α1抗トリプシン欠損症増強療法市場：製品タイプ、エンドユーザー、地域別、2026～2032年

α1抗トリプシン欠損症増強療法市場の評価-2026～2032年

α1抗トリプシン欠損症の有病率の増加と、肺疾患のコントロールにおける増強療法の利点に対する意識の高まりは、α1抗トリプシン欠損症増強療法市場の成長を促進する主要な力となっています。Verified Market Researchのアナリストによると、α1抗トリプシン欠損症増強療法市場は、2024年に約15億9,000万米ドルと評価され、予測期間中に26億9,000万米ドルの評価に達すると推定されます。

α1抗トリプシン欠損症患者のアンメット・メディカル・ニーズに対応するための治療オプションの強化に対するニーズの高まりと、継続的な研究開発の取り組みが、α1抗トリプシン欠損症増強療法市場を前進させています。これにより、市場は2024～2032年にかけてCAGR 6.8％で成長します。

α1抗トリプシン欠損症増強療法市場定義/概要

α1抗トリプシン欠損症増強療法は、体内でα1抗トリプシン（AAT）タンパク質が十分に作られず、肺障害や肝障害のリスクが高まる遺伝性の問題に対処する治療法です。この治療法では、ヒト血漿から生成した純粋なAATタンパク質を定期的に静脈注射し、血液中のAAT濃度を高めています。この治療法は、特に肺気腫や慢性閉塞性肺疾患（COPD）のような肺疾患が起こりやすい人の場合、白血球が生成する酵素によるダメージから肺を守ろうとするものです。

さらに、増強療法はα1抗トリプシン欠損症患者の肺疾患の進行を減少させ、増悪の回数を最小限に抑えることが判明しています。また、この欠乏症は肝疾患の面でも有益であることが調査から示唆されています。

市場の成長に影響を与える促進要因は何か？

α1抗トリプシン欠乏症（AATD）は、重度の肺疾患や肝臓疾患を引き起こす遺伝性疾患です。ヘルスケア専門家や一般市民の間でAATDに対する認識が高まっているため、診断の早期化と頻度が高まっています。このことは、遺伝子スクリーニング法の世界の増加と相まって、増強療法の対象となる患者層を拡大し、市場の成長をもたらしています。

洗練されたバイオテクノロジー的アプローチと遺伝子治療の進歩は、AATD増強療法市場の主要な促進要因です。これらの開発は、既存の治療の有効性と安全性を向上させるだけでなく、新たな治療法の発見を容易にします。例えば組換えDNA技術の革新は、より安定した効果的なバージョンのα-1抗トリプシンを提供する可能性を提供し、患者がAATD症状をよりよく管理し、全体的な健康を増進することを可能にします。

さらに、ヘルスケアインフラを改善し、希少医薬品の調査と登録を促進する政府の試みも、AATD増強療法市場を後押ししています。多くの地域では、AATD治療は有利な償還規則や希少疾病研究に対する優遇措置によって支えられており、患者にとってより購入しやすい価格となっています。こうした施策は、患者の経済的負担を劇的に軽減すると同時に、補強療法の普及率を高める可能性を秘めています。

市場が直面する主要課題とは？

α1抗トリプシン欠損症（AATD）に対する補強療法は法外に高価であることが多く、特に医療制度が未発達な地域や保険適用が不十分な国では、その利用が制限されています。高額なのは、α-1抗トリプシンタンパク質の製造方法が困難であることに加え、医薬品がオーファンドラッグ（希少疾病用医薬品）に分類されているためです。この経済的障壁は、患者が必要な治療法にアクセスすることを阻み、市場の成長可能性を制限しています。

さらに、現在のところ、AATD増強療法の選択肢は限られており、その有用性と費用対効果については意見が分かれています。従来型薬剤療法に反応しない患者もおり、肺疾患の進行を止める増強療法の効果も様々です。また、生涯にわたる治療が必要であることは、患者のコンプライアンスや満足度の点で難点です。治療選択肢の数が限られていることに加え、医薬品の有効性がさまざまであることが、市場の拡大と患者の転帰に課題をもたらしています。

