Alpha-1 Antitrypsin Deficiency Disease Treatment Market – By Treatment Type, By Route of Administration, By Age Group, By End-use – Global Forecast, 2024 – 2032

Alpha-1 Antitrypsin Deficiency Disease Treatment Market Size

The global alpha-1 antitrypsin deficiency disease treatment market size accounted for USD 2.6 billion in 2023 and is expected to grow at 10.1% of CAGR from 2024 to 2032. The growth of the market is driven by several factors, including the rising prevalence of AATD-related respiratory and liver diseases, increased awareness and screening programs, advancements in diagnostic technologies, and the development of effective treatment options.
 

Additionally, the growing number of cases of chronic obstructive pulmonary disease (COPD), liver cirrhosis, and other conditions related to AATD is significantly boosting market growth. For instance, according to the American Lung Association, there are between 80,000 to 100,000 people in the U.S. living with alpha-1 antitrypsin (AAT) deficiency, putting them at greater risk for developing COPD. This highlights the critical need for effective interventions and therapies.
 

Furthermore, advancements in diagnostic technologies have been crucial for the early detection and treatment of AATD-related disorders. New diagnostic tools now allow healthcare providers to identify AATD earlier, enabling timely and personalized treatment plans, thereby propelling market growth.
 

AAT is a protein made in the liver that helps protect the lungs. Alpha-1 antitrypsin deficiency increases the risk of lung and other diseases. If the body does not produce enough AAT, the lungs become more vulnerable to damage from smoking, pollution, and dust. Treatment for AATD includes diagnostics, therapeutics, and preventive measures targeting AATD-related conditions. Treatment options include augmentation therapy with purified alpha-1 antitrypsin protein, gene therapy, inhaled therapies, liver transplantation for severe cases, and lifestyle changes.

 

Alpha-1 Antitrypsin Deficiency Disease Treatment Market Trends

The expansion of the market is significantly driven by the increasing prevalence of AATD, a genetic disorder that can lead to serious conditions such as chronic obstructive pulmonary disease (COPD) and liver disease. AATD affects approximately 1 in 2,500 individuals globally. However, the condition remains underdiagnosed, though awareness among healthcare providers and the public is growing, leading to increased identification and diagnosis rates. This heightened awareness, coupled with improved diagnostic capabilities, is driving demand for effective treatments.
 

As more individuals are diagnosed, the need for therapeutic options, including augmentation therapy, gene therapy, and other innovative treatments, is expanding. Pharmaceutical companies are responding by investing in the development of new and more effective therapies to meet this growing demand, recognizing the significant market potential. Additionally, government initiatives and support for rare diseases like AATD are contributing to market growth. Regulatory incentives, such as orphan drug designations, provide opportunities for companies to innovate and bring new treatments to market.
 

Alpha-1 Antitrypsin Deficiency Disease Treatment Market Analysis

Based on treatment type, the market is segmented into medication, therapy, and surgery. The medication segment is further divided into alpha-1 proteinase inhibitor, bronchodilators, corticosteroids, and other medications. The medication segment dominated the market with USD 1.9 billion in 2023.
 

• The medication segment in the alpha-1 antitrypsin deficiency (AATD) treatment market focuses on managing symptoms and preventing complications associated with this genetic disorder. AATD can lead to severe lung conditions like emphysema and chronic obstructive pulmonary disease (COPD), making medications essential in patient care.
   
• Bronchodilators are commonly used to relax the muscles around the airways, improving breathing in patients with AATD-related COPD.
   
• Corticosteroids, another important class of drugs in this segment, are used to reduce inflammation in the airways, thereby preventing exacerbations and further lung damage.
   
• Additionally, antibiotics are often prescribed to treat respiratory infections, to which AATD patients are particularly susceptible due to their compromised lung function.
   

Based on route of administration, the alpha-1 antitrypsin deficiency disease treatment market is divided into parenteral, intranasal, and oral. The parenteral segment accounted for significant market share of 58.1% in 2023.
 

• The parenteral route of administration plays a critical role in the treatment of alpha-1 antitrypsin deficiency (AATD). This method delivers therapeutic agents directly into the bloodstream, ensuring rapid and efficient drug absorption, which is particularly important for patients requiring immediate therapeutic intervention.
   
• In the AATD treatment market, the primary parenteral therapy is augmentation therapy, where patients receive infusions of purified human alpha-1 antitrypsin (AAT) protein. The parenteral route is favored because it quickly achieves the necessary plasma concentrations of AAT, reducing the risk of progressive lung damage.  
   

Based on age group, the alpha-1 antitrypsin deficiency disease treatment market is divided into pediatric and adult. The adult segment dominated the market with USD 2.3 billion in 2023.
 

• The adult age group constitutes a significant portion of the AATD treatment market, driven by increased disease awareness and advancements in therapeutic options.
   
• AATD, a genetic disorder leading to severe respiratory and liver complications, often manifests in adulthood, particularly in individuals aged 30 to 50. This age group is most affected due to the cumulative effects of AATD, which can result in chronic obstructive pulmonary disease (COPD) and emphysema.
   
• Moreover, the market for the adult age group is expanding due to improved diagnostic techniques that allow for earlier detection and intervention, which is critical for managing symptoms and improving quality of life.
   

Based on end-use, the alpha-1 antitrypsin deficiency disease treatment market is divided into hospitals, specialty clinics, and other end-users. The hospitals segment is expected to reach 3.6 billion by the end of 2032.
 

• Hospitals play a crucial role in the AATD treatment market, serving as the primary setting for diagnosis, treatment initiation, and disease management. Hospitals are often the first point of contact for patients with AATD, especially those experiencing severe symptoms such as emphysema or liver disease.
   
• Additionally, the complexity of AATD necessitates a multidisciplinary approach involving pulmonologists, hepatologists, and genetic counselors, who are typically based within hospital settings.
   
• Furthermore, hospitals are key players in advancing AATD treatments through clinical trials and research initiatives. They provide the necessary infrastructure for testing new therapies, such as gene editing and stem cell approaches, which hold promise for more effective and lasting treatments.
   

North America alpha-1 AATD treatment market is expected to grow at a 9.8% CAGR, to reach USD 2.9 billion by 2032.
 

• The U.S. market in North America is characterized by significant advancements in therapeutic interventions, driven by robust research and development activities. The U.S. market is propelled by the high prevalence of AATD and the growing geriatric population, which is more susceptible to the disease.
   
• Additionally, the U.S. market benefits from government initiatives and support from organizations such as the Alpha-1 Foundation, which funds research and advocates for patient access to effective treatments.
   
• Moreover, the integration of telemedicine and digital health tools enhances patient monitoring and treatment adherence, further driving market expansion.
   

The UK alpha-1 antitrypsin deficiency disease treatment market is experiencing robust growth in Europe market.
 

• The AATD treatment market in the UK is by a strong emphasis on public healthcare through the National Health Service (NHS), which plays a pivotal role in ensuring patient access to advanced treatments.
   
• In the UK, AATD is recognized as a significant health concern, particularly due to its potential to cause chronic obstructive pulmonary disease (COPD) and liver disease.
   
• The market also benefits from a well-established healthcare system, which ensures that patients have access to necessary treatments through government-funded programs. Additionally, patient advocacy groups, such as the Alpha-1 UK Support Group, play a crucial role in raising awareness and supporting individuals affected by AATD.
   

The Asia Pacific alpha-1 antitrypsin deficiency disease treatment market is witnessing substantial growth of 10.6% during the analysis period.
 

• The Asia Pacific region is emerging as a rapidly growing market for AATD treatments, driven by increasing healthcare expenditure, rising awareness, and improving healthcare infrastructure.
   
• Countries like Japan, China, and South Korea are leading the charge in the Asia-Pacific AATD treatment market. These nations have seen a rise in research activities and partnerships with global pharmaceutical companies, aimed at bringing advanced therapies to the region.
   
• Furthermore, the region's market growth is also supported by government initiatives to improve rare disease management and the increasing adoption of Western medical practices.
   

Alpha-1 Antitrypsin Deficiency Disease Treatment Market Share

The market is characterized by a highly competitive landscape, marked by rapid innovation and technological progress. Prominent pharmaceutical companies hold a significant share of the market, driven by their extensive portfolios of diagnostic tests and advanced treatment options, including a variety of medications and emerging therapeutic modalities. Strategic initiatives, such as mergers, acquisitions, and collaborations, further strengthen the market positions of key players, allowing them to expand their product offerings and access new markets.
 

Alpha-1 Antitrypsin Deficiency Disease Treatment Market Companies

The company profile section includes both companies that have commercial drugs available in the market as well as those that are onto clinical phase development. Prominent players operating in the alpha-1 antitrypsin deficiency disease treatment industry include:

• Arrowhead Pharmaceuticals, Inc.
• CHIESI Farmaceutici S.p.A.
• CSL Behring
• Epicrispr Biotechnologies, Inc.
• GlaxoSmithKline plc
• Grifols S.A.
• Intellia Therapeutics, Inc.
• Kamada Pharmaceuticals
• Mayo Foundation for Medical Education and Research
• National Jewish Health
• Shire Plc (Takeda Pharmaceutical Company Limited)
• The General Hospital Corporation
• The Johns Hopkins Hospital
   

Alpha-1 Antitrypsin Deficiency Disease Treatment Industry News:

• In July 2024, GSK announced the ongoing clinical development of GSK-5462688, an investigational treatment for alpha-1 antitrypsin deficiency, which is currently in Phase II clinical trials. GSK-5462688 is designed to address the genetic disorder alpha-1 antitrypsin deficiency, which can lead to severe lung and liver disease. The outcome of this trial is anticipated to significantly impact GSK's position in the treatment landscape for genetic disorders.

• In July 2024, Intellia Therapeutics received authorization to initiate a Phase 1/2 clinical trial for NTLA-3001, a groundbreaking gene editing treatment targeting alpha-1 antitrypsin deficiency. NTLA-3001 represents Intellia’s first wholly owned CRISPR-based in vivo targeted gene insertion candidate to progress into clinical trials. This strategy is expected to add value to the company’s business portfolio.
   

The alpha-1 antitrypsin deficiency disease treatment market research report includes an in-depth coverage of the industry with estimates & forecast in terms of revenue in USD Million from 2021 - 2032 for the following segments:

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Market, By Treatment Type

• Medication
  • Alpha-1 proteinase inhibitor
  • Bronchodilators
  • Corticosteroids
  • Other medications
• Therapy
  • Augmentation therapy
  • Oxygen therapy
  • Other therapies
• Surgery

Market, By Route of Administration

• Parenteral
• Intranasal
• Oral

Market, By Age Group

• Pediatric
• Adult

Market, By End-use

• Hospitals
• Specialty clinics
• Other end-users

The above information is provided for the following regions and countries:

• North America
  • U.S.
  • Canada
• Europe
  • Germany
  • UK
  • France
  • Spain
  • Italy
  • Netherlands
  • Rest of Europe
• Asia-Pacific
  • China
  • Japan
  • India
  • Australia
  • South Korea
  • Rest of Asia-Pacific
• Latin America
  • Brazil
  • Mexico
  • Argentina
  • Rest of Latin America
• Middle East and Africa
  • South Africa
  • Saudi Arabia
  • UAE
  • Rest of Middle East and Africa