Alpha-1 Antitrypsin Deficiency Market Insight, Epidemiology and Market Forecast - 2032

Key Highlights

• The prevalence of alpha-1 antitrypsin deficiency has been increasing, but due to underreporting and underdiagnoses, it is difficult to gather accurate epidemiological data. Many people with alpha-1 antitrypsin deficiency go undetected or receive incorrect diagnoses for other respiratory diseases such as COPD or asthma.
• The epidemiological scenario is further complicated by genetic variability and the high illness occurrence in particular communities. As a result, the disorder's actual prevalence is underestimated. However, the diagnosis can improve in the future due to increased disease understanding and further advancements in diagnostic technologies and biomarkers development.
• Ongoing research and an increase in disease understanding have led to the identification of therapies with effective and convenient routes of administration, with the potential to improve patient's QoL. Approval of augmentation therapies, continuous innovations, and improvement in formulations have enhanced the treatment scenario for alpha-1 antitrypsin deficiency. Several augmentation therapies are approved in the US market for alpha-1 antitrypsin deficiency-associated lung diseases; however, no therapies have been yet approved for alpha-1 antitrypsin deficiency-associated liver diseases. Currently, four augmentation therapies, ARALAST-NP, GLASSIA, PROLASTIN-C, and ZEMAIRA are available in the US market.
• The prevalence of alpha-1 antitrypsin deficiency in Japan is estimated to be low compared to the US and Europe, resulting in fewer patients. The Japanese treatment landscape has only one approved therapy by PMDA, Grifols' LYNSPAD (PROLASTIN-C), for treating alpha-1 antitrypsin deficiency.
• Alpha-1 antitrypsin deficiency is associated with poor health-related quality of life, increased healthcare resource utilization, reduced productivity, and substantial economic burden, which increases with disease severity.
• In 2022, the US had the largest market size of alpha-1 antitrypsin deficiency among the 7MM countries, accounting for approximately USD 931.32 million, expected to increase further by 2032.

This “Alpha-1 Antitrypsin Deficiency (AATD) - Market Insights, Epidemiology, and Market Forecast - 2032” report delivers an in-depth understanding of alpha-1 antitrypsin deficiency, historical and forecasted epidemiology, as well as the alpha-1 antitrypsin deficiency market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

The alpha-1 antitrypsin deficiency market report provides current treatment practices, emerging drugs, market share of individual therapies, and current and forecasted 7MM alpha-1 antitrypsin deficiency market size from 2019 to 2032. The report also covers alpha-1 antitrypsin deficiency treatment practices/algorithms and unmet medical needs to curate the best opportunities and assess the market's potential.

Geography Covered

• The United States
• EU4 (Germany, France, Italy, and Spain) and the United Kingdom
• Japan

Study Period: 2019-2032

Alpha-1 Antitrypsin Deficiency Understanding and Treatment Algorithm

Alpha-1 Antitrypsin Deficiency Overview

Alpha-1 antitrypsin deficiency is an inherited autosomal codominant genetic disorder characterized by a deficiency of the alpha-1 antitrypsin protein (AAT) that is essential for protecting the lungs and liver from damage caused by enzymes, and its deficiency can lead to serious respiratory and liver-related complications. It is primarily caused by mutations in the SERPINA1 gene that provides instructions for making AAT protein, which protects the body from a powerful enzyme called neutrophil elastase. The most common allele of the SERPINA1 gene, called M, produces normal levels of alpha-1 antitrypsin and is present in 85-90% of individuals. Other versions of the SERPINA1 gene lead to reduced levels of AAT. The most prevalent deficiency alleles are S and Z. For example, the S allele produces moderately low levels of this protein, and the Z allele produces very little AAT. Individuals with two copies of the Z allele (ZZ) in each cell will likely have alpha-1 antitrypsin deficiency and are at high risk of developing lung disease and liver disease associated with alpha-1 antitrypsin deficiency. The Z allele is present in almost 90% of severe alpha-1 antitrypsin deficiency cases.

