Alpha-1 Antitrypsin Deficiency (A1ATD) Market

DelveInsight’s ‘Alpha-1 Antitrypsin Deficiency (AATD) – Market Insights, Epidemiology, and Market Forecast – 2032’ report delivers an in-depth understanding of the historical and forecasted epidemiology as well as the market trends of alpha-1 antitrypsin deficiency (AATD) in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom), and Japan.

 

The alpha-1 antitrypsin deficiency (AATD) market report provides current treatment practices, emerging drugs, market share of the individual therapies, and current and forecasted alpha-1 antitrypsin deficiency (AATD) market size from 2019 to 2032 segmented by 7MM. The report also covers the current alpha-1 antitrypsin deficiency (AATD) treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geographies Covered

  • The United States
  • EU5 (Germany, France, Italy, Spain, and the United Kingdom)
  • Japan

Study Period: 2019–2032

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

Alpha-1 Antitrypsin Deficiency (AATD) Overview

According to National Organization for Rare Disorders (NORD), alpha-1 antitrypsin deficiency (AATD) is a rare hereditary (autosomal recessive) metabolic disorder characterized by low levels of a protein called alpha-1 antitrypsin (A1AT), found in the blood. This deficiency may predispose an individual to several illnesses and most commonly manifests as a 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 frequent among individuals with Wegener’s granulomatosis, now called polyangiitis with granulomatosis.

 

According to the Genetic and Rare Disease Information Centre (GARD), symptoms of AATD may include shortness of breath, excessive cough with phlegm/sputum production, wheezing, decrease in exercise capacity and a persistent low energy state or tiredness, chest pain that increases when breathing in, symptoms may be chronic or occur with acute respiratory tract infections, such as a cold or the flu. When the liver is affected by A1AT deficiency, symptoms may include tiredness, loss of appetite, weight loss, swelling of the feet or belly, yellowish discoloration of the skin (jaundice) or the white part of the eyes, vomiting of blood, or blood in stools.

 

The etiology and pathogenesis of AATD involve a complex interaction between genetic and environmental factors resulting in immune-mediated reactions affecting the lungs, liver, and skin. The disease is due to variants in SERPINA1 (14q32.13). Major gene sequencing efforts carried out in disease populations disclosed more than 100 rare SERPINA1 variants, which may cause the absence of circulating A1AT (null alleles), poor AAT secretion from hepatocytes (deficiency alleles), or even form a modified enzyme inhibitory activity (dysfunctional alleles).

 

There is currently no curative treatment. Treatment is similar to that used for COPD and emphysema and aims at reducing symptoms and slowing progression. Long-acting bronchodilators, antibiotics, corticosteroid inhalations, and long-acting beta-agonists are the mainstay of treatment. Another treatment option in severe cases involves augmentation therapy with the administration of purified human AAT to reach normal physiological levels.

 

Smoking and exposure to tobacco smoke should be avoided. Vaccines for common respiratory disorders may be beneficial. Lung transplantation may be required for end-stage lung disease. Likewise, liver transplantation can be considered for advanced liver disease. Panniculitis usually responds to treatment with intravenous augmentation of AAT. Several treatments for lung and liver disease are currently in clinical trials. Exercise programs (pulmonary rehabilitation) and good nutrition may help increase the overall quality of daily living. People with emphysema must avoid smoking, employment that exposes the patient to lung irritants, and the use of nonmedical aerosol sprays. Preventing infection as possible with yearly influenza and periodic pneumococcal vaccinations is also recommended

 

Alpha-1 Antitrypsin deficiency (AATD) Diagnosis and Treatment

It covers the details of conventional and current medical therapies and diagnoses available in the alpha-1 antitrypsin deficiency (AATD) market to treat the condition. It also provides country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.

Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology

The alpha-1 antitrypsin deficiency (AATD) epidemiology division provides insights into the historical and current patient pool and the forecasted trend for every 7MM. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of KOL. The report also provides the prevalent patient pool, trends, and assumptions.