目次

第1章 α1抗トリプシン欠損症増強療法の世界市場導入

• 市場概要
• 調査範囲
• 前提条件

第2章 エグゼクティブサマリー

第3章 VERIFIED MARKET RESEARCHの調査手法

• データマイニング
• バリデーション
• 一次資料
• データソース一覧

第4章 α1抗トリプシン欠損症増強療法の世界市場展望

• 概要
• 市場力学
  • 促進要因
  • 抑制要因
  • 機会
• ポーターのファイブフォースモデル
• バリューチェーン分析

第5章 α1抗トリプシン欠損症増強療法の世界市場：製品タイプ別

• 概要
• グラシア
• アララストNP
• プロラスチンC
• ゼマイラ/レスプレザ

第6章 α1抗トリプシン欠損症増強療法の世界市場：エンドユーザー別

• 概要
• 病院
• 専門クリニック
• オンラインストア

第7章 α1抗トリプシン欠損症増強療法の世界市場：地域別

• 概要
• 北米
  • 米国
  • カナダ
  • メキシコ
• 欧州
  • ドイツ
  • 英国
  • フランス
  • その他の欧州
• アジア太平洋
  • 中国
  • 日本
  • インド
  • その他のアジア太平洋
• その他
  • ラテンアメリカ
  • 中東

第8章 α1抗トリプシン欠損症増強療法の世界市場競争情勢

• 概要
• 各社の市場ランキング
• 主要開発戦略

第9章 企業プロファイル

• Takeda Pharmaceutical Company Limited
• CSL Behring
• Paramount Therapeutics
• Vertex Pharmaceuticals Incorporated
• Shire plc
• Shanghai RAAS BIoTechnology Co.
• JCR Pharmaceuticals Co.
• Instituto Butantan
• Lonza Group Ltd
• Samsung BioLogics

第10章 付録

• 関連調査

Alpha-1 Antitrypsin Deficiency Augmentation Therapy Market Valuation - 2026-2032

The growing prevalence of Alpha-1 Antitrypsin deficiency and growing awareness of the benefits of augmentation therapy in controlling lung disorders are key forces fueling the growth of the Alpha-1 Antitrypsin deficiency augmentation therapy market. According to the analyst from Verified Market Research, the Alpha-1 Antitrypsin deficiency augmentation therapy market is estimated to reach a valuation of USD 2.69 Billion over the forecast subjugating around USD 1.59 Billion valued in 2024.

The growing need for enhanced therapy options to address the unmet medical needs of Alpha-1 Antitrypsin deficiency patients, together with continuing research & development initiatives, is propelling the Alpha-1 Antitrypsin deficiency augmentation therapy market forward. It enables the market to grow at a CAGR of 6.8% from 2024 to 2032.

Alpha-1 Antitrypsin Deficiency Augmentation Therapy Market: Definition/Overview

Alpha-1 Antitrypsin Deficiency Augmentation Therapy is a medical treatment that addresses a hereditary problem in which the body does not create enough alpha-1 antitrypsin (AAT) protein, which increases the risk of lung and liver disorders. The therapy entails injecting pure AAT protein generated from human plasma intravenously regularly to boost AAT levels in the blood. The therapy seeks to protect the lungs against damage caused by enzymes generated by white blood cells, especially in people who are prone to lung disorders such as emphysema and chronic obstructive pulmonary disease (COPD) due to the deficit.

Furthermore, augmentation therapy has been found to decrease the progression of lung illness and minimize the number of exacerbations in patients with Alpha-1 Antitrypsin Deficiency. Also, research suggests that this deficiency may have benefits in terms of liver disease

What are the Drivers Influencing the Growth of the Market?

Alpha-1 Antitrypsin Deficiency (AATD) is a hereditary disorder that can cause severe lung and liver illnesses. The growing awareness of AATD among healthcare professionals and the general public is resulting in earlier and more frequent diagnoses. This, combined with the global increase in genetic screening methods, is broadening the patient pool eligible for augmentation therapy, resulting in market growth.

The advancement of sophisticated biotechnological approaches and genetic therapies is a major driver of the AATD augmentation therapy market. These developments not only improve the efficacy and safety of existing medications but also make it easier to discover new treatment choices. Innovations in recombinant DNA technology, for example, offer the potential to provide more stable and effective versions of alpha-1 antitrypsin, allowing patients to better manage their AATD symptoms and enhance their overall health.

Furthermore, government attempts to improve healthcare infrastructure and facilitate the research and registration of orphan pharmaceuticals are also propelling the AATD augmentation therapy market. In many areas, AATD medicines are backed by advantageous reimbursement rules and incentives for rare illness research, making them more affordable to patients. These policies have the potential to dramatically lower patients' financial burdens while also increasing the adoption rate of augmentation therapy.

What are the Main Challenges Faced by the Market?

Augmentation therapy for Alpha-1 Antitrypsin Deficiency (AATD) is frequently prohibitively expensive, limiting their availability, particularly in locations with underdeveloped healthcare systems or in nations where insurance coverage for such medicines is inadequate. The high cost is due in part to the difficult manufacturing methods involved in making the alpha-1 antitrypsin protein, as well as the medicines' classification as orphan pharmaceuticals, which are created for disorders that afflict only a small fraction of the population. This financial barrier stops patients from accessing needed therapies, limiting the market's growth potential.

Furthermore, currently, AATD augmentation therapy options are limited, and there is continuous disagreement over their usefulness and cost-effectiveness. Some individuals do not respond well to conventional medications, and the effectiveness of augmentation therapy in halting the course of lung disease varies. Also, the requirement for lifelong treatment is a difficulty in terms of patient compliance and satisfaction. The limited number of treatment alternatives, as well as the varied efficacy of medicines, pose challenges to market expansion and patient outcomes.

Category-wise Acumens

What Factors Contribute to the Increasing Demand for Prolastin C Products?

According to VMR analyst, the prolastin C segment is expected to dominate the segment within the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. Prolastin-C, one of the first augmentation medicines available for AATD, has a long track record of success and safety. Several studies and real-world evidence have supported its usage in AATD patients to reduce the progression of emphysema. This robust evidence foundation has made it a popular choice among healthcare providers and people, adding to its market dominance.

Prolastin-C, as a market pioneer in AATD augmentation therapy, enjoys strong brand recognition and loyalty from physicians and patients. Years of great experiences and belief in the product's effectiveness and safety are common reasons for loyalty. Many patients and healthcare providers prefer to continue with a tried-and-true therapy rather than transition to newer alternatives with which they are unfamiliar.

Furthermore, the manufacturer of Prolastin-C has created comprehensive patient support initiatives, which have greatly improved its market position. These initiatives may include financial help, instructional materials, and individualized patient support services. Such assistance is critical for individuals suffering from chronic illnesses such as AATD, as it can enhance medication adherence, access to therapy, and overall patient happiness. These programs transform Prolastin-C from a medicine into a holistic treatment solution, which can be a deciding factor for many patients and caregivers when selecting an augmentation therapy.

How does Comprehensive Care Propel the Growth of Hospitals in the Market?

The hospital segment is estimated to hold the largest share of the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. Hospitals provide a wide range of medical treatments, including integrated care for patients with complicated illnesses such as AATD. They house interdisciplinary teams made up of pulmonologists, hepatologists, genetic counselors, and other specialists who can work together to provide tailored therapy options. Hospitals' capacity to provide comprehensive care from diagnosis to treatment and follow-up in one location makes them the preferred choice for many patients, driving market share.

Hospitals are often outfitted with advanced diagnostic equipment and treatment facilities required for the proper care of AATD. This includes specialist equipment for genetic testing, lung function tests, and access to the most recent augmentation medicines. The availability of cutting-edge healthcare technology, as well as the ability to perform sophisticated treatments such as intravenous infusion of alpha-1 antitrypsin, contribute to their market dominance.

Furthermore, hospitals have more strong systems in place to handle insurance and payment policies, making it easier for patients to receive covered services. Because of the intricacy and expensive cost of AATD augmentation therapy, many patients rely heavily on insurance. Hospitals' ability to properly manage these financial issues can remove considerable hurdles to treatment, increasing patient appeal and, as a result, market share.

Country/Region-wise Acumens

How Does High Healthcare Infrastructure Drive the Market in North America?

