Familial amyloid polyneuropathy (FAP) or transthyretin (TTR) amyloid neuropathy is a genetic disorder caused by the deposition of insoluble amyloid fibrils around the peripheral nerves and in various tissues, including the heart muscle. Transthyretin is a transporter of retinol and thyroid hormone; majority of which is made in liver and small amount of the protein is made in brain and eyes. The prevalence of familial amyloid polyneuropathy is very low across the globe. According to a review article published in The Lancet Neurology, although FAP cases has been reported throughout the world, but in Europe there exists strong genotypic heterogeneity. The article also mentions that 30 different TTR gene variants are reported in Japan and France. 
Currently over 30 different proteins are found to cause amyloid deposition. Familial amyloid polyneuropathy is characterized by slowly progressive, peripheral sensorimotor polyneuropathy and autonomic dysfunction. Symptoms for the disorder include limb weakness, and loss of sensation in case of peripheral neuropathy, bowel disturbance and sexual dysfunction in case of autonomic neuropathy. Cardiomyopathy may also cause heart dysfunction and even failure. Patients with familial amyloid polyneuropathy are born with a mutation in the TTR gene which leads to the disorder. Although majority of the symptoms are experience in the middle age, some patients may experience a symptomless condition and my just transfer the mutated gene to their offspring. The disorder is mainly diagnosed through tissue biopsy of abdominal fats, heart or rectum. Genetic tests are also recommended for detecting mutation in TTR gene. 
Liver transplant has proved to be the most successful cure for the disorder. But with long waiting periods for liver transplants, there is a need for effective treatment of familial amyloid polyneuropathy. In Europe, only one drug has been approved for the treatment of FAP- Vyndaqel (tafamidis) by Pfizer. However the drug is under Phase III clinical trials to seek U.S. FDA approval. It has also been found that nonsteroidal anti-inflammatory drug (NSAID) diflunisal is effective in restricting the progression of neurological impairment. Although not FDA approved, the drug is prescribed by many physicians as an off label prescription. In 2014, GlaxoSmithKline paid U.S. based Isis Pharmaceuticals USD 1 million for the advancement of the Phase III study of ISIS-TTRRx in patients with familial amyloid polyneuropathy. ISIS-TTRRx is an antisense drug which reduces the amount of mutant. Treatment with ISIS-TTRRx lowers the amount of disease causing protein found in the blood.
Although many companies initiate clinical studies in future, drug approval is subjected to various uncertainties. Due to high cost of the treatment, a small portion of the probable customer base is actually able to afford the treatment. This is a major restraint for development of the market in developing countries. Owing to early initiation of the market in the European countries, currently Europe leads the familial amyloid polyneuropathy market. As Vyndaqel (tafamidis) receives approval from regulatory authorities in the U.S. the market is expected to grow rapidly as cost of drugs in the U.S. is higher as compared to Europe. Moreover as companies succeed in research activates, more product will be added to the market. Advent of gene therapy is also expected to boost the market growth in the future.

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.

Custom Market Research Services

TMR offers custom market research services that help clients to get information on their business scenario required where syndicated solutions are not enough.


Familial Amyloid Polyneuropathy

Pre Book