Global Transthyretin Amyloidosis Treatment Market Size is valued at USD 5.5 Bn in 2024 and is predicted to reach USD 11.7 Bn by the year 2034 at a 8.0% CAGR during the forecast period for 2025 to 2034.
Transthyretin Amyloidosis Treatment Market Size, Share & Trends Analysis Report By Type (ATTR-PN, ATTR-CM), By Therapy (Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax), Supportive Therapy, Pipeline Therapy), By Disease Type (Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type), Wild Type Amyloidosis), By Distribution Channel, By Region, And By Segment Forecasts, 2026 to 2035
Transthyretin amyloidosis (ATTR) is an uncommon and progressive condition resulting from the misfolding of transthyretin (TTR) proteins, which leads to amyloid accumulation in organs and tissues, mostly impacting the heart and nervous system.
Patients undergoing transthyretin amyloidosis treatment are expected to be driven by the rising elderly patient demographic and the growing prevalence of transthyretin amyloidosis among these patients. According to a 2022 study published in the European Journal of Heart Failure, patients aged 70 years and older were found to have transthyretin amyloidosis (ATTR).
Furthermore, the ageing population is driving the industry, especially in developed nations, as the condition is more prevalent in older people. For instance, transthyretin amyloidosis (ATTRwt) of the wild-type primarily affects older males; postmortem reports indicate that transthyretin amyloid cardiac deposits are present in 25% of patients over the age of 80. Therefore, it is anticipated that the incidence rates of transthyretin amyloidosis illness will rise in tandem with the worldwide elderly population, driving the transthyretin amyloidosis treatment market expansion.
The transthyretin amyloidosis treatment market is segmented based on type, therapy, disease type, and distribution channel. Based on type, the market is segmented into ATTR-PN and ATTR-CM. By therapy, the market is divided into Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax), Supportive Therapy, and Pipeline Therapy. By disease type, the market is segmented into Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type) and Wild Type Amyloidosis. The distribution channel segment includes Hospital Pharmacies, Specialty Pharmacies, Retail Pharmacies, and Online Pharmacies.
The ATTR-CM category is expected to hold a major global market share in 2023. This is caused by the high frequency of disease and the rapid improvement in diagnostic rates. Although the precise frequency of the condition is unknown, different autopsy reports indicate that between 22% and 25% of individuals 80 years of age and older had heart TTR amyloid accumulation. Even yet, patients with ATTR-CM amyloidosis typically have modest amyloid deposition. Additionally, the introduction of new medications to treat ATTR-CM is anticipated to accelerate the growth of the transthyretin amyloidosis treatment market. For instance, the U.S. FDA approved Pfizer Inc.'s VYNDAQEL and VYNDAMAX in May 2019 for the treatment of ATTR-CM.
The majority of hospitals focus on the diagnosis, investigation, and treatment of the illness. For instance, the goal of Massachusetts-based Brigham and Women's Hospital is to identify and treat diseases that have been misdiagnosed. It launched the Amyloidosis Program, providing patient-centred care, access to cutting-edge treatments, and new clinical trials. The group of professionals would raise awareness among patients and educate doctors and patients. These programs would help the transthyretin amyloidosis treatment market expand even further.
The North American transthyretin amyloidosis treatment market is expected to note the highest market share in revenue in the near future. Strong reimbursement rules, a well-established healthcare infrastructure, and a high level of patient and healthcare professional knowledge regarding rare diseases are among the region's many strengths. The industry's expansion in North America is further aided by the existence of significant market participants and continuous research and development initiatives. Furthermore, the region's early adoption of cutting-edge medicines and a strong focus on innovation have increased its market share in transthyretin amyloidosis treatment. In addition, the Middle East and Africa are projected to grow rapidly in the global transthyretin amyloidosis treatment market. The high unmet treatment demands, increasing approval rates, and rising ailment prevalence are expected to boost the transthyretin amyloidosis treatment market in the following years. Some important factors that are expected to be the key drivers of growth are a large target population, sensible healthcare reforms, and an increase in the awareness of illnesses.
| Report Attribute | Specifications |
| Market Size Value In 2024 | USD 5.5 Bn |
| Revenue Forecast In 2034 | USD 9.4 Bn |
| Growth Rate CAGR | CAGR of 8.0% from 2025 to 2034 |
| Quantitative Units | Representation of revenue in US$ Million and CAGR from 2025 to 2034 |
| Historic Year | 2021 to 2024 |
| Forecast Year | 2025-2034 |
| Report Coverage | The forecast of revenue, the position of the company, the competitive market structure, growth prospects, and trends |
| Segments Covered | By Type, Therapy, Disease Type, And Distribution Channel |
| Regional Scope | North America; Europe; Asia Pacific; Latin America; Middle East & Africa |
| Country Scope | U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico; France; Italy; Spain; South East Asia; South Korea |
| Competitive Landscape | Ionis Pharmaceuticals, Inc., Pfizer Inc., AstraZeneca, Johnson & Johnson Services, Inc., Alnylam Pharmaceuticals, Inc., Astellas Pharma, Inc.. BridgeBio Pharma, Inc., Bristol-Myers Squibb Company, Acrotech Biopharma, Prothena, SOM Biotech, and Others |
| Customization Scope | Free customization report with the procurement of the report and modifications to the regional and segment scope. Particular Geographic competitive landscape. |
| Pricing And Available Payment Methods | Explore pricing alternatives that are customized to your particular study requirements. |
This study employed a multi-step, mixed-method research approach that integrates:
This approach ensures a balanced and validated understanding of both macro- and micro-level market factors influencing the market.
Secondary research for this study involved the collection, review, and analysis of publicly available and paid data sources to build the initial fact base, understand historical market behaviour, identify data gaps, and refine the hypotheses for primary research.
Secondary data for the market study was gathered from multiple credible sources, including:
These sources were used to compile historical data, market volumes/prices, industry trends, technological developments, and competitive insights.
Primary research was conducted to validate secondary data, understand real-time market dynamics, capture price points and adoption trends, and verify the assumptions used in the market modelling.
Primary interviews for this study involved:
Interviews were conducted via:
Primary insights were incorporated into demand modelling, pricing analysis, technology evaluation, and market share estimation.
All collected data were processed and normalized to ensure consistency and comparability across regions and time frames.
The data validation process included:
This ensured that the dataset used for modelling was clean, robust, and reliable.
The bottom-up approach involved aggregating segment-level data, such as:
This method was primarily used when detailed micro-level market data were available.
The top-down approach used macro-level indicators:
This approach was used for segments where granular data were limited or inconsistent.
To ensure accuracy, a triangulated hybrid model was used. This included:
This multi-angle validation yielded the final market size.
Market forecasts were developed using a combination of time-series modelling, adoption curve analysis, and driver-based forecasting tools.
Given inherent uncertainties, three scenarios were constructed:
Sensitivity testing was conducted on key variables, including pricing, demand elasticity, and regional adoption.