Alpha-1 Antitrypsin Deficiency Market - A Global and Regional Analysis: Focus on Treatment, Distribution Channel, and Regional Analysis - Analysis and Forecast, 2025-2035

Industry Overview

The global alpha-1 antitrypsin deficiency (AATD) market is experiencing growth, fueled by advances in diagnosis, treatment options, and increasing awareness of the condition. AATD is a genetic disorder where the body produces insufficient alpha-1 antitrypsin, a protein that protects the lungs and liver. Its deficiency can lead to chronic obstructive pulmonary disease (COPD), emphysema, and liver cirrhosis. The condition often remains underdiagnosed, which presents an opportunity for early detection and intervention.

The primary treatment for AATD is augmentation therapy, which involves the infusion of AAT protein to restore deficient levels in the bloodstream. This therapy is designed to protect the lungs and slow the progression of emphysema. Other treatment options include symptomatic management, such as bronchodilators and corticosteroids. Research continues to explore gene therapies and novel treatments to better address the underlying genetic causes of AATD.

The market is expanding due to rising awareness about AATD, improvements in diagnostic tools, and innovations in treatment options. Key drivers include the increasing prevalence of the disease, particularly in older populations, and the growing availability of specialized therapies. However, challenges such as high treatment costs and the need for long-term care remain significant factors impacting the global alpha-1 antitrypsin deficiency market growth.

In summary, the global alpha-1 antitrypsin deficiency market is poised for growth, with a strong focus on improving diagnostic accuracy and providing effective treatments, offering significant opportunities for stakeholders in the healthcare and pharmaceutical industries.

Impact

Increasing demand for Alpha-1 Antitrypsin Deficiency therapies is anticipated to support the growth of the global alpha-1 antitrypsin deficiency market during the forecast period 2025-2035.
The global alpha-1 antitrypsin deficiency market is expected to grow at a significant rate due to advancements in diagnostic technologies, the development of innovative therapies, and increasing awareness among patients and healthcare providers.

Market Segmentation:

Segmentation 1: by Treatment

Augmentation Therapy
Bronchodilators
Corticosteroids
Oxygen Therapy

Segmentation 2: by Distribution Channel

Hospitals Pharmacy
Retail Pharmacies
Online Pharmacies

Segmentation 3: by Region

North America
Europe
Asia-Pacific
Rest-of-the-World

The North America alpha-1 antitrypsin deficiency market is expected to lead globally due to advanced healthcare infrastructure, high awareness, and significant research investments. The market is primarily driven by augmentation therapy, which involves the administration of alpha-1 proteinase inhibitors to manage disease progression. Enhanced genetic testing and early diagnosis have improved treatment outcomes. Hospitals and specialty clinics serve as key treatment settings, offering specialized care. The region's favorable reimbursement policies and commitment to research and development further support market growth, positioning North America as a dominant force in the global alpha-1 antitrypsin deficiency treatment landscape.

Recent Developments:

Regulatory Activities: In March 2025, Beam Therapeutics announced that the U.S. FDA cleared its Investigational New Drug (IND) application for BEAM-302, a base-editing therapy for AATD. This marks the first clinical advancement of a base-editing approach for AATD, with initial Phase 1/2 trial data showing promising results in correcting the PiZ mutation associated with the disease.
Regulatory Activities: In February 2025, Grifols completed enrollment of the second cohort in its Phase 1/2 study for Alpha-1 15%, a subcutaneous AATD treatment. This development could provide patients with a more convenient administration option, enhancing treatment adherence and quality of life.
Acquisition: In May 2024, Sanofi completed its acquisition of Inhibrx, Inc., gaining control of INBRX-101, a recombinant human AAT-Fc fusion protein under development for AATD treatment. INBRX-101 aims to normalize serum AAT levels with less frequent dosing, potentially offering a significant advancement in AATD therapy.

