies inaccessible to a substantial portion of HAE patients, especially those without comprehensive health insurance coverage. This leads to disparities in access to care, with only a fraction of those in need benefiting from the latest advancements in HAE therapeutics.

For individuals who manage to access HAE therapies, the financial strain can be overwhelming. Copayments, deductibles, and out-of-pocket expenses can quickly accumulate, potentially forcing patients to make difficult choices between their healthcare needs and other essential expenses.

Access to HAE treatments is even more challenging in regions with limited healthcare resources and lower income levels. This exacerbates global healthcare disparities, where patients in wealthier nations have greater access to cutting-edge treatments, while those in less affluent regions struggle to secure even basic care.

High treatment costs can impact patients' adherence to prescribed regimens. Some individuals may skip doses or discontinue treatment due to financial constraints, jeopardizing their health and increasing the risk of severe HAE attacks.

The high cost of HAE therapies places a strain on healthcare systems and insurance providers. As demand for these treatments increases with growing awareness and diagnoses, managing the financial aspects of providing these therapies becomes an ongoing challenge for healthcare stakeholders.

Complex Diagnosis and Misdiagnosis

complex diagnosis and misdiagnosis pose significant challenges to the Global Hereditary Angioedema Therapeutics Market. HAE is a rare genetic disorder characterized by recurrent and unpredictable swelling episodes, which can affect various body parts, including the skin, gastrointestinal tract, and airways. Despite advancements in our understanding of the disease, diagnosing HAE remains a complex and often elusive task.

As HAE affects only a small percentage of the population, many healthcare providers may lack experience in recognizing its symptoms. Furthermore, the symptoms of HAE can mimic those of other more common conditions, such as allergies or gastrointestinal disorders, leading to misdiagnosis or delayed diagnosis. This delay can have serious consequences for patients, as they may receive inappropriate treatments that do not address the root causes of their symptoms.

Misdiagnosis or delayed diagnosis can result in patients not receiving appropriate HAE-specific therapies promptly. This delay can lead to increased suffering, more severe attacks, and a higher risk of life-threatening complications.

Misdiagnosed patients may undergo unnecessary medical procedures, tests, or treatments, consuming healthcare resources that could be better utilized elsewhere. This inefficiency can strain healthcare systems and contribute to rising healthcare costs.

The accurate identification of HAE patients is crucial for conducting effective clinical trials of new therapies. Misdiagnosis or inclusion of patients with different conditions can skew trial results, making it difficult to evaluate the safety and efficacy of potential treatments.

Complex diagnosis and misdiagnosis underscore the importance of patient advocacy and education. Patient advocacy groups and organizations play a crucial role in raising awareness, providing resources, and supporting individuals in their journey to accurate diagnosis and appropriate treatment.

Key Market Trends

Personalized Medicine Approaches

Hereditary angioedema (HAE) is a rare genetic disorder characterized by recurrent episodes of severe swelling in various body parts, including the extremities, face, gastrointestinal tract, and airway. This condition is caused by mutations in the SERPING1 gene, leading to deficient or dysfunctional C1 inhibitor (C1-INH) proteins, which play a crucial role in regulating inflammatory pathways.

Personalized medicine has significantly advanced the management of HAE by tailoring treatments to individual genetic profiles and disease manifestations. The process begins with precise genetic testing to identify specific mutations in the SERPING1 gene, which helps determine the subtype of HAE a patient has. This accurate diagnosis is essential, as it guides the selection of targeted therapies that address the underlying pathophysiological mechanisms.

For instance, patients with Type I HAE, characterized by low levels of functional C1-INH, may benefit from C1-INH replacement therapies such as Berinert or Cinryze. These treatments work by supplementing the deficient protein, thereby preventing or reducing the frequency of swelling episodes. Conversely, patients with normal C1-INH levels but dysfunctional protein (Type II HAE) might respond better to therapies that inhibit the kallikrein-kinin pathway, such as lanadelumab (Takhzyro), a monoclonal antibody that targets plasma kallikrein to prevent bradykinin formation, a key mediator of swelling in HAE. 

