Transthyretin amyloid polyneuropathy, also known as transthyretin-related hereditary amyloidosis, is a rare inherited, autosomal-dominant, adult-onset, and slowly neurodegenerative progressive disease. Transthyretin amyloid polyneuropathy is characterized by the extracellular deposition of amyloid fibrils, most commonly due to mutated transthyretin (TTR). TTR gene mutations lead to destabilization and dissociation of TTR tetramers into variant TTR monomers, which form amyloid fibrils that deposit in peripheral nerves and various organs, giving rise to peripheral and autonomic neuropathy and several nondisease specific symptoms. The penetrance and age at onset of transthyretin amyloid polyneuropathy among people carrying the same mutation vary between countries. The symptomatology and clinical course of transthyretin amyloid polyneuropathy can be highly variable.
According to reports published on transthyretin amyloidosis, it has been estimated that nearly 1.1 per 100,000 individuals in the world are living with transthyretin amyloid polyneuropathy. The age group of patients suffering from the disorder is between 30 and 40 years of age. Familial amyloid polyneuropathy (TTR-FAP) leads to 100 different types of mutations in the transthyretin gene, which leads to protein misfolding. There is only a 50% chance of transferring the mutation to the next generation from an affected parent. The global transthyretin amyloid polyneuropathy therapeutics market is likely to be driven by an increase in the prevalence of disease, increase in awareness, improvement in diagnostic procedures, improving health care services, rapid economic growth in developing countries, and rise in research and development activity. However, major restraining factors for the transthyretin amyloid polyneuropathy therapeutics market are less awareness in developing economies, misdiagnosis of the disease, high cost associated with disease diagnosis and treatment, limited clinical trials, and lack of efficient medication.
The transthyretin amyloid polyneuropathy therapeutics can be segmented based on treatment, end-user, and region. In terms of treatment, the market can be classified into drug therapy and surgery. GlaxoSmithKline (GSK) is currently in Phase II of development of a novel therapy for systemic amyloidosis, which directly targets the amyloid deposits themselves through a mechanism directed toward serum amyloid P component (SAP). Based on end-user, the market can be classified into hospital pharmacies, retail pharmacies, and online pharmacies.
In terms of region, the transthyretin amyloid polyneuropathy therapeutics market can be segmented into North America, Latin America, Europe, Asia Pacific, and Middle East & Africa. North America dominates the global transthyretin amyloid polyneuropathy therapeutics market due to new product innovation, high health care expenditure, and government awareness programs. In March 2018, Ionis Pharmaceuticals, Inc. and its affiliate, Akcea Therapeutics, Inc. announced Phase III clinical data showing that inotersen-treated patients with hereditary ATTR (hATTR) amyloidosis who were treated for up to 27 months in the NEURO-TTR and open-label extension (OLE) studies continued to demonstrate sustained benefit in measures of quality of life and neuropathy. Europe is a prominent region of the transthyretin amyloid polyneuropathy therapeutics market. Within Europe, the incidence of TTR-FAP is highly variable. There are large areas in Portugal and Sweden in which the disease is endemic and is localized to one small area, with a traceable family history with a single genetic mutation. The market in Asia Pacific is expected to expand at a considerable rate due to a rapid rise in 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 expected to offer minimal growth opportunities to the global transthyretin amyloid polyneuropathy therapeutics market due to low awareness among people.
Major players operating in the global transthyretin amyloid polyneuropathy therapeutics market include Pfizer, Inc., GlaxoSmithKline Plc., Corino Therapeutics 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 polyneuropathy is expected to be a major factor driving the market over the forecast period. For instance, RNAi Therapeutics for transthyretin amyloid polyneuropathy by Alnylam Pharmaceuticals. A robust drug pipeline is expected to boost the transthyretin amyloid polyneuropathy 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
- 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.