Alpha-1 Antitrypsin Deficiency Market: Epidemiology, Therapies, Companies, DelveInsight | Arrowhead Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies

The Alpha-1 Antitrypsin Deficiency market size in the 7MM was around USD 830 million in 2023 and is expected to increase with a significant CAGR during the forecast period.

Therapies for Alpha-1 Antitrypsin Deficiency, including SAR447537 (INBRX-101), WVE-006, BEAM-302, NTLA-3001, KB408, and others, are expected to drive growth in the Alpha-1 Antitrypsin Deficiency market in the coming years.

DelveInsight has released a comprehensive report titled "Alpha-1 Antitrypsin Deficiency - Market Insights, Epidemiology, and Market Forecast-2034," providing detailed analysis of the disease, historical and projected epidemiology, and market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

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Some of the key facts of the Alpha-1 Antitrypsin Deficiency Market Report:

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In 2023, the Alpha-1 Antitrypsin Deficiency (AATD) market across the seven major markets (7MM) was valued at approximately USD 830 million and is expected to grow at a significant compound annual growth rate (CAGR) during the forecast period. The United States held the largest share, accounting for around 84% of the total market value that year.

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There were an estimated 224,750 prevalent AATD cases in the 7MM in 2023, with Japan showing notably lower prevalence compared to Europe and the U.S. Currently, no curative treatments exist for AATD, and the main therapeutic approach is augmentation (or replacement) therapy, approved specifically for managing lung complications associated with the disease. In the U.S., four augmentation therapies are available: Grifols' PROLASTIN-C, Takeda's ARALAST, CSL Behring's ZEMAIRA, and Kamada/Takeda's GLASSIA. In Europe, approved formulations include RESPREEZA (equivalent to ZEMAIRA in the U.S.), PROLASTIN, PROLASTINA, PROLASPLAN, PLITALFA, and ALFALASTIN in selected countries. In Japan, PROLASTIN-C is marketed as LYNSPAD.

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Among these, PROLASTIN by Grifols led the augmentation therapy market in 2023 based on sales, establishing it as the dominant product in the segment. In 2020, the distribution of AATD cases across the 7MM indicated that the U.S. accounted for approximately 59% of total prevalence, while the EU4 and the UK made up 40%, and Japan represented just 1%. By 2023, diagnosed prevalent cases in the U.S. were estimated at around 10,700, with numbers projected to increase by 2034.

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In March 2025, Beam Therapeutics Inc. (Nasdaq: BEAM) announced FDA approval of its investigational new drug (IND) application for BEAM-302, a potential therapy for AATD. Key companies such as Arrowhead Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, and others are actively developing new treatments to enhance the AATD therapeutic landscape. Promising pipeline candidates include SAR447537 (INBRX-101), WVE-006, BEAM-302, NTLA-3001, KB408, and others.

Alpha-1 Antitrypsin Deficiency Overview

According to the National Organization for Rare Disorders (NORD), alpha-1 antitrypsin deficiency (AATD) is an inherited disorder characterized by abnormally low levels of the alpha-1 antitrypsin (AAT) protein in the blood. This deficiency increases the risk of several health problems, most commonly manifesting as chronic obstructive pulmonary disease (COPD), including bronchiectasis, as well as liver conditions such as cirrhosis and hepatocellular carcinoma. In less common instances, AATD can also cause a skin disorder called panniculitis.

The condition arises from a combination of genetic mutations and environmental factors, which contribute to immune-mediated damage in the lungs, liver, and skin. Mutations in the SERPINA1 gene lead to reduced production or malfunction of the AAT protein. Without adequate AAT, proteolytic enzymes that are normally regulated by this protein can harm tissues, resulting in progressive lung damage, such as emphysema, and potential liver and skin complications.

Alpha-1 Antitrypsin Deficiency Market Outlook

Alpha-1 Antitrypsin Deficiency (AATD) is an inherited condition that mainly impacts the lungs and liver. Early detection and appropriate medical management are essential to enhance patient outcomes and quality of life. At present, no curative or disease-modifying treatments exist.

The primary approved therapy is augmentation (replacement) therapy, which addresses lung-related complications by boosting AAT protein levels through plasma-derived proteins from healthy donors. This treatment is specifically indicated for lung diseases associated with AATD, such as COPD and emphysema. Symptom management may also involve off-label medications, including bronchodilators, corticosteroids, and antibiotics. In severe cases, surgical options like lung volume reduction, bullectomy, or lung transplantation may be considered, though these procedures carry significant risks.

While AATD can also cause liver disease, no approved therapies currently target liver-related complications. Supportive care focuses on maintaining nutrition and providing essential nutrients to support liver function.

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Alpha-1 Antitrypsin Deficiency Marketed Drugs

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PROLASTIN-C LIQUID/LYNSPAD: Grifols

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GLASSIA: Kamada Pharmaceuticals/Takeda

Alpha-1 Antitrypsin Deficiency Emerging Drugs

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Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals

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Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals and Takeda

Scope of the Alpha-1 Antitrypsin Deficiency Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Alpha-1 Antitrypsin Deficiency Companies: Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, and others

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Alpha-1 Antitrypsin Deficiency Therapeutic Assessment: Alpha-1 Antitrypsin Deficiency current marketed and Alpha-1 Antitrypsin Deficiency emerging therapies

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Alpha-1 Antitrypsin Deficiency Market Dynamics: Alpha-1 Antitrypsin Deficiency market drivers and Alpha-1 Antitrypsin Deficiency market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Alpha-1 Antitrypsin Deficiency Unmet Needs, KOL's views, Analyst's views, Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement

To know what's more in our Alpha-1 Antitrypsin Deficiency report, https://www.delveinsight.com/report-store/alpha-1-antitrypsin-deficiency-a1atd-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Key benefits of the Alpha-1 Antitrypsin Deficiency Market Report:

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Alpha-1 Antitrypsin Deficiency market report covers a descriptive overview and comprehensive insight of the Alpha-1 Antitrypsin Deficiency Epidemiology and Alpha-1 Antitrypsin Deficiency market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).

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The Alpha-1 Antitrypsin Deficiency market report provides insights into the current and emerging therapies.

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The Alpha-1 Antitrypsin Deficiency market report provides a global historical and forecasted market covering drug outreach in 7MM.

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The Alpha-1 Antitrypsin Deficiency market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Alpha-1 Antitrypsin Deficiency market.

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Table of Contents

1. Report Introduction

2. Executive Summary

3. SWOT analysis

4. Alpha-1 Antitrypsin Deficiency Patient Share (%) Overview at a Glance

5. Alpha-1 Antitrypsin Deficiency Market Overview at a Glance

6. Alpha-1 Antitrypsin Deficiency Disease Background and Overview

7. Alpha-1 Antitrypsin Deficiency Epidemiology and Patient Population

8. Country-Specific Patient Population of Alpha-1 Antitrypsin Deficiency

9. Alpha-1 Antitrypsin Deficiency Current Treatment and Medical Practices

10. Unmet Needs

11. Alpha-1 Antitrypsin Deficiency Emerging Therapies

12. Alpha-1 Antitrypsin Deficiency Market Outlook

13. Country-Wise Alpha-1 Antitrypsin Deficiency Market Analysis (2020-2034)

14. Market Access and Reimbursement of Therapies

15. Market drivers

16. Market barriers

17. Appendix

18. Alpha-1 Antitrypsin Deficiency Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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Related Reports:

Alpha-1 Antitrypsin Deficiency Pipeline Insights, DelveInsight

"Alpha-1 Antitrypsin Deficiency Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Alpha-1 Antitrypsin Deficiency market. A detailed picture of the Alpha-1 Antitrypsin Deficiency pipeline landscape is provided, which includes the disease overview and Alpha-1 Antitrypsin Deficiency treatment guidelines.

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

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