The personalized approach extends beyond initial treatment selection to ongoing disease management. Therapeutic regimens are regularly adjusted based on the patient's response, attack frequency, and lifestyle considerations. This dynamic management strategy aims to optimize efficacy while minimizing adverse effects, thereby improving the patient's quality of life. Moreover, advancements in self-administration techniques and the development of subcutaneous formulations have empowered patients to manage their condition more effectively and with greater convenience.

In summary, personalized medicine has revolutionized the treatment landscape for hereditary angioedema by enabling precise, individualized therapeutic strategies. Through detailed genetic insights and tailored interventions, patients experience improved outcomes and a better quality of life, highlighting the importance of continued research and development in this field.

Advancements in Diagnostic Techniques

Advancements in diagnostic techniques are significantly boosting the Global Hereditary Angioedema Therapeutics Market. HAE is a rare genetic disorder characterized by recurrent and unpredictable swelling episodes, and the accuracy and speed of diagnosis have long been a challenge. However, recent developments in diagnostic techniques have revolutionized the way HAE is identified and managed.

Genetic testing is one of the most crucial advancements in HAE diagnosis. By analyzing a patient's DNA, healthcare providers can identify specific genetic mutations associated with HAE, allowing for precise subtype classification. This genetic information is invaluable in tailoring treatment plans to address the underlying genetic and biochemical factors contributing to the disease. Genetic testing has significantly reduced the risk of misdiagnosis or delayed diagnosis, ensuring that patients receive the most appropriate care from the outset.

Furthermore, advancements in biomarker identification have enhanced the accuracy and efficiency of HAE diagnosis. Biomarkers are specific molecules or proteins in the body that can indicate the presence and severity of the disease. Researchers have identified certain biomarkers associated with HAE, such as elevated levels of bradykinin. These biomarkers can be detected through blood tests, providing an additional diagnostic tool that helps confirm HAE diagnoses and assess disease activity.

The adoption of these advanced diagnostic techniques has several implications for the HAE therapeutics market. First, it reduces the time to diagnosis, allowing patients to start treatment sooner, which is crucial for managing the disease effectively. Second, it minimizes the risk of misdiagnosis or incorrect subtype classification, preventing patients from receiving inappropriate treatments. Third, it fosters confidence among healthcare providers, ensuring they can make informed decisions about the most suitable therapies for their patients.

Segmental Insights

Drug Class Insights

Based on the Drug Class, C1 Esterase Inhibitor emerged as the dominant segment in the Global Hereditary Angioedema Therapeutics Market in 2024. C1-INH replacement therapy has a long history of success in managing HAE attacks. It directly addresses the underlying cause of HAE, which is the deficiency or dysfunction of C1-INH, a protein involved in regulating the bradykinin pathway. By restoring adequate levels of C1-INH, these therapies effectively prevent and treat HAE attacks. C1-INH therapies offer various administration options, including intravenous (IV) infusion and subcutaneous (SC) injection. This flexibility allows healthcare providers to tailor treatment plans to individual patient preferences and needs. For instance, some patients may prefer the convenience of self-administered SC injections, while others may opt for IV infusion in a clinical setting.

Distribution Channel Insights

Based on the Distribution Channel, the Retail Pharmacy emerged as the dominant segment in the Global Hereditary Angioedema Therapeutics Market in 2024. Retail pharmacies are widely accessible to the general population. They are found in local communities and neighborhoods, making it convenient for patients to access their prescribed medications without having to travel long distances or visit specialized medical facilities. Retail pharmacies offer a convenient and familiar environment for patients to pick up their HAE medications. Patients can combine their regular visits to the pharmacy with other shopping or errands, saving them time and effort. 

