Summary

Overview
Global type 1 alpha-1 antitrypsin deficiency treatment market reached US$ xx billion in 2022 and is expected to reach US$ xx billion by 2030, growing with a CAGR of xx% during the forecast period 2023-2030.
The global type 1 alpha-1 antitrypsin deficiency treatment market has witnessed significant growth and transformations over the years, with various factors influencing its dynamics. The chance of developing lung and additional illnesses increases if the person have an AAT deficiency. AAT is a protein produced in the liver that aids in lung defense. The lungs are more vulnerable to injury from smoking, air pollution, and environmental dust if the body fails to produce enough AAT.
Government investments and research, particularly in developing economies, will continue to drive utilization of advanced products or therapies and boost the global type 1 alpha-1 antitrypsin deficiency treatment market. The respective innovative products includes the use of novel treatments such as augmentation therapy for better management.
Market Dynamics: Drivers & Restraints
Growing initiatives and regulatory approvals for treatment
Developed economies have been witnessing rapid growth in betterment of their healthcare sectors, driven by high investments, income levels, and infrastructure development. Several countries have experienced substantial demand for type 1 alpha-1 antitrypsin deficiency treatment owing to the positive outcomes in research. Various regulatory approvals for product launch among different countries will be a crucial factor driving the growth of the market.
On November 24, 2021, Alpha-1 antitrypsin deficiency (AATD) patients in Spain now have a new home-infusion treatment option thanks to Grifols, one of the top manufacturers of plasma-derived medications in the world with more than 110 years of experience improving people's health and quality of life. Within Grifols' AlfaCare initiative, this new service seeks to improve treatment accessibility and the convenience of its administration. Through the use of qualified specialists, it provides AATD patients with an integrated, tailored program that includes instruction, emotional support, and help.
On May 16, 2023, according to Kamada Ltd., an industrial phase global biopharmaceutical company with a collection of marketed medications indicated for rare and serious illnesses, Glassia, an Alpha-1 Proteinase Inhibitor (Human), received approval for advertising in Switzerland for ongoing augmentation in addition to maintenance treatment in adulthood having clinically diagnosed emphysema resulting from severe inherited Alpha-1 antitrypsin deficiency (AA).
Furthermore, the rising utilization of novel therapies or drugs for the management of alpha-1 antitrypsin deficiency. Rising awareness among people and increasing research for innovative product development will be a major factor driving the growth of the type 1 alpha-1 antitrypsin deficiency treatments market.
Side effects associated with the augmentation therapy
Alphas have documented a number of adverse effects of augmentation therapy, however the majority never suffer any substantial problems. Fatigue or flu-like symptoms are the most typical side effect, and they might last up to 24 hours after an infusion. These symptoms can frequently be lessened or even eliminated by slowing down the infusion rate.
Some of the indications that some Alphas experience that resemble minor allergic responses include itching, hives, rash, dyspnea, heaviness in the chest, and wheezing. Many individuals can continue receiving augmentation therapy if they take an allergy medication like Benadryl before their infusions. Very seldom do side effects become so severe that an Alpha has to entirely stop taking their augmentation prescription.
Segment Analysis
The global type 1 alpha-1 antitrypsin deficiency treatment market is segmented based on treatment, route of administration, end user and region.
Augmentation Therapy segment accounted for approximately 45% of market share
Patients with significant type 1 alpha-1 antitrypsin (AAT) insufficiency who have emphysema may benefit from augmentation therapy, sometimes referred to as replacement therapy. The level of alpha-1 antitrypsin (AAT) in the lungs of patients having AAT deficiency is increased using alpha-1 antitrypsin protein obtained from the blood of healthy donors. AAT guards against lung damage caused by the immune system in healthy people. AAT augmentation therapy helps individuals with AAT deficiency increase the quantity of AAT in their lungs, preventing additional lung damage.
On July 13, 2023, a phase 3 clinical research to determine whether weekly administration of two different Grifols dosing regimens results in a more gradual loss of lung tissue in people with emphysema who have alpha-1-antitrypsin (AAT) deficiency (alpha-1). According to Grifols, one of the major manufacturers of medicines produced from plasma, Prolastin-C has enrolled all 339 of its target patients.
According to SPARTA, the biggest randomized, placebo-controlled, double-blind study on AAT augmentation treatment to date, Prolastin-C has the potential to significantly slow the progression of emphysema in alpha-1 patients through raising AAT protein levels through weekly administration of two effective dose levels in comparison to placebo.
Geographical Penetration
North America accounted for around 40% of market share in 2022
Due to the rising need for type 1 alpha-1 antitrypsin deficiency treatment in healthcare, manufacturers in North America have chances of increasing their operations. There are many producers and suppliers in North America and owing to the quick economic growth of the region, industrial production has expanded, driving the demand for type 1 alpha-1 antitrypsin deficiency treatment.
Increasing expenditure on healthcare and rising research studies, advancement of technologies and different kinds of therapies for management, and increase in pharmaceutical or biotechnology business establishment across the region are also contributing to the growth of type 1 alpha-1 antitrypsin deficiency treatment market share of this region. The market in this area is growing as people become more aware of various novel innovative therapies such as Prolastin-C. The aforementioned elements further attest to North America's hegemonic position in the world.
North America continues to be a key player in the global type 1 alpha-1 antitrypsin deficiency treatment market, with United States leading the way. Government initiatives promoting infrastructure development and investment, and a focus on rising advancements have fueled the demand for type 1 alpha-1 antitrypsin deficiency treatment in the U.S. United States have been proactive in executing several initiatives or researches, stimulating type 1 alpha-1 antitrypsin deficiency treatment demand.
COVID-19 Impact Analysis
The outbreak of the COVID-19 pandemic in late 2019 created unprecedented challenges for industries worldwide, including the global type 1 alpha-1 antitrypsin deficiency treatment market. As countries grappled with lockdowns, supply chain disruptions and reduced economic activity, the pharmaceutical sector, with a significant consumer of various device designs, was significantly impacted. The widespread lockdowns and restrictions imposed by the pandemic, which began in early 2020, had an effect on a number of initiatives all around the world.
Major medical device industries came to a standstill and shifted their attention towards the management of COVID-19, leading to a slump in demand for type 1 alpha-1 antitrypsin deficiency treatment. Now several research studies have been initiated and companies have again started trial for their products safety and efficacy. Overall, the impact of the pandemic on the global type 1 alpha-1 antitrypsin deficiency treatment market is expected to be relatively moderate, with the market continuing to grow steadily due to the ongoing need and research for novel therapies, like augmenting therapies and other programs for management of type-1 alpha-1 antitrypsin deficiency.
By Treatment
• Augmentation Therapy
o Prolastin-C
o Aralast
o Zemaira
o Glassia
• Medication
o Bronchodilators
 Ipratropium Bromide
 Theophylline
o Antibiotics
 Doxycycline
 Dapsone
• Oxygen Therapy
• Smoking Cessation Therapy
• Others
By Route of Administration
• Inhaled
• Intravenous
• Others
By End User
• Hospitals
• Ambulatory Surgical Centers
• Others
By Region
• North America
o U.S.
o Canada
o Mexico
• Europe
o Germany
o UK
o France
o Italy
o Spain
o Rest of Europe
• South America
o Brazil
o Argentina
o Rest of South America
• Asia-Pacific
o China
o India
o Japan
o Australia
o Rest of Asia-Pacific
• Middle East and Africa
Key Developments
• On April 4, 2023, in Takeda's Phase 3 REDWOOD clinical investigation of fazirsiran (TAK-999/ARO-AAT) as the management of alpha-1 antitrypsin deficiency related liver disease (AATD-LD), Arrowhead Pharmaceuticals Inc. stated that the first patient has been dosed. Takeda will provide Arrowhead a $40 million breakthrough payment as soon as this crucial trial begins. In July 2021, the U.S. Food and Drug Administration awarded Fazirsiran Breakthrough Therapeutic Designation for the management of AATD-LD.
• On October 17, 2022, Alvelestat (MPH-966), an experimental oral neutrophil elastase inhibitor, has been given U.S. Food and Drug Administration (FDA) Fast Track designation by Mereo BioPharma Group plc, a clinical-stage biopharmaceutical firm with a focus on oncology and rare disorders. Around the end of the year, Mereo plans to meet with the FDA at the End-of-Phase 2 meeting to explore the planning of a registration-enabling trial for alvelestat as a cure for AATD-associated lung illness, including the potential for an expedited approval pathway.
Competitive Landscape
The major global players in the market include Kamada Pharmaceuticals, Takeda Pharmaceutical Company Limited, CSL, Vertex Pharmaceuticals Incorporated, Grifols, Arrowhead Pharmaceuticals, Inc., Dicerna Pharmaceuticals, Inc, Inhibrx, Inc., Wave Life Sciences and Mereo Biopharma Group PLC among others.
Why Purchase the Report?
• To visualize the global type 1 alpha-1 antitrypsin deficiency treatment market segmentation based on treatment, route of administration, end user and region as well as understand key commercial assets and players.
• Identify commercial opportunities by analyzing trends and co-development.
• Excel data sheet with numerous data points of type 1 alpha-1 antitrypsin deficiency treatment market-level with all segments.
• PDF report consists of a comprehensive analysis after exhaustive qualitative interviews and an in-depth study.
• Product mapping available as excel consisting of key products of all the major players.
The global type 1 alpha-1 antitrypsin deficiency treatment market report would provide approximately 61 tables, 58 figures and 186 Pages.
Target Audience 2023
• Manufacturers/ Buyers
• Industry Investors/Investment Bankers
• Research Professionals
• Emerging Companies

ページTOPに戻る

Table of Contents

1. Methodology and Scope
1.1. Research Methodology
1.2. Research Objective and Scope of the Report
2. Definition and Overview
3. Executive Summary
3.1. Snippet by Treatment
3.2. Snippet by Route of Administration
3.3. Snippet by End User
3.4. Snippet by Region
4. Dynamics
4.1. Impacting Factors
4.1.1. Drivers
4.1.1.1. Growing initiatives and regulatory approvals for treatment
4.1.1.2. Rising technological advancements and developments
4.1.2. Restraints
4.1.2.1. Side effects associated with the augmentation therapy
4.1.3. Opportunity
4.1.4. Impact Analysis
5. Industry Analysis
5.1. Porter's Five Force Analysis
5.2. Supply Chain Analysis
5.3. Pricing Analysis
5.4. Regulatory Analysis
5.5. Pricing Analysis
5.6. SWOT Analysis
5.7. Patent Analysis
5.8. Russia-Ukraine War Impact Analysis
5.9. DMI Opinion
6. COVID-19 Analysis
6.1. Analysis of COVID-19
6.1.1. Scenario Before COVID
6.1.2. Scenario During COVID
6.1.3. Scenario Post COVID
6.2. Pricing Dynamics Amid COVID-19
6.3. Demand-Supply Spectrum
6.4. Government Initiatives Related to the Market During Pandemic
6.5. Manufacturers Strategic Initiatives
6.6. Conclusion
7. By Treatment
7.1. Introduction
7.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
7.1.2. Market Attractiveness Index, By Treatment
7.2. Augmentation Therapy*
7.2.1. Introduction
7.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
7.2.3. Prolastin-C
7.2.4. Aralast
7.2.5. Zemaira
7.2.6. Glassia
7.3. Medication
7.3.1. Bronchodilators
7.3.1.1. Ipratropium Bromide
7.3.1.2. Theophylline
7.3.2. Antibiotics
7.3.2.1. Doxycycline
7.3.2.2. Dapsone
7.4. Oxygen Therapy
7.5. Smoking Cessation Therapy
7.6. Others
8. By Route of Administration
8.1. Introduction
8.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
8.1.2. Market Attractiveness Index, By Route of Administration
8.2. Inhaled *
8.2.1. Introduction
8.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
8.3. Intravenous
8.4. Others
9. By End User
9.1. Introduction
9.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
9.1.2. Market Attractiveness Index, By End User
9.2. Hospitals*
9.2.1. Introduction
9.2.2. Market Size Analysis and Y-o-Y Growth Analysis (%)
9.3. Ambulatory Surgical Centers
9.4. Others
10. By Region
10.1. Introduction
10.1.1. Market Size Analysis and Y-o-Y Growth Analysis (%), By Region
10.1.2. Market Attractiveness Index, By Region
10.2. North America
10.2.1. Introduction
10.2.2. Key Region-Specific Dynamics
10.2.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
10.2.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.2.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.2.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.2.6.1. U.S.
10.2.6.2. Canada
10.2.6.3. Mexico
10.3. Europe
10.3.1. Introduction
10.3.2. Key Region-Specific Dynamics
10.3.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
10.3.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.3.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.3.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.3.6.1. Germany
10.3.6.2. UK
10.3.6.3. France
10.3.6.4. Italy
10.3.6.5. Spain
10.3.6.6. Rest of Europe
10.4. South America
10.4.1. Introduction
10.4.2. Key Region-Specific Dynamics
10.4.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
10.4.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.4.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.4.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.4.6.1. Brazil
10.4.6.2. Argentina
10.4.6.3. Rest of South America
10.5. Asia-Pacific
10.5.1. Introduction
10.5.2. Key Region-Specific Dynamics
10.5.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
10.5.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.5.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
10.5.6. Market Size Analysis and Y-o-Y Growth Analysis (%), By Country
10.5.6.1. China
10.5.6.2. India
10.5.6.3. Japan
10.5.6.4. Australia
10.5.6.5. Rest of Asia-Pacific
10.6. Middle East and Africa
10.6.1. Introduction
10.6.2. Key Region-Specific Dynamics
10.6.3. Market Size Analysis and Y-o-Y Growth Analysis (%), By Treatment
10.6.4. Market Size Analysis and Y-o-Y Growth Analysis (%), By Route of Administration
10.6.5. Market Size Analysis and Y-o-Y Growth Analysis (%), By End User
11. Competitive Landscape
11.1. Competitive Scenario
11.2. Market Positioning/Share Analysis
11.3. Mergers and Acquisitions Analysis
12. Company Profiles
12.1. Kamada Pharmaceuticals*
12.1.1. Company Overview
12.1.2. Product Portfolio and Description
12.1.3. Financial Overview
12.1.4. Key Developments
12.2. Takeda Pharmaceutical Company Limited
12.3. CSL
12.4. Vertex Pharmaceuticals Incorporated
12.5. Grifols
12.6. Arrowhead Pharmaceuticals, Inc.
12.7. Dicerna Pharmaceuticals, Inc.
12.8. Inhibrx, Inc.
12.9. Wave Life Sciences
12.10. Mereo Biopharma Group PLC
LIST NOT EXHAUSTIVE
13. Appendix
13.1. About Us and Services
13.2. Contact Us