 

Key Findings

The disease epidemiology covered in the report provides historical and forecasted alpha-1 antitrypsin deficiency (AATD) epidemiology segmented as the prevalent cases of Alpha-1 Antitrypsin Deficiency (AATD), diagnosed cases of Alpha-1 Antitrypsin Deficiency (AATD), the prevalence of Alpha-1 Antitrypsin Deficiency (AATD) based on symptoms, the genotype of Alpha-1 Antitrypsin Deficiency (AATD) diagnosed patients and comorbidity associated with Alpha-1 Antitrypsin Deficiency (AATD). The report includes the prevalent alpha-1 antitrypsin deficiency (AATD) scenario in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country-wise Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology

The epidemiology segment also provides the alpha-1 antitrypsin deficiency (AATD) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

 

The total prevalent patient population of alpha-1 antitrypsin deficiency (AATD) in the 7MM countries was nearly 190,000 cases in 2021.

 

As per the estimates, the US had the highest prevalent patient population of alpha-1 antitrypsin deficiency (AATD) in 2021. Among the EU5 countries, the UK had the highest total patient population of alpha-1 antitrypsin deficiency (AATD), with more than 20,000 cases, followed by Germany in 2021. On the other hand, France had the lowest total prevalent patient population of alpha-1 antitrypsin deficiency (AATD) with approximately 10,000 cases in 2021.

Alpha-1 Antitrypsin Deficiency (AATD) Drug Chapters

The drug chapter segment of the alpha-1 antitrypsin deficiency (AATD) report encloses the detailed analysis of alpha-1 antitrypsin deficiency (AATD) marketed drugs and late-stage (Phase III, Phase II/III, Phase II, and Phase I/II) pipeline drugs. It also helps understand the alpha-1 antitrypsin deficiency (AATD) clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

 

The current standard of care lacks efficiency in treating alpha-1 antitrypsin deficiency (AATD) that permanently cures AATD. Treatment of AATD has traditionally been lung-centric and has aimed to delay the onset of emphysema, reduce the frequency of pulmonary exacerbations, and improve QOL. The only treatment option for AATD-associated liver disease was liver transplantation, which had good outcomes in children and adults with AATD. However, this is a treatment reserved for advanced diseases. The treatment options available to AATD patients at present are broadly similar to patients with COPD unrelated to AATD, with symptoms managed by bronchodilators, inhaled steroids, pulmonary rehabilitation, possibly lung volume reduction treatment, and lung transplantation.

 

Thus, there is an immense need to raise awareness to improve AATD diagnosis and treatment. Also, proper understanding is needed regarding the role of AATD in liver disorders and skin disorders.

Products detail in the report…

 

Alpha-1 Antitrypsin Deficiency (AATD) Emerging Drugs

Drug developers are gradually shifting their attention toward alpha-1 antitrypsin deficiency (AATD) to meet the patient pool’s current demands and counter the unmet needs of the therapeutic market.

 

Several companies are working robustly on many new therapies, such as ARO-AAT (Arrowhead Pharmaceutical), Alvelestat (Mereo Biopharma), and Inhaled AAT (Kamada).

 

ARO-AAT (Arrowhead Pharmaceutical) is a second-generation subcutaneously administered compound that knocks down the alpha-1 antitrypsin (AAT) gene transcript and reduces the hepatic production of the mutant AAT protein. Currently, the company is conducting Phase II clinical trials to evaluate the safety, tolerability, and pharmacodynamic effect of the drug in AATD patients.

 

Alvelestat (Mereo Biopharma) is an oral drug researched in people with AATD lung disease. It inhibits the neutrophil elastase enzyme and protects AATD patients from further lung damage. Currently, the drug is being evaluated in Phase II to treat AATD. The US FDA has granted ODD to alvelestat for treating AATD.

Products detail in the report…

Alpha-1 Antitrypsin Deficiency (AATD) Market Outlook

The alpha-1 antitrypsin deficiency (AATD) market outlook of the report builds a detailed comprehension of the historical, current, and forecasted alpha-1 antitrypsin deficiency (AATD) market trends by analyzing the impact of current therapies on the market, unmet needs, and demand for better technology.

