Alpha-1 Antitrypsin Deficiency Market Expected to Experience Major Growth by 2034, According to DelveInsight | Kamada Pharma, Mereo BioPharma/AstraZeneca, Grifols Therapeutics LLC, Takeda, Vertex

The Key Alpha-1 Antitrypsin Deficiency Companies in the market include – 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.

 

DelveInsight’s “Alpha-1 Antitrypsin Deficiency Market Insights, Epidemiology, and Market Forecast-2034″ report offers an in-depth understanding of the Alpha-1 Antitrypsin Deficiency, historical and forecasted epidemiology as well as the Alpha-1 Antitrypsin Deficiency market trends in the United States, EU4 (Germany, Spain, Italy, France) the United Kingdom and Japan.

 

To Know in detail about the Alpha-1 Antitrypsin Deficiency market outlook, drug uptake, treatment scenario and epidemiology trends, Click here; Alpha-1 Antitrypsin Deficiency Market Forecast

 

Some of the key facts of 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.

 

Alpha-1 Antitrypsin Deficiency Overview

Alpha-1 Antitrypsin Deficiency (AATD) is a genetic disorder that affects the liver and lungs. It occurs when there is a deficiency in the production of alpha-1 antitrypsin (AAT) protein, which is mainly produced by the liver and helps protect the lungs from damage caused by inflammation. Without enough AAT, the walls of the lungs can be damaged by enzymes released from white blood cells, leading to conditions such as emphysema and chronic obstructive pulmonary disease (COPD).

 

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Alpha-1 Antitrypsin Deficiency Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

 

Alpha-1 Antitrypsin Deficiency Epidemiology Segmentation:

The Alpha-1 Antitrypsin Deficiency market report proffers epidemiological analysis for the study period 2020–2034 in the 7MM segmented into:

  • Total Prevalence of Alpha-1 Antitrypsin Deficiency

  • Prevalent Cases of Alpha-1 Antitrypsin Deficiency by severity

  • Gender-specific Prevalence of Alpha-1 Antitrypsin Deficiency

  • Diagnosed Cases of Episodic and Chronic Alpha-1 Antitrypsin Deficiency

 

Download the report to understand which factors are driving Alpha-1 Antitrypsin Deficiency epidemiology trends @ Alpha-1 Antitrypsin Deficiency Epidemiology Forecast

 

Alpha-1 Antitrypsin Deficiency Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Alpha-1 Antitrypsin Deficiency market or expected to get launched during the study period. The analysis covers Alpha-1 Antitrypsin Deficiency market uptake by drugs, patient uptake by therapies, and sales of each drug. 

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Alpha-1 Antitrypsin Deficiency Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

 

Alpha-1 Antitrypsin Deficiency Therapies and Key Companies

  • Alpha-1 15%: Grifols Therapeutics LLC

  • Fazirsiran Injection: Takeda

  • VX-864: Vertex Pharmaceuticals

  • WVE-006: Wave Life Sciences

  • BEAM-302: Beam Therapeutics

  • Fazirsiran Injection (TAK-999, ARO-AAT): Arrowhead Pharmaceuticals

  • INBRX-101: Inhibrx, Inc.

  • Belcesiran: Dicerna Pharmaceuticals

  • Alpha-1 MP: Grifols Therapeutics LLC

 

Discover more about therapies set to grab major Alpha-1 Antitrypsin Deficiency market share @ Alpha-1 Antitrypsin Deficiency Treatment Landscape

 

Alpha-1 Antitrypsin Deficiency Market Strengths

  • Ongoing research and an increase in disease understanding have led to the identification of therapies with effective and convenient routes of administration, including subcutaneous, inhalation, and oral, with the potential to improve patient’s QoL.

  • Active patient registries and support organizations provide information, advocacy, and support to alpha-1 antitrypsin deficiency individuals

 

Alpha-1 Antitrypsin Deficiency Market Opportunities

  • Several preclinical and early-phase assets utilizing novel approaches like RNA editing and gene therapy are under investigation, and conducting further research and clinical trials may offer curative therapy.

  • No therapies are approved for liver disease associated with alpha-1 antitrypsin deficiency, allowing pharma players to capitalize on this untapped market by grabbing first mover advantage.

 

Scope of the Alpha-1 Antitrypsin Deficiency Market Report

  • Study Period: 2020–2034

  • Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

  • 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

  • Alpha-1 Antitrypsin Deficiency Therapeutic Assessment: Alpha-1 Antitrypsin Deficiency current marketed and Alpha-1 Antitrypsin Deficiency emerging therapies

  • Alpha-1 Antitrypsin Deficiency Market Dynamics: Alpha-1 Antitrypsin Deficiency market drivers and Alpha-1 Antitrypsin Deficiency market barriers 

  • Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies

  • Alpha-1 Antitrypsin Deficiency Unmet Needs, KOL’s views, Analyst’s views, Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement 

 

To know more about Alpha-1 Antitrypsin Deficiency companies working in the treatment market, visit @ Alpha-1 Antitrypsin Deficiency Clinical Trials and Therapeutic Assessment

 

Table of Contents 

1. Alpha-1 Antitrypsin Deficiency Market Report Introduction

2. Executive Summary for Alpha-1 Antitrypsin Deficiency

3. SWOT analysis of Alpha-1 Antitrypsin Deficiency

4. Alpha-1 Antitrypsin Deficiency Patient Share (%) Overview at a Glance

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

6. Alpha-1 Antitrypsin Deficiency Disease Background and Overview

7. Alpha-1 Antitrypsin Deficiency Epidemiology and Patient Population

8. Country-Specific Patient Population of Alpha-1 Antitrypsin Deficiency 

9. Alpha-1 Antitrypsin Deficiency Current Treatment and Medical Practices

10. Alpha-1 Antitrypsin Deficiency Unmet Needs

11. Alpha-1 Antitrypsin Deficiency Emerging Therapies

12. Alpha-1 Antitrypsin Deficiency Market Outlook

13. Country-Wise Alpha-1 Antitrypsin Deficiency Market Analysis (2020–2034)

14. Alpha-1 Antitrypsin Deficiency Market Access and Reimbursement of Therapies

15. Alpha-1 Antitrypsin Deficiency Market Drivers

16. Alpha-1 Antitrypsin Deficiency Market Barriers

17.  Alpha-1 Antitrypsin Deficiency Appendix

18. Alpha-1 Antitrypsin Deficiency Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

 

 

About DelveInsight 

DelveInsight is a leading Healthcare Business Consultant, and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

It also offers Healthcare Consulting Services, which benefits in market analysis to accelerate the business growth and overcome challenges with a practical approach.

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