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Transthyretin Amyloidosis Treatment Market – By Type (ATTR-CM, ATTR-PN), Disease Type (Hereditary, Wild Type), Drug Type (Tafamidis, Patisiran), Route of Administration (Oral, Parenteral), Distribution Channel (Hospital Pharmacy, Retail Drug Store) – Global Forecast 2024 – 2032

  • Report ID: GMI8484
  • Published Date: Mar 2024
  • Report Format: PDF

Transthyretin Amyloidosis Treatment Market Size

Transthyretin Amyloidosis Treatment Market size was valued at USD 5.9 billion in 2023 and is expected to reach USD 11.2 billion by 2032, growing at a CAGR of 7.5% over the analysis period. Increasing prevalence of transthyretin amyloidosis, a rare genetic disorder, coupled with advancements in treatment options for rare diseases are fuelling market expansion.

Transthyretin Amyloidosis Treatment Market

Additionally, as the disease is more common in older individuals, rising aging population, particularly in developed countries, is contributing to market growth. For instance, wild-type transthyretin amyloidosis (ATTRwt) mainly affects older men, and certain autopsy studies have shown that 25% of patients aged above 80 years old have transthyretin amyloid myocardial deposits. Thus, as the global geriatric population rises, the incidence rates of transthyretin amyloidosis disease are expected to increase, thereby fostering the market growth.

Transthyretin amyloidosis (ATTR) treatment refers to the medical interventions aimed at managing and slowing the progression of ATTR amyloidosis, a rare, progressive, and often fatal disease caused by the buildup of abnormal transthyretin protein in tissues and organs. Treatment approaches include medications to stabilize or reduce the production of transthyretin and supportive therapies to manage symptoms and complications.

Transthyretin Amyloidosis Treatment Market Trends

  • Advancements in diagnostic techniques has significantly contributed to the market growth. With the development of more sensitive imaging modalities such as cardiac MRI and nuclear scintigraphy, along with improved genetic testing methods, early and accurate diagnosis has become more achievable.
  • For instance, recent advancements in nuclear cardiac imaging with technetium pyrophosphate scan, the diagnosis of ATTR-CM is possible without cardiac biopsy. Therefore, more patients are being screened and diagnosed with ATTR-CM.
  • These advancements not only facilitate timely treatment initiation but also enable better patient management and monitoring. Additionally, the introduction of novel biomarkers and the ongoing research into new diagnostic technologies promise further improvements in the diagnosis and management of transthyretin amyloidosis, propelling the market growth, and in turn propelling the rare disease treatment market as well.

Transthyretin Amyloidosis Treatment Market Analysis

Transthyretin Amyloidosis Treatment Market, By Type, 2021 – 2032 (USD Billion)

Based on the type, the global market is classified into ATTR amyloidosis with cardiomyopathy (ATTR-CM) and ATTR amyloidosis with polyneuropathy (ATTR-PN). The ATTR-CM dominated the market with revenue of USD 4.7 billion in 2023.

  • ATTR-CM is a form of amyloidosis characterized by the deposition of abnormal transthyretin protein in the heart, leading to heart failure. This segment's prominence is driven by the rising prevalence of ATTR-CM, coupled with advancements in diagnostics techniques that aid in timely intervention.
  • Further, the development of novel therapeutics, including gene silencing therapies and stabilizers of transthyretin protein has also fueled the growth of this segment.
  • Additionally, increasing awareness among healthcare professionals and patients about the disease and its treatment options has contributed to the ATTR-CM segment's high market share.


Transthyretin Amyloidosis Treatment Market, By Disease Type (2023)

Based on disease type, the transthyretin amyloidosis treatment market is classified into hereditary transthyretin amyloidosis and wild type transthyretin amyloidosis. The hereditary transthyretin amyloidosis is further segmented into polyneuropathy, cardiomyopathy, and mixed type. The segment held a dominant business share of 61.7% in 2023.

  • Hereditary transthyretin amyloidosis (hATTR) holds a significant market share in the market, driven by its genetic predisposition and progressive nature. hATTR is the most common form of transthyretin amyloidosis, accounting for a significant majority of cases. This prevalence increases the demand for targeted therapies specific to hATTR.
  • Additionally, the genetic nature of hATTR necessitates long-term treatment, further driving the segment growth.
  • Moreover, the severity and progressive nature of hATTR, often leads to debilitating neuropathy and cardiac complications, emphasizing the urgent need for effective treatments, spurring research and development efforts in this area.

Based on drug type, the transthyretin amyloidosis treatment market is classified into tafamidis, patisiran, inotersen, and other drug types. The tafamidis segment is expected to hold the highest revenue throughout the forecast period.

  • High market share of tafamidis drug can be attributed to its high efficacy and safety profile. It works by stabilizing the transthyretin protein, preventing its breakdown into amyloid fibrils, and thereby slowing the progression of the disease.
  • The drug's success in clinical trials, coupled with its approval by regulatory bodies around the world, has made it a cornerstone in the management of transthyretin amyloidosis, contributing to its high market share in the treatment landscape.

Based on route of administration, the transthyretin amyloidosis treatment market is classified into oral and parenteral. The oral segment is anticipated to grow at a CAGR of 7.2% between 2024 – 2032.

  • Oral medications offer convenience and ease of administration, leading to better patient compliance and treatment outcomes.
  • Additionally, oral medications have a more favorable safety profile compared to other treatment modalities, reducing the risk of adverse events.
  • The development of novel oral therapies targeting transthyretin amyloidosis has further boosted the growth of this segment, providing patients with more treatment options, and potentially improving their quality of life.