Alpha-1 Antitrypsin Deficiency Diagnosis

Diagnosing alpha-1 antitrypsin deficiency involves a combination of clinical evaluation, laboratory tests, and genetic testing. It typically involves blood tests measuring AAT levels, liver function tests, liver biopsy pulmonary function tests, imaging studies (chest X-rays or CT scans), and genetic testing identifying specific mutations.

Alpha-1 antitrypsin deficiency is often missed or delayed diagnosed as COPD, emphysema, chronic bronchitis, or AAT-associated liver disease as they share common respiratory and hepatitis symptoms. Thus, differential diagnosis may identify specific markers in the blood or other biological samples associated with alpha-1 antitrypsin deficiency. Furthermore, an early diagnosis of alpha-1 antitrypsin deficiency is essential for preventing disease progression, timely intervention, and optimizing treatment management.

Further details related to country-based variations are provided in the report.

Alpha-1 Antitrypsin Deficiency treatment

Current treatment for alpha-1 antitrypsin deficiency includes both medicinal and surgical therapies. Both of these categories provide symptomatic relief to the subjects based on the level and severity of their conditions. The primary purpose of treatment is to improve the manifestation of liver and lung disease developed due to alpha-1 antitrypsin deficiency. Additionally, to manage alpha-1 antitrypsin deficiency-associated liver disease symptoms, shunts may be inserted to lower the pressure within the blood vessels in the liver, and dilated veins in the food tube (esophagus) may be clipped or banded to lower the risk of bleeding. Thus, behavioral, lifestyle modification, surgical treatment, bronchodilators, inhaled corticosteroids and oxygen therapy are recommended for individuals with alpha-1 antitrypsin deficiency.

Treatment is similar to that used for COPD and emphysema and aims at reducing symptoms and slowing progression. Long-acting bronchodilators, antibiotics, corticosteroid inhalations, and long-acting beta-agonists are the mainstay of treatment. Another treatment option in severe cases involves augmentation therapy with the administration of purified human AAT to reach normal physiological levels. The US FDA has approved six augmentation therapy drugs: PROLASTIN, ARALAST, ARALAST NP, ZEMAIRA, PROLASTIN-C, and GLASSIA, of which the latter four are currently available. While in Europe, ALFALASTIN, PROLASTIN, and RESPREEZA are approved. Further, Japan has only one approved therapy i.e., PROLASTIN-C, authorized as LYNSPAD to treat severe alpha-1 antitrypsin deficiency.

Alpha-1 Antitrypsin Deficiency Epidemiology

As the market is derived using a patient-based model, the alpha-1 antitrypsin deficiency epidemiology chapter in the report provides historical as well as forecasted epidemiology segmented by total prevalent cases of alpha-1 antitrypsin deficiency, genotype-specific prevalent cases of alpha-1 antitrypsin deficiency, and comorbidity-associated prevalent cases of alpha-1 antitrypsin deficiency in the 7MM covering the United States, EU4 countries (Germany, France, Italy, and Spain) and the United Kingdom, and Japan from 2019 to 2032.

• In 2022, the total diagnosed prevalent cases of alpha-1 antitrypsin deficiency were estimated to be approximately 220,352 cases in the 7MM. These cases are expected to increase by 2032.
• Among the 7MM, the US accounted for nearly 60% of the total diagnosed prevalent cases of alpha-1 antitrypsin deficiency in 2022. These cases are expected to increase during the study period (2019-2032).
• As per The epidemiology model, the genotype of alpha-1 antitrypsin deficiency was divided into three groups: PiZZ, PiSZ, and others (including PiMZ and other less common genotypes). In 2022, the US had approximately 119,274, 7,316, and 5,937 cases of PiZZ, PiSZ, and other genotypes, respectively. These cases are expected to increase during the study period (2019-2032).
• In EU4 and the UK, there were 87,801 prevalent cases of alpha-1 antitrypsin deficiency in 2022. These cases are expected to increase during the forecast period.
• Among the EU4 and the UK, as per the report estimates, the UK accounted for the highest prevalent alpha-1 antitrypsin deficiency cases, approximately 22,597, while Italy reported for least prevalent alpha-1 antitrypsin deficiency cases, nearly 12,219 in 2022. The analyst estimates that by 2032, these cases are expected to change.