The North American region is estimated to dominate the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. North America, particularly the United States, has a sophisticated healthcare infrastructure that provides extensive access to cutting-edge medical technologies, facilities, and specialized healthcare experts. This region has one of the highest healthcare expenditures per capita internationally, allowing for major investment in uncommon diseases such as AATD. The availability of specialized treatment centers, as well as an emphasis on early detection and management of genetic diseases, are critical for effective AATD treatment, leading to the region's market domination.

Furthermore, North America is home to some of the world's leading pharmaceutical and biotechnology businesses, which are pioneering research and development (R&D) in uncommon diseases such as AATD. The region's strong emphasis on innovation, combined with large R&D investments and a favorable regulatory environment for drug research and approval, has resulted in the introduction of several augmentation medicines. This ecosystem not only speeds up the availability of new medications but also ensures a consistent pipeline of innovative therapies for AATD.

What Factor Contributes to the Growth of the Market in Asia Pacific?

Asia Pacific is estimated to exhibit the highest growth within the Alpha-1 Antitrypsin deficiency augmentation therapy market during the forecast period. In Asia-Pacific, healthcare professionals and the general public are becoming more aware of rare disorders, such as AATD. Patient advocacy groups, healthcare providers, and government health departments have all worked to educate the public and medical community, contributing to the growth in awareness. Increased awareness leads to higher screening and diagnosis rates, which expands the patient population eligible for augmentation therapy. Early diagnosis is critical for efficiently managing AATD, which drives the demand for augmentation therapy.

Furthermore, many Asian-Pacific countries are experiencing rapid economic expansion, which is driving up healthcare spending at both the government and individual levels. As disposable incomes rise and health insurance coverage expands, more people will be able to afford modern treatments, including expensive therapy for illnesses such as AATD. Also, regional governments are increasingly realizing the necessity of tackling rare diseases, which has resulted in increased funding for healthcare services as well as support for orphan medication research and approval.

Competitive Landscape

The competitive landscape of the Alpha-1 Antitrypsin deficiency augmentation therapy market is marked by ongoing research and development initiatives aimed at improving therapeutic efficacy and accessibility. Also, the introduction of biosimilar goods, as well as the prospect of generic competition, offer market participants both difficulties and opportunities.

Some of the prominent players operating in the Alpha-1 Antitrypsin deficiency augmentation therapy market include:

Takeda Pharmaceutical Company Limited

CSL Behring

Paramount Therapeutics

Vertex Pharmaceuticals Incorporated

Shire plc

Chiesi Farmaceutici SpA

Shanghai RAAS Biotechnology Co.

JCR Pharmaceuticals Co.

Instituto Butantan

Lonza Group Ltd

Samsung BioLogics

Boehringer Ingelheim

Latest Developments:

In February 2024, Grifols conducted Cohort 1 in a clinical investigation of Alpha-1 15, a first-in-human subcutaneous dosage alternative for AATD, to provide patients with a more comfortable home administration option. This development has substantial implications for patient convenience and autonomy.

In November 2024, Intellia Therapeutics intended to focus on NTLA-3001, a CRISPR-mediated in vivo gene insertion candidate for AATD-associated lung illness. This novel technique seeks to permanently restore functional A1AT protein expression with a single dose, potentially removing the need for weekly intravenous infusions or lung transplants in extreme cases. A Clinical Trial Application (CTA) submission for a Phase 1 study of NTLA-3001 is anticipated for the first quarter of 2024.

In June 2022, Takeda and Arrowhead Pharmaceuticals announced the findings of a Phase 2 study of Fazirsiran in AATD patients in the New England Journal of Medicine. The study found significant decreases in liver globule burden and improvements in indicators of liver damage. This collaboration seeks to co-develop and maybe co-commercialize Fazirsiran, which offers a promising novel therapy for AATD.

