Alpha-1 Antitrypsin Deficiency Treatment Market 2034: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, and Companies by DelveInsight

Alpha-1 Antitrypsin Deficiency Companies are Kamada Pharmaceuticals, Arrowhead Pharmaceuticals, Takeda, Mereo BioPharma, AstraZeneca, Inhibrx, Novo Nordisk, and others.
(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

* Among the 7MM countries, Alpha-1 Antitrypsin Deficiency market size was valued approximately ~USD 1,238 million in 2023 and is anticipated to grow with a significant CAGR during the study period (2020-2034)
* In April 2024, Wave Life Sciences Ltd. (Nasdaq: WVE), a clinical-stage biotechnology company focused on advancing RNA medicines to improve human health, announced the approval of its first clinical trial application (CTA) for the RestorAATion-2 trial of WVE-006. This first-in-class RNA editing oligonucleotide is being developed for treating alpha-1 antitrypsin deficiency (AATD). WVE-006 is a GalNAc-conjugated, subcutaneously administered therapy that does not utilize a lipid nanoparticle (LNP) delivery system.
* In April 2024, Enlivex Therapeutics has announced promising topline safety and efficacy results from its Phase II clinical trial of Allocetra, an off-the-shelf cell therapy, for the treatment of sepsis. The placebo-controlled, randomized trial aims to identify the optimal dose of Allocetra when administered in combination with the standard care for sepsis caused by various infections.
* In 2023, the total market size of AATD in the US was around USD 986.01 million, making up roughly 80% of the total market revenue for the 7MM.
* In 2023, the total market size of AATD in the EU4 and the UK was estimated to be around USD 252.30 million. Within this group, Germany led the market with approximately USD 68.49 million, followed by France at around USD 53.12 million, and the UK at nearly USD 49.52 million.
* In 2023, the total market size of AATD in Japan was about USD 0.43 million.
* Among the therapies currently in use, ZEMAIRA/RESPREEZA held the largest market share, generating approximately USD 496.31 million in revenue in 2023 across the 7MM.
* In March 2024, Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines through base editing, announced that the United Kingdom Medicines and Healthcare Products Regulatory Agency has cleared its clinical trial authorization (CTA) application for BEAM-302, an in vivo base editor intended as a potential treatment for patients with alpha-1 antitrypsin deficiency (AATD).
* In September 2023, The US Food and Drug Administration has approved Krystal Biotech's Investigational New Drug (IND) application for a Phase I clinical trial of KB408, an investigational treatment for alpha-1 antitrypsin deficiency (AATD).
* In July 2023, Grifols has achieved the target enrollment of 339 subjects in the Phase III SPARTA (Study of Prolastin-C Randomized Therapy with Alpha-1 Augmentation) trial for treating emphysema. This placebo-controlled, double-blind, randomized study aims to evaluate the safety and efficacy of 60mg/kg and 120mg/kg doses of Alpha1-Proteinase inhibitor Prolastin-C, administered weekly, in slowing the progression of lung tissue loss in emphysema patients.
* In May 2023, Inhibrx, Inc. (Nasdaq: INBX), a clinical-stage biopharmaceutical company focused on developing therapies for oncology and rare diseases, announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to INBRX-101, an optimized recombinant human AAT-Fc fusion protein, for treating patients with emphysema due to alpha-1 antitrypsin deficiency (AATD).
* DelveInsight's epidemiology model indicates that in the 7MM, the total diagnosed prevalent cases of AATD were around 221 thousand in 2023. This number is expected to increase during the forecast period (2024-2034) due to heightened awareness, improved screening, and advancements in genetic testing.
* In 2023, the US had the highest number of diagnosed prevalent cases of AATD, with around 133 thousand cases, while Japan had the fewest, with only 23 cases.
* In 2023, among the EU4 and the UK, the UK had the highest number of diagnosed prevalent cases of AATD, with around 23 thousand cases, followed by Germany with approximately 20 thousand cases, and France with nearly 18 thousand cases.
* Key Alpha-1 Antitrypsin Deficiency Companies: Kamada Pharmaceuticals, Mereo BioPharma/AstraZeneca, Grifols Therapeutics LLC, Takeda, Vertex Pharmaceuticals, Wave Life Sciences, Beam Therapeutics, Arrowhead Pharmaceuticals, Inhibrx, Inc., Dicerna Pharmaceuticals, Grifols Therapeutics LLC, and others
* Key Alpha-1 Antitrypsin Deficiency Therapies: Inhaled Alpha 1-Antitrypsin (AAT), Alvelestat (MPH966), Alpha-1 15%, VX-864, WVE-006, BEAM-302, Fazirsiran Injection (TAK-999, ARO-AAT), INBRX-101, Belcesiran, Alpha-1 MP, and others
* The Alpha-1 Antitrypsin Deficiency market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Alpha-1 Antitrypsin Deficiency pipeline products will significantly revolutionize the Alpha-1 Antitrypsin Deficiency market dynamics.

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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:

* Total AATD Prevalent Cases
* AATD Genotype-specific Prevalent Cases
* AATD Comorbidity-associated Prevalent Cases

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.

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Alpha-1 Antitrypsin Deficiency Pipeline Therapies and Key Companies

* Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals
* Fazirsiran (ARO-AAT/TAK-999): Arrowhead Pharmaceuticals/Takeda
* Alvelestat (MPH-966): Mereo BioPharma/AstraZeneca
* VX-864: Vertex Pharmaceuticals -- INBRX-101: Inhibrx
* Belcesiran (DCR-A1AT): Novo Nordisk

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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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