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Alpha-1 Antitrypsin Deficiency Treatment Market 2034: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, And Companies By DelveInsight
"Alpha-1 Antitrypsin Deficiency Treatment Market"
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
Discover which therapies are expected to grab the major AATD market share @ Alpha-1 Antitrypsin Deficiency Market Report
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:
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.
To know more about AATD treatment guidelines, visit @ Alpha-1 Antitrypsin Deficiency Prevalence
Alpha-1 Antitrypsin Deficiency Pipeline Therapies and Key Companies
Learn more about the FDA-approved drugs for AATD @ Drugs for Alpha-1 Antitrypsin Deficiency Treatment
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.
Request for sample report @ Alpha-1 Antitrypsin Treatment Market
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.
Media ContactCompany Name:DelveInsight Business Research LLPContact Person: Ankit NigamEmail:Send EmailPhone: +14699457679Address:304 S. Jones Blvd #2432 City: AlbanyState: New YorkCountry: United StatesWebsite:https://www.Delveinsight.Com/consulting/market-assessment-services
Press Release Distributed by ABNewswire.ComTo view the original version on ABNewswire visit: Alpha-1 Antitrypsin Deficiency Treatment Market 2034: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, and Companies by DelveInsight
The Alpha-1 Foundation Commemorates Its 30th Anniversary
CORAL GABLES, Fla., Feb. 3, 2025 /PRNewswire/ -- The Alpha-1 Foundation (A1F) is proud to commemorate its 30th anniversary of supporting patients with Alpha-1 Antitrypsin Deficiency (Alpha-1) while searching for a cure. On January 31, 1995, A1F was incorporated as a not-for-profit organization by its three Alpha-1 patient co-founders, John W. Walsh, Sandy Lindsey, and Susan Stanley, to find a cure for Alpha-1. This milestone celebrates and honors the legacy of those who have impacted the Alpha-1 community over three decades, especially Alphas and their families who A1F serves.
Alpha-1 Antitrypsin Deficiency (Alpha-1) is a rare genetic (inherited) condition— passed from parents to their children through their genes. Alpha-1 may result in serious lung disease in adults and/or liver disease at any age.
A1F was created because the National Heart, Lung and Blood Institute (NHLBI) of the National Institutes of Health (NIH) was nearing the conclusion of its seven-year study on Alpha-1 and leading Alpha-1 advocate John W. Walsh recognized that no further resources would be put towards Alpha-1 research. It became clear future research and the hope for a cure would depend on Alpha-1 patients taking the lead in building the resources and infrastructure needed to drive progress and advance the field to find a cure for this debilitating condition.
"A1F has spent 30 years working for the Alpha-1 community, to improve the lives of Alphas and to find a cure for Alpha-1. It has invested over $100 million in Alpha-1 research at 130 institutions around the world, spurring countless innovations and impacts. Together, we are creating a legacy of hope, possibility, and progress; one that will resonate for generations to come," said Scott Santarella, President & CEO of the Alpha-1 Foundation.
For three decades, with the insight of the Alpha-1 community, A1F has designed and launched innovative, patient-focused programs and services supporting Alphas along their journey; including investing in a solid infrastructure to promote pioneering scientific research, and the establishment of collaborative partnerships to advance the development of novel therapies. The impact of A1F's work over 30 years has exponentially improved the quality of life for those affected by Alpha-1.
"As an Alpha-1 patient, it is truly an exciting time celebrating the milestones and achievements of this outstanding organization and recognizing the last 30 years. We are fortunate to have the best research and therapeutic development landscape ever seen and A1F is well-positioned to accelerate that even further," said Jon Hagstrom, Chair of the A1F Board of Directors.
Story continues
A1F has been committed to finding a cure for Alpha-1 while improving the lives of those affected worldwide since inception. In pursuit of this mission, A1F has devoted substantial resources to addressing the social and scientific challenges of Alpha-1. The founders set out to establish an innovative scientific infrastructure that would support a powerful grant program aimed at investing in evidenced-based research to find a cure for Alpha-1.
A unique and innovative aspect of A1F is that it started and has continued to be a patient-driven and directed organization; most of the A1F Board of Directors are either diagnosed with Alpha-1 or have a family member diagnosed with Alpha-1.
With the help of the Alpha-1 community, A1F has designed and launched patient-centric educational programs and developed a solid infrastructure to promote research and the development of new therapies for improving the quality of life for those diagnosed with Alpha-1. It has fostered collaborations with investigators throughout North America, Europe, the Middle East, and Australia, working closely with the National Institutes of Health (NIH), the U.S. Food and Drug Administration (FDA), the pharmaceutical industry, and Alpha-1 patients to expedite development of improved treatments.
A1F's achievements in its first 30 years can be attributed to the ability to bring all constituents and stakeholders to the table with a patient-focused approach to accelerate novel approaches to this condition.
Recognized for good operating practices, accountability, and responsible governance, A1F has received the National Health Council's Full Compliance Certification and the Charity Seal from the Better Business Bureau's Wise Giving Alliance. These are the highest accreditations attainable in the not-for-profit and voluntary health sectors. A1F has also received an Exceptional 4-Star rating by Charity Navigator, the highest rating given by that organization. GuideStar has also awarded the Platinum Seal of Transparency, their highest level of recognition, to A1F.
About the Alpha-1 Foundation
The Alpha-1 Foundation, founded in 1995, is committed to finding a cure for Alpha-1 Antitrypsin Deficiency (Alpha-1) and to improving the lives of people affected by the condition worldwide. A1F has invested over $100 million to support Alpha-1 research and programs at 130 institutions in North America, Europe, the Middle East and Australia.
For more information, visit www.Alpha1.Org
Contact: Jeanne Kushner
Senior Director of Communications & Policy
877-228-7321
jkushner@alpha1.Org
CisionView original content to download multimedia:https://www.Prnewswire.Com/news-releases/the-alpha-1-foundation-commemorates-its-30th-anniversary-302366504.Html
SOURCE Alpha-1 Foundation
COPD Causes And Symptoms
Narrator
These men and women are fighting a common enemy: COPD – Chronic Obstructive Pulmonary Disease. Their lungs don't work normally…every breath taken can be a struggle. What do they believe to be the cause?Bill Durham COPD Patient
Too many cigarettes. Smoked too long, about 40 years.Loretta Freeman COPD Patient
I started smoking when I was 17.Joseph Burlas COPD Patient
My wife says it's from 40 years of smoking.Narrator
That's true for about 85-percent of COPD cases.Gerald Staton, MD
We do see occasional patients that have various industrial exposures that can seem to contribute to that, but often they smoke cigarettes too and so it's probably a combination of factors.Narrator
Consider what happened to 59-year-old Gregory Wright. As a firefighter he was exposed to burning plastics, asbestos and chemicals. Then he worked at an airport breathing in noxious fumes and gases.Gregory Wright, COPD Patient
The doctor going over my medical history – did you ever smoke? Yes. I smoked for over 30 years.Narrator
Another troublesome combination:Gerald Staton, MD
Asthma seems to be a predisposition that if you are an asthmatic as a child and then smoke cigarettes then you have a fairly significantly higher risk of developing COPD later in life.Narrator
Then there's a particularly lethal mix: a genetic, or hereditary predisposition due to alpha 1 antitrypsin deficiency.Diane Kay, COPD Patient
I was diagnosed with alpha 1 in '99 but I had symptoms for years and being a flight attendant I had a difficult time keeping up with the rest of the crews.Narrator
Although rare, people like Diane Kay have low levels of a so-called lung protector protein that's produced by the liver.Diane Kay, COPD Patient
It took me forever to walk up the driveway from getting the mail. I had to take breaks, and you just think you getting older and at that the time I was a smoker. So you think you know you're older and you smoking and you know. What do you expect?Narrator
She was susceptible to colds, bronchitis and laryngitis and it took her forever to get over these illnesses. The early COPD symptoms are often missed:Gerald Staton, MD
Even people in their 30's and 40's will have the so-called smoker's cough. They think that's normal. They think everybody gets up in the morning and coughs up stuff, that's not normal. That's the beginning of symptoms.Narrator
Shortness of breath tends to develop in their late 40's and 50's. Diane finally had to give up her job:Diane Kay, COPD Patient
They kind of frown on flight attendants walking on an airplane with an oxygen mask (laughs).Narrator
Eventually it became so difficult for Diane to breathe that she ended up needing a lung transplant. Her advice: don't smoke.Diane Kay, COPD Patient
It basically caused me to have a transplant. If I hadn't been a smoker or worked in an environment where there is second hand smoke I probably wouldn't have had a transplant I never would have gotten as sick as I got, as quickly as I did.Narrator
For WebMD, I'm Rhonda Rowland.Popular Posts
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