The United States hereditary angioedema (HAE) therapeutics market size is a dynamic and rapidly evolving sector of the healthcare industry. With a growing understanding of the genetic causes and underlying mechanisms of HAE, new treatment options are constantly being developed and approved. According to recent research, the US HAE therapeutics market is expected to grow at a compound annual growth rate (CAGR) of 8.20% between 2024 and 2032. This growth is driven by a combination of factors, including an increasing prevalence of HAE, a rising awareness of the condition among healthcare professionals and patients, and a growing demand for effective treatment options.
Understanding Hereditary Angioedema
HAE is caused by a mutation in the gene that codes for C1 inhibitor (C1-INH), a protein that regulates the activation of the complement and the kallikrein-kinin systems. These systems are involved in the inflammatory response and the production of bradykinin, a peptide that causes blood vessels to dilate and leak fluid into the surrounding tissues. When C1-INH is deficient or dysfunctional, the complement and the kallikrein-kinin systems are overactivated, leading to excessive bradykinin and swelling. There are three types of HAE, depending on the level and function of C1-INH:
- Type I HAE: The most common type, accounting for about 85% of cases. It is characterized by low levels of C1-INH in the blood.
- Type II HAE: Accounting for about 15% of cases. It is characterized by normal or high levels of C1-INH, but with reduced function.
- Type III HAE: A rare and poorly understood type, affecting mostly women. It is characterized by normal levels and function of C1-INH, but with other genetic or hormonal factors that trigger swelling.
The symptoms of HAE vary from person to person, but typically include swelling of the skin, mucous membranes, and internal organs, accompanied by pain, nausea, vomiting, diarrhea, and fever. The frequency and severity of attacks also vary, ranging from once every few months to several times a week. The triggers of attacks can include stress, trauma, infection, hormonal changes, medications, and foods. However, some attacks may occur without any apparent trigger. The diagnosis of HAE is based on the clinical history, physical examination, and laboratory tests that measure the level and function of C1-INH and the complement components.
Current Treatment Options
The treatment of HAE aims to prevent or reduce the frequency and severity of attacks, and to provide rapid relief of symptoms during an attack. There are two main approaches to HAE treatment: acute treatment and prophylactic treatment. Acute treatment is used to treat an ongoing attack, while prophylactic treatment is used to prevent or reduce the occurrence of future attacks.
Acute Treatment Options
The acute treatment options for HAE include antihistamines, corticosteroids, ecallantide, and icatibant. Antihistamines and corticosteroids are commonly used to treat allergic reactions, but they have limited efficacy and safety in HAE, as they do not target the underlying cause of swelling. They are administered by subcutaneous injection and have shown to be effective and well-tolerated in reducing the duration and severity of HAE attacks. However, they are expensive and require careful monitoring for potential side effects, such as anaphylaxis and angioedema.
Prophylactic Treatment Options
The prophylactic treatment options for HAE include C1-INH replacement therapy, bradykinin B2 receptor antagonist, and kallikrein inhibitor. C1-INH replacement therapy is the standard of care for HAE, as it restores the deficient or dysfunctional C1-INH and normalizes the complement and the kallikrein-kinin systems. It is administered by intravenous infusion, either on-demand or on a regular schedule, depending on the patient’s needs and preferences. It has proven to be effective and safe in preventing and reducing the frequency and severity of HAE attacks. However, it is also costly and requires access to a medical facility or home infusion service. They are administered by subcutaneous injection, either every two or four weeks, depending on the drug and the patient’s response. They have demonstrated to be effective and well-tolerated in reducing the frequency and severity of HAE attacks. However, they are also expensive and require careful monitoring for potential side effects, such as injection site reactions and hypersensitivity reactions.
Market Analysis
The US HAE therapeutics market is a dynamic and growing market, with a high unmet need for effective and convenient therapies. According to a report by Grand View Research, the US HAE therapeutics market size was valued at USD 1.8 billion in 2020 and is expected to grow at a CAGR of 8.20% between 2024 and 2032.
Key Players and Their Market Share
The US HAE therapeutics market is dominated by a few key players that have established a strong presence and reputation in the field. These include:
- Shire (now part of Takeda): The leading player in the market, with a market share of about 70% in 2020. Shire offers a portfolio of products for both acute and prophylactic treatment of HAE, such as Cinryze, Firazyr, Kalbitor, and Haegarda.
- CSL Behring: The second-largest player in the market, with a market share of about 20% in 2020. CSL Behring offers a product for prophylactic treatment of HAE, called Berinert, which is also approved for acute treatment in some countries.
- Dyax (now part of Sanofi): The third-largest player in the market, with a market share of about 10% in 2020. Dyax offers a product for acute treatment of HAE, called Kalbitor, which is also in development for prophylactic treatment.
- Other players: The remaining players in the market include BioCryst, Pharming, and Ionis, which offer products for acute or prophylactic treatment of HAE, such as Orladeyo, Ruconest, and Tegsedi, respectively.
Regulatory Landscape and Approval Process for HAE Therapies in the US
The US Food and Drug Administration (FDA) is the regulatory authority that oversees the approval and marketing of HAE therapies in the US. The FDA has also issued guidance documents and draft guidance documents for the clinical development and evaluation of HAE therapies, which outline the general principles and specific recommendations for the design, conduct, and analysis of clinical trials, as well as the endpoints, criteria, and methods for assessing the safety and efficacy of HAE therapies. The FDA has approved several HAE therapies in the past decade, based on the evidence from randomized controlled trials, open-label studies, and real-world data, that demonstrate the clinical benefit and favorable risk-benefit profile of these therapies.
Challenges and Opportunities
Despite the advances and innovations in the HAE therapeutics market, there are still several challenges and opportunities that need to be addressed and explored to improve the diagnosis and treatment of HAE in the US. These include:
Challenges in Diagnosing and Treating HAE
One of the major challenges in HAE is the delay and difficulty in diagnosing the condition, due to the rarity, variability, and complexity of the disorder. Many patients remain undiagnosed or misdiagnosed for years, and receive inappropriate or ineffective treatments, such as antihistamines or corticosteroids, that may worsen their condition or cause adverse effects. Moreover, some patients may not have access to specialized care or diagnostic tests, especially in rural or remote areas, or may face barriers such as stigma, discrimination, or lack of awareness. Therefore, there is a need for increasing the education and training of healthcare professionals and patients, as well as the availability and affordability of diagnostic tools and services, to facilitate the early and accurate diagnosis of HAE.
Another challenge in HAE is the variability and unpredictability of the response to treatment, as different patients may have different types, triggers, and severity of HAE, and may respond differently to different therapies. Therefore, there is a need for developing and implementing personalized and precision medicine approaches, that take into account the individual characteristics, preferences, and goals of each patient, and tailor the treatment accordingly. This may involve the use of biomarkers, genetic testing, pharmacogenomics, and patient-reported outcomes, to guide the selection, dosing, and monitoring of therapies, and to optimize the outcomes and satisfaction of patients.
Future Directions
The future of HAE treatment landscape in the US is bright and promising, as there are several emerging therapies and trends that may transform the diagnosis and treatment of HAE in the coming years. These include:
Emerging Therapies and Their Potential Impact
As mentioned above, there are several emerging therapies in development for HAE, that may offer novel and improved options for HAE patients. Some of these therapies are expected to be approved and launched in the US market in the near future, such as BCX7353, CSL312, and NTLA-2001, while others are in the early stages of clinical development, such as DTX401, ATN-249, and PHA121. These therapies may have a significant impact on the HAE treatment landscape, as they may expand the range and diversity of therapies, address the unmet needs and gaps in the current treatment landscape, and improve the outcomes and satisfaction of HAE patients. However, they may also pose challenges and uncertainties, such as the safety and efficacy profiles, the cost and reimbursement issues, and the market competition and penetration, that need to be carefully evaluated and monitored.
Role of Personalized Medicine in HAE Treatment
As mentioned above, there is a need for developing and implementing personalized and precision medicine approaches in HAE treatment, that take into account the individual characteristics, preferences, and goals of each patient, and tailor the treatment accordingly. This may involve the use of biomarkers, genetic testing, pharmacogenomics, and patient-reported outcomes, to guide the selection, dosing, and monitoring of therapies, and to optimize the outcomes and satisfaction of patients. Personalized medicine may also enable the identification and stratification of subgroups of patients, based on their genetic, molecular, or clinical features, and the development and evaluation of targeted therapies, that are specific and effective for these subgroups. Personalized medicine may have a significant role in HAE treatment, as it may enhance the understanding and management of the disorder, improve the quality and efficiency of care, and reduce the variability and unpredictability of the response to treatment.