Transthyretin Amyloidosis Treatment Market Size & Growth Analysis to 2030

The global transthyretin amyloidosis treatment market size was valued at over USD 6 billion in 2024 and projected to grow at a robust CAGR of around 14% during the forecast period from 2025 to 2030. The market is experiencing significant growth due to advancements in diagnostics, increasing awareness about the disease, and a surge in the availability of innovative therapies. Transthyretin amyloidosis is a rare, life-threatening condition caused by the deposition of misfolded transthyretin proteins in tissues and organs, leading to polyneuropathy (ATTR-PN) and cardiomyopathy (ATTR-CM). Targeted therapies, such as transthyretin stabilizers and gene silencers, are driving market growth by addressing the disease’s root cause. These therapies are complemented by supportive treatments aimed at symptom management. Additionally, the increasing development of pipeline therapies, including gene editing and novel drugs, offers hope for improved patient outcomes, further fueling market expansion.

The market is segmented by disease type, with hereditary transthyretin amyloidosis and wild-type amyloidosis representing the primary categories. Hospital and specialty pharmacies dominate the distribution channels due to the specialized nature of the treatments, though online pharmacies are emerging as a convenient alternative for patients. North America and Europe are key regional markets, driven by well-established healthcare infrastructure and robust R&D investments, while the Asia-Pacific region is expected to witness the fastest growth owing to increasing awareness and improving healthcare access. Collaborations between pharmaceutical companies and research institutions are accelerating the development of cutting-edge therapies, making the market highly dynamic and competitive.

Market Snapshot:

Market Drivers:

The global transthyretin amyloidosis treatment market is experiencing significant growth, driven by several key factors. One major contributor is the increasing prevalence of the disease, particularly among the aging population. As the global population ages, the incidence of transthyretin amyloidosis, especially its wild-type form (ATTR-CM), is rising, leading to a higher demand for effective treatments. Additionally, advancements in diagnostic techniques have improved early detection rates, enabling timely intervention and management of the disease. This heightened awareness and improved diagnosis are propelling the market forward.

Another significant driving factor is the development and approval of novel targeted therapies. Pharmaceutical companies are investing heavily in research and development to introduce innovative treatments that address the underlying causes of transthyretin amyloidosis. For instance, the U.S. Food and Drug Administration (FDA) granted orphan drug designation to vutrisiran, a gene-silencing therapy designed to reduce the production of transthyretin protein. Such advancements not only enhance patient outcomes but also expand the therapeutic options available, thereby driving market growth. Furthermore, increasing product approvals and launches, coupled with a growing geriatric population, are major factors contributing to the expansion of the transthyretin amyloidosis treatment market.

Market Trends:

Emergence of Gene-Silencing Therapies: Gene-silencing therapies are revolutionizing the treatment landscape for transthyretin amyloidosis. Products such as vutrisiran and patisiran are leading the way by targeting the genetic root cause of the disease. These therapies work by inhibiting the production of transthyretin (TTR) proteins that misfold and accumulate in organs, causing severe damage. The development of these targeted treatments marks a shift from traditional symptom-focused care to addressing the underlying disease mechanism, offering patients a chance at significantly improved quality of life and potentially slowing disease progression. With FDA approvals and promising clinical results, gene-silencing therapies are expected to become a cornerstone of treatment, driving market growth.

Focus on Personalized Medicine: There is a growing trend toward personalized or precision medicine in the treatment of transthyretin amyloidosis. The disease manifests in various forms, with hereditary transthyretin amyloidosis (ATTR) and wild-type amyloidosis (ATTR-CM) being the most common types. As a result, pharmaceutical companies are focusing on developing tailored therapies based on the specific subtype of the disease a patient has. By customizing treatments for individual genetic profiles and disease progression stages, these therapies aim to increase efficacy and minimize side effects. Personalized treatments not only offer hope for better management of the disease but also align with the broader healthcare industry’s shift toward individualized care.

Digital Health Integration: With the rise of digital health technologies, the management of transthyretin amyloidosis is becoming more patient-centric and efficient. Wearable devices, telemedicine platforms, and mobile apps are increasingly used to monitor patients’ conditions, track treatment adherence, and facilitate real-time consultations with healthcare providers. These tools allow for better monitoring of disease progression, helping doctors adjust treatment plans and identify any complications early. As digital health solutions gain acceptance in the healthcare sector, they enhance patient engagement, optimize resource use, and improve overall disease management. This integration of technology is expected to become a major trend, offering more holistic care and improving patient outcomes.

Expanding Pipeline and Collaborations: The treatment pipeline for transthyretin amyloidosis is expanding, with a range of new therapies currently in clinical trials. Pharmaceutical companies are increasingly investing in R&D to develop more advanced and effective treatments, including next-generation gene therapies, small molecules, and biologics. In addition, collaborations between biotech firms, pharmaceutical companies, and academic institutions are accelerating the pace of innovation. Partnerships aimed at co-developing novel treatments or combining existing therapies are also gaining momentum. These efforts are vital for bringing cutting-edge treatments to market faster, enriching the available options for both hereditary and wild-type amyloidosis patients, and driving growth in the market.