In EU4 and the UK, comorbidities associated with alpha-1 antitrypsin deficiency were divided into lung, liver, and other diseases. Comorbidity associated with lung diseases in alpha-1 antitrypsin deficiency ranked first with nearly 70,546 cases, other diseases with approximately 8,939 cases, and liver disease with nearly 8,316 cases in 2022

• As per the analysis, in 2022, Japan accounted for the lowest prevalent cases of alpha-1 antitrypsin deficiency among the 7MM, making up nearly 0.01% of the total cases.
• In 2022, among the total cases of alpha-1 antitrypsin deficiency, the PiZZ genotype was found in around 21 cases, while only one case was found for both PiSZ and other genotypes. The PiZZ genotype cases in Japan are expected to decrease by 2032.

Alpha-1 Antitrypsin Deficiency Drug Chapters

The drug chapter segment of the alpha-1 antitrypsin deficiency report encloses a detailed analysis of alpha-1 antitrypsin deficiency-marketed drugs and late-stage (Phase III and Phase II) pipeline drugs. It also helps understand the alpha-1 antitrypsin deficiency clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug, and the latest news and press releases.

Marketed Drugs

PROLASTIN-C LIQUID/LYNSPAD: Grifols Therapeutics

PROLASTIN-C LIQUID is an alpha1-proteinase inhibitor (human) (alpha1-PI) indicated for chronic augmentation and maintenance therapy in adults with clinical evidence of emphysema due to severe hereditary deficiency of alpha1-proteinase inhibitor. PROLASTIN increases the antigenic and functional levels of serum and antigenic lung epithelial lining fluid levels of alpha1-proteinase inhibitor. PROLASTIN-C is prepared from pooled human plasma of a healthy person with some modifications and refinements of the cold ethanol method of Cohn.

In January 2021, MHLW granted manufacturing and marketing approval to Grifols' LYNSPAD (sold as PROLASTIN-C in other markets) for treating alpha-1 antitrypsin deficiency in Japan. In September 2017, PROLASTIN-C liquid was approved by the US FDA for chronic augmentation and maintenance therapy in adults with clinical evidence of emphysema due to severe alpha-1 antitrypsin deficiency.

The recommended dose of PROLASTIN-C LIQUID is 60 mg/kg body weight IV once per week. This dose is intended to increase and maintain a level of functional alpha1-proteinase inhibitor in the epithelial lining of the lower respiratory tract, providing adequate anti-elastase activity in the lungs of individuals with alpha-1 antitrypsin deficiency.

Note: Detailed marketed drugs assessment will be provided in the final report.

Emerging Drugs

Inhaled Alpha 1-Antitrypsin (AAT): Kamada Pharmaceuticals

Inhaled AAT, an investigational medicine researched for alpha-1 antitrypsin deficiency patients, aims to replace the deficient AAT protein in the lung. It acts as an immunomodulator (serine peptidase inhibitor) that prevents inflammation and tissue degradation by regulating the imbalance of proteases and antiproteases in the lungs. Inhaled AAT has received ODD from the US FDA and EMA for treating alpha-1 antitrypsin deficiency.

The Phase II trial met its primary and secondary outcome points, showing that inhaled AAT significantly increased the ELF-AAT, neutrophil elastase (NE)-AAT, and ANEC complexes concentration in subjects receiving the 80 mg and 160 mg doses. Currently, the company is conducting a Phase III clinical trial to evaluate the safety and efficacy of Kamada AAT for Inhalation in the EU and has completed Phase II and II/III trials in the US and EU, respectively.

Note: Detailed emerging therapies assessment will be provided in the final report.

Drug Class Insights

Alpha-1 antitrypsin deficiency is a genetic disorder that can lead to lung and liver disease. It is an inherited condition caused by mutations in the SERPINA1 gene, which provides instructions for making an AAT or alpha-1 protein. The disease is under-recognized and a genetic risk factor that predisposes individuals to lung diseases like COPD, emphysema, bronchitis, liver disease (chronic hepatitis, cirrhosis, and hepatoma), and skin disease (panniculitis).

Augmentation or replacement therapy is approved for treating alpha-1-related lung disease. Weekly IV supplementation with the AAT protein purified from pooled healthy donors' plasma is infused to improve the protein level in the blood and lungs. Augmentation therapy is suitable only for individuals with a respiratory disease without severe lung function loss and no liver disorders resulting from accumulating the incorrectly folded AAT protein in hepatocytes. For AAT associated with liver disease, there is no specific treatment available except liver transplantation.

Alpha-1 Antitrypsin Deficiency Market Outlook

Although alpha-1 is an inherited condition, some individuals with alpha-1 antitrypsin deficiency may lead perfectly healthy lives unaffected by their genetically altered alpha-1 antitrypsin gene; however, others develop serious complications. The major associated diseases of the lung and liver occur due to the imbalance in concentrations of AAT in the liver and lungs of the affected individual. Early diagnosis and appropriate medical care are important for managing the condition and improving the quality of life for individuals with alpha-1 antitrypsin deficiency.

PROLASTIN, launched in the US in February 1988, was the first and only FDA-approved therapy for treating alpha-1 by augmenting patients' levels of the AAT protein. PROLASTIN was eventually phased out and replaced by PROLASTIN-C, a product that applies the latest methods to purifying AAT from plasma. The major difference is that a vial of PROLASTIN containing about 1,000 mg of AAT is diluted with 40 mL (about 1 and 1/3 ounce) of water, while the same dose of PROLASTIN-C is diluted with 20 mL (about 2/3 ounce). This decreased volume may reduce the infusion time, often in half. The manufacturer also reports that PROLASTIN-C has a higher purity than PROLASTIN, i.e., there are lower levels of plasma proteins other than active AAT in each vial than in PROLASTIN. The package insert states that the purity of PROLASTIN-C is 90%.

The US FDA approved the progression from PROLASTIN to PROLASTIN-C based on two studies comparing the safety and biochemical efficacy of the two product versions. Biochemical efficacy is the ability of these products to raise the level of alpha-1 antitrypsin protein in the blood and the lungs. In September 2017, the US FDA approved PROLASTIN-C Liquid, the first liquid formulation of an alpha-1 antitrypsin deficiency replacement therapy, launched in 2018 in the US market.

Currently, liver transplants are only curative therapy for patients with liver-associated disease and are recommended in patients with severe progressive liver damage. Usually, liver transplants are done in children with the type of infantile hepatitis associated with severe alpha-1 antitrypsin deficiency. A transplant completely replaces the liver cells that produce mutated alpha-1 in these patients, correcting the protein abnormality.

To be continued in the report.

The current market has been segmented into different commonly used therapies based on the prevailing treatment pattern across the 7MM, which presents minor variations in the overall prescription pattern. The current standard of care includes ursodeoxycholic acid and acetaminophen, while several off-label medications are utilized, including bronchodilators, corticosteroids, antibiotics, and others. Further, approved therapies, including Grifols' PROLASTIN/PROLASTIN-C/LYNSPAD, Takeda's ARALAST, CSL Behring's ZEMAIRA/RESPREEZA, and Kamada/Takeda's GLASSIA are also considered in the alpha-1 antitrypsin deficiency forecast model.

Key players Kamada Pharmaceuticals, Arrowhead Pharmaceuticals, Takeda, Vertex Pharmaceuticals, Mereo BioPharma, AstraZeneca, Krystal Biotech, and others are evaluating their lead candidates in different stages of clinical development. They aim to investigate their products to treat alpha-1 antitrypsin deficiency.

