Hereditary Transthyretin Amyloidosis (hATTR) - Competitive Landscape, Market Insights, Epidemiology and Forecast to 2030

Hereditary Transthyretin Amyloidosis (hATTR) - Competitive Landscape, Market Insights, Epidemiology and Forecast to 2030 delivers an in-depth understanding of the Hereditary Transthyretin Amyloidosis (hATTR), historical and forecasted epidemiology as well as the hATTR market trends in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom) and Japan.

The Hereditary Transthyretin Amyloidosis (hATTR) market report provides current treatment practices, emerging drugs, Hereditary Transthyretin Amyloidosis (hATTR) market share of the individual therapies, current and forecasted Hereditary Transthyretin Amyloidosis (hATTR) market Size from 2017 to 2030 segmented by seven major markets. The report also covers current Hereditary Transthyretin Amyloidosis (hATTR) treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses underlying potential of the market.

Geographies Covered

• The United States
• EU5 (Germany, France, Italy, Spain and the United Kingdom)
• Japan

Study Period: 2017-2030

Hereditary Transthyretin Amyloidosis (hATTR) Disease Understanding and Treatment Algorithm

Hereditary Transthyretin Amyloidosis (hATTR) Overview

Hereditary Transthyretin Amyloidosis (hATTR) is caused by the misfolding of protein monomers derived from the tetrameric protein transthyretin (TTR). Mutations in the gene for TTR frequently result in instability of TTR and subsequent fibril formation. hATTR can be sub-classified into familial amyloid polyneuropathy (FAP) and familial amyloid cardiomyopathy (FAC). The deposition of TTR amyloid fibrils in various tissues leads to ATTR, including Familial ATTR (hereditary ATTR) and non-hereditary ATTR types. Familial ATTR is a rare disorder with unequal distribution all around the world.

Familial transthyretin amyloidosis (FTA) is caused by changes (mutations) in the TTR gene. This gene is responsible for making a protein called transthyretin which transports vitamin A and a hormone called thyroxin too many parts of the body. Mutations in TTR lead to a transthyretin protein that is not made correctly. The faulty protein then folds up to form amyloid. Amyloid builds up in various parts of the body causing nerve and tissue damage. Most people who have FTA have inherited the TTR mutation from a family member. However, a few people with FTA will have no family history of the disease and have a new (de novo) mutation in the TTR gene.

Hereditary Transthyretin Amyloidosis (hATTR) Diagnosis

Disease heterogeneity and its rarity make a diagnosis of hATTR amyloidosis challenging. However, making a correct diagnosis is vital to determining prognosis, treatment, and appropriate patient and family counseling. Timely diagnosis is also important because it allows patients the opportunity to receive appropriate care as early as possible in the disease course.

Diagnosis can be confirmed via biopsy of the affected tissue or organ followed by staining with Congo red to confirm the presence of amyloid. The most sensitive test used for diagnosing cardiac involvement in hATTR amyloidosis is endomyocardial biopsy. However, it is not widely available. Therefore, other diagnostic tests are used for identifying cardiac involvement in hATTR amyloidosis, including electrocardiography (ECG), echocardiography, and cardiac magnetic resonance imaging (cMRI). Nuclear cardiology using scintigraphic tracers can also provide diagnostic value by allowing clinicians to visualize amyloid infiltration throughout the body.

Hereditary Transthyretin Amyloidosis (hATTR) Treatment

It covers the details of conventional and current medical therapies available in the Hereditary Transthyretin Amyloidosis (hATTR) market for the treatment of the condition. It also provides the country-wise treatment guidelines and algorithm across the United States, Europe and Japan.

The market report gives a thorough understanding of Hereditary Transthyretin Amyloidosis (hATTR) by including details such as disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides Hereditary Transthyretin Amyloidosis (hATTR) treatment algorithms and treatment guidelines for Hereditary Transthyretin Amyloidosis (hATTR) in the US, Europe, and Japan.

Hereditary Transthyretin Amyloidosis (hATTR) Epidemiology

The Hereditary Transthyretin Amyloidosis (hATTR) epidemiology division provide the insights about historical and current Hereditary Transthyretin Amyloidosis (hATTR) patient pool and forecasted trend for each seven major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the diagnosed patient pool and their trends along with assumptions undertaken.

Key Findings

Although the exact prevalence of hATTR is difficult to determine, existing estimates suggest a worldwide prevalence of 50,000 individuals, with varying phenotypic presentations of disease. ATTR is a rare condition and TTR-FAP is an ultra-rare condition with the global prevalence that currently effects an estimated 8,000 to 10,000 people worldwide. According to Lin et al., 2019, in the US, prevalence of hATTR amyloidosis with polyneuropathy is estimated to be 10,000-15,000 patients, although fewer than 3,000 have been diagnosed.

Hereditary Transthyretin Amyloidosis has the highest diagnosed prevalence in the United States, while Japan has the lowest diagnosed number over the forecast period.

