UK ATTR Market Analysis


  • Type: Rare Disease Area
  • Published : April 2021
  • Region: Europe
  • Country: UK
  • Report ID : 2567

  • Format: PPT, PDF

UK Transthyretin Amyloidosis Therapeutics Market: Segmented by Product Types, Genetic Based Therapies, TTR Stabilizer, CRISPR/Cas Gene Editing Therapy, and Product Application – Size, Share, Impact, Growth, Trends, and Forecasts (2019 – 2028)

Published Date: June 2021


SKU: 2567 Categories: ,

Report Overview

The UK Transthyretin Amyloidosis (ATTR) Therapeutics market size stood at around USD 0.012 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Transthyretin Amyloidosis (ATTR) is caused by the transthyretin (TTR) protein and occurs in two forms, either hereditary or wild type. Transthyretin amyloidosis cardiomyopathy (ATTR-CM) is an increasingly recognized cause of heart failure in older individuals. The United Kingdom has the lowest prevalent population in 2016.

Till 2019, 711 patients with wild-type ATTR-CM, 205 with hereditary ATTR-CM associated with the V122I variant (V122I-hATTR-CM), and 118 with non-V122I-hATTR-CM at the UK National Amyloidosis Centre were studied. Patients with V122I-hATTR-CM were more impaired functionally and had worse measures of cardiac disease at the time of diagnosis, a greater decline in quality of life, and poorer survival in comparison with the other subgroups.

Market Growth Drivers

The growth will be driven by the surge in the development of drug therapies of varying mechanisms, and the overall rise in diagnosed patient population due to a much-needed increase in awareness and understanding of the disease among clinicians and the public.

The main driver of the ATTR market expansion will be the approval and launch of the RNA interference therapeutics for both ATTR polyneuropathy and ATTR cardiomyopathy. Another prominent contributor to sales growth is the increase in diagnosed prevalence, the increase will be caused by improved understanding and awareness of the disease alongside campaigns by drug developers to increase genetic testing.

The ATTR market currently has unlimited competition and holds room for new entrants.

The key market opportunities lie in addressing unmet needs through the development of efficacious therapies for clearing already deposited amyloid and increasing the number of therapies approved for ATTR cardiomyopathy.

Market Restraints

The major barrier for the ATTR market will be the huge annual cost of therapy for new pipeline drugs and the lack of options for advanced disease states, the stage at which the majority of patients are diagnosed.


1. UK Transthyretin Amyloidosis Therapeutics Market Overview……………..
A. Market Size
2. Market Growth Drivers and Restraints……………………………………………
Market Growth Drivers
A. Development of Drug Therapies of varying Mechanisms
B. Approval and Launch of RNA Interference Therapeutics
C. Increase in the Incidence and Prevalence Rate
Market Restraints
A. Huge Cost of the Treatment
B. Lack of Options for the Advanced Disease States
3. Major Types of Transthyretin Lymphoma…………………………………
A. Transthyretin (TTR) Familial Amyloid Cardiomyopathy
B. Transthyretin (TTR) Familial Amyloid Polyneuropathy
4. ATTR Therapeutics Market Segmentation…………………………………
A. By Products Type
I. Hereditary ATTR
II. Wild-Type ATTR
B. By Genetic Based Therapies
I. Patisiran
II. Inotersen
III. Vutrisiran
C. By TTR Stabilizer
I. AG-10
D. By CRISPR/Cas Gene Editing Therapy
I. NLTA-2001
E. By Product Application
I. Ambulatory and Surgical Centres
II. Hospitals and Clinics
III. Diagnostic Centres
IV. Others
5. ATTR Major Drugs Market Share………………………………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles………………………………………………………………….
A. Alnylam Company overview, Product & Services, Strategies & Financials
B. Pfizer Company overview, Product & Services, Strategies & Financials
C. Ionis Company, Product & Services, Strategies & Financials
D. Corino Company, Product & Services, Strategies & Financials
E. Proclara Company, Product & Services, Strategies & Financials
F. Arcturus Company, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth……………………………………………
A. New Trends and Development of ATTR Therapeutics Market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Products Type:

Various kind of drugs and other treatments options available to treat Hereditary ATTR (hATTR) and Wild type ATTR.

Hereditary (Familial) ATTR amyloidosis has an alteration (mutation) in the DNA that can be passed down from generation to generation. As a result, the TTR protein becomes more unstable and prone to forming amyloid fibrils. The most common mutation in the UK is called TTR Thr60Ala (T60A), often seen in people with Irish ancestry. Val122Ile associated hereditary ATTR amyloidosis mainly affects the heart and may also cause carpal tunnel syndrome.

Wild-type ATTR amyloidosis – Wild-type involves no aberrant DNA and cannot be handed down through the generations. Instead, the normal TTR protein becomes unstable as you get older, misfolds, and creates amyloid fibrils.

By Genetic Based Therapies:

On the basis of genetic-based therapies, the ATTR disease therapeutics market can be categorised as

  • Patisiran
  • Inotersen
  • Vutrisiran
  • Others

Regulatory authorities in the UK approved Patisiran (a small interfering RNA drug) and Inotersen (an antisense oligonucleotide drug) for treating neuropathy caused by hereditary ATTR amyloidosis. The APOLLO-B study is evaluating the efficacy and safety of Patisiran in patients with ATTR amyloidosis (hereditary or wild type) with cardiomyopathy. Vutrisiran (AL-YTTRSCO2) is a second-generation RNA inhibitor belonging to the small interfering RNA drug class. AKCEA-TTR-LRx is a second-generation RNA inhibitor, belonging to the antisense oligonucleotide drug class. Out of these, Patisiran and Inotersen, aim to “switch off” the TTR gene in the liver cells, so that TTR (both mutant and wild-type) is simply not produced, hence these two drugs are more often used to treat ATTR in the UK.

By TTR Stabilizer:

AG10 is a new selective TTR stabilizer drug being developed to treat ATTR amyloidosis. The Eidos AG10-301 study is evaluating the efficacy and safety of AG-10 in patients with symptomatic ATTR amyloidosis cardiomyopathy (hereditary and wild type).

By CRISPR/Cas Gene Editing Therapy:

CRISPR/cas9 technology is a novel gene-editing tool that allows scientists to alter genes very precisely and efficiently. NTLA-2001 is the first CRISPR therapy to be administered systemically (intravenously) to edit genes inside the body. NLTA-2001 uses a lipid nanoparticle delivery system to deliver the gene-editing CRISPR protein to the liver.  The goal is to permanently delete the gene for TTR in a single course of treatment.

By Product Application:

As per the product application, the ATTR disease treatment market can be categorised as

  • Ambulatory and Surgical Centres
  • Hospitals and Clinics
  • Diagnostic Centres
  • Others

Clinical evaluation of patients can usually be completed over 1-2 days during which hospitals or other accommodation can be arranged. Treatment is usually administered at patients’ local hospitals or at other regional centres in conjugation with advice form and reviews at the National Amyloidosis Centre. A small proportion of cases are managed directly at the Royal Free Hospital. Most patients with amyloidosis need long term surveillance, with six-monthly or annual specialist follow up in the shorter term.

Top Major Players

Many new players are emerging in the transthyretin amyloidosis (ATTR) therapeutics market, as a result of a number of growth opportunities. Some of the major key players prominent in the market are Pfizer Inc, Ionis Pharmaceuticals, Alnylam Pharmaceuticals, Corino Therapeutics, Proclara Bioscience, Arcturus Therapeutics.

Patisiran and Inotersen sold under the brand name ONPATTRO and TEGSEDI marketed by Alnylam Pharmaceuticals and Ionis Pharmaceuticals respectively.

VYNDAQEL (tafamidis), a once-daily 61 mg oral capsule for the treatment of wild type and hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

Research Methodology

Insights10 will provide you reports within 10 key parameters which are:

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or disease type
  4. Market segmentation
  5. Market share
  6. Competitive landscape
  7. Key company profiles
  8. Healthcare policies & regulatory framework
  9. Reimbursement scenario
  10. Factors driving future growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision making for business stakeholders. Our focused approach to develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

Our analysis methodology involves three critical stages:

Stage I: Market Data Collection

Primary Interviews: We have developed a network of experts, freelancers and researchers across countries through which we engage with local experts to gather key data points and assumptions around each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

Secondary Data Collection: We have developed strong expertise and experience in secondary data collection methods for developing unique data sets and research material. We gather data from multiple reliable sources to maintain the high level of accuracy and consistency. The market data is analysed and forecasted using appropriate statistical and coherent models. The report offers an overall analysis of the market size, growth, market share as well as segment-level analysis of the specific market. Our report includes precise, to the point information related to the overall market, competition, growth drivers, challenges, regulatory updates, and competition.

Data Sources: We have access to multiple highly reliable free and subscription data sources. We have many years of experience to understand which sources are more dependable for what and which to prefer for the reliable and latest information. The key sources of information include the following, but are not limited to:

  • Statistical Databases
  • Company Websites/Annual Reports
  • Trade Publications
  • Online Databases
  • Published Research Reports
  • Whitepapers
  • Press Releases of Key Market Players

Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameter and micro indicators. The macro parameters includes changes in government policies, demand and supply of the market, government intervention programs and major market share. The micro indicators GDP growth, market size, market volume etc. We also understand nuances specific to each country like US, Canada, India, Germany, etc. and have worked across 60+ countries and hence no only understand global trends, but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape and policy and regulatory scenario and the quantitative analysis covers different market estimates and forecasts.

Data Triangulation & Validation:

Data triangulation of various sources and results of the research are carried out by benchmarking with reliable sources such as industry statistics, statistical databases and the company level averages etc.

We make sure to finalize the numbers in alignment to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results are then, interpreted by considering following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
  • Analyzing current needs and determining penetration to estimate market

Insights: Our reports deliver actionable insights backed with supporting facts and figures to assist you in achieving exemplary growth. Our in-depth analyses are interspersed with relevant insights and statistics to offer an executive-level view of a given market. The description helps in correlating many minor factors affecting the market and its impact to the different segments within the market.
Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

Presentation & Reporting: The market research report is presented in different forms such as charts by using a scientific approach for easy understanding. Historic, current and future analysis is provided for each market in terms of both value and volume. The size of the market is interpreted in the US Dollar value and the respective unit, based on the product, for volume consumptions.
The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

To request a free sample copy this report, please complete the form below.

    We value your inquiry and offer free customization with every report to fulfil your exact research needs.

    This website is secure and your personal details are safe.

    This report addresses

    • Market intelligence to enable effective decision making
    • Growth opportunities and trend analysis
    • Competitive benchmarking
    • Historical data & forecasts
    • Regional opportunities

    Need more?

    • Speak to our analyst to know how this research was put together
    • Add more countries or regions as part of customisation
    • Understand how this report can have direct impact on your revenue

    Get a custom report based on your requirements