• The total market size of alpha-1 antitrypsin deficiency in the 7MM was approximately USD 1,169.50 million in 2022 and is projected to increase during the forecast period (2023-2032).
• The market size of alpha-1 antitrypsin deficiency in the US will increase during the study period due to increasing awareness of the disease and the launch of the emerging therapy. The market size was approximately USD 931.32 million in 2022.
• Among the 7MM, EU4 and the UK captured around 20% of the total 7MM market size in 2022.
• According to the publisher's estimates, among EU4 and the UK, Germany accounted for the highest market with approximately USD 64.47 million in 2022, while Italy accounted for the lowest market in 2022.
• According to the publisher's analysis, in the US, PROLASTIN/PROLASTIN-C/LYNSPAD has the highest market share among all the therapies in 2022, with a revenue of approximately USD 345.60 million, followed by ZEMAIRA/RESPREEZA with approximately USD 343.10, and others in 2022 respectively.
• In 2022, the market size of alpha-1 antitrypsin deficiency in Japan was nearly 0.03% of the total market size in the 7MM.
• Among the emerging therapies, Mereo BioPharma/AstraZeneca's alvelestat (MPH-966) is a neutrophil elastase enzyme inhibitor that helps protect people with alpha-1 antitrypsin deficiency by slowing progressive lung damage. It is expected to enter the US market by 2028 and is predicted to have a medium-fast uptake during the forecast period.

Alpha-1 Antitrypsin Deficiency Drugs Uptake

This section focuses on the uptake rate of potential drugs expected to be launched in the market during 2019-2032. For example, Kamada Pharmaceutical's inhaled alpha 1-antitrypsin, an immunomodulator (serine peptidase inhibitor), is expected to enter the US market by 2028 and is projected to have a medium uptake during the forecast period.

Further detailed analysis of emerging therapies drug uptake in the report.

Alpha-1 Antitrypsin Deficiency Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase III, Phase II, and Phase I. It also analyzes key players involved in developing targeted therapeutics.

Pipeline development activities

The report covers information on collaborations, acquisitions and mergers, licensing, and patent details for emerging therapies for alpha-1 antitrypsin deficiency.

KOL Views

To keep up with current market trends, we take KOLs and SMEs' opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry Experts contacted for insights on alpha-1 antitrypsin deficiency evolving treatment landscape, patient reliance on conventional therapies, patient therapy switching acceptability, drug uptake, along with challenges related to accessibility, including Medical/scientific writers, Medical Professionals, Professors, Directors, and Others.

This analysts connected with 50+ KOLs to gather insights; however, interviews were conducted with 15+ KOLs in the 7MM. Centers like the University of Florida, the University of Birmingham, Paracelsus Medical University, The University Hospital Centre of Tours, Fondazione IRCCS Policlinico San Matteo, and Hokkaido University were contacted. Their opinion helps understand and validate current and emerging therapy treatment patterns or alpha-1 antitrypsin deficiency market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Physician's View

According to our primary research analysis, the current treatment guideline recommends the use of augmentation therapies along with several off-label drugs for symptomatic relief and managing lung-related complications; however, no therapies are approved to address liver-associated disease, and off-label therapies and liver transplant in severe cases are recommended. Furthermore, while these treatments do not cure symptoms, they lessen them. Surgical options such as lung volume reduction surgery and bullectomy are advised for patients in the more severe stages of the disease. Patients with very advanced emphysema may also need lung transplant surgery, which entails removing the damaged lung and replacing it with a healthy lung but carries a high risk of rejection and infection.

The current pipeline is novel molecules like AAT corrector, Neutrophil elastase enzyme inhibitor GalNAc-based RNA interference, and immunomodulator (serine peptidase inhibitor) targeting novel pathways in alpha-1 antitrypsin deficiency that have shown significant improvements in ELF, AAT antigenic, and anti-neutrophil elastase inhibitory (ANEC) levels with improved safety profiles in early-stage trials. There is hope that the entry of these small molecules, biologics, and peptides will provide options based on patient-specific needs. However, there is still a need for therapies that cure the disease rather than relieve the symptoms.

Qualitative Analysis

The publisher performs Qualitative and market Intelligence analysis using various approaches, such as SWOT and Conjoint Analysis. In the SWOT analysis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided. These pointers are based on the Analyst's discretion and assessment of the patient burden, cost analysis, and existing and evolving treatment landscape.

Conjoint Analysis analyzes multiple emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.

In efficacy, the trial's primary and secondary outcome measures are evaluated; for instance, in alpha-1 antitrypsin deficiency trials, efficacy scores are according to change from baseline in the concentration of antigenic AAT in the lung epithelial lining fluid measured by ELISA and ANEC, change from baseline in total, soluble, and insoluble Z-AAT liver concentrations, change in plasma functional AAT levels, and change in blood neutrophil elastase activity.

Further, the therapies' safety is evaluated wherein the acceptability, tolerability, and adverse events are majorly observed. It sets a clear understanding of the side effects posed by the drug in the trials. In addition, the scoring is also based on the route of administration, order of entry and designation, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.

Market Access and Reimbursement

PROLASTIN-C LIQUID is an alpha1-proteinase inhibitor (human) (alpha1-PI) indicated for chronic augmentation and maintenance therapy in adults with clinical evidence of emphysema due to severe hereditary deficiency of alpha1-proteinase inhibitor.

The PROLASTIN DIRECT Program is managed by EVERSANA, the only specialty pharmacy that fills prescriptions for PROLASTIN-C LIQUID. The program provides the patient with a team dedicated to alpha-1 care that will assist with securing insurance coverage, coordinating infusions, and managing one's health with their physician's guidance. The program's patient service coordinator is the primary point of contact the patient can rely on for everything related to the treatment. It assists alpha-1 expert nurses and pharmacists 24 h a day, 7 days a week. One can take comfort in knowing that the PROLASTIN DIRECT program handles every detail of the patient's infusion, from shipping medicine, scheduling infusion, and administering infusion everything is handled.

UnitedHealthcare Commercial Medical Benefit Drug Policy, some certificates of coverage allow for coverage of experimental/investigational/unproven treatments for life-threatening illnesses when certain conditions are met. Additionally, alpha1-proteinase inhibitors such as ARALAST NP, GLASSIA, PROLASTIN-C, and ZEMAIRA are medically necessary for the treatment of emphysema due to congenital deficiency alpha1-proteinase inhibitors in patients. The member-specific benefit plan document must be consulted to make coverage decisions for this service. Some states mandate benefit coverage for off-label use of medications for some diagnoses or under some circumstances when certain conditions are met. Where such mandates apply, they supersede language in the benefit document or the medical or drug policy. Benefit coverage for an otherwise unproven service for treating serious rare diseases may occur when certain conditions are met.

In November 2018, Grifols initiated a support program for patients with alpha-1 antitrypsin deficiency in Spain. AlfaCare is a comprehensive, personalized assistance program that offers training, counseling, and a range of other services to alpha-1 antitrypsin deficiency patients to encourage new habits and help them better manage their disease.

The report provides detailed insights on the country-wise accessibility and reimbursement scenarios, cost-effectiveness scenarios, programs making accessibility easier and out-of-pocket costs more affordable, insights on patients insured under federal or state government prescription drug programs, etc.

Scope of the Report

• The report covers a segment of key events, an executive summary, and a descriptive overview of alpha-1 antitrypsin deficiency, explaining its causes, signs and symptoms, pathogenesis, and currently available therapies.
• Comprehensive insight into the epidemiology segments and forecasts, the future growth potential of diagnosis rate, disease progression, and treatment guidelines have been provided.
• Additionally, an all-inclusive account of the current and emerging therapies and the elaborative profiles of late-stage and prominent therapies will impact the current treatment landscape.
• A detailed review of the alpha-1 antitrypsin deficiency market, historical and forecasted market size, market share by therapies, detailed assumptions, and rationale behind our approach is included in the report, covering the 7MM drug outreach.
• The report provides an edge while developing business strategies by understanding trends through SWOT analysis, expert insights/KOL views, patient journey, and treatment preferences that help shape and drive the 7MM alpha-1 antitrypsin deficiency market.

Alpha-1 Antitrypsin Deficiency Report Insights

• Patient Population
• Therapeutic Approaches
• Alpha-1 Antitrypsin Deficiency Pipeline Analysis
• Alpha-1 Antitrypsin Deficiency Market Size and Trends
• Existing and Future Market Opportunity

Alpha-1 Antitrypsin Deficiency Report Key Strengths

• Ten years Forecast
• The 7MM Coverage
• Alpha-1 Antitrypsin Deficiency Epidemiology Segmentation
• Key Cross Competition
• Attribute analysis
• Drugs Uptake and Key Market Forecast Assumptions

Alpha-1 Antitrypsin Deficiency Report Assessment

• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness
• Qualitative Analysis (SWOT and Conjoint Analysis)

Key Questions Answered

Market Insights

• What was the total market size of alpha-1 antitrypsin deficiency, the market size of Alpha-1 antitrypsin deficiency by therapies, and market share (%) distribution in 2019, and what would it look like by 2032? What are the contributing factors for this growth?
• How will alvelestat (MPH-966) and fazirsiran (ARO-AAT/TAK-999) affect the treatment paradigm of alpha-1 antitrypsin deficiency?
• How will PROLASTIN/PROLASTIN-C/LYNSPAD compete with similar-class products and off-label therapies?
• Which drug is going to be the largest contributor by 2032?
• What are the pricing variations among different geographies for approved and off-label therapies?
• How would future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights

• What are the disease risks, burdens, and unmet needs of alpha-1 antitrypsin deficiency? What will be the growth opportunities across the 7MM with respect to the patient population pertaining to alpha-1 antitrypsin deficiency?
• What is the historical and forecasted alpha-1 antitrypsin deficiency patient pool in the United States, EU4 (Germany, France, Italy, and Spain) and the UK, and Japan?
• Out of the countries mentioned above, which country would have the highest diagnosed prevalent alpha-1 antitrypsin deficiency population during the forecast period (2023-2032)?
• What factors are contributing to the growth of alpha-1 antitrypsin deficiency cases?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies

• What are the current options for the treatment of alpha-1 antitrypsin deficiency? What are the current guidelines for treating alpha-1 antitrypsin deficiency?
• How many companies are developing therapies for the treatment of alpha-1 antitrypsin deficiency?
• How many emerging therapies are in the mid-stage and late stage of development for treating alpha-1 antitrypsin deficiency?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitations of existing therapies?
• What is the cost burden of current treatment on the patient?
• Patient acceptability in terms of preferred treatment options as per real-world scenarios?
• What are the accessibility issues of approved therapy in the US?
• What is the 7MM historical and forecasted market of alpha-1 antitrypsin deficiency?

Reasons to Buy

• The report will help develop business strategies by understanding the latest trends and changing treatment dynamics driving the alpha-1 antitrypsin deficiency market.
• Insights on patient burden/disease prevalence, evolution in diagnosis, and factors contributing to the change in the epidemiology of the disease during the forecast years.
• Understand the existing market opportunities in varying geographies and the growth potential over the coming years.
• The distribution of historical and current patient share is based on real-world prescription data in the US, EU4 (Germany, France, Italy, and Spain) and the UK, and Japan.
• Identifying upcoming solid players in the market will help devise strategies to help get ahead of competitors.
• Detailed analysis and ranking of class-wise potential current and emerging therapies under the conjoint analysis section to provide visibility around leading classes.
• Highlights of Access and Reimbursement policies for alpha-1 antitrypsin deficiency, barriers to accessibility of approved therapy, and patient assistance programs.
• To understand Key Opinion Leaders' perspectives around the accessibility, acceptability, and compliance-related challenges of existing treatment to overcome barriers in the future.
• Detailed insights on the unmet needs of the existing market so that the upcoming players can strengthen their development and launch strategy.