TABLE OF CONTENTS

1 INTRODUCTION OF GLOBAL ALPHA1 ANTITRYPSIN DEFICIENCY AUGMENTATION THERAPY MARKET

• 1.1 Overview of the Market
• 1.2 Scope of Report
• 1.3 Assumptions

2 EXECUTIVE SUMMARY

3 RESEARCH METHODOLOGY OF VERIFIED MARKET RESEARCH

• 3.1 Data Mining
• 3.2 Validation
• 3.3 Primary Interviews
• 3.4 List of Data Sources

4 GLOBAL ALPHA1 ANTITRYPSIN DEFICIENCY AUGMENTATION THERAPY MARKET OUTLOOK

• 4.1 Overview
• 4.2 Market Dynamics
  • 4.2.1 Drivers
  • 4.2.2 Restraints
  • 4.2.3 Opportunities
• 4.3 Porters Five Force Model
• 4.4 Value Chain Analysis

5 GLOBAL ALPHA1 ANTITRYPSIN DEFICIENCY AUGMENTATION THERAPY MARKET, BY PRODUCT TYPE

• 5.1 Overview
• 5.2 Glassia
• 5.3 Aralast NP
• 5.4 Prolastin C
• 5.5 Zemaira/Respreeza

6 GLOBAL ALPHA1 ANTITRYPSIN DEFICIENCY AUGMENTATION THERAPY MARKET, BY END-USER

• 6.1 Overview
• 6.2 Hospitals
• 6.3 Specialty Clinics
• 6.4 Online Stores

7 GLOBAL ALPHA1 ANTITRYPSIN DEFICIENCY AUGMENTATION THERAPY MARKET, BY GEOGRAPHY

• 7.1 Overview
• 7.2 North America
  • 7.2.1 U.S.
  • 7.2.2 Canada
  • 7.2.3 Mexico
• 7.3 Europe
  • 7.3.1 Germany
  • 7.3.2 U.K.
  • 7.3.3 France
  • 7.3.4 Rest of Europe
• 7.4 Asia Pacific
  • 7.4.1 China
  • 7.4.2 Japan
  • 7.4.3 India
  • 7.4.4 Rest of Asia Pacific
• 7.5 Rest of the World
  • 7.5.1 Latin America
  • 7.5.2 Middle East

8 GLOBAL ALPHA1 ANTITRYPSIN DEFICIENCY AUGMENTATION THERAPY MARKET COMPETITIVE LANDSCAPE

• 8.1 Overview
• 8.2 Company Market Ranking
• 8.3 Key Development Strategies

9 COMPANY PROFILES

• 9.1 Takeda Pharmaceutical Company Limited
  • 9.1.1 Overview
  • 9.1.2 Financial Performance
  • 9.1.3 Product Outlook
  • 9.1.4 Key Developments
• 9.2 CSL Behring
  • 9.2.1 Overview
  • 9.2.2 Financial Performance
  • 9.2.3 Product Outlook
  • 9.2.4 Key Developments
• 9.3 Paramount Therapeutics
  • 9.3.1 Overview
  • 9.3.2 Financial Performance
  • 9.3.3 Product Outlook
  • 9.3.4 Key Developments
• 9.4 Vertex Pharmaceuticals Incorporated
  • 9.4.1 Overview
  • 9.4.2 Financial Performance
  • 9.4.3 Product Outlook
  • 9.4.4 Key Developments
• 9.5 Shire plc
  • 9.5.1 Overview
  • 9.5.2 Financial Performance
  • 9.5.3 Product Outlook
  • 9.5.4 Key Developments
• 9.6 Shanghai RAAS Biotechnology Co.
  • 9.6.1 Overview
  • 9.6.2 Financial Performance
  • 9.6.3 Product Outlook
  • 9.6.4 Key Developments
• 9.7 JCR Pharmaceuticals Co.
  • 9.7.1 Overview
  • 9.7.2 Financial Performance
  • 9.7.3 Product Outlook
  • 9.7.4 Key Developments
• 9.8 Instituto Butantan
  • 9.8.1 Overview
  • 9.8.2 Financial Performance
  • 9.8.3 Product Outlook
  • 9.8.4 Key Developments
• 9.9 Lonza Group Ltd
  • 9.9.1 Overview
  • 9.9.2 Financial Performance
  • 9.9.3 Product Outlook
  • 9.9.4 Key Developments
• 9.10 Samsung BioLogics
  • 9.10.1 Overview
  • 9.10.2 Financial Performance
  • 9.10.3 Product Outlook
  • 9.10.4 Key Developments

10 Appendix

• 10.1 Related Research