Demand – Drivers and Limitations

The following are the drivers for the global alpha-1 antitrypsin deficiency market:

Increasing Awareness and Early Diagnosis
Advancements in Treatment Options

The global alpha-1 antitrypsin deficiency market is expected to face some limitations too, due to the following challenges:

High Treatment Costs
Limited Awareness in Emerging Markets

How Can This Report Add Value to an Organization?

Product/Innovation Strategy:Product launches and innovations in the global alpha-1 antitrypsin deficiency market are focused on advancing treatment options to improve patient care. These innovations aim to enhance the efficacy of therapies and streamline the detection and management of the disease. Key players in the global alpha-1 antitrypsin deficiency market, such as Grifols, CSL Behring, Takeda, GlaxoSmithKline plc, and Pfizer Inc., have been involved in offering of therapies for alpha-1 antitrypsin deficiency.

Competitive Strategy:Enterprises led by market leaders in the global alpha-1 antitrypsin deficiency market are continuously working on updating their product portfolios with innovative treatments to maintain competitiveness. A detailed competitive benchmarking of the key players in the global alpha-1 antitrypsin deficiency market has been conducted, providing insights into how these companies compare in terms of product offerings, market share, and innovation. This benchmarking provides readers with a clear understanding of the market landscape and the positions of the leading players. Additionally, comprehensive competitive strategies, such as partnerships, agreements, and collaborations, will help readers identify untapped revenue opportunities in the global alpha-1 antitrypsin deficiency market.

Some of the prominent names established in this market are:

Grifols
CSL Behring
Takeda Pharmaceuticals
LFB Biotechnologies
GlaxoSmithKline plc
AstraZeneca
Merck
Pfizer Inc.

