Alpha-1 Antitrypsin Deficiency Market Size and Trend Report including Epidemiology and Pipeline Analysis, Competitor Assessment, Unmet Needs, Clinical Trial Strategies and Forecast, 2021-2031

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Alpha-1 Antitrypsin Deficiency Market Report Overview

The Alpha-1 Antitrypsin Deficiency (AATD) market size in the 2MM (US and Germany) was valued at $1.36 billion in 2021 and is forecast to grow at a CAGR of more than 12% during the forecast period, 2021-2031. The introduction of 4 new pipeline agents are key drivers of this market growth. AATD is an inherited disease that causes an increased risk of chronic obstructive pulmonary disorder (COPD), hepatic failure, skin problems (panniculitis), and inflammation of the blood vessels (vasculitis). Pulmonary problems almost always occur in adults and skin problems may occur in adults and children. In pediatric cases of AATD, liver disease, as opposed to COPD, is the most common form in which the disease presents itself.

Currently, marketed therapies are successfully used in AATD patients with clinically evident emphysema. There is currently a lack of efficacious pipeline therapies that are able to address the hepatic complications in AATD without inducing off-target effects. Market exclusivity for rare disease therapeutics presents an opportunity to develop novel, efficacious therapies that can gain a significant share of the AATD market.

AATD Market Overview in the 2MM, 2021-2031

AATD Market Overview in the 2MM, 2021-2031

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What are the market drivers of the AATD market?

The AATD market is expected to have several novel entrants across the forecast period. These novel therapies will provide significant opportunities to address the need for long-term efficacious therapies. The uptake of therapies in the AATD pipeline by physicians will be driven by clinical data demonstrating efficacy and ease of administration as well as price. The entrance of RNAi therapies will likely increase treatment at the primary care level, as physicians will be able to directly treat patients with abnormal liver function tests and low AAT serum levels. This will increase market size for AATD as a greater number of patients will have access to treatment.

What are the classifications in the AATD market?

AATD is classified by the different gene variants inherited. The Z allele is the most common variant of the gene and causes AATD. The gene for producing normal AAT is known as the M gene. The S allele is a less common variant that causes AATD. However, inheriting both S and Z alleles also increases the likelihood of AATD. A patient who inherits the Z allele from each parent has a very low level of AATD, allowing the neutrophil elastase enzyme to damage the lung tissue.

What are the key phases of the AATD market clinical trials?

The key phases of the AATD market clinical trials are completed, terminated, ongoing, withdrawn, and planned. Most of the trials are in the completed stage. The increasing number of completed trials highlights the increasingly crowded and mature AATD market. AATD clinical research and investigation is dominated by proteinase inhibitors and RNAi therapeutics that are being trialed as monotherapies.

AATD Market by Phases

AATD Market by Phases

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What are the key pipeline products in the AATD market?

AATD has products in the late-stage pipeline at Phase II in the 2 major markets (2MM) of the US and Germany. The Alpha-1 Proteinase Inhibitor (A1-PI) therapy is currently in Phase II in the US only and the Proteinase Inhibitor therapy PHP-303 is in Phase II in the EU only. In addition, the NE therapy Alvelestat, is currently in Phase II in both the EU and US. There are two RNAi therapies set to launch towards the end of the forecast period and are novel to the AATD market in their MoA.

Who are the key players in the AATD market?

Some of the key players in the AATD market are Takeda, Kamada Pharmaceuticals, Grifols, CSL, Baxalta, pH Pharma Co, Mereo Biopharma, Novo Nordisk, and Arrowhead Pharmaceuticals.

Market report scope

Market Size (Year – 2021) $1.36 billion in the 2MM (US and Germany)
Growth rate CAGR of >12%
Forecast period 2021-2031
Classifications Z Allele and S Allele
Key phases Completed, Terminated, Ongoing, Withdrawn, and Planned
Key players Takeda, Kamada Pharmaceuticals, Grifols, CSL, Baxalta, pH Pharma Co, Mereo Biopharma, Novo Nordisk, and Arrowhead Pharmaceuticals

This report provides:

  • Overview of AATD, including epidemiology, symptoms, diagnosis, and disease management.
  • Annualized AATD therapeutics market revenue, annual cost of therapy, treatment usage pattern in 2021, and forecast for ten years to 2031.
  • Key topics including strategic competitor assessment, market characterization, unmet needs, clinical trial mapping, and implications for the AATD therapeutics market.
  • Pipeline analysis, comprehensive data assessing emerging trends and mechanisms of action under development for AATD treatment. The most promising candidates in late-stage development are profiled.
  • Analysis of the current and future market competition in the global AATD therapeutics market and insightful review of the key industry drivers, restraints, and challenges. Each trend is independently researched to provide a qualitative analysis of its implications.

Reasons to Buy

  • Develop and design your in-licensing and out-licensing strategies through a review of pipeline products and technologies, and by identifying the companies with the most robust pipeline.
  • Develop business strategies by understanding the trends shaping and driving the global AATD therapeutics market.
  • Drive revenues by understanding the key trends, innovative products and technologies, market segments, and companies likely to impact the global AATD therapeutics market in the future.
  • Formulate effective sales and marketing strategies by understanding the competitive landscape and by analyzing the performance of various competitors.
  • Identify emerging players with potentially strong product portfolios and create effective counterstrategies to gain a competitive advantage.
  • Organize your sales and marketing efforts by identifying the market categories and segments that present maximum opportunities for consolidations, investments, and strategic partnerships.

Kamada Pharmaceuticals
pH Pharma Co
Mereo Biopharma
Novo Nordisk
Arrowhead Pharmaceuticals

Table of Contents

Table of Contents (PowerPoint Deck)

1 Executive Summary

1.1 Executive Summary

2 Disease Overview

2.1 Overview of Alpha-1 Antitrypsin Deficiency (AATD)

2.2 AATD SWOT Analysis

2.3 Classification of AATD

3 Epidemiology

3.1 Diagnosed Prevalent Cases of Alpha-1 Antitrypsin Deficiency, Men and Women, 2021–31

3.2 Sources and Methodology

4 Current Treatment Options

4.1 Treatment Paradigm

4.2 Marketed Products

4.3 Product Profiles: Alpha-1 Proteinase Inhibitors (Glassia, Prolastin-C, Zemaira/Respreeza, Aralast NP)

5 Unmet Needs and Opportunities

5.1 Unmet Needs in AATD

5.2 Earlier AATD Disease Diagnosis

5.3 AATD Treatments with Improved Efficacy

5.4 Reduced Cost for AATD Treatments

5.5 Treatment Dosage for AATD Patients

5.6 Patient Compliance

6 Pipeline Assessment

6.1 AATD Pipeline Overview

6.2 Leading Pipeline Agents

6.3 Product Profiles: Alpha-1 proteinase inhibitor (human) second generation, PHP-303, Alvelestat, Belcesiran, ARO-AAT (Fazirsiran Sodium)

6.4 Pipeline Products – Review Designations

6.5 AATD: Clinical Trials (Phase II/III) Overview

7 R&D Strategies

7.1 Trends in Clinical Trial Design in AATD

7.2 Trends in Deal-Making in AATD

8 Market Outlook

8.1 AATD Sales Forecast

8.2 AATD Market Forecast

8.3 Market Drivers and Barriers

9 Appendix

9.1 Primary Research: KOL Information

9.2 About the Authors

10 Contact Us

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