Global Expansion and Access: While the treatment landscape for transthyretin amyloidosis has traditionally been more developed in regions like North America and Europe, there is a growing focus on expanding access to these therapies in emerging markets. Countries in Asia-Pacific and Latin America are seeing improvements in healthcare infrastructure, better diagnostic capabilities, and increased awareness of rare diseases like amyloidosis. These factors are helping to improve early diagnosis and expand patient access to treatment options. Additionally, pharmaceutical companies are working on making treatments more affordable and accessible in these regions, which is expected to fuel market growth. As healthcare systems continue to evolve, more patients worldwide will be able to benefit from the latest advancements in transthyretin amyloidosis care.

Market Opportunities:

The Transthyretin Amyloidosis Treatment Market presents several promising opportunities driven by recent advancements and increasing disease awareness. The approval of new therapies, such as BridgeBio’s Attruby (acoramidis) for treating transthyretin amyloid cardiomyopathy (ATTR-CM), has expanded treatment options and is expected to generate significant revenue. This development not only offers patients more choices but also stimulates market growth by introducing competitive pricing and fostering innovation.

Additionally, the success of gene-silencing drugs like vutrisiran underscores the potential for transformative treatments in this space. Clinical trials have demonstrated that vutrisiran can significantly reduce mortality rates and improve patients’ quality of life. Such breakthroughs open avenues for further research and development, presenting opportunities for pharmaceutical companies to invest in novel therapies and expand their portfolios. The combination of regulatory approvals and scientific advancements positions the market for substantial growth and innovation in the coming years.

Market Restraints:

The global transthyretin amyloidosis treatment market faces several restraining factors that are expected to hinder its growth. One of the primary challenges is the high cost of advanced therapies, particularly gene-silencing treatments like vutrisiran and patisiran, which is estimated to limit patient access, especially in low- and middle-income regions. This financial barrier, coupled with limited insurance coverage for these specialized treatments, is expected to result in significant healthcare disparities. Additionally, while awareness of transthyretin amyloidosis is increasing, it remains a rare and often underdiagnosed condition, which is expected to delay treatment initiation and affect the overall market demand. The complexity of the disease, combined with the limited availability of healthcare professionals skilled in diagnosing and managing amyloidosis, is further expected to complicate timely intervention. Moreover, the lack of long-term data on the safety and efficacy of newer therapies is estimated to lead to hesitancy among healthcare providers and patients, slowing the adoption of these treatments.

Market Insights:

The global transthyretin amyloidosis treatment market is bifurcated into type, therapy, disease, distribution channel, and geography. On the basis of therapy, the targeted therapy segment dominated the global market in the benchmark year 2024. This is primarily driven by the success of gene-silencing therapies such as patisiran and vutrisiran, which have revolutionized the treatment of transthyretin amyloidosis by targeting the root cause of the disease. These therapies work by inhibiting the production of transthyretin (TTR) proteins, which are responsible for the amyloid deposits that cause organ damage in patients. The efficacy of these therapies in reducing the accumulation of amyloid deposits and improving organ function has made them the cornerstone of treatment for both hereditary and wild-type forms of transthyretin amyloidosis. As these targeted therapies continue to show positive clinical results, their adoption is expected to increase, driving the growth of this segment.

In addition to their clinical success, targeted therapies are also poised to dominate due to their potential for long-term disease management. Unlike supportive therapies, which only alleviate symptoms without addressing the underlying disease, targeted treatments provide a more comprehensive solution by slowing or halting disease progression. This makes them highly attractive to both patients and healthcare providers. With increasing regulatory approvals and advancements in gene-silencing technologies, the targeted therapy segment is expected to capture the largest share of the market. As more patients gain access to these innovative treatments, the segment’s market share is expected to continue to grow, further solidifying its dominance in the transthyretin amyloidosis treatment landscape.

The transthyretin amyloidosis treatment market research report presents the analysis of each segment from 2020 to 2030 considering 2023 as the base year for the research. The compounded annual growth rate (CAGR) for each respective segment is calculated for the forecast period from 2025 to 2030.

Historical & Forecast Period

• 2020-23 – Historical Year
• 2024 – Base Year
• 2025-2030 – Forecast Period

Market Segmentation:

By Type:

• ATTR with Polyneuropathy (ATTR-PN)
• ATTR with Cardiomyopathy (ATTR-CM)

By Therapy:

• Targeted Therapy
• Supportive Therapy
• Pipeline Therapy

By Disease:

• Hereditary Transthyretin Amyloidosis
• Wild Type Amyloidosis

By Distribution Channel:

• Hospital Pharmacies
• Specialty Pharmacies
• Retail Pharmacies
• Online Pharmacies

By Region:

• North America
• Europe
• Asia Pacific
• Latin America
• Middle East & Africa

Regional Analysis:

Geographically, the North America dominated the global transthyretin amyloidosis treatment market in the benchmark year 2024. This dominance is primarily driven by the high prevalence of transthyretin amyloidosis in the United States and Canada, along with advanced healthcare infrastructure, a high level of awareness, and access to cutting-edge medical treatments. North America has been a leader in the approval and adoption of innovative therapies, such as patisiran and vutrisiran, both of which were initially approved by the FDA. These gene-silencing therapies have proven highly effective in treating transthyretin amyloidosis, contributing to the region’s growth. Furthermore, the presence of major pharmaceutical companies and research institutions in North America accelerates the development and commercialization of new therapies, positioning the region as the hub for the latest treatment options.

In addition to strong healthcare infrastructure, North America’s dominance is also attributed to the well-established reimbursement systems, which facilitate patient access to expensive therapies. Insurance coverage for specialized treatments is more comprehensive in North America compared to other regions, allowing a larger number of patients to receive these life-changing therapies. The ongoing research and development in this region are expected to lