Ultra Market Research | United States Hereditary Transthyretin-Mediated Amyloidosis Market
United States Hereditary Transthyretin-Mediated Amyloidosis Market
Report ID : 1171
Category : United-States(US)
No Of Pages : 156
Published on: June 2025
Status: Published
Format :
Key Question Answer
Global Market Outlook
In-depth analysis of global and regional trends
Analyze and identify the major players in the market, their market share, key developments, etc.
To understand the capability of the major players based on products offered, financials, and strategies.
Identify disrupting products, companies, and trends.
To identify opportunities in the market.
Analyze the regional penetration of players, products, and services in the market.
Comparison of major players financial performance.
Evaluate strategies adopted by major players.
Recommendations
Introduction
The United States Hereditary Transthyretin‑Mediated Amyloidosis Market is centered around treatments of hereditary transthyretin amyloidosis (hATTR), a rare inherited disorder in which misfolded transthyretin protein aggregates lead to nerve and heart damage. Therapeutic uses involve RNA interference (e.g., vutrisiran/Amvuttra, patisiran/Onpattro), antisense oligonucleotides (e.g., inotersen/Tegsedi, eplontersen/Wainua), and stabilizers (e.g., tafamidis/Vyndaqel, acoramidis/Attruby). The last few years witnessed accelerated drug approvals and increasing awareness, with U.S. market value increasing from around USD 1.36 billion in 2024 to a forecast USD 2.14 billion by 2029 at ~9.4% CAGR (researchandmarkets.com). The market is dominated by current therapies such as Vyndaqel, and newer players like Amvuttra and Attruby target growing patient spaces .
Segmentation
Type
Hereditary (hATTR)
Polyneuropathy
Early stage
Advanced
Others
Cardiomyopathy
Mild
Severe
Mixed
Others
Wild‑type (wATTR)
Cardiomyopathy
Mild
Severe
Others
Therapy Class
RNAi therapies
Patisiran (Onpattro)
Vutrisiran (Amvuttra)
Others
Antisense oligonucleotides
Inotersen (Tegsedi)
Eplontersen (Wainua)
Others
Stabilizers
Tafamidis (Vyndaqel/Vyndamax)
Acoramidis (Attruby)
Others
Supportive care
Symptomatic treatments
Patient support services
Others
Distribution Channel
Hospital pharmacies
Specialty/Retail pharmacies
Online pharmacies
Others
End‑User
Hospitals/clinics
Neurologists
Cardiologists
Specialty treatment centers
Home healthcare
Others
Market Players
Key active companies in the United States Hereditary Transthyretin‑Mediated Amyloidosis Market:
Alnylam Pharmaceuticals (USA)
Ionis Pharmaceuticals (USA)
Pfizer Inc. (USA)
BridgeBio Pharma (USA)
AstraZeneca (UK)
Prothena Corporation (Ireland)
Eidos Therapeutics (USA)
Akcea Therapeutics (USA)
GSK plc (UK)
Bristol‑Myers Squibb (USA)
Novo Nordisk (Denmark)
Regeneron Pharmaceuticals (USA)
Corino Therapeutics (USA)
Attralus, Inc. (USA)
Intellia Therapeutics (USA)
Drivers
United States Hereditary Transthyretin‑Mediated Amyloidosis Market growth is bolstered by the spurt in FDA approvals and market launch of sophisticated treatments: RNAi (Amvuttra, Onpattro), antisense oligonucleotides (Wainua, Tegsedi), and stabilizers (Attruby). Moreover, the growing geriatric population and enhanced diagnostic efficiency—in terms of genetic screening—are raising disease awareness and early diagnosis (imarcgroup.com, en.wikipedia.org, grandviewresearch.com, researchandmarkets.com, coherentmarketinsights.com). High disease burden, particularly of hATTR‑CM and hATTR‑PN, drives demand. In addition, robust R&D spending and collaborations (for example, Ionis–AstraZeneca, Alnylam's pipeline) are driving quicker therapeutic advances and improving patient availability .
Restraints
Obstacles are high therapy prices—prices vary from ~$100K (Amvuttra) to more than $260K (Vyndaqel) per year—which can limit payer reimbursement and restrict access (reuters.com). Underdiagnosis of the disease and nonspecific early symptoms lead to therapy delay. Emerging safety issues—e.g., platelet risks with inotersen—justify REMS programs, which increase patient management complexity . Finally, oral competition and entrenched incumbents might moderate uptake of newer injectables in some patient segments.
Opportunity
The United States Hereditary Transthyretin‑Mediated Amyloidosis Market is poised to gain from promising pipeline therapies (e.g., next-gen gene editing, new RNAi/ASOs), broader indications into cardiomyopathy, and greater payer acceptance of value-based pricing models. Early genetic testing programs and family-health outreach programs—such as Alnylam's "Family Health History Road Trip"—can drive diagnosis rates . Moreover, expanding patient support infrastructure and telehealth enable enhanced treatment compliance and expand market access.
Trend
A prevalent trend is the move towards RNA therapies with reduced dosing frequency. Amvuttra (vutrisiran) provides quarterly subcutaneous dosing compared to daily or monthly regimens (en.wikipedia.org). Oral stabilizers like angiotensin inhibitors are becoming increasingly accepted—Attruby (acoramidis) was FDA-approved in late 2024 (reuters.com). Strategic initiatives like the distribution agreement between Ionis and AstraZeneca for Wainua extend market reach. Telemedicine integration and remote monitoring platforms are also emerging as useful patient-management tools.
Table 9–23: Financial Overview of Top Market Players
10.2 List of Figures
Figure 1: United States Hereditary Transthyretin‑Mediated Amyloidosis Market Growth Trend (2024–2030)
Figure 2: RNAi vs ASO Market Share Forecast
Figure 3: Patient Demographics Distribution
Figure 4: Treatment Uptake by Therapeutic Class
Figure 5: Revenue Contribution by Distribution Channel
Figure 6: Competitive Landscape – Market Share by Company
Figure 7–20: Therapy Type Breakdown, Application Breakdown, Pipeline Progression
Research Methodology
The process of market research at Ultra Market Research is an iterative in application and usually follows following path. Information from secondary used to build data models, then results from data models are validated from primary participants. Then cycle repeats where, according to inputs from primary participants, additional secondary research is done, and new Information is again incorporated into data model. The process continues till desired level of Information is not generated
To calculate the market size, the report considers the revenue generated from the sales of Ultra Market Research providers. The revenue generated from the sales of Ultra Market Research has been calculated through primary and secondary research. The report also presents the key players operating in the Ultra Market Research market across the globe identified through secondary research and a corresponding detailed analysis of the top vendors in the market. The market size calculation also includes distribution channel segmentation determined using secondary sources and verified through primary sources.
Secondary Research
The secondary research source that are typically referred to include, but are not limited to:
Company websites, annual reports, financial reports, broker reports, investor presentations and SEC filings
Internal and external proprietary databases, relevant patent and regulatory databases
National government documents, statistical databases and market reports
News articles, press releases and web-casts specific to the companies operating in the market
The source for secondary research includes but is not limited to: Factiva, Hoovers and Statista
Inner Circle Represents – Stage of Research Process
Middle Circle Represents – Source of Information
Outer Circle Represents – Information Derived from that source
Primary Research We conduct primary interviews on an ongoing basis with industry participants and commentators in order to validate data and analysis. A typical research interview fulfills the following functions:
It provides first-hand Information on the market size, market trends, growth trends, competitive landscape, future outlook etc.
Helps in validating and strengthening the secondary research findings
Further develops the analysis team’s expertise and market understanding
Primary research involves E-mail interactions, telephonic interviews as well as face-to-face interviews for each market, category, segment and sub-segment across geographies
The participants who typically take part in such a process include, but are not limited to:
Industry participants: CEOs, VPs, marketing/ distribution channel managers, market intelligence managers and national sales managers
Purchasing managers, technical personnel, distributors and resellers
Outside experts: Investment bankers, valuation experts, research analysts specializing in specific markets
Key opinion leaders specializing in different areas corresponding to different industry distribution channels
Models Where no hard data is available, we use modeling and estimates in order to produce comprehensive data sets. A rigorous methodology is adopted in which the available hard data is cross referenced with the following data distribution channels to produce estimates:
Demographic data: Population split by segment
Macro-economic indicators: GDP, etc.
Industry indicators: Expenditure, distribution channel stage & infrastructure, sector growth and facilities.
Data is then cross checked by the expert panel.
2..1 Company Share Analysis Model
Company share analysis is used to derive the size of Global market. As well as study of revenues of companies for last three to five years also provide the base for forecasting the market size and its growth rate. This model is built in following steps: 2..2 Revenue Based Modeling
Revenue based models can be built in two ways – Top-Down or Bottom-Up irrespective of industry. Market size estimated from company share analysis acts as a validation point for bottom-up approach where as it acts as starting point for top-down approach.
2.1 Research Limitations Inflation is not a part of pricing in this report. Prices of United States Hereditary Transthyretin‑Mediated Amyloidosis Market and its derivatives vary in each region and hence similar revenue ratio does not follow for each individual region. The same price for each distribution channel has been taken into account while estimating and forecasting market revenue Globally. Regional average price has been considered while breaking down this market by application in each region. This report provides market size of CANOMAD syndrome Market for the past year and forecasts for the next six years. United States Hereditary Transthyretin‑Mediated Amyloidosis Market size is given in terms of revenue. Market revenue is defined in USD Million. Market numbers are given on the basis of different United States Hereditary Transthyretin‑Mediated Amyloidosis Market categories. Market size and forecasts for each major application is provided in the context of Global market. The numbers provided in this report are derived on the basis of demand for United States Hereditary Transthyretin‑Mediated Amyloidosis Market from different application industries in different regions.