 

This segment gives a thorough detail of the alpha-1 antitrypsin deficiency (AATD) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need for the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market, and KOL view. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

 

As per DelveInsight, the alpha-1 antitrypsin deficiency (AATD) market in 7MM is expected to change in the study period 2019–2032.

 

Key Findings

This section includes a glimpse of the alpha-1 antitrypsin deficiency (AATD) market in 7MM. In the 7MM, the alpha-1 antitrypsin deficiency (AATD) market size was approximately USD 1,200 million in 2021.

 

The United States: Market Outlook

This section provides the total alpha-1 antitrypsin deficiency (AATD) market size. It also provides the market size of alpha-1 antitrypsin deficiency (AATD) by therapies in the United States.

 

The United States accounts for the highest market size of alpha-1 antitrypsin deficiency (AATD) than the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

 

EU5 Countries: Market Outlook

This section provides the total alpha-1 antitrypsin deficiency (AATD) market size. It also provides alpha-1 antitrypsin deficiency (AATD) market size by therapies in Germany, France, Italy, Spain, and the United Kingdom.

 

Japan Market Outlook

This section provides the total alpha-1 antitrypsin deficiency (AATD) market size. It also provides the market size of alpha-1 antitrypsin deficiency (AATD) by therapies in Japan.

Alpha-1 Antitrypsin Deficiency (AATD) Drugs Uptake

This section focuses on the uptake rate of potential drugs recently launched or expected to get launched in the market during the study period 2019–2032. The analysis covers alpha-1 antitrypsin deficiency (AATD) market uptake by drugs, patient uptake by therapies, and sales of each drug.

 

This helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on market share and size, which again will be useful in investigating factors important in the market uptake and making financial and regulatory decisions.

Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stages. It also analyses alpha-1 antitrypsin deficiency (AATD)’s key players in developing targeted therapeutics.

 

Major players include Arrowhead Pharmaceuticals, Kamada, and Mereo Biopharma, whose key products are expected to launch in the US market by 20XX.

 

Pipeline Development Activities

The report covers information on collaborations, acquisitions, mergers, licensing, and patent details for emerging alpha-1 antitrypsin deficiency (AATD) therapies.

KOL Views

To keep up with current market trends, we take KOLs and SMEs’ opinions working in the alpha-1 antitrypsin deficiency (AATD) domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies treatment patterns or alpha-1 antitrypsin deficiency (AATD) market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

We perform a competitive and market intelligence analysis of the alpha-1 antitrypsin deficiency (AATD) market using various competitive intelligence tools: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

  • The report covers the descriptive overview of alpha-1 antitrypsin deficiency (AATD), explaining its causes, signs and symptoms, pathophysiology, and currently available therapies
  • Comprehensive insight is provided into the alpha-1 antitrypsin deficiency (AATD) epidemiology and treatment in the 7MM
  • Additionally, an all-inclusive account of both the current and emerging therapies for alpha-1 antitrypsin deficiency (AATD) is provided, along with the assessment of new therapies that will impact the current treatment landscape
  • A detailed review of the alpha-1 antitrypsin deficiency (AATD) market; historical and forecasted, is included in the report, covering drug outreach in the 7MM
  • The report provides an edge while developing business strategies by understanding trends shaping and driving the global alpha-1 antitrypsin deficiency (AATD) market

Report Highlights

  • Recently, the alpha-1 antitrypsin deficiency (AATD) market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market
  • The companies and academics are working to assess challenges and seek opportunities that could influence alpha-1 antitrypsin deficiency (AATD) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
  • Major players are involved in developing therapies for alpha-1 antitrypsin deficiency (AATD). The launch of emerging therapies will significantly impact the alpha-1 antitrypsin deficiency (AATD) market
  • For alpha-1 antitrypsin deficiency (AATD), a better understanding of disease pathogenesis will also contribute to developing novel therapeutics
  • Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competitor, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the R&D activities

Alpha-1 Antitrypsin Deficiency (AATD) Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Alpha-1 Antitrypsin Deficiency (AATD) Pipeline Analysis
  • Alpha-1 Antitrypsin Deficiency (AATD) Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Alpha-1 Antitrypsin Deficiency (AATD) Report Key Strengths

  • 11-year Forecast
  • 7MM Coverage
  • Alpha-1 Antitrypsin Deficiency (AATD) Epidemiology Segmentation
  • Key Competitors
  • Highly Analyzed Market
  • Drugs Uptake

Alpha-1 Antitrypsin Deficiency (AATD) Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Pipeline Product Profiles
  • Market Attractiveness

Key Questions

Market Insights:

  • What was the market share percentage distribution, and how would it look by 2032?
  • What would be the total market size and market size of alpha-1 antitrypsin deficiency (AATD) by therapies across the 7MM forecast period (2019–2032)?
  • What are the key findings about the market across 7MM, and which country will have the largest alpha-1 antitrypsin deficiency (AATD) market size during the forecast period (2019–2032)?
  • At what CAGR is the alpha-1 antitrypsin deficiency (AATD) market expected to grow in the 7MM forecast period (2019–2032)?
  • What would be the alpha-1 antitrypsin deficiency (AATD) market outlook across the 7MM forecast period (2019–2032)?
  • What would be the alpha-1 antitrypsin deficiency (AATD) market growth until 2032 and the resultant market size by 2032?
  • How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

 

Epidemiology Insights:

  • What are the disease risk, burdens, and unmet needs of alpha-1 antitrypsin deficiency (AATD)?
  • What is the historical patient pool of alpha-1 antitrypsin deficiency (AATD) covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What would be the forecasted patient pool of alpha-1 antitrypsin deficiency (AATD) covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
  • What will be the growth opportunities in the 7MM concerning the patient population of alpha-1 antitrypsin deficiency (AATD)?
  • Out of all the 7MM, which country would have the highest prevalence of alpha-1 antitrypsin deficiency (AATD) during the forecast period (2019–2032)?
  • At what CAGR is the population expected to grow in the 7MM during the forecast period (2019–2032)?

 

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:

  • What are the current options to treat alpha-1 antitrypsin deficiency (AATD)?
  • What are the current treatment guidelines for treating alpha-1 antitrypsin deficiency (AATD) in the US, Europe, and Japan?
  • What are the alpha-1 antitrypsin deficiency (AATD) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety, efficacy, etc.?
  • How many companies are developing therapies to treat alpha-1 antitrypsin deficiency (AATD)?
  • How many therapies are developed by each company to treat alpha-1 antitrypsin deficiency (AATD)?
  • How many emerging therapies are in the mid-stage and late stages of development to treat alpha-1 antitrypsin deficiency (AATD)?
  • What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, and licensing activities related to the Alpha-1 Antitrypsin deficiency (AATD) therapies?
  • What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
  • What are the clinical studies for alpha-1 antitrypsin deficiency (AATD) and its status?
  • Which key designations have been granted for the emerging therapies for alpha-1 antitrypsin deficiency (AATD)?
  • What are the global historical and forecasted alpha-1 antitrypsin deficiency (AATD) markets?

Reasons to buy

  • The report will help in developing business strategies by understanding trends shaping and driving the alpha-1 antitrypsin deficiency (AATD) market
  • To understand the future market competition in the alpha-1 antitrypsin deficiency (AATD) market and an insightful review of the key market drivers and barriers
  • Organize sales and marketing efforts by identifying the best opportunities for alpha-1 antitrypsin deficiency (AATD) in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan
  • Identifying upcoming solid players in the market will help devise strategies that will help get ahead of competitors
  • Organize sales and marketing efforts by identifying the best opportunities for the alpha-1 antitrypsin deficiency (AATD) market
  • To understand the future market competition in the alpha-1 antitrypsin deficiency (AATD) market

1. Key Insights

2. Report Introduction

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

3.1. Market Share (%) Distribution of Alpha-1 Antitrypsin Deficiency in 2019

3.2. Market Share (%) Distribution of Alpha-1 Antitrypsin Deficiency in 2032

4. Executive Summary of Alpha-1 Antitrypsin Deficiency

5. Disease Background and Overview

5.1. Introduction

5.2. Clinical Manifestations

5.3. Etiology

5.4. Pathogenesis

5.5. Diagnosis

5.5.1. Biomarkers

5.5.2. Differential Diagnosis

5.6. Treatment and Management

5.6.1. Treatment algorithm

5.6.1.1. Management of AATD-associated lung disease

5.6.1.2. Management of AATD-associated liver disease

6. Epidemiology and Patient Population

6.1. Key Findings

6.2. Total Prevalence of AATD in the 7MM

6.3. Assumption and Rationale

6.4. The United States

6.4.1. Prevalence of AATD in the United States

6.4.2. Prevalence of AATD based on Symptoms in the United States

6.4.3. Diagnosed Population of AATD in the United States

6.4.4. The Genotype of AATD Diagnosed patients in the United States

6.4.5. Comorbidity associated with AATD in the United States

6.5. The EU5

6.5.1. Prevalence of AATD in the EU5

6.5.2. Prevalence of AATD based on Symptoms in the EU5

6.5.3. Diagnosed Population of AATD in the EU5

6.5.4. The Genotype of AATD Diagnosed patients in the EU5

6.5.5. Comorbidity associated with AATD in the EU5

6.6. Japan

6.6.1. Prevalence of AATD in Japan

6.6.2. Prevalence of AATD based on Symptoms in Japan

6.6.3. Diagnosed Population of AATD in Japan

6.6.4. The Genotype of AATD Diagnosed patients in Japan

6.6.5. Comorbidity associated with AATD in Japan

7. Patient Journey of Alpha-1 Antitrypsin Deficiency

8. Marketed Products

8.1. Key cross Competition

8.2. PROLASTIN-C: Grifols

8.2.1. Product description

8.2.2. Regulatory milestones

8.2.3. Other development activities

8.2.4. Safety and efficacy of PROLASTIN-C

8.2.5. Side effects of PROLASTIN-C

8.2.6. Product profile

8.3. ZEMAIRA: CSL Behring

8.3.1. Product description

8.3.2. Regulatory milestones

8.3.3. Safety and efficacy of ZEMAIRA

8.3.4. Side effects of ZEMAIRA

8.3.5. Product profile

8.4. GLASSIA: Kamada

8.4.1. Product description

8.4.2. Regulatory milestones

8.4.3. Other development activities

8.4.4. Safety and efficacy of GLASSIA

8.4.5. Side effects of GLASSIA

8.4.6. Product profile

8.5. ARALAST: Shire

8.5.1. Product description

8.5.2. Regulatory milestones

8.5.3. Other development activities

8.5.4. Safety and efficacy of ARALAST

8.5.5. Side effects of ARALAST

8.5.6. Product profile

8.6. ALFALASTIN: LFB Biotechnologies

8.6.1. Product description

8.6.2. Regulatory milestones

8.6.3. Safety and efficacy of ALFALASTIN

8.6.4. Side effects of ALFALASTIN

8.6.5. Product profile

8.7. TRYPSONE: Grifols

8.7.1. Product description

8.7.2. Regulatory milestones

8.7.3. Safety and efficacy of TRYPSONE

8.7.4. Side effects of TRYPSONE

8.7.5. Product profile

9. Emerging Therapies

9.1. Key Cross: Emerging

9.2. ARO-AAT (Fazirsiran): Arrow Head Pharmaceuticals

9.2.1. Drug description

9.2.2. Other development activities

9.2.3. Clinical development

9.2.3.1. Clinical trial information

9.2.4. Safety and efficacy

9.2.5. Product profile

9.2.6. Analyst comments

9.3. Alvelestat (MPH-966): Mereo Biopharma

9.3.1. Drug description

9.3.2. Other development activities

9.3.3. Clinical development

9.3.3.1. Clinical trial information

9.3.4. Safety and efficacy

9.3.5. Product profile

9.3.6. Analyst comments

9.4. Inhaled AAT: Kamada Ltd

9.4.1. Drug description

9.4.2. Other development activities

9.4.3. Clinical development

9.4.3.1. Clinical trial information

9.4.4. Safety and efficacy

9.4.5. Product profile

9.4.6. Analyst comments

10. Other Assets

10.1. Key Cross

10.2. Belcesiran (DCR-A1AT): Dicerna Pharmaceuticals

10.2.1. Drug description

10.2.2. Other development activities

10.2.3. Clinical development

10.2.3.1. Clinical trial information

10.2.4. Product Profile

10.3. INBRX-101: CHIESI Farmaceutici

10.3.1. Drug description

10.3.2. Other development activities

10.3.3. Clinical development

10.3.3.1. Clinical trial information

10.3.4. Product Profile

11. Alpha-1 Antitrypsin Deficiency (AATD): Seven Major Market Analysis

11.1. Key Findings

11.2. Total Market Size of AATD in the 7MM

11.3. Market Outlook

11.4. Attribute Analysis

11.5. The United States Market Size

11.5.1. Total Market Size of AATD in the United States

11.5.2. Market Size of AATD by Therapies in the United States

11.6. The EU5 Market Size

11.6.1. Total Market Size of AATD in the EU5

11.6.2. Market Size of AATD in the EU5 by Therapies

11.7. Japan Market Size

11.7.1. Total Market Size of AATD in Japan

11.7.2. Market Size of AATD in Japan by Therapies

12. SWOT Analysis

13. KOL Views

14. Unmet Needs

15. Market Access

16. Acronyms and Abbreviations

17. Appendix

17.1. Bibliography

17.2. Report Methodology

18. DelveInsight Capabilities

19. Disclaimer

20. About DelveInsight

List of Table

Table 1: Summary of AATD, Epidemiology, and Key Events (2019–2032)

Table 2: Symptoms of AATD Associated Liver Disease by Age Groups

Table 3: Common Genotypes Associated With AATD

Table 4: Recommendations for the diagnosis of AATD by Alpha-1 Foundation

Table 5: Genetic Disorders Associated With Liver Disease in the Differential Diagnosis of AATD

Table 6: Summary of 2016 Alpha-1 Foundation recommendations on AATD management

Table 7: Summary of ATS/ERS and US Guidelines on AATD-associated Lung Disease Management

Table 8: Summary of ATS/ERS and US Guidelines on AATD-associated Liver Disease Management

Table 9: Total Prevalent Cases of AATD in the 7MM (2019–2032)

Table 10: Prevalent cases of AATD in the United States (2019–2032)

Table 11: Prevalent cases of AATD based on Symptoms in the United States (2019–2032)

Table 12: Diagnosed Population of AATD in the United States (2019–2032)

Table 13: Genotype of AATD Diagnosed patients in the United States (2019–2032)

Table 14: Comorbidity associated with AATD in the United States (2019–2032)

Table 15: Prevalent cases of AATD in the EU5 (2019–2032)

Table 16: Prevalent cases of AATD based on Symptoms in the EU5 (2019–2032)

Table 17: Diagnosed Population of AATD in the EU5 (2019–2032)

Table 18: Genotype of AATD Diagnosed patients in the EU5 (2019–2032)

Table 19: Comorbidity associated with AATD in the EU5 (2019–2032)

Table 20: Prevalence of AATD in Japan (2019–2032)

Table 21: Prevalence of AATD based on Symptoms in Japan (2019–2032)

Table 22: Diagnosed Population of AATD in Japan (2019–2032)

Table 23: The Genotype of AATD Diagnosed patients in Japan (2019–2032)

Table 24: Comorbidity associated with AATD in Japan (2019–2032)