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Executive Summary
Scope of Study
1. Global Alpha-1 Antitrypsin Deficiency Market: Market Outlook
1.1 Industry Outlook
1.1.1 Market Overview and Ecosystem
1.1.2 Market Trends
1.1.3 Epidemiological Analysis of Alpha-1 Antitrypsin Deficiency
1.1.3.1 By Region
1.1.4 Clinical Trials
1.1.4.1 By Phase
1.1.4.2 By Sponsor Type
1.1.5 Regulatory Landscape / Compliance
1.1.5.1 Legal Requirement and Framework in the U.S.
1.1.5.2 Legal Requirement and Framework in the E.U.
1.1.5.3 Legal Requirement and Framework in Japan
1.1.5.4 Legal Requirement and Framework in Rest-of-the-World
1.2 Market Dynamics
1.2.1 Impact Analysis
1.2.2 Market Drivers
1.2.3 Market Restraints
1.2.4 Market Opportunities
2. Global Alpha-1 Antitrypsin Deficiency Market, By Treatment, $Million, 2023-2035
2.1 Overview
2.2 Augmentation Therapy
2.3 Bronchodilators
2.4 Corticosteroids
2.5 Oxygen Therapy
3. Global Alpha-1 Antitrypsin Deficiency Market, By Distribution Channel, $Million, 2023-2035
3.1 Overview
3.2 Hospitals Pharmacy
3.3 Retail Pharmacies
3.4 Online Pharmacies
4. Global Alpha-1 Antitrypsin Deficiency Market, By Region, $Million, 2023-2035
4.1 North America
4.1.1 Key Findings
4.1.2 Market Dynamics
4.1.3 Market Sizing and Forecast
4.1.3.1 North America Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)
4.1.3.2 North America Alpha-1 Antitrypsin Deficiency Market (by Country)
4.1.3.2.1 U.S.
4.1.3.2.2 Canada
4.2 Europe
4.2.1 Key Findings
4.2.2 Market Dynamics
4.2.3 Market Sizing and Forecast
4.2.3.1 Europe Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)
4.2.3.2 Europe Alpha-1 Antitrypsin Deficiency Market (by Country)
4.2.3.2.1 U.K.
4.2.3.2.2 Germany
4.2.3.2.3 France
4.2.3.2.4 Italy
4.2.3.2.5 Spain
4.2.3.2.6 Rest of Europe
4.3 Asia-Pacific
4.3.1 Key Findings
4.3.2 Market Dynamics
4.3.3 Market Sizing and Forecast
4.3.3.1 Asia-Pacific Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)
4.3.3.2 Asia-Pacific Alpha-1 Antitrypsin Deficiency Market (by Country)
4.3.3.2.1 Japan
4.3.3.2.2 China
4.3.3.2.3 Rest of Asia-Pacific
4.4 Rest-of-the-World
4.4.1 Key Findings
4.4.2 Market Dynamics
4.4.3 Market Sizing and Forecast
4.4.3.1 Rest-of-the-World Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel)
5. Global Alpha-1 Antitrypsin Deficiency Market - Competitive Landscape and Company Profiles
5.1 Competitive Landscape
5.1.1 Growth Share Matrix (2024)
5.1.1.1 By Treatment
5.1.2 Key Strategies and Developments by Company
5.1.2.1 Funding Activities
5.1.2.2 Mergers and Acquisitions
5.1.2.3 Regulatory Approvals
5.1.2.4 Partnerships, Collaborations and Business Expansions
5.1.3 Key Developments Analysis
5.2 Company Profiles
5.2.1 Grifols
5.2.1.1 Company Overview
5.2.1.2 Product Portfolio
5.2.1.3 Target Customers/End Users
5.2.1.4 Analyst View
5.2.2 CSL Behring
5.2.2.1 Company Overview
5.2.2.2 Product Portfolio
5.2.2.3 Target Customers/End Users
5.2.2.4 Analyst View
5.2.3 Takeda Pharmaceuticals
5.2.3.1 Company Overview
5.2.3.2 Product Portfolio
5.2.3.3 Target Customers/End Users
5.2.3.4 Analyst View
5.2.4 LFB Biotechnologies
5.2.4.1 Company Overview
5.2.4.2 Product Portfolio
5.2.4.3 Target Customers/End Users
5.2.4.4 Analyst View
5.2.5 GlaxoSmithKline plc
5.2.5.1 Company Overview
5.2.5.2 Product Portfolio
5.2.5.3 Target Customers/End Users
5.2.5.4 Analyst View
5.2.6 AstraZeneca
5.2.6.1 Company Overview
5.2.6.2 Product Portfolio
5.2.6.3 Target Customers/End Users
5.2.6.4 Analyst View
5.2.7 Merck
5.2.7.1 Company Overview
5.2.7.2 Product Portfolio
5.2.7.3 Target Customers/End Users
5.2.7.4 Analyst View
5.2.8 Pfizer Inc.
5.2.8.1 Company Overview
5.2.8.2 Product Portfolio
5.2.8.3 Target Customers/End Users
5.2.8.4 Analyst View
6. Research Methodology
List of Figures
Figure: Global Alpha-1 Antitrypsin Deficiency Market, Market Overview
Figure: Global Alpha-1 Antitrypsin Deficiency Market, Epidemiological Analysis, U.S.
Figure: Global Alpha-1 Antitrypsin Deficiency Market Coverage
Figure: Global Alpha-1 Antitrypsin Deficiency Market Key Trends, Impact Analysis, 2023-2035
Figure: Global Alpha-1 Antitrypsin Deficiency Market, Competitive Landscape, January 2022-April 2025
List of Tables
Table: Global Alpha-1 Antitrypsin Deficiency Market, Regulatory Scenario
Table: Global Alpha-1 Antitrypsin Deficiency Market Dynamics, Impact Analysis
Table: Global Alpha-1 Antitrypsin Deficiency Market (by Treatment), $Million, 2023-2035
Table: Global Alpha-1 Antitrypsin Deficiency Market (by Distribution Channel), $Million, 2023-2035

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