PUBLISHER: DelveInsight | PRODUCT CODE: 2082945
PUBLISHER: DelveInsight | PRODUCT CODE: 2082945
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) 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.
Comprehensive unmet needs insights in Alpha-1 Antitrypsin Deficiency (AATD) and their strategic implications are provided in the full report.
Key Findings from Alpha-1 Antitrypsin Deficiency (AATD) Epidemiological Analysis and Forecast
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.
Alpha-1 Antitrypsin Deficiency (AATD) Drug Updates
Drug Class Insights
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.
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.
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.
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.
Market Insights