Table 25: Key cross of emerging drugs

Table 26: ARO-AAT, Clinical Trial Description, 2022

Table 27: Alvelestat (MPH-966), Clinical Trial Description, 2022

Table 28: Inhaled AAT, Clinical Trial Description, 2022

Table 29: Belcesiran, Clinical Trial Description, 2022

Table 30: INBRX-101, Clinical Trial Description, 2022

Table 31: Total Market Size of AATD in the 7MM in USD Million (2019–2032)

Table 32: Weighted Score Based on Ranking

Table 33: Market Size of AATD in the United States in USD Million (2019–2032)

Table 34: Market Size of AATD in the United States by Therapies in USD Million (2019–2032)

Table 35: The EU5 Market Size of AATD in USD Million (2019–2032)

Table 36: Market Size of AATD in the EU5 by Therapies in USD Million (2019–2032)

Table 37: Total Market Size of AATD in Japan in USD Million (2019–2032)

Table 38: Market Size of AATD in Japan by Therapies in USD Million (2019–2032)

List of Figures

Figure 1: Symptoms Associated With AATD

Figure 2: Panniculitis Associated With AATD

Figure 3: Other Clinical Manifestations of AATD

Figure 4: Risk Factors

Figure 5: Risk of AATD if Both Parents are Carriers

Figure 6: Risk of AATD if One Parent has AATD

Figure 7: Potential Mechanism for Increased Smoking Induced-disease Risk in Individuals With the PI*MZ Genotype

Figure 8: Pathogenesis of AATD-associated Lung Disease

Figure 9: Pathogenesis of AATD-associated Liver Disease

Figure 10: Diagnostic and Monitoring Methods for AATD

Figure 11: ATS/ERS Diagnostic Recommendations for AATD

Figure 12: ATS/ERS Recommendations (to be discussed) for AATD

Figure 13: ATS/ERS Diagnostic Recommendations (not recommended) for AATD

Figure 14: Treatment Algorithm of AATD

Figure 15: Total Prevalent Cases of AATD in the 7MM (2019–2032)

Figure 16: Prevalent cases of AATD in the United States (2019–2032)

Figure 17: Prevalent cases of AATD based on Symptoms in the United States (2019–2032)

Figure 18: Diagnosed Population of AATD in the United States (2019–2032)

Figure 19: Genotype of AATD Diagnosed patients in the United States (2019–2032)

Figure 20: Comorbidity associated with AATD in the United States (2019–2032)

Figure 21: Prevalent cases of AATD in the EU5 (2019–2032)

Figure 22: Prevalent cases of AATD based on Symptoms in the EU5 (2019–2032)

Figure 23: Diagnosed Population of AATD in the EU5 (2019–2032)

Figure 24: Genotype of AATD Diagnosed patients in the EU5 (2019–2032)

Figure 25: Comorbidity associated with AATD in the EU5 (2019–2032)

Figure 26: Prevalence of AATD in Japan (2019–2032)

Figure 27: Prevalence of AATD based on Symptoms in Japan (2019–2032)

Figure 28: Diagnosed Population of AATD in Japan (2019–2032)

Figure 29: The Genotype of AATD Diagnosed patients in Japan (2019–2032)

Figure 30: Comorbidity associated with AATD in Japan (2019–2032)

Figure 31: Total Market Size of AATD in the 7MM (2019–2032)

Figure 32: Market Size of AATD in the United States (2019–2032)

Figure 33: Market Size of AATD in the United States by Therapies (2019–2032)

Figure 34: The EU5 Market Size of AATD (2019–2032)

Figure 35: Market Size of AATD in the EU5 by Therapies (2019–2032)

Figure 36: Total Market Size of AATD in Japan (2019–2032)

Figure 37: Market Size of AATD in Japan by Therapies (2019–2032)

Arrow Head Pharmaceuticals
Mereo Biopharma
Kamada Ltd
Grifols
CSL Behring
Shire
LFB Biotechnologies

 

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