The disease epidemiology covered in The report provides historical as well as forecasted Hereditary Transthyretin Amyloidosis (hATTR) epidemiology [segmented as Hereditary Transthyretin Amyloidosis (hATTR) Diagnosed Prevalent Population, and Type-Specific Prevalent Population of Hereditary Transthyretin Amyloidosis (hATTR)] scenario of Hereditary Transthyretin Amyloidosis (hATTR) in 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom), and Japan from 2017 to 2030.

Country-wise Hereditary Transthyretin Amyloidosis (hATTR) Epidemiology

The epidemiology segment also provides the Hereditary Transthyretin Amyloidosis (hATTR) epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain and the United Kingdom) and Japan.

Hereditary Transthyretin Amyloidosis (hATTR) Drug Chapters

Drug chapter segment of the Hereditary Transthyretin Amyloidosis (hATTR) report encloses the detailed analysis of Hereditary Transthyretin Amyloidosis (hATTR) marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the Hereditary Transthyretin Amyloidosis (hATTR) 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.

Hereditary Transthyretin Amyloidosis (hATTR) Marketed Drugs

Vyndaqel/ Vyndamax: Pfizer
Vyndaqel (tafamidis meglumine 20 mg) and Vyndamax (tafamidis 61 mg) are oral transthyretin stabilizers that selectively bind to transthyretin, stabilizing the tetramer of the transthyretin transport protein and slowing the formation of amyloid. The tafamidis 61 mg capsule corresponds to an 80 mg tafamidis meglumine dose (4x 20mg capsules) and was developed for patient convenience to enable a single capsule for daily administration. Vyndamax 61 mg and Vyndaqel 20 mg are not substitutable on a per milligram basis.

Products details in the report...

Hereditary Transthyretin Amyloidosis (hATTR) Emerging Drugs

Vutrisiran: Alnylam Pharmaceuticals
Vutrisiran is an investigational, subcutaneously-administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary (hATTR) and wild-type (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, blocking the production of wild-type and mutant transthyretin (TTR) protein before it is made. Quarterly administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s next-generation delivery platform known as the Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform.

Products details in the report...

Hereditary Transthyretin Amyloidosis (hATTR) Market Outlook

The Hereditary Transthyretin Amyloidosis (hATTR) market outlook of The report helps to build the detailed comprehension of the historic, current and forecasted Hereditary Transthyretin Amyloidosis (hATTR) market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.

This segment gives a through detail of Hereditary Transthyretin Amyloidosis (hATTR) market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to the report, Hereditary Transthyretin Amyloidosis (hATTR) market in 7MM is expected to change in the study period 2017-2030.

Hereditary Transthyretin Amyloidosis (hATTR) Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Hereditary Transthyretin Amyloidosis (hATTR) market or expected to get launched in the market during the study period 2017-2030. The analysis covers Hereditary Transthyretin Amyloidosis (hATTR) market uptake by drugs; patient uptake by therapies; and sales of each drug.

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows the comparison of the drugs on the basis of market share and Size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Hereditary Transthyretin Amyloidosis (hATTR) Pipeline Development Activities

The report provides insights into different therapeutic candidates in Phase II, and Phase III stage. It also analyses Hereditary Transthyretin Amyloidosis (hATTR) key players involved in developing targeted therapeutics.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Hereditary Transthyretin Amyloidosis (hATTR) emerging therapies.

Reimbursement Scenario in Hereditary Transthyretin Amyloidosis (hATTR)

Approaching reimbursement proactively can have a positive impact both during the late stages of product development and well after product launch. In report we take reimbursement into consideration to identify economically attractive indications and market opportunities. When working with finite resources, the ability to select the markets with the fewest reimbursement barriers can be a critical business & price strategy.

KOL Views

To keep up with current market trends, we take KOLs and SME’s opinion working in Hereditary Transthyretin Amyloidosis (hATTR) domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps to understand and validate current and emerging therapies treatment patterns or Hereditary Transthyretin Amyloidosis (hATTR) market trend. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis

The publisher performs a Competitive & Market Intelligence analysis of the Hereditary Transthyretin Amyloidosis (hATTR) Market by using various Competitive Intelligence tools that includes - SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report

• The report covers the descriptive overview of Hereditary Transthyretin Amyloidosis (hATTR), explaining its causes, signs and symptoms, pathophysiology and currently available therapies
• Comprehensive insight has been provided into the Hereditary Transthyretin Amyloidosis (hATTR) epidemiology and treatment in the 7MM
• Additionally, an all-inclusive account of both the current and emerging therapies for Hereditary Transthyretin Amyloidosis (hATTR) are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape
• A detailed review of Hereditary Transthyretin Amyloidosis (hATTR) market; historical and forecasted is included in The report, covering drug outreach in the 7MM
• The report provides an edge while developing business strategies, by understanding trends shaping and driving the global Hereditary Transthyretin Amyloidosis (hATTR) market

Report Highlights

• In the coming years, Hereditary Transthyretin Amyloidosis (hATTR) market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the Size of the market to enable the drug manufacturers to penetrate more into the market
• The companies and academics are working to assess challenges and seek opportunities that could influence Hereditary Transthyretin Amyloidosis (hATTR) R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition
• Major players are involved in developing therapies for Hereditary Transthyretin Amyloidosis (hATTR). Launch of emerging therapies, will significantly impact the Hereditary Transthyretin Amyloidosis (hATTR) market
• A better understanding of disease pathogenesis will also contribute to the development of novel therapeutics for Hereditary Transthyretin Amyloidosis (hATTR)
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends and comparative analysis of pipeline products with detailed clinical profiles, key cross-competition, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities

Hereditary Transthyretin Amyloidosis (hATTR) Report Insights

• Patient Population
• Therapeutic Approaches
• Hereditary Transthyretin Amyloidosis (hATTR) Pipeline Analysis
• Hereditary Transthyretin Amyloidosis (hATTR) Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Hereditary Transthyretin Amyloidosis (hATTR) Report Key Strengths

• 11 Years Forecast
• 7MM Coverage
• Hereditary Transthyretin Amyloidosis (hATTR) Epidemiology Segmentation
• Key Cross Competition
• Highly Analyzed Market
• Drugs Uptake

Hereditary Transthyretin Amyloidosis (hATTR) Report Assessment

• Current Treatment Practices
• Unmet Needs
• Market Product Profiles
• Pipeline Product Profiles
• Market Drivers and Barriers
• SWOT Analysis

Key Questions

Market Insights:

• What was the Hereditary Transthyretin Amyloidosis (hATTR) Market share (%) distribution in 2017 and how it would look like in 2030?
• What would be the Hereditary Transthyretin Amyloidosis (hATTR) total market Size as well as market Size by therapies across the 7MM during the forecast period (2017-2030)?
• What are the key findings pertaining to the market across 7MM and which country will have the largest Hereditary Transthyretin Amyloidosis (hATTR) market Size during the forecast period (2017-2030)?
• At what CAGR, the Hereditary Transthyretin Amyloidosis (hATTR) market is expected to grow in 7MM during the forecast period (2017-2030)?
• What would be the Hereditary Transthyretin Amyloidosis (hATTR) market outlook across the 7MM during the forecast period (2017-2030)?
• What would be the Hereditary Transthyretin Amyloidosis (hATTR) market growth till 2030 and what will be the resultant market Size in the year 2030?
• How would the market drivers, barriers and future opportunities affect the market dynamics and a subsequent analysis of the associated trends?

Epidemiology Insights:

• What is the disease risk, burden and unmet needs of the Hereditary Transthyretin Amyloidosis (hATTR)?
• What is the historical Hereditary Transthyretin Amyloidosis (hATTR) patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan?
• What would be the forecasted patient pool of Hereditary Transthyretin Amyloidosis (hATTR) in 7 major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan?
• What will be the growth opportunities in the 7MM with respect to the patient population pertaining to Hereditary Transthyretin Amyloidosis (hATTR)?
• Out of all 7MM countries, which country would have the highest prevalent population of Hereditary Transthyretin Amyloidosis (hATTR) during the forecast period (2017-2030)?
• At what CAGR the population is expected to grow in 7MM during the forecast period (2017-2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the treatment of Hereditary Transthyretin Amyloidosis (hATTR) along with the approved therapy?
• What are the current treatment guidelines for the treatment of Hereditary Transthyretin Amyloidosis (hATTR) in USA, Europe and Japan?
• What are the Hereditary Transthyretin Amyloidosis (hATTR) marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy etc.?
• How many companies are developing therapies for the treatment of Hereditary Transthyretin Amyloidosis (hATTR)?
• How many therapies are developed by each company for the treatment of Hereditary Transthyretin Amyloidosis (hATTR)?
• How many emerging therapies are in mid stage, and late stage of development for the treatment of Hereditary Transthyretin Amyloidosis (hATTR)?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hereditary Transthyretin Amyloidosis (hATTR) therapies?
• What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hereditary Transthyretin Amyloidosis (hATTR) and their status?
• What are the key designations that have been granted for the emerging therapies for Hereditary Transthyretin Amyloidosis (hATTR)?
• What are the global historical and forecasted market of Hereditary Transthyretin Amyloidosis (hATTR)?

Reasons to Buy

• The report will help in developing business strategies by understanding trends shaping and driving the Hereditary Transthyretin Amyloidosis (hATTR) market
• To understand the future market competition in the Hereditary Transthyretin Amyloidosis (hATTR) market and Insightful review of the key market drivers and barriers
• Organize sales and marketing efforts by identifying the best opportunities for Hereditary Transthyretin Amyloidosis (hATTR) in US, Europe (Germany, Spain, Italy, France and United Kingdom) and Japan
• Identification of strong upcoming players in market will help in devising strategies that will help in getting ahead of competitors
• Organize sales and marketing efforts by identifying the best opportunities for Hereditary Transthyretin Amyloidosis (hATTR) market
• To understand the future market competition in the Hereditary Transthyretin Amyloidosis (hATTR) market