Alpha-1 Antitrypsin Deficiency Market is Anticipated to Expand Rapidly by 2034 Owing to the Emergence of Innovative Gene and RNA-based Therapies | DelveInsight

The market dynamics for Alpha-1 Antitrypsin Deficiency (AATD) are witnessing steady growth driven by the increasing prevalence of respiratory and liver diseases associated with AATD, rising awareness and diagnosis rates, and growing advancements in gene-editing and RNA-based therapies. Additionally, the launch of emerging therapies such as Fazirsiran (ARO-AAT/TAK-999), Inhaled Alpha 1-Antitrypsin (AAT), Alvelestat (MPH-966), WVE-006, KB408, and others will further fuel the market.
DelveInsight, a leading market research firm, announces the release of its latest report, "DelveInsight's Alpha-1 Antitrypsin Deficiency (AATD) Market Insights, Epidemiology, and Market Forecast 2034." This comprehensive report provides an in-depth understanding of Alpha-1 Antitrypsin Deficiency, including historical and forecasted epidemiology, market trends, and treatment scenarios across the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom, and Japan.

Key Takeaways from the Alpha-1 Antitrypsin Deficiency Market

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The market size for Alpha-1 Antitrypsin Deficiency in the leading markets is expected to grow significantly by 2034.

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The United States accounted for the highest Alpha-1 Antitrypsin Deficiency treatment market size in 7MM in 2024, in comparison to the other major markets, i.e., EU4 countries, the United Kingdom, and Japan.

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Based on DelveInsight's assessment in 2024, the 7MM had approximately 225,950 prevalent cases of AATD, which are expected to rise during the forecast period (2025-2034).

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According to DelveInsight's analysis, the number of prevalent cases of AATD in the US was estimated to be nearly 134,340 in 2024.

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Data suggests that the majority of AATD patients remain undiagnosed, with nearly 90% of cases being missed, while only 5-10% of patients receive a confirmed diagnosis.

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In 2024, Japan accounted for approximately 2,950 cases of AATD, with prevalence significantly lower compared to the US and Europe.

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Leading Alpha-1 Antitrypsin Deficiency companies, such as Kamada Pharmaceuticals, Arrowhead Pharmaceuticals and Takeda, Mereo BioPharma/AstraZeneca, Sanofi/Inhibrx Biosciences, Wave Life Sciences, Beam Therapeutics, Grifols, Krystal Biotech, BioMarin, AlveoGene, Korro Bio, Spexis/Santhera Pharmaceutical, and others, are developing new Alpha-1 Antitrypsin Deficiency treatment drugs that can be available in the Alpha-1 Antitrypsin Deficiency market in the coming years.

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The promising Alpha-1 Antitrypsin Deficiency therapies in clinical trials include Fazirsiran (ARO-AAT/TAK-999), Inhaled Alpha 1-Antitrypsin (AAT), Alvelestat (MPH-966), INBRX-101/SAR447537, WVE-006, KB408, BEAM-302, Alpha-1 AT 15%, and others.

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Key Factors Driving the Alpha-1 Antitrypsin Deficiency Market

Rising Awareness and Diagnosis Rates:

Increasing awareness among healthcare professionals and the growing use of genetic and biomarker-based diagnostic testing are improving the diagnosis of AATD. Enhanced screening for patients with COPD, emphysema, and liver disorders is expected to contribute to market growth.

Growing Advancements in Gene and RNA-based Therapies:

Emerging therapies targeting the root cause of AATD through RNA interference, RNA editing, gene correction, and inhaled protein replacement approaches are transforming the treatment landscape. Therapies such as Fazirsiran, WVE-006, and BEAM-302 are attracting significant market attention due to their potential disease-modifying and curative capabilities.

Increasing Demand for Improved and Convenient Treatment Approaches:

Current augmentation therapies require frequent intravenous administration and are associated with high treatment burden. New inhaled, oral, subcutaneous, and self-administered therapies are expected to improve patient compliance, convenience, and quality of life.

Alpha-1 Antitrypsin Deficiency Competitive Landscape

Several Alpha-1 Antitrypsin Deficiency drugs in development include Fazirsiran (ARO-AAT/TAK-999) (Arrowhead Pharmaceuticals and Takeda), Inhaled Alpha 1-Antitrypsin (AAT) (Kamada Pharmaceuticals), Alvelestat (MPH-966) (Mereo BioPharma/AstraZeneca), INBRX-101/SAR447537 (Sanofi/Inhibrx Biosciences), WVE-006 (Wave Life Sciences), KB408 (Krystal Biotech), and BEAM-302 (Beam Therapeutics).

These candidates target diverse mechanisms such as reduction of mutant Z-AAT protein production, immunomodulation through inhaled AAT replacement, neutrophil elastase inhibition, RNA editing, and gene correction technologies, aiming to provide long-term disease modification with improved efficacy and convenience compared to current augmentation therapies.

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Recent Developments in the Alpha-1 Antitrypsin Deficiency Market

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In March 2025, the FDA approved an update to GLASSIA's US Prescribing Information to include 1-, 4-, and 5-gram single-dose vials, available from July 2025, aiming to simplify weight-based dosing and improve convenience for patients.

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In November 2024, Korro Bio announced submission to the Bellberry HREC for a Phase I/II clinical study of KRRO-110 for AATD.

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In October 2024, Wave Life Sciences highlighted proof-of-mechanism data for WVE-006 for the treatment of AATD during Annual Research Day 2024.

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In September 2024, Krystal Biotech amended the protocol for its KB408 Phase I SERPENTINE-1 study to include mandatory bronchoscopies for measuring alpha-1 antitrypsin expression.

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In July 2024, Beam Therapeutics entered into a settlement agreement related to BEAM-302 development and commercialization activities.

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According to Takeda's FY2024 presentation released in May 2025, the company anticipates regulatory submissions for Fazirsiran in AATD-related liver disease between 2027 and 2029.

What is Alpha-1 Antitrypsin Deficiency?

Alpha-1 Antitrypsin Deficiency (AATD) is a rare inherited genetic disorder caused by mutations in the SERPINA1 gene, leading to low levels or dysfunctional alpha-1 antitrypsin protein in the blood. The condition primarily affects the lungs and liver, resulting in diseases such as emphysema, chronic obstructive pulmonary disease (COPD), cirrhosis, and liver failure. AATD is often underdiagnosed because its symptoms resemble those of asthma, COPD, and other liver disorders. Early diagnosis and appropriate management are essential to improve patient outcomes and quality of life.

Alpha-1 Antitrypsin Deficiency Epidemiology Segmentation

The Alpha-1 Antitrypsin Deficiency epidemiology section provides insights into the historical and current Alpha-1 Antitrypsin Deficiency patient pool and forecasted trends for the leading markets. The Alpha-1 Antitrypsin Deficiency market report proffers epidemiological analysis for the study period 2020-2034 in the leading markets, segmented into:

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Total Prevalent Cases of Alpha-1 Antitrypsin Deficiency

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Total Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency

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Genotype-specific Cases of Alpha-1 Antitrypsin Deficiency

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Comorbidity-associated Cases of Alpha-1 Antitrypsin Deficiency

Scope of the Alpha-1 Antitrypsin Deficiency Market Report

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

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Alpha-1 Antitrypsin Deficiency Market Dynamics: Key Market Forecast Assumptions of Emerging Alpha-1 Antitrypsin Deficiency Drugs and Market Outlook

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Key Companies: Kamada Pharmaceuticals, Arrowhead Pharmaceuticals and Takeda, Mereo BioPharma/AstraZeneca, Sanofi/Inhibrx Biosciences, Wave Life Sciences, Beam Therapeutics, Grifols, Krystal Biotech, BioMarin, AlveoGene, Korro Bio, Spexis/Santhera Pharmaceutical, and others

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Key Therapies: Fazirsiran (ARO-AAT/TAK-999), Inhaled Alpha 1-Antitrypsin (AAT), Alvelestat (MPH-966), INBRX-101/SAR447537, WVE-006, KB408, BEAM-302, Alpha-1 AT 15%, PROLASTIN-C LIQUID/LYNSPAD, ZEMAIRA/RESPREEZA, GLASSIA, ARALAST NP, and others

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Competitive Intelligence Analysis: SWOT analysis and Market entry strategies

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

To know more about Alpha-1 Antitrypsin Deficiency companies working in the treatment market, visit @ Alpha-1 Antitrypsin Deficiency Clinical Trials and Therapeutic Assessment [https://www.delveinsight.com/sample-request/alpha-1-antitrypsin-deficiency-a1atd-market?utm_source=abnewswire&utm_medium=pressrelease&utm_campaign=kspr]

Table of Contents

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Alpha-1 Antitrypsin Deficiency Market Report Introduction

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Executive Summary for Alpha-1 Antitrypsin Deficiency

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SWOT Analysis of Alpha-1 Antitrypsin Deficiency

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

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Alpha-1 Antitrypsin Deficiency Market Overview at a Glance

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Alpha-1 Antitrypsin Deficiency Background and Overview

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Alpha-1 Antitrypsin Deficiency Epidemiology and Patient Population

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Country-Specific Patient Population of Alpha-1 Antitrypsin Deficiency

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Alpha-1 Antitrypsin Deficiency Current Treatment and Medical Practices

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Alpha-1 Antitrypsin Deficiency Unmet Needs

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Alpha-1 Antitrypsin Deficiency Emerging Therapies

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Alpha-1 Antitrypsin Deficiency Market Outlook

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

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Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement of Therapies

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Alpha-1 Antitrypsin Deficiency Market Drivers

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Alpha-1 Antitrypsin Deficiency Market Barriers

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Alpha-1 Antitrypsin Deficiency Appendix

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Alpha-1 Antitrypsin Deficiency Report Methodology

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DelveInsight Capabilities

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Disclaimer

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About DelveInsight

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. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve.

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