Download Free Sample Report

Regional Insights

North America emerged as the dominant region in the Global Hereditary Angioedema Therapeutics Market in 2024. This is due to a combination of advanced healthcare infrastructure, strong regulatory support, and high awareness levels among healthcare professionals and patients. The region benefits from a well-established framework for rare disease management, ensuring timely diagnosis and access to advanced therapies. The widespread availability of genetic testing and biomarker-based diagnostics has contributed to early detection, enabling effective treatment initiation. Leading pharmaceutical companies continue to invest in research and development, bringing innovative therapies such as monoclonal antibodies and gene-based treatments to market. Regulatory bodies like the U.S. Food and Drug Administration (FDA) have played a crucial role by granting fast-track approvals and orphan drug designations, accelerating the commercialization of novel treatments. Favorable reimbursement policies and patient assistance programs have further improved treatment accessibility, reducing financial barriers. Additionally, the presence of key market players, active patient advocacy organizations, and increasing adoption of personalized medicine approaches have driven market expansion. The introduction of subcutaneous and oral therapies has enhanced patient adherence, reinforcing North America’s leadership in the HAE therapeutics market. With ongoing advancements and continued investments, the region is expected to maintain its dominant position in the coming years.

Recent Developments

• In February 2025, Astria Therapeutics, Inc. announced the launch of the ALPHA-ORBIT Phase 3 trial for navenibart, a potential long-acting therapy for hereditary angioedema (HAE). Designed for rapid and sustained attack prevention, navenibart offers a low treatment burden with administration every 3 to 6 months.
• In October 2024, Intellia Therapeutics initiated a Phase III HAELO trial for NTLA-2002, its investigational gene-editing therapy for hereditary angioedema (HAE). The multinational, placebo-controlled study will evaluate the therapy’s efficacy and safety in 60 adult patients with Type I or II HAE, with attack frequency reduction as the primary endpoint.
• In October 2024, Astria Therapeutics, Inc. announced that the European Commission (EC) granted Orphan Medicinal Product Designation (OMPD) to navenibart for hereditary angioedema (HAE) treatment. Navenibart, a monoclonal antibody inhibitor of plasma kallikrein, showed a 90-96% reduction in monthly attack rates in the Phase 1b/2 ALPHA-STAR trial, demonstrating strong safety and tolerability.
• In September 2024, Astria Therapeutics, Inc. announced that its monoclonal antibody navenibart (STAR-0215) received Orphan Drug Designation from the U.S. FDA for hereditary angioedema (HAE) treatment. Phase 1b/2 ALPHA-STAR trial results showed 90-96% reduction in attack rates over six months with a favorable safety profile.
• In June 2024, Ionis Pharmaceuticals entered a license agreement granting Otsuka Pharmaceutical exclusive rights to donidalorsen in the Asia-Pacific region for hereditary angioedema (HAE). Ionis will lead global development, while Otsuka handles regional regulatory approvals and commercialization in Asia-Pacific and Europe.

Key Market Players

• Shire plc
• Sanofi SA
• Pharming Healthcare Inc.
• Attune Pharmaceuticals
• Adverum Biotechnologies
• Arrowhead Pharmaceuticals
• Ionis Pharmaceuticals
• BioCryst Pharmaceuticals Inc.
• CSL Behring.
• CENTOGENE N.V

 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:

o   C1 Esterase Inhibitor

o   Selective Bradykinin B2 Receptor Antagonist

o   Kallikrein Inhibitor

o   Other Drug Classes

• Hereditary Angioedema Therapeutics Market, By Distribution Channel:

o   Hospital Pharmacy

o   Retail Pharmacy

o   Other Distribution Channels

• Hereditary Angioedema Therapeutics Market, By Application:

o   Prophylaxis

o   On-demand

• Hereditary Angioedema Therapeutics Market, By Region:

o   North America

§  United States

§  Canada

§  Mexico

o   Europe

§  France

§  United Kingdom

§  Italy

§  Germany

§  Spain

o   Asia-Pacific

§  China

§  India

§  Japan

§  Australia

§  South Korea

o   South America

§  Brazil

§  Argentina

§  Colombia

o   Middle East & Africa

§  South Africa

§  Saudi Arabia

§  UAE

Competitive Landscape

Company Profiles: Detailed analysis of the major companies present in the Global Hereditary Angioedema Therapeutics Market.

Available Customizations:

Global Hereditary Angioedema Therapeutics Market   report with the given market data, TechSci Research offers customizations according to a company's specific needs. The following customization options are available for the report:

Company Information

• Detailed analysis and profiling of additional market players (up to five).