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Global Transthyretin Amyloidosis Treatment Market Share, Size, Growth, Analysis: By Drug Type: Tafamidis, Diflunisal, Patisiran, Inotersen, Others; By Disease Type: Hereditary Type Transthyretin Amyloidosis, Wild Type Transthyretin Amyloidosis; By Distribution Channel: Hospital Pharmacies, Others; Regional Analysis; Supplier Landscape; 2023-2031

Global Transthyretin Amyloidosis Treatment Market Outlook

The growth of the transthyretin amyloidosis treatment market is driven by the rising prevalence of hereditary transthyretin amyloidosis syndrome. The prevalence of the disease is likely to grow at a CAGR of 15% during the forecast period of 2023-2031.

 

Transthyretin Amyloidosis Syndrome: Insights, Types, Causes, Symptoms, and Treatment

Transthyretin amyloidosis is a protein disorder affecting the pumping system of the heart. In this disorder, there is the build-up of irregular proteins called fibrils in the heart. These proteins harden the left ventricle and the main pumping segment, causing difficulty in regular blood flow. This disease is also called cardiac amyloidosis, amyloidosis ATTR, and TTR amyloidosis.

 

The two primary transthyretin amyloidosis disorders are hereditary ATTR-CM and wild-type ATTR-CM. In hereditary ATTR-CM, amyloids are formed due to the mutational changes in the TTR gene, while in wild-type ATTR-CM, the cause is unknown, mainly affecting the cardiovascular and nervous systems.

 

Transthyretin amyloidosis syndrome is a hereditary disorder caused due to changes in the TTR gene. This results in the production of improper TTR proteins, which misfold themselves and form deposits of amyloid fibrils. These fibrils flow with the blood passage and accumulate in the nerves or veins of the heart.

 

The most common symptoms of transthyretin amyloidosis syndrome include bloated stomach, heart palpitations, irregular heart rate, coughing, dizziness, shortness of breath and fatigue., among others.

 

There is no approved treatment available for the management of transthyretin amyloidosis syndrome yet but certain medications have been proven beneficial for treating this syndrome.

 

According to the transthyretin amyloidosis treatment market research report, the market can be categorised into the following segments:

 

Market Breakup by Drug Type

  • Tafamidis
  • Diflunisal
  • Patisiran
  • Inotersen
  • Others

 

Market Breakup by Disease Type

  • Hereditary Type Transthyretin Amyloidosis
    • Polyneuropathy
    • Cardiomyopathy
    • Mixed Type
  • Wild Type Transthyretin Amyloidosis

 

Market Breakup by Distribution Channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

 

Market Breakup by Region

  • North America
    • United States of America
    • Canada
  • Europe
    • United Kingdom
    • Germany
    • France
    • Italy
    • Others
  • Asia Pacific
    • China
    • Japan
    • India
    • ASEAN
    • Australia
    • Others
  • Latin America
    • Brazil
    • Argentina
    • Mexico
    • Others
  • Middle East and Africa
    • Saudi Arabia
    • United Arab Emirates
    • Nigeria
    • South Africa
    • Others

 

Transthyretin Amyloidosis Treatment Market Scenario

The growth of the market is primarily driven by the rising prevalence of hereditary transthyretin amyloidosis syndrome. With the increasing prevalence, the demand for the available drugs increases, contributing significantly to the market growth.

 

In developed countries, the increasing awareness about the transthyretin amyloidosis syndrome is anticipated to boost the expansion of the market. 

 

The novel product launches and approvals are also aiding the market growth. For example, in 2019, the United States FDA approved the two drugs manufactured by Pfizer. This will help in the generation of more significant revenues and profits.

 

Additionally, evolving healthcare infrastructure sector, faster economic growth of countries, and significant improvement in diagnostic procedures are attributed to the development of the transthyretin amyloidosis treatment market during the forecast period.

 

North America Accounts for the Largest Share

North America is anticipated to dominate all the regional markets due to the increasing prevalence of transthyretin amyloidosis syndrome in the United States. According to the National Organisation for Rare Disorders, nearly one in 100,000 North Americans are affected by transthyretin amyloidosis syndrome. Thus, this rising number of cases augments the transthyretin amyloidosis treatment market growth.

