(Albany, USA) DelveInsight’s Alpha-1 Antitrypsin Deficiency Market Insights report includes a comprehensive understanding of current treatment practices, alpha-1 antitrypsin deficiency emerging drugs, market share of individual therapies, and current and forecasted market size from 2020 to 2034, segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan].
Key Takeaways from the Alpha-1 Antitrypsin Deficiency Market Report
Discover which therapies are expected to grab the major AATD market share @ Alpha-1 Antitrypsin Deficiency Market Report
Alpha-1 Antitrypsin Deficiency Overview
Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary autosomal codominant genetic disorder characterized by a shortage of the alpha-1 antitrypsin protein (AAT). This protein is crucial for safeguarding the lungs and liver from enzyme-induced damage, and its insufficiency can result in severe respiratory and liver-related complications. The condition primarily arises from mutations in the SERPINA1 gene, responsible for producing the AAT protein, which shields the body from the potent enzyme neutrophil elastase.
AATD encompasses symptoms such as breathlessness, persistent coughing, wheezing, and a diminished capacity for exercise. The emergence and intensity of these AATD symptoms can widely differ among individuals, even those sharing identical genetic mutations. While some individuals may exhibit no symptoms, others may face considerable health challenges. Timely detection of AATD is crucial for preventing the progression of the disease, enabling prompt intervention, and optimizing treatment management.
The diagnosis of AATD typically involves blood tests to measure AAT levels and genetic testing to identify specific mutations. AATD is frequently misdiagnosed or delayed in diagnosis as other conditions such as COPD, emphysema, chronic bronchitis, or AAT-associated liver disease, given the common respiratory and hepatitis symptoms they share. Therefore, a differential diagnosis may pinpoint particular markers in blood or other biological samples associated with AATD.
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Alpha-1 Antitrypsin Deficiency Epidemiology Segmentation
The AATD epidemiology section provides insights into the historical and current AATD patient pool and forecasted trends for the 7MM. It helps recognize the causes of current and forecasted patient trends by exploring numerous studies and views of key opinion leaders.
The AATD market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:
Alpha-1 Antitrypsin Deficiency Treatment Market
The existing AATD treatment landscape lacks definitive and disease-altering remedies. Symptomatic relief and the management of lung-related complications such as COPD or emphysema often rely on various off-label drugs, including bronchodilators, corticosteroids, and antibiotics. Augmentation therapy, also known as replacement therapy, is an approved approach for addressing AAT-related lung disease. In the severe stages of the ailment, surgical interventions like lung volume reduction surgery and bullectomy are recommended. For individuals with highly advanced emphysema, lung transplant surgery may be necessary, entailing the removal of a damaged lung and its replacement with a healthy one; however, this procedure comes with significant risks, including the potential for infection and rejection.
IV replacement therapy, primarily employed in the treatment of lung disorders, employs AAT protein sourced from the blood plasma of healthy human donors. This approach is applied to enhance alpha-1 levels in individuals diagnosed with emphysema, to elevate the concentration of alpha-1 protein in the lungs. The objective is to safeguard the lungs from the harmful impacts of necrotizing panniculitis (NP). The therapy involves a weekly IV infusion and can be administered on a lifelong basis until alternative treatments become accessible.
FDA-approved augmentation therapies available for purchase in the US market include PROLASTIN-C by Grifols, ARALAST by Takeda, ZEMAIRA by CSL Behring, and GLASSIA by Kamada. PROLASTIN, introduced in 1988, has maintained a commendable safety record. Subsequently, ARALAST and ZEMAIRA entered the market in 2003, followed by GLASSIA in 2010. Each of these therapies received approval based on their safety profiles and their ability to augment alpha-1 levels in the blood and lungs, comparable to PROLASTIN.
To know more about AATD treatment guidelines, visit @ Alpha-1 Antitrypsin Deficiency Prevalence
Alpha-1 Antitrypsin Deficiency Pipeline Therapies and Key Companies
Learn more about the FDA-approved drugs for AATD @ Drugs for Alpha-1 Antitrypsin Deficiency Treatment
Alpha-1 Antitrypsin Deficiency Market Dynamics
The dynamics of the alpha-1 antitrypsin deficiency market are expected to change in the coming years. Ongoing research and an increased understanding of diseases have resulted in the identification of therapies featuring effective and convenient routes of administration, such as subcutaneous, inhalation, and oral methods, with the potential to enhance the quality of life for patients. Active patient registries and supportive organizations play a crucial role in providing information, advocacy, and assistance to individuals with alpha-1 antitrypsin deficiency. Meanwhile, exploration of novel approaches like RNA editing and gene therapy in several preclinical and early-phase assets is underway, offering promising avenues for curative therapies. Despite the absence of approved therapies for liver disease associated with alpha-1 antitrypsin deficiency, this unmet need presents a strategic opportunity for pharmaceutical players to seize a first-mover advantage in the untapped AATD market.
Furthermore, many potential therapies are being investigated for the treatment of AATD, and it is safe to predict that the treatment space will significantly impact the AATD market during the forecast period. Moreover, the anticipated introduction of emerging therapies with improved efficacy and a further improvement in the diagnosis rate are expected to drive the growth of the AATD market in the 7MM.
However several factors may impede the growth of the alpha-1 antitrypsin deficiency market. The majority of AATD cases remain undiagnosed, with an average delay of over 5 years until diagnosis. Unfortunately, there is no curative treatment for this condition, compelling patients to depend on long-term medication or undergo transplant surgeries in severe cases. However, transplants pose risks of rejection and are associated with mortality or morbidity. The rarity and under diagnosis of the disease create challenges in enrolling a sufficient number of patients for clinical trials, while undefined outcome measures further hinder the development of these trials. Additionally, AATD is frequently misdiagnosed due to its similarities with other conditions such as COPD, asthma, and emphysema.
Moreover, AATD treatment poses a significant economic burden and disrupts patients’ overall well-being and QOL. Furthermore, the AATD market growth may be offset by failures and discontinuation of emerging therapies, unaffordable pricing, market access and reimbursement issues, and a shortage of healthcare specialists. In addition, the undiagnosed, unreported cases and the unawareness about the disease may also impact the AATD market growth.
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Table of Content:
1. Key Insights
2. Report Introduction
3. Executive Summary
4. Alpha-1 Antitrypsin Deficiency Market Overview at a Glance
4.1. Market Share Distribution by Therapies (%) in 2020 in the 7MM
4.2. Market Share Distribution by Therapies (%) in 2034 in the 7MM
5. Alpha-1 Antitrypsin Deficiency Epidemiology and Market Forecast Methodology
6. Key Events
7. Disease Background and Overview
8. AATD Epidemiology and Patient Population
9. Alpha-1 Antitrypsin Deficiency Patient Journey
10. Marketed Alpha-1 Antitrypsin Deficiency Drugs
11. Emerging Alpha-1 Antitrypsin Deficiency Drugs
12. AATD Market: Seven Major Market Analysis
13. Alpha-1 Antitrypsin Deficiency Unmet Needs
14. Alpha-1 Antitrypsin Deficiency SWOT Analysis
15. Alpha-1 Antitrypsin Deficiency KOL Views
16. Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement
17. Appendix
18. DelveInsight Capabilities
19. Disclaimer
20. About DelveInsight
About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.
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