알파-1 안티트립신 결피증 : 시장 인사이트, 역학 및 시장 예측(2036년)

Alpha-1 Antitrypsin Deficiency (AATD) Insights and Trends

• According to DelveInsight's analysis, the AATD market size was found to be ~USD 1,000 million in the leading markets (the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan) in 2025.
• AATD is a hereditary disorder characterised by low levels of a protein called Alpha-1 Antitrypsin (A1AT), which is found in the blood. This deficiency may predispose an individual to several illnesses and most commonly manifests as Chronic Obstructive Pulmonary Disease (COPD) (including bronchiectasis) and liver disease (especially cirrhosis and hepatoma), or more rarely, as a skin condition called panniculitis.
• AATD is a genetic disorder that manifests as lung and/or liver disease. Because symptoms of AATD overlap with those of common pulmonary and hepatic conditions, AATD is often misdiagnosed, which has resulted in substantial underdiagnosis of AATD worldwide.
• Secondary studies estimate that the prevalence of AATD in the United States and Europe ranges from 1 in 2,500 to 1 in 5,000 for the PI*ZZ genotype in the general population, while highlighting that only a small proportion of affected individuals have been formally diagnosed.
• AATD with the MZ genotype, present in approximately 3.5% of the global population and affecting over 35 million individuals, is frequently underdiagnosed, with its symptoms often overlooked or misinterpreted.
• Approximately 15% of individuals with Alpha-1 develop cirrhosis, and overall, about 1 in 10 develop liver disease due to AATD.
• The current treatment landscape lacks curative therapies. Augmentation therapy, also called replacement therapy, is approved for treating alpha-1-related lung disease. Currently, augmentation therapy with periodic IV infusion of pooled human serum AAT is used in individuals who have established emphysema. Currently, four augmentation therapy products are available in the US for AATD: PROLASTIN-C, ARALAST, ZEMAIRA, and GLASSIA, all alpha1-proteinase inhibitors.
• In selected European countries, augmentation therapies for Alpha-1 Antitrypsin Deficiency include alpha1-proteinase inhibitors such as RESPREEZA (marketed as ZEMAIRA in the US), PROLASTIN, PROLASTINA, PROLASPLAN, PLITALFA, and ALFALASTIN. In Japan, PROLASTIN-C is marketed under the brand name LYNSPAD.
• Bronchodilators, corticosteroids, and oxygen therapy are also recommended, as they halt respiratory symptoms. Further, lung transplantation may be an appropriate option for individuals with end-stage lung disease, and liver transplantation is the definitive treatment for severe AATD-associated liver disease to restore AAT levels in the body.
• There are no licensed pharmacological treatments available for patients with AATD-associated liver disease. Fazirsiran appears as a potentially transformative approach targeting the disease at its source by reducing abnormal Z-AAT protein production in the liver, potentially offering a transformative alternative to liver transplants, currently the only effective option.

DelveInsight's 'Alpha-1 Antitrypsin Deficiency (AATD) - Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of AATD, historical and forecasted epidemiology, as well as the AATD market trends in the United States, EU4 (Germany, Spain, Italy, and France), the United Kingdom, and Japan.

The Alpha-1 Antitrypsin Deficiency (AATD) market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates AATD patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in AATD and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.

Key Factors Driving the Alpha-1 Antitrypsin Deficiency (AATD) Market

Rising Alpha-1 Antitrypsin Deficiency (AATD) Prevalence

The prevalence of AATD is gradually increasing, acting as a key driver of market growth. In the United States, there were approximately ~11,500 diagnosed prevalent cases in 2025, with numbers expected to rise further by 2036. This increase is largely attributable to enhanced screening initiatives, greater clinical awareness of AATD among healthcare providers, and advancements in genetic and molecular diagnostic techniques, rather than a true rise in disease incidence.

Rising Opportunities in Targeted Therapies in Alpha-1 Antitrypsin Deficiency (AATD)

Rising opportunities in targeted therapies for AATD are driven by increasing focus on disease-modifying approaches beyond conventional augmentation therapy. Advances in gene therapy, RNA-based therapeutics, and inhaled recombinant AAT are expanding the treatment pipeline. Improved genetic screening and early diagnosis are also increasing the identified patient pool eligible for targeted interventions. Additionally, strong unmet needs in lung and liver disease progression control are attracting significant R&D and strategic investments from biotech and pharmaceutical companies.

Emerging Alpha-1 Antitrypsin Deficiency (AATD) Competitive Landscape

Emerging AATD therapies such as Fazirsiran, Alvelestat, Efdoralprin alfa, WVE-006, BEAM-302, and others are shifting the market from conventional augmentation therapy toward targeted and potentially curative approaches. RNA-based and gene-editing therapies like Fazirsiran and BEAM-302 could significantly reduce long-term disease burden by addressing the underlying genetic defect. Oral and recombinant options are also improving convenience and adherence, increasing overall treatment uptake. Collectively, these innovations are expected to reshape the AATD market with strong growth in disease-modifying and precision medicine segments.

Alpha-1 Antitrypsin Deficiency (AATD) Understanding and Treatment Algorithm

Alpha-1 Antitrypsin Deficiency (AATD) Overview and Diagnosis

AATD is an inherited disorder marked by low levels of alpha-1 antitrypsin, a protein that protects tissues from enzyme damage. The condition most commonly leads to lung diseases such as COPD, including emphysema and bronchiectasis, and liver diseases such as cirrhosis and hepatocellular carcinoma. In rare cases, it may also present as a skin condition called panniculitis. The deficiency allows proteolytic enzymes to damage tissues, particularly the lungs, resulting in progressive destruction of alveoli. It can also cause liver damage due to the accumulation of abnormal protein in liver cells. Disease progression is often accelerated by smoking and environmental or occupational exposures.

Alpha-1 Antitrypsin Deficiency (AATD) Diagnosis

Diagnosis is typically made using a combination of blood tests and genetic testing. The first step involves measuring serum alpha-1 antitrypsin levels, where low levels indicate possible deficiency. This is confirmed through genotyping or phenotyping to identify variants such as PIZZ or PIMZ. Additional assessments, like liver function tests and pulmonary function tests, help evaluate disease impact. Early diagnosis is especially important in patients with unexplained COPD, liver disease, or a family history of AATD.

Alpha-1 Antitrypsin Deficiency (AATD) Treatment

AATD treatment primarily aims to manage and slow the progression of lung and liver disease associated with the condition. Management includes behavioural and lifestyle modifications, especially smoking cessation, along with medical and supportive therapies. Standard COPD-like treatments such as long-acting bronchodilators, inhaled corticosteroids, long-acting beta-agonists, and antibiotics are commonly used for symptom control, although they do not restore serum AAT levels.

A disease-specific option is intravenous augmentation (replacement) therapy with alpha1-proteinase inhibitor, which helps increase circulating AAT levels and is approved for AATD-related lung disease. In advanced cases, lung transplantation may be considered for end-stage respiratory failure, while liver transplantation remains the only definitive treatment for severe AATD-associated liver disease and also restores normal AAT production.

Currently, there is no curative therapy for AATD-related liver disease, although experimental approaches (e.g., rapamycin, carbamazepine) have shown potential in enhancing degradation of misfolded A1AT in preclinical studies and are under investigation.

Alpha-1 Antitrypsin Deficiency (AATD) Unmet Needs

The section "unmet needs of Alpha-1 Antitrypsin Deficiency (AATD)" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.

1. Delayed and underdiagnosis due to low awareness and non-specific symptoms

2. Lack of curative therapy, with current treatments being mainly supportive

3. Limited effectiveness of augmentation therapy in severe or advanced disease

4. No approved targeted therapy for AATD-related liver disease

5. High treatment burden, including lifelong infusions and monitoring

6. Restricted access and cost challenges for augmentation and transplant options, and others.....

Comprehensive unmet needs insights in Alpha-1 Antitrypsin Deficiency (AATD) and their strategic implications are provided in the full report.

Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology

Key Findings from Alpha-1 Antitrypsin Deficiency (AATD) Epidemiological Analysis and Forecast

• Based on DelveInsight's assessment in 2025, the 7MM had ~227,000 prevalent cases of AATD. These are expected to rise due to the rising prevalence of respiratory illnesses, particularly COPD and liver diseases associated with AATD.
• Among the 7MM, the US accounted for the highest number (~60%) of diagnosed prevalent AATD cases in 2025.
• In 2025, among the EU4 and the UK, the UK had the highest diagnosed prevalent cases of AATD, which accounted for around 10% of the total AATD cases in the 7MM, followed by Germany and others.
• In the US, with approximately 10,200 cases, Pi*ZZ was the most common genotype, followed by Pi*SZ, with approximately, and other (PiMZ, SS, etc.) genotypes in 2025. These cases are expected to increase during the study period.
• The prevalence of AATD in Japan is significantly lower than in Europe and the United States.
• Since AATD's symptoms might be mistaken for those of other illnesses, including asthma and COPD, it is frequently misdiagnosed and unnoticed, particularly in the early stages of the disease. Data suggests that the majority of AATD patients go undiagnosed (upto 90%); about 5-10% of AATD patients receive a diagnosis.
• In the US, among AATD-associated comorbid cases, lung disease accounts for the majority (~75%), followed by other diseases, while liver disease represents the smallest proportion (~8%).

Alpha-1 Antitrypsin Deficiency (AATD) Drug Chapters & Competitive Analysis

The AATD drug chapter provides a detailed, market-focused review of approved therapies and the emerging pipeline across Phase I-III clinical trials. It covers the mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, and strategic partnerships for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the AATD treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the AATD therapeutics market.

Approved Therapies for Alpha-1 Antitrypsin Deficiency (AATD)

Alpha-1 Proteinase Inhibitor (PROLASTIN-C LIQUID/LYNSPAD): Grifols

PROLASTIN-C LIQUID/LYNSPAD, developed by Grifols, is a biologic augmentation therapy used for the treatment of AATD-related emphysema. It works by replacing the deficient A1PI in patients, thereby helping to protect lung tissue from further enzymatic damage. The therapy is administered IV and is specifically indicated for patients with AATD-associated emphysema.

Regulatory approvals for this product vary across regions, with PROLASTIN-C liquid approved in the US (2009, 2017 updates), LYNSPAD approved in Japan (2021), and European approval granted in 2024 (Germany, Denmark). It is a biologic therapy and represents a key disease-modifying option for lung manifestations of AATD.

Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Analysis

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

Fazirsiran is being developed to treat the liver disease associated with AATD, a rare genetic disorder that severely damages the liver and lungs of affected individuals. Fazirsiran is designed to knock down the hepatic production of the mutant alpha-1 antitrypsin (Z-AAT) protein, the cause of progressive liver disease in AATD patients. Reducing production of the inflammatory Z-AAT protein is expected to halt the progression of liver disease and potentially allow it to regenerate and repair. Fazirsiran is currently in Phase III of clinical trials. Arrowhead Pharmaceuticals received a Breakthrough Designation (BTD) from the US FDA for ARO-AAT to treat AAT-LD. Takeda is conducting multiple Phase III studies for the treatment of AATD liver disease, including the REDWOOD study.

• Grifols
• CSL Behring
• Kamada Pharmaceuticals
• Takeda Pharmaceuticals
• Arrowhead Pharmaceuticals
• Mereo BioPharma
• Sanofi
• Wave Life Sciences
• Beam Therapeutics, and others

Alpha-1 Antitrypsin Deficiency (AATD) Drug Updates

• According to Takeda's FY2025 presentation released in January 2026, the company anticipates submitting regulatory filings for fazirsiran in AATD-related liver disease between 2027 and 2029.
• In March 2026, Beam Therapeutics announced updated safety and efficacy data from the ongoing Phase I/II trial of BEAM-302 and the selection of 60 mg as the optimal biological dose to advance into pivotal development to support potential accelerated approval.
• As per the March 2026 presentation by Mereo BioPharma, Alvelestat (MPH-966) is a Phase II anticipated with Phase III initiation planned and potential partnering opportunities under evaluation in 2026.
• As per the March 2026 presentation, WVE-006 (SERPINA1) for AATD is expected to deliver data from the 400 mg multidose cohort and 600 mg single-dose cohort of the RESTORAAtion-2 study at the ATS conference in May 2026, followed by anticipated regulatory feedback in mid-2026 regarding a potential accelerated approval pathway.
• In December 2025, Kamada announced that the independent Data and Safety Monitoring Board (DSMB) advised the Company that, based on a prespecified interim futility analysis, the Phase III InnovAATe trial of Inhaled AAT for the treatment of AATD is unlikely to demonstrate a statistically significant benefit in its primary endpoint - lung function measured by FEV1. Based on the futility analysis outcome, the Company will discontinue the trial.
• In December 2025, the European Medicines Agency (EMA) granted orphan designation to efdoralprin alfa for the potential treatment of AATD-related emphysema, a rare respiratory condition with great unmet medical need.
• In October 2025, Positive results from the global ElevAATe Phase II study showed that efdoralprin alfa (SAR447537, formerly known as INBRX-101) met all primary and key secondary endpoints when dosed every three weeks (Q3W) or four weeks (Q4W) in adults with AATD emphysema, a rare disease.

Drug Class Insights

Alpha-1 Antitrypsin Deficiency (AATD) Market Outlook

The market outlook for AATD is progressively expanding, driven by high unmet medical needs and continuous therapeutic advancements.

AATD is a genetic disorder that can lead to progressive lung and liver disease. Early diagnosis and appropriate clinical management are essential for improving patient outcomes and quality of life. Despite advancements in supportive care, the current treatment landscape remains largely non-curative and limited in disease-modifying options. At present, augmentation therapy (AAT replacement therapy) is the only approved disease-specific treatment for AATD-related lung disease. These therapies involve intravenous administration of purified AAT protein derived from healthy human plasma, designed to restore circulating and pulmonary AAT levels and slow emphysema progression, although definitive disease-modifying benefits remain limited in real-world evidence.

In addition to augmentation therapy, several off-label treatments are widely used for symptomatic management and control of COPD-related complications. These include bronchodilators, inhaled corticosteroids, and antibiotics for infection control. In advanced disease stages, surgical interventions such as lung volume reduction surgery and bullectomy may be considered, while patients with end-stage emphysema may require lung transplantation, which carries significant risks including infection, rejection, and long-term immunosuppression.

AATD-associated liver disease remains a major unmet need, as no approved disease-specific therapies currently exist. Management is primarily supportive, focusing on maintaining adequate nutrition and addressing complications to preserve liver function. The global augmentation therapy landscape is heterogeneous across regions, with significant variation in availability and reimbursement. In the United States, four FDA-approved intravenous augmentation therapies are available. In Europe, augmentation therapy availability varies by country and regulatory pathway, with products such as RESPREEZA (CSL Behring), PROLASTIN variants (including PROLASTINA/PLITALFA/ALFALASTIN in select markets) approved in several national jurisdictions. In Japan, due to the relatively low diagnosed prevalence of AATD, LYNSPAD (marketed as PROLASTIN-C in Japan) is currently the only approved augmentation therapy.

However, recent clinical setbacks are also shaping the near-term outlook. In December 2025, Kamada announced that the independent DSMB recommended discontinuation of its Phase III InnovAATe trial of inhaled AAT therapy, following a prespecified interim futility analysis indicating that the study was unlikely to meet its primary endpoint of lung function improvement (FEV1). This outcome represents a key setback for inhaled augmentation strategies, reinforcing the challenges of demonstrating meaningful clinical benefit beyond established IV plasma-derived therapies.

Despite such setbacks, the long-term outlook remains positive, with RNAi therapies (e.g., Fazirsiran), small molecule agents (e.g., Alvelestat), engineered biologics (e.g., INBRX-101/SAR447537), and RNA editing therapies (e.g., WVE-006) expected to drive the next wave of innovation. These modalities are increasingly focused on disease modification rather than symptomatic lung support, potentially reshaping treatment paradigms over the forecast period (2022-2036).

Overall, the launch of first-in-class therapies, improved diagnostic approaches, and increasing disease awareness are expected to drive steady growth in the 7MM AATD market from 2022 to 2036, with strong commercial implications for both marketed products and emerging pipelines.

• According to the estimates, the largest market size of AATD was captured by the United States, i.e., ~USD 880 million in 2025.
• Regional market dynamics for AATD show the United States leading in revenue, while Japan and Europe demonstrate steady growth, driven by rising prevalence, improved diagnostics, and increasing adoption of advanced therapies.
• The entry of mid- to late-stage candidates such as Fazirsiran (ARO-AAT/TAK-999), Alvelestat (MPH-966), and others is expected to intensify competition in the AATD treatment landscape during the forecast period.

Drug Class/Insights into Leading Emerging and Marketed Therapies in Alpha-1 Antitrypsin Deficiency (AATD) (2022-2036 Forecast)

The AATD treatment landscape is steadily evolving from lifelong plasma-derived augmentation toward disease-modifying, RNA-based, and gene-editing approaches aimed at addressing both lung and liver pathology. While current management remains centred on intravenous A1-PI replacement, the pipeline increasingly focuses on reducing mutant protein accumulation, correcting genetic defects, and enabling durable clinical benefit.

• Plasma-derived augmentation therapies (disease-replacement biologics): Agents such as PROLASTIN-C LIQUID/LYNSPAD (Grifols), ZEMAIRA/RESPREEZA (CSL Behring), GLASSIA (Kamada/Takeda), and ARALAST NP (Takeda) act via alpha-1 proteinase inhibitor (A1-PI) replacement, restoring antiprotease activity in the lungs. These remain the standard of care for emphysema in AATD, providing symptomatic benefit and slowing disease progression. However, they are lifelong IV therapies and do not modify underlying genetic or liver disease pathology.
• RNA interference (RNAi) therapies (disease-modifying liver-directed agents): Agents such as Fazirsiran (ARO-AAT/TAK-999) act by silencing mutant Z-AAT protein production in hepatocytes, reducing toxic protein accumulation. This represents a next-generation disease-modifying approach targeting liver disease progression in PiZZ AATD and is currently in late-stage (Phase III) development.
• Small molecule therapies (anti-inflammatory / lung protection): Agents such as Alvelestat (MPH-966) act via neutrophil elastase inhibition, reducing protease-mediated lung tissue destruction. These oral therapies offer a convenient alternative or adjunct to IV augmentation, targeting downstream inflammatory pathways rather than replacing deficient protein.

Alpha-1 Antitrypsin Deficiency (AATD) Drug Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the AATD market's uptake by drugs, patient uptake by therapy, and sales of each drug.

Marketed plasma-derived A1-PI augmentation therapies such as PROLASTIN-C LIQUID/LYNSPAD (Grifols), ZEMAIRA/RESPREEZA (CSL Behring), GLASSIA (Kamada/Takeda), and ARALAST NP (Takeda) are expected to maintain steady uptake, supported by their established role as the standard of care for emphysema in AATD. However, their uptake growth is expected to be limited by the need for lifelong IV administration and lack of disease-modifying effect.

In the emerging segment, RNA interference therapy such as Fazirsiran (ARO-AAT/TAK-999) is expected to witness fast uptake, driven by its strong disease-modifying potential and ability to directly reduce mutant Z-AAT protein production, addressing a key unmet need in AATD-related liver disease. Small molecule therapy such as Alvelestat (MPH-966) is projected to show medium uptake, supported by its oral route of administration and role in reducing neutrophil elastase-mediated lung damage, making it a convenient adjunct or alternative to augmentation therapy.

Engineered biologic therapy such as INBRX-101 / SAR447537 is expected to achieve moderate uptake, reflecting its potential as a next-generation A1-PI replacement with improved functional properties, although IV administration may moderate rapid adoption.

Overall, the market is expected to progressively shift from supportive protein replacement therapies toward RNA-based, small-molecule, and gene-editing approaches, with uptake increasingly driven by disease modification potential, route of administration convenience, and long-term clinical benefit.

Alpha-1 Antitrypsin Deficiency (AATD) Therapies Price Scenario & Trends

Pricing and analogue assessment of AATD therapies highlights evolving price dynamics structures. This section summarises the cost of approved treatments, the closest and most appropriate analogue selection for emerging therapies, and the understanding of how pricing influences market access, adherence, and long-term uptake.

• Pricing of Alpha-1 Antitrypsin Deficiency (AATD) Approved Drugs

The average sales price of GLASSIA is USD 5.099 per 10 mg. Annual treatment costs for AATD patients thus range from USD 80,000 to USD 120,000, varying by dosage and administration schedule.

Further details are provided in the final report....

Industry Experts and Physician Views for Alpha-1 Antitrypsin Deficiency (AATD)

To keep up with AATD market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on the AATD emerging therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in AATD, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.

DelveInsight's analysts connected with 10+ KOLs to gather insights; however, interviews were conducted with 6+ KOLs in the 7MM. Centres such as the University of North Carolina at Chapel Hill, the University of Tokyo Hospital, and King's College London, etc. were contacted. Their opinion helps understand and validate current and emerging AATD therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritisation in AATD.

Qualitative Analysis: SWOT and Conjoint Analysis

We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and conjoint analysis.

In the SWOT analysis of AATD, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.

Conjoint analysis analyses emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyse the effectiveness of therapy.

The team of analysts analyses promising emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. In efficacy, the trial's primary and secondary outcome measures are evaluated, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are mainly observed. In addition, the scoring is also based on the route of administration, order of entry, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Scope of the Report:

• The report covers a segment of key events, an executive summary, a descriptive overview of Alpha-1 Antitrypsin Deficiency (AATD), explaining its causes, signs and symptoms, pathogenesis, and currently available treatments.
• Comprehensive insight has been provided into the epidemiology segments and forecasts, the future growth potential of the diagnosis rate, and disease progression along treatment guidelines.
• Additionally, an all-inclusive account of both the current and emerging treatments, along with the elaborate profiles of late-stage and prominent therapies, will have an impact on the current treatment landscape.
• A detailed review of the Alpha-1 Antitrypsin Deficiency (AATD) market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis and expert insights/KOL views, patient journey, and treatment preferences that help in shaping and driving the 7MM Alpha-1 Antitrypsin Deficiency (AATD) market.

Report Insights

• Alpha-1 Antitrypsin Deficiency (AATD) Patient Population Forecast
• Alpha-1 Antitrypsin Deficiency (AATD) Therapeutics Market Size
• Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Analysis
• Alpha-1 Antitrypsin Deficiency (AATD) Market Size and Trends
• Alpha-1 Antitrypsin Deficiency (AATD) Market Opportunity (Current and forecasted)

Report Key Strengths

• Epidemiology-based (Epi-based) Bottom-up Forecasting
• Artificial Intelligence (AI)-enabled Market Research Report
• 11-year forecast
• Alpha-1 Antitrypsin Deficiency (AATD) Market Outlook (North America, Europe, Asia-Pacific)
• Patient Burden Trends (by geography)
• Alpha-1 Antitrypsin Deficiency (AATD) Treatment Addressable Market (TAM)
• Alpha-1 Antitrypsin Deficiency (AATD) Competitive Landscape
• Alpha-1 Antitrypsin Deficiency (AATD) Major Companies Insights
• Alpha-1 Antitrypsin Deficiency (AATD) Price Trends and Analogue Assessment
• Alpha-1 Antitrypsin Deficiency (AATD) Therapies Drug Adoption/Uptake
• Alpha-1 Antitrypsin Deficiency (AATD) Therapies Peak Patient Share Analysis

Report Assessment

• Alpha-1 Antitrypsin Deficiency (AATD) Current Treatment Practices
• Alpha-1 Antitrypsin Deficiency (AATD) Unmet Needs
• Alpha-1 Antitrypsin Deficiency (AATD) Clinical Development Analysis
• Alpha-1 Antitrypsin Deficiency (AATD) Emerging Drugs Product Profiles
• Alpha-1 Antitrypsin Deficiency (AATD) Market Attractiveness
• Alpha-1 Antitrypsin Deficiency (AATD) Qualitative Analysis (SWOT and Conjoint Analysis)

FAQs:

Market Insights

• What was the Alpha-1 Antitrypsin Deficiency (AATD) market size, the market size by therapies, market share (%), distribution in 2025, and what would it look like by 2036? What are the contributing factors for this growth?
• What are the anticipated pricing variations among different geographies for the emerging therapies in the future?
• What can be the future treatment paradigm of Alpha-1 Antitrypsin Deficiency (AATD)?
• What are the disease risks, burdens, and unmet needs of Alpha-1 Antitrypsin Deficiency (AATD)? What will be the growth opportunities across the 7MM concerning the patient population with Alpha-1 Antitrypsin Deficiency (AATD)?
• Who is the major future competitor in the market, and how will the competitors affect their market share?
• What are the current options for the treatment of Alpha-1 Antitrypsin Deficiency (AATD)? What are the current guidelines for treating Alpha-1 Antitrypsin Deficiency (AATD) in the US, Europe, and Japan?

Reasons to Buy:

• The report will help in developing business strategies by understanding the latest trends and changing treatment dynamics driving the Alpha-1 Antitrypsin Deficiency (AATD) market.
• Bottom up forecasting builds from the affected population to product forecasts, delivering a robust, data driven approach ideal for new therapies and novel classes.
• Insights on patient burden/disease incidence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• Identifying strong upcoming players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• To understand KOLs' perspectives on the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights into the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.
• This Artificial Intelligence (AI) enabled report summarises and simplifies complex datasets within the report into clear, actionable insights for stakeholders, investors, and healthcare providers, enabling faster, data driven decisions.

Table of Contents

1. Key Insights

2. Report Introduction

3. Executive Summary

4. Key Events

• 4.1. Upcoming Key Catalysts
• 4.2. Key Transactions And Collaborations
• 4.3. News Flow
• 4.4. Key Conference Highlights

5. Epidemiology and Market Forecast Methodology

6. Alpha-1 Antitrypsin Deficiency (AATD) Market Overview at a Glance

• 6.1. Clinical Landscape Analysis (By Phase, Molecule Type, and RoA)
• 6.2. Market Share (%) Distribution of Alpha-1 Antitrypsin Deficiency (AATD) by Therapies in the 7MM, in 2025
• 6.3. Market Share (%) Distribution of Alpha-1 Antitrypsin Deficiency (AATD) by Therapies in the 7MM, in 2036

7. Disease Background and Overview of Alpha-1 Antitrypsin Deficiency (AATD)

• 7.1. Introduction
• 7.2. Types
• 7.3. Symptoms
• 7.4. Causes
• 7.5. Pathophysiology
• 7.6. Diagnosis
• 7.7. Treatment

8. Treatment Guidelines for Alpha-1 Antitrypsin Deficiency (AATD)

• 8.8.1. European Respiratory Society Statement on Diagnosis, Clinical Management, and Treatment of Pulmonary Disease in AATD
• 8.8.2. French Clinical Practice Guidelines for the Diagnosis and Management of Lung Disease With AATD
• 8.8.3. COPD Foundation: Clinical Practice Guidelines for the Diagnosis and Management of AATD
• 8.8.1. European Respiratory Society Statement on Diagnosis, Clinical Management, and Treatment of Pulmonary Disease in AATD

9. Epidemiology and Patient Population of Alpha-1 Antitrypsin Deficiency (AATD)

• 9.1. Key Findings
• 9.2. Assumptions and Rationale
• 9.3. Total Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in the 7MM
• 9.4. The United States
  • 9.4.1. Total Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in the United States
  • 9.4.2. Total Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in the United States
  • 9.4.3. Genotype-specific Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in the United States
  • 9.4.4. Comorbidity-associated Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in the United States
  • 9.4.5. Total Treated Cases of Alpha-1 Antitrypsin Deficiency (AATD) in the United States
• 9.5. EU4 and the UK
  • 9.5.1. Total Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in EU4 and the UK
  • 9.5.2. Total Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in EU4 and the UK
  • 9.5.3. Genotype-specific Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in EU4 and the UK
  • 9.5.4. Comorbidity-associated Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in EU4 and the UK
  • 9.5.5. Total Treated Cases of Alpha-1 Antitrypsin Deficiency (AATD) in EU4 and the UK
• 9.6. Japan
  • 9.6.1. Total Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in Japan
  • 9.6.2. Total Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in Japan
  • 9.6.3. Genotype-specific Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in Japan
  • 9.6.4. Comorbidity-associated Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency (AATD) in Japan
  • 9.6.5. Total Treated Cases of Alpha-1 Antitrypsin Deficiency (AATD) in Japan

10. Patient Journey of Alpha-1 Antitrypsin Deficiency (AATD)

11. Marketed Therapies

• 11.1. Marketed Competitive Landscape of Alpha-1 Antitrypsin Deficiency (AATD)
• 11.2. Alpha-1 Proteinase Inhibitor (PROLASTIN-C LIQUID/LYNSPAD): Grifols
  • 11.2.1. Drug Description
  • 11.2.2. Regulatory Milestones
  • 11.2.3. Other Developmental Activities
  • 11.2.4. Summary of Pivotal Trials
  • 11.2.5. Clinical Development
    • 11.2.5.1. Clinical Trial Information
  • 11.2.6. Safety and Efficacy
  • 11.2.7. Analyst's Views

12. Emerging Therapies

• 12.1. Emerging Competitive Landscape of Alpha-1 Antitrypsin Deficiency (AATD)
• 12.2. Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals and Takeda Pharmaceuticals
  • 12.2.1. Drug Description
  • 12.2.2. Other Developmental Activities
  • 12.2.3. Clinical Development
    • 12.2.3.1. Clinical Trials Information
  • 12.2.4. Safety and Efficacy
  • 12.2.5. Analyst's Views
• 12.3. Alvelestat (MPH966): Mereo BioPharma
  • 12.3.1. Drug Description
  • 12.3.2. Other Developmental Activity
  • 12.3.3. Clinical Development
    • 12.3.3.1. Clinical Trials Information
  • 12.3.4. Safety and Efficacy
  • 12.3.5. Analyst's Views

13. Alpha-1 Antitrypsin Deficiency (AATD): 7MM Analysis

• 13.1. Key Findings
• 13.2. Market Outlook of Alpha-1 Antitrypsin Deficiency (AATD)
• 13.3. Key Market Forecast Assumptions
  • 13.3.1. Cost Assumptions
  • 13.3.2. Pricing Trends
  • 13.3.3. Analogue Assessment
  • 13.3.4. Launch Year and Therapy Uptakes
• 13.4. Conjoint Analysis of Alpha-1 Antitrypsin Deficiency (AATD)
• 13.5. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) in the 7MM
• 13.6. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) by Therapies in the 7MM
• 13.7. The United States
  • 13.7.1. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) in the United States
  • 13.7.2. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) by Therapies in the United States
• 13.8. EU4 and the UK
  • 13.8.1. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) in EU4 and the UK
  • 13.8.2. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) by Therapies in EU4 and the UK
• 13.9. Japan
  • 13.9.1. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) in Japan
  • 13.9.2. Total Market Size of Alpha-1 Antitrypsin Deficiency (AATD) by Therapies in Japan

14. Unmet Needs of Alpha-1 Antitrypsin Deficiency (AATD)

15. SWOT Analysis of Alpha-1 Antitrypsin Deficiency (AATD)

16. KOL Views of Alpha-1 Antitrypsin Deficiency (AATD)

17. Market Access and Reimbursement of Alpha-1 Antitrypsin Deficiency (AATD)

• 17.1. The United States
• 17.2. EU4 and the UK
  • 17.2.1. Germany
  • 17.2.2. France
  • 17.2.3. Italy
  • 17.2.4. Spain
  • 17.2.5. United Kingdom
• 17.3. Japan
• 17.4. Summary and comparison of Market Access and Pricing Policy Developments in 2025
• 17.5. Market Access and Reimbursement of Alpha-1 Antitrypsin Deficiency (AATD) Therapies

18. Appendix

• 18.1. Bibliography
• 18.2. Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight