Transthyretin amyloid cardiomyopathy is a rare inherited and slowly progressive cardiac disease, in which a protein-based infiltrate deposits in tissues as beta-pleated sheets. The disease is characterized by an abnormal buildup of deposits of a mutant and wild-type transthyretin (TTR) protein in the heart called TTR amyloid fibrils in the body's organs and tissues. This leads to diastolic dysfunction from restrictive cardiomyopathy and eventual heart failure. In the U.S., the most common mutation (V122I) is seen predominantly among individuals of African descent.  There are three types of transthyretin amyloid cardiomyopathy: light-chain amyloidosis, mutant transthyretin amyloidosis, and wild-type transthyretin amyloidosis. All three forms of transthyretin amyloid cardiomyopathy are progressive and fatal. For patients with wild-type transthyretin amyloidosis cardiomyopathy and mutant transthyretin amyloidosis cardiomyopathy, symptoms usually manifest later in life (after the age of 50). The median period of survival is three to five years from the time of diagnosis.

The global transthyretin amyloid cardiomyopathy therapeutics market is estimated to expand at a rapid pace in the coming years. A rise in the prevalence of transthyretin amyloid cardiomyopathy, increase in research and development, introduction of novel disease therapies, and rise in demand for effective therapies among patients are projected to fuel the growth of the global transthyretin amyloid cardiomyopathy therapeutics market. In February 2019, Eidos Therapeutics, Inc. initiated its ATTRibute-CM, a Phase 3 study of AG10 in patients with transthyretin amyloid cardiomyopathy. The design of the ATTRibute-CM study, which incorporates feedback from the FDA, includes two potentially registrational endpoints. The prevalence of transthyretin amyloid cardiomyopathy is highly variable among countries across the globe. Despite inherent uncertainty, studies suggests that the prevalence of transthyretin amyloid cardiomyopathy is underestimated, particularly for the mutant transthyretin amyloid cardiomyopathy subgroup. A rise in the prevalence of cardiovascular diseases and increase in health care insurance coverage are other factors likely to accelerate the growth of the global transthyretin amyloid cardiomyopathy therapeutics market. According to the World Health Organization, cardiovascular diseases accounted for 17.7 million deaths in 2015, representing 31% of all global deaths. However, stricter regulation for product approvals and high product cost are likely to restrain the transthyretin amyloid cardiomyopathy therapeutics market.

The transthyretin amyloid cardiomyopathy therapeutics market can be segmented based on treatment, disease, end-user, and region. In terms of treatment, the market can be classified into drug therapy and medical devices. The drug therapy segment can be sub-segmented into symptom-directed treatment and slow progression drugs. Based on disease, the transthyretin amyloid cardiomyopathy therapeutics market can be classified into wild type transthyretin amyloidosis and mutant transthyretin amyloidosis. In terms of end-user, the market can be categorized into hospital pharmacies, retail pharmacies, and online pharmacies.

Based on region, the transthyretin amyloid cardiomyopathy therapeutics market can be segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominates the global transthyretin amyloid cardiomyopathy therapeutics market due to new product innovation, high health care expenditure, and government awareness programs. In January 2019, the U.S. Food and Drug Administration (FDA) accepted the filing of Pfizer Inc.’s New Drug Applications (NDAs) for tafamidis for the treatment of transthyretin amyloid cardiomyopathy. Pfizer has submitted two NDAs based on two forms of tafamidis: meglumine salt and free acid. Europe is a prominent region of the transthyretin amyloid cardiomyopathy therapeutics market. The market in Asia Pacific is expected to grow at a higher rate due to a rapid rise in the population, rise in the prevalence of chronic diseases, increase in health care coverage, and rise in investment in research and development. Emerging regions such as Latin America and Middle East & Africa are projected to offer growth opportunities to the global transthyretin amyloid cardiomyopathy therapeutics market due to low awareness among people.

Major players operating in the global transthyretin amyloid cardiomyopathy therapeutics market include Pfizer, Inc., Ionis Pharmaceuticals Inc., Ltd., Eidos Therapeutics, Inc., Alnylam Pharmaceuticals, Prothena Corporation plc, and Regeneron Pharmaceuticals, Inc. Approvals and launches of novel therapies for the treatment of transthyretin amyloid cardiomyopathy are expected to be a major factor driving the market growth over the forecast period. For instance, the introduction of RNAi Therapeutics for transthyretin amyloid cardiomyopathy by Alnylam Pharmaceuticals. A robust pipeline of drugs is estimated to boost the transthyretin amyloid cardiomyopathy therapeutics market during the forecast period.

This study by TMR is all-encompassing framework of the dynamics of the market. It mainly comprises critical assessment of consumers' or customers' journeys, current and emerging avenues, and strategic framework to enable CXOs take effective decisions.

Our key underpinning is the 4-Quadrant Framework EIRS that offers detailed visualization of four elements:

  • Customer Experience Maps
  • Insights and Tools based on data-driven research
  • Actionable Results to meet all the business priorities
  • Strategic Frameworks to boost the growth journey

The study strives to evaluate the current and future growth prospects, untapped avenues, factors shaping their revenue potential, and demand and consumption patterns in the global market by breaking it into region-wise assessment.

The following regional segments are covered comprehensively:

  • North America
  • Asia Pacific
  • Europe
  • Latin America
  • The Middle East and Africa

The EIRS quadrant framework in the report sums up our wide spectrum of data-driven research and advisory for CXOs to help them make better decisions for their businesses and stay as leaders.

Below is a snapshot of these quadrants.

1. Customer Experience Map

The study offers an in-depth assessment of various customers’ journeys pertinent to the market and its segments. It offers various customer impressions about the products and service use. The analysis takes a closer look at their pain points and fears across various customer touchpoints. The consultation and business intelligence solutions will help interested stakeholders, including CXOs, define customer experience maps tailored to their needs. This will help them aim at boosting customer engagement with their brands.

2. Insights and Tools

The various insights in the study are based on elaborate cycles of primary and secondary research the analysts engage with during the course of research. The analysts and expert advisors at TMR adopt industry-wide, quantitative customer insights tools and market projection methodologies to arrive at results, which makes them reliable. The study not just offers estimations and projections, but also an uncluttered evaluation of these figures on the market dynamics. These insights merge data-driven research framework with qualitative consultations for business owners, CXOs, policy makers, and investors. The insights will also help their customers overcome their fears.

3. Actionable Results

The findings presented in this study by TMR are an indispensable guide for meeting all business priorities, including mission-critical ones. The results when implemented have shown tangible benefits to business stakeholders and industry entities to boost their performance. The results are tailored to fit the individual strategic framework. The study also illustrates some of the recent case studies on solving various problems by companies they faced in their consolidation journey.

4. Strategic Frameworks

The study equips businesses and anyone interested in the market to frame broad strategic frameworks. This has become more important than ever, given the current uncertainty due to COVID-19. The study deliberates on consultations to overcome various such past disruptions and foresees new ones to boost the preparedness. The frameworks help businesses plan their strategic alignments for recovery from such disruptive trends. Further, analysts at TMR helps you break down the complex scenario and bring resiliency in uncertain times.

The report sheds light on various aspects and answers pertinent questions on the market. Some of the important ones are:

1. What can be the best investment choices for venturing into new product and service lines?

2. What value propositions should businesses aim at while making new research and development funding?

3. Which regulations will be most helpful for stakeholders to boost their supply chain network?

4. Which regions might see the demand maturing in certain segments in near future?

5. What are the some of the best cost optimization strategies with vendors that some well-entrenched players have gained success with?

6. Which are the key perspectives that the C-suite are leveraging to move businesses to new growth trajectory?

7. Which government regulations might challenge the status of key regional markets?

8. How will the emerging political and economic scenario affect opportunities in key growth areas?

9. What are some of the value-grab opportunities in various segments?

10. What will be the barrier to entry for new players in the market?

Note: Although care has been taken to maintain the highest levels of accuracy in TMR’s reports, recent market/vendor-specific changes may take time to reflect in the analysis.

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Transthyretin Amyloid Cardiomyopathy Therapeutics Market

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