 

Therapeutic Landscape

There is no specific treatment regimen for the complete eradication of transthyretin amyloidosis syndrome. However, several medications are used to minimise the symptoms and control the severity of the disease. The main goal of the transthyretin amyloidosis treatment is to stop syndrome progression by hindering the growth of amyloidosis deposits.

 

Standard Medications and Therapeutic Interventions

The exact treatment for transthyretin amyloidosis syndrome depends on the syndrome subtype, its severity, and the patient’s morphology. In cardiac amyloidosis, medications are prescribed to alleviate cardiovascular symptoms. The most used drugs are Tafamidis to treat cardiomyopathy, diuretics to reduce swelling, and blood thinners for thinning the blood concentrations.

 

In severe cases of transthyretin amyloidosis syndrome, organ removal or replacement is considered to be the last therapeutic option. Heart transplants and liver transplants are two major surgical procedures that are used to limit the build-up of amyloidosis deposits.

 

Cell therapies also find wide application in treating the modified TTR gene. Novel therapeutic options incorporating genetic therapies are under process and, once approved, can pave the way for the significant growth of the transthyretin amyloidosis treatment market during the forecast period.

 

Clinical Trial Landscape

With extensive research and developmental activities, there is the possibility to discover new therapeutic interventions. The ongoing clinical trials focus on finding novel TTR stabilisers and siRNA drugs. The existing medications, including doxycycline, have also shown some promising effects in fibril reabsorption and degradation.

 

Emerging Drugs and Gene Alteration Technologies in the Transthyretin Amyloidosis Treatment Market

Inotersen, a TTR gene inhibitor, is under Phase II clinical trials to test its safety and efficacy. FDA has already approved this drug for treating ATTR-related polyneuropathy. However, the undergoing clinical trials are ongoing for ATTR cardiomyopathy.

 

Tafamidis, a specific tetramer stabiliser, works by blocking the rate-limiting step in the transthyretin’s genesis. This drug is under post-marketing surveillance to monitor its clinical indications, doses, and side effects.

 

Doxycycline-tauroursodeoxycholic acid (Doxy/TUDCA) has shown positive results in in-vitro studies by limiting the syndrome’s progression. This medication is under Phase III clinical trials for further evaluation.

 

PRX004, a monoclonal antibody, is specifically discovered to target transthyretin amyloid deposits and is under the initial phases of clinical testing. Further assessment and evaluation are needed to test its safety and efficacy in animal models.

 

Currently, many drugs are in various stages of clinical trials. These novel treatments are anticipated to enhance the prognosis of patients with a precise diagnosis of transthyretin amyloidosis syndrome. Technological advancements are propelling the discovery of new therapeutic agents, thus contributing to the share in the market.

 

Competitive Landscape

The report gives an in-depth analysis of the key players involved in the transthyretin amyloidosis treatment market, sponsors manufacturing the therapies, and putting them through trials to get FDA approvals. The companies included in the market are as follows:

 

  • Sorrento Therapeutics, Inc.
  • Astellas Pharma Inc.
  • AstraZeneca plc
  • Alexion Pharmaceuticals, Inc.
  • Spectrum Pharmaceuticals, Inc.
  • Pfizer Inc.
  • Alnylam Pharmaceuticals, Inc.
  • Ionis Pharmaceuticals, Inc.
  • Eidos Therapeutics, Inc.
  • Corino Therapeutics, Inc.
  • Prothena Corporation plc

 

Key Highlights of the Report

REPORT FEATURES DETAILS
Base Year 2022
Historical Period 2016-2022
Forecast Period 2023-2031
Scope of the Report

Historical and Forecast Trends, Industry Drivers and Constraints, Historical and Forecast Market Analysis by Segment:

  • Drug Type
  • Disease Type
  • Distribution Channel
  • Region
Breakup by Drug Type
  • Tafamidis
  • Diflunisal
  • Patisiran
  • Inotersen
  • Others
Breakup by Disease Type
  • Hereditary Type Transthyretin Amyloidosis
  • Wild Type Transthyretin Amyloidosis
Breakup by Distribution Channel
  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies
Breakup by Region
  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East and Africa
Market Dynamics
  • SWOT
  • Porter's Five Forces
  • Key Indicators for Demand
  • Key Indicators for Price
Supplier Landscape
  • Market Structure
  • Company Profiles
    • Company Overview
    • Product Portfolio
    • Demographic Reach and Achievements
    • Mergers and Acquisitions
    • Certifications
Companies Covered
  • Sorrento Therapeutics, Inc.
  • Astellas Pharma Inc.
  • AstraZeneca plc
  • Alexion Pharmaceuticals, Inc.
  • Spectrum Pharmaceuticals, Inc.
  • Pfizer Inc.
  • Alnylam Pharmaceuticals, Inc.
  • Ionis Pharmaceuticals, Inc.
  • Eidos Therapeutics, Inc.
  • Corino Therapeutics, Inc.
  • Prothena Corporation plc

 

*At Expert Market Research, we strive to always give you current and accurate information. The numbers depicted in the description are indicative and may differ from the actual numbers in the final EMR report.

1    Preface
    1.1    Objectives of the Study
        1.1.1    Research Objectives
        1.1.2    Key Findings of the Report
    1.2    Limitations of the Study and Scope for Future Research
2    Research Methodology
3    Executive Summary
4    Global Transthyretin Amyloidosis Overview 

    4.1    Guidelines and Stages
    4.2    Pathophysiology
    4.3    Screening and Diagnosis
    4.4    Treatment Pathway 
5    Patient Profile
    5.1    Patient Profile Overview
    5.2    Patient Psychology and Emotional Impact Factors
    5.3    Risk Assessment and Treatment Success Rate
6    Global Transthyretin Amyloidosis Treatment Market 
    6.1    Global Transthyretin Amyloidosis Treatment Market Overview   
    6.2    Global Transthyretin Amyloidosis Treatment Market Analysis 
        6.2.1    Market Overview
            6.2.1.1    Global Transthyretin Amyloidosis Treatment Market Historical Value (2016-2022) 
            6.2.1.2    Global Transthyretin Amyloidosis Treatment Market Forecast Value (2023-2031)
    6.3     Global Transthyretin Amyloidosis Treatment Market by Drug Type
        6.3.1    Market Overview
            6.3.1.1    Tafamidis
            6.3.1.2    Diflunisal
            6.3.1.3    Patisiran
            6.3.1.4    Inotersen
            6.3.1.5    Others
    6.4    Global Transthyretin Amyloidosis Treatment Market by Disease Type
        6.4.1    Market Overview
            6.4.1.1    Hereditary Type Transthyretin Amyloidosis 
                6.4.1.1.1    Polyneuropathy
                6.4.1.1.2    Cardiomyopathy
                6.4.1.1.3    Mixed Type
            6.4.1.2    Wild Type Transthyretin Amyloidosis
    6.5    Global Transthyretin Amyloidosis Treatment Market by Distribution Channel
        6.5.1    Market Overview
            6.5.1.1    Hospital Pharmacies
            6.5.1.2    Retail Pharmacies
            6.5.1.3    Online Pharmacies  
    6.6    Global Transthyretin Amyloidosis Treatment Market by Region
        6.6.1    Market Overview
            6.6.1.1    North America 
                6.6.1.1.1    Historical Trend (2016-2022)
                6.6.1.1.2    Forecast Trend (2023-2031)
            6.6.1.2    Europe
                6.6.1.2.1    Historical Trend (2016-2022)
                6.6.1.2.2    Forecast Trend (2023-2031)
            6.6.1.3    Asia Pacific
                6.6.1.3.1    Historical Trend (2016-2022)
                6.6.1.3.2    Forecast Trend (2023-2031)
            6.6.1.4    Latin America
                6.6.1.4.1    Historical Trend (2016-2022)
                6.6.1.4.2    Forecast Trend (2023-2031)
            6.6.1.5    Middle East and Africa
                6.6.1.5.1     Historical Trend (2016-2022)
                6.6.1.5.2    Forecast Trend (2023-2031)
7    North America Transthyretin Amyloidosis Treatment Market
    7.1    United States of America
        7.1.1    Historical Trend (2016-2022)
        7.1.2    Forecast Trend (2023-2031)
    7.2    Canada 
        7.2.1    Historical Trend (2016-2022)
        7.2.2    Forecast Trend (2023-2031)
8    Europe Transthyretin Amyloidosis Treatment Market
    8.1    United Kingdom
        8.1.1    Historical Trend (2016-2022)
        8.1.2    Forecast Trend (2023-2031)
    8.2    Germany
        8.2.1    Historical Trend (2016-2022)
        8.2.2    Forecast Trend (2023-2031)
    8.3    France
        8.3.1    Historical Trend (2016-2022)
        8.3.2    Forecast Trend (2023-2031)
    8.4    Italy
        8.4.1    Historical Trend (2016-2022)
        8.4.2    Forecast Trend (2023-2031)
9    Asia Pacific Transthyretin Amyloidosis Treatment Market
    9.1    China
        9.1.1    Historical Trend (2016-2022)
        9.1.2    Forecast Trend (2023-2031)
    9.2    Japan
        9.2.1    Historical Trend (2016-2022)
        9.2.2    Forecast Trend (2023-2031)
    9.3    India
        9.3.1    Historical Trend (2016-2022)
        9.3.2    Forecast Trend (2023-2031)
    9.4    ASEAN
        9.4.1    Historical Trend (2016-2022)
        9.4.2    Forecast Trend (2023-2031)
    9.5    Australia
        9.5.1    Historical Trend (2016-2022)
        9.5.2    Forecast Trend (2023-2031)
10    Latin America Transthyretin Amyloidosis Treatment Market
    10.1    Brazil
        10.1.1    Historical Trend (2016-2022)
        10.1.2    Forecast Trend (2023-2031)
    10.2    Argentina
        10.2.1    Historical Trend (2016-2022)
        10.2.2    Forecast Trend (2023-2031)
    10.3    Mexico
        10.3.1    Historical Trend (2016-2022)
        10.3.2    Forecast Trend (2023-2031)
11    Middle East and Africa Transthyretin Amyloidosis Treatment Market
    11.1    Saudi Arabia
        11.1.1    Historical Trend (2016-2022)
        11.1.2    Forecast Trend (2023-2031)
    11.2    United Arab Emirates
        11.2.1    Historical Trend (2016-2022)
        11.2.2    Forecast Trend (2023-2031)
    11.3    Nigeria
        11.3.1    Historical Trend (2016-2022)
        11.3.2    Forecast Trend (2023-2031)
    11.4    South Africa
        11.4.1    Historical Trend (2016-2022)
        11.4.2    Forecast Trend (2023-2031)
12    Current Scenario Evaluation and Regulatory Framework
    12.1    Emerging Therapies and Clinical Trials Synopsis
    12.2    Patent Landscape
        12.2.1    Patent Overview
            12.2.1.1    Patent Status and Expiry
            12.2.1.2    Timelines from Drug Development to Commercial Launch
            12.2.1.3    New Drug Application
                12.2.1.3.1    Documentation and Approval Process
    12.3    Cost of Treatment
    12.4    Regulatory Framework
        12.4.1    Regulatory Overview
            12.4.1.1    US FDA
            12.4.1.2    EU EMA
            12.4.1.3    INDIA CDSCO
            12.4.1.4    JAPAN PMDA
            12.4.1.5    Others
13    Challenges and Unmet Needs
    13.1    Treatment Pathway Challenges
    13.2    Compliance and Drop-Out Analysis
    13.3    Awareness and Prevention Gaps
14    Global Transthyretin Amyloidosis Treatment Market Dynamics
    14.1    Market Drivers and Constraints
    14.2    SWOT Analysis
    14.3    Porter’s Five Forces Model
    14.4    Key Demand Indicators
    14.5    Key Price Indicators
    14.6    Industry Events, Initiatives and Trends
    14.7    Value Chain Analysis
15    Supplier Landscape
    15.1    Sorrento Therapeutics, Inc.
        15.1.1    Company Overview
        15.1.2    Product Portfolio
        15.1.3    Demographic Reach and Achievements
        15.1.4    Mergers and Acquisitions
        15.1.5    Certifications
    15.2    Astellas Pharma Inc.
        15.2.1    Company Overview
        15.2.2    Product Portfolio
        15.2.3    Demographic Reach and Achievements
        15.2.4    Mergers and Acquisitions
        15.2.5    Certifications
    15.3     AstraZeneca plc
        15.3.1    Company Overview
        15.3.2    Product Portfolio
        15.3.3    Demographic Reach and Achievements
        15.3.4    Mergers and Acquisitions
        15.3.5    Certifications
    15.4     Alexion Pharmaceuticals, Inc.
        15.4.1    Company Overview
        15.4.2    Product Portfolio
        15.4.3    Demographic Reach and Achievements
        15.4.4    Mergers and Acquisitions
        15.4.5    Certifications
    15.5    Spectrum Pharmaceuticals, Inc.
        15.5.1    Company Overview
        15.5.2    Product Portfolio
        15.5.3    Demographic Reach and Achievements
        15.5.4    Mergers and Acquisitions
        15.5.5    Certifications
    15.6    Pfizer Inc.
        15.6.1    Company Overview
        15.6.2    Product Portfolio
        15.6.3    Demographic Reach and Achievements
        15.6.4    Mergers and Acquisitions
        15.6.5    Certifications
    15.7    Alnylam Pharmaceuticals, Inc.
        15.7.1    Company Overview
        15.7.2    Product Portfolio
        15.7.3    Demographic Reach and Achievements
        15.7.4    Mergers and Acquisitions
        15.7.5    Certifications 
    15.8    Ionis Pharmaceuticals, Inc.
        15.8.1    Company Overview
        15.8.2    Product Portfolio
        15.8.3    Demographic Reach and Achievements
        15.8.4    Mergers and Acquisitions
        15.8.5    Certifications 
    15.9    Eidos Therapeutics, Inc.
        15.9.1    Company Overview
        15.9.2    Product Portfolio
        15.9.3    Demographic Reach and Achievements
        15.9.4    Mergers and Acquisitions
        15.9.5    Certifications
    15.10    Corino Therapeutics, Inc.
        15.10.1    Company Overview
        15.10.2    Product Portfolio
        15.10.3    Demographic Reach and Achievements
        15.10.4    Mergers and Acquisitions
        15.10.5    Certifications 
    15.11    Prothena Corporation plc
        15.11.1    Company Overview
        15.11.2    Product Portfolio
        15.11.3    Demographic Reach and Achievements
        15.11.4    Mergers and Acquisitions
        15.11.5    Certifications
16    Recommendations and Discussion
17    Global Transthyretin Amyloidosis Treatment Drugs Distribution Model (Additional Insight)

    17.1    Overview 
    17.2    Potential Distributors 
    17.3    Key Parameters for Distribution Partner Assessment
18    Payment Methods (Additional Insight)
    18.1    Government Funded
    18.2    Private Insurance
    18.3    Out-of-Pocket

 

*Additional insights provided are customisable as per client requirements.

Key Questions Answered in the Report

The prevalence of the disease is likely to grow at a CAGR of 15% during the forecast period of 2023-2031.

The increasing prevalence of hereditary transthyretin amyloidosis syndrome, evolving healthcare infrastructure, and rapid improvements in diagnostic procedures are driving the growth of the market.

Based on the drug type, the market can be segmented into tafamidis, diflunisal, patisiran, and inotersen, among others.

Based on the disease type, the market is divided into hereditary and wild-type transthyretin amyloidosis syndrome.

Based on the distribution channel, the market can be categorised into hospital pharmacies, online pharmacies, and retail pharmacies.

The regional markets can be categorised into North America, Latin America, Europe, Asia Pacific, and the Middle East and Africa. North America is anticipated to hold the maximum share of the global market.

The key players in the market include Sorrento Therapeutics, Inc., Astellas Pharma Inc., AstraZeneca plc, Alexion Pharmaceuticals, Inc., Spectrum Pharmaceuticals, Inc., Pfizer Inc., Alnylam Pharmaceuticals, Inc., Ionis Pharmaceuticals, Inc., Eidos Therapeutics, Inc., Corino Therapeutics, Inc., and Prothena Corporation plc, among others.

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