Based on distribution channel, the transthyretin amyloidosis treatment market is classified into hospital pharmacy, retail drug stores, and online pharmacy. The hospital pharmacy segment is expected to be valued at USD 5 billion by 2032.

  • High segment growth is primarily fueled by the well-equipped nature of the hospital pharmacies to handle these specialized medications, ensuring that patients receive the exact formulations prescribed by their healthcare providers.
  • Moreover, hospital pharmacies are staffed by pharmacists with clinical expertise who work closely with healthcare providers to ensure accurate and safe medication management for patients with complex condition of transthyretin amyloidosis.
  • Further, high volume of patients visiting hospitals, drives the segmental growth of hospital pharmacy segment.


North America Transthyretin Amyloidosis Treatment Market, 2020 – 2032 (USD Billion)

In 2023, North America held a market share of 37.6% in the global transthyretin amyloidosis treatment and is expected to dominate throughout the forecast period.

  • The region boasts of a well-established healthcare infrastructure, high awareness about rare diseases among healthcare professionals and patients, and robust reimbursement policies. Additionally, the presence of key market players and ongoing research and development activities contribute to the growth of this market in North America.
  • Further, the region's strong focus on innovation and early adoption of advanced therapies further enhances its market share in transthyretin amyloidosis treatment.

Transthyretin Amyloidosis Treatment Market Share

The competitive landscape of the market is characterized by the presence of established pharmaceutical companies vying for market share. Key players are engaging in strategic initiatives such as mergers, acquisitions, and partnerships to strengthen their product portfolios. The companies are actively adopting multi-pronged approaches to address the rising demand for new treatments and strengthen their market presence.

Transthyretin Amyloidosis Treatment Market Companies

Prominent players operating in the transthyretin amyloidosis treatment industry are as mentioned below:

  • Acrotech Biopharma, LLC
  • Alnylam Pharmaceuticals, Inc.
  • AstraZeneca PLC
  • Astellas Pharma Inc.
  • BridgeBio Pharma, Inc.
  • Bristol-Myers Squibb Company
  • Ionis Pharmaceuticals, Inc.
  • Johnson & Johnson
  • Prothena Biosciences Limited
  • Pfizer Inc.
  • SOM Innovation Biotech, S.L.

Transthyretin Amyloidosis Treatment Industry News:

  • In January 2022, AstraZeneca received Orphan Drug Designation (ODD) in the U.S. by the Food and Drug Administration (FDA) for Eplontersen drug for the treatment of transthyretin-mediated amyloidosis. This helped the company to expand its product portfolio.
  • In June 2022, Alnylam Pharmaceuticals, Inc. received FDA authorization for AMVUTTRA (vutrisiran) for the treatment of Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis. This helped the company to market its products in the U.S. and expand its customer base.

The transthyretin amyloidosis treatment market research report includes an in-depth coverage of the industry with estimates & forecast in terms of revenue in USD Million from 2018 - 2032 for the following segments:

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Market, By Type

  • ATTR amyloidosis with polyneuropathy (ATTR-PN)
  • ATTR amyloidosis with cardiomyopathy (ATTR-CM)

Market, By Disease Type

  • Hereditary transthyretin amyloidosis
    • Polyneuropathy
    • Cardiomyopathy
    • Mixed type 
  • Wild type transthyretin amyloidosis

Market, By Drug Type

  • Tafamidis
  • Patisiran
  • Inotersen
  • Other drug types

Market, By Route of Administration

  • Oral
  • Parenteral

Market, By Distribution Channel

  • Hospital pharmacy
  • Retail drug stores
  • Online pharmacy

The above information is provided for the following regions and countries:

  • North America
    • U.S.
    • Canada 
  • Europe
    • Germany
    • UK
    • France
    • Spain
    • Italy
    • Rest of Europe
  • Asia Pacific
    • Japan
    • China
    • India
    • Australia
    • Rest of Asia Pacific
  • Latin America
    • Brazil
    • Mexico
    • Rest of Latin America
  • Middle East and Africa
    • South Africa
    • Saudi Arabia
    • Rest of Middle East and Africa


Authors: Mariam Faizullabhoy, Gauri Wani

Frequently Asked Questions (FAQ) :

Transthyretin amyloidosis treatment industry was valued at USD 5.9 billion in 2023 and is poised to expand at 7.5% CAGR between 2024 and 2032 owing to the increasing prevalence of transthyretin amyloidosis, a rare genetic disorder, coupled with advancements in treatment options for rare diseases.

The ATTR-CM segment generated USD 4.7 billion in revenue in 2023 and is estimated to grow at a notable CAGR from 2024-2032 owing to the rising advancements in diagnostics techniques that aid in timely intervention.

North America accounted 37.6% revenue share of the transthyretin amyloidosis treatment industry in 2023 and is set to expand at a considerable growth rate through 2032 due to well-established healthcare infrastructure, and high awareness about rare diseases among healthcare professionals and patients.

Some of the top transthyretin amyloidosis treatment companies are Acrotech Biopharma, LLC, AstraZeneca PLC, Astellas Pharma Inc., BridgeBio Pharma, Inc., Bristol-Myers Squibb Company, Ionis Pharmaceuticals, Inc., Johnson & Johnson, Prothena Biosciences Limited, Pfizer Inc., SOM Innovation Biotech, S.L.

Transthyretin Amyloidosis Treatment Market Scope

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Premium Report Details

  • Base Year: 2023
  • Companies covered: 11
  • Tables & Figures: 299
  • Countries covered: 19
  • Pages: 170
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