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

Alpha-1 Antitrypsin Deficiency therapies, such as SAR447537 (INBRX-101), WVE-006, BEAM-302, NTLA-3001, KB408, and others, are expected to boost the Alpha-1 Antitrypsin Deficiency Market in the upcoming years.

DelveInsight has launched a new report on "Alpha-1 Antitrypsin Deficiency - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Alpha-1 Antitrypsin Deficiency, historical and forecasted epidemiology as well as the Alpha-1 Antitrypsin Deficiency market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

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

In 2023, the Alpha-1 Antitrypsin Deficiency (AATD) market across the seven major markets (7MM) was valued at approximately USD 830 million and is projected to grow at a notable compound annual growth rate (CAGR) over the forecast period. The United States represented the largest share, contributing around 84% of the total market value that year.
There were an estimated 224,750 prevalent cases of AATD in the 7MM in 2023, with significantly lower prevalence observed in Japan compared to Europe and the U.S.
Currently, there are no curative treatments for AATD. The primary therapeutic option is augmentation therapy (also known as replacement therapy), which is approved specifically for treating lung complications associated with the condition. 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, various formulations such as RESPREEZA (ZEMAIRA in the U.S.), PROLASTIN, PROLASTINA, PROLASPLAN, PLITALFA, and ALFALASTIN are approved in select countries. In Japan, PROLASTIN-C is sold under the brand name LYNSPAD.
Among these therapies, PROLASTIN by Grifols led the market in 2023 based on sales, making it the dominant product in the augmentation therapy segment for AATD.
In 2020, the distribution of Alpha-1 Antitrypsin Deficiency (AATD) prevalent cases across the seven major markets (7MM) showed that the United States accounted for around 59% of the total, while the EU4 and the UK comprised 40%, and Japan represented just 1%.
By 2023, the number of diagnosed prevalent AATD cases in the U.S. was estimated at approximately 10,700, with this figure projected to rise by 2034.
In March 2025, Beam Therapeutics Inc. (Nasdaq: BEAM) announced that the U.S. Food and Drug Administration (FDA) had approved its investigational new drug (IND) application for BEAM-302, a potential therapy for alpha-1 antitrypsin deficiency (AATD).
Key Alpha-1 Antitrypsin Deficiency companies such as Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, and others are evaluating new drugs for Alpha-1 Antitrypsin Deficiency to improve the treatment landscape.
Promising Alpha-1 Antitrypsin Deficiency therapies 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 a genetic condition marked by abnormally low levels of the alpha-1 antitrypsin (AAT) protein in the bloodstream. This deficiency increases susceptibility to various health issues, most commonly presenting as chronic obstructive pulmonary disease (COPD), including bronchiectasis, and liver disorders such as cirrhosis and hepatocellular carcinoma. In rarer cases, it can also lead to a skin condition known as panniculitis.

The underlying causes and mechanisms of AATD involve a complex interplay of genetic mutations and environmental influences, triggering immune-mediated damage to the lungs, liver, and skin. Mutations in the *SERPINA1* gene result in reduced production or malfunction of the AAT protein. Without sufficient AAT, proteolytic enzymes-normally regulated by this protein-can damage tissues, leading to progressive lung destruction (such as emphysema) and potential liver and skin complications.

Alpha-1 Antitrypsin Deficiency Market Outlook

Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary disorder that primarily affects the lungs and liver. Early diagnosis and proper medical management are crucial for improving patient outcomes and quality of life. Currently, there are no curative or disease-modifying treatments available.

The main approved treatment is augmentation (replacement) therapy, which is used to manage lung-related complications by increasing AAT protein levels using plasma-derived proteins from healthy donors. This therapy is specifically approved for alpha-1-related lung disease, such as COPD and emphysema. Symptom management often includes off-label use of medications like bronchodilators, corticosteroids, and antibiotics. In advanced cases, surgical interventions such as lung volume reduction, bullectomy, or even lung transplantation may be considered, though these come with significant risks.

Although AATD is also linked to liver disease, no therapies are currently approved to treat liver-related complications. Supportive care focuses on nutritional maintenance and providing essential nutrients to support liver function.

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Alpha-1 Antitrypsin Deficiency Marketed Drugs
PROLASTIN-C LIQUID/LYNSPAD: Grifols
GLASSIA: Kamada Pharmaceuticals/Takeda

Alpha-1 Antitrypsin Deficiency Emerging Drugs
Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals
Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals and Takeda

Scope of the Alpha-1 Antitrypsin Deficiency Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Alpha-1 Antitrypsin Deficiency Companies: Arrow Head Pharmaceuticals, Mereo Biopharma, Kamada Ltd, Grifols, CSL Behring, Shire, LFB Biotechnologies, and others
Alpha-1 Antitrypsin Deficiency Therapeutic Assessment: Alpha-1 Antitrypsin Deficiency current marketed and Alpha-1 Antitrypsin Deficiency emerging therapies
Alpha-1 Antitrypsin Deficiency Market Dynamics: Alpha-1 Antitrypsin Deficiency market drivers and Alpha-1 Antitrypsin Deficiency market barriers
Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies
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, visit https://www.delveinsight.com/report-store/alpha-1-antitrypsin-deficiency-a1atd-market?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Key benefits of the Alpha-1 Antitrypsin Deficiency Market Report:
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).
The Alpha-1 Antitrypsin Deficiency market report provides insights into the current and emerging therapies.
The Alpha-1 Antitrypsin Deficiency market report provides a global historical and forecasted market covering drug outreach in 7MM.
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.

Contact Info:

Kritika Rehani, Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
www.delveinsight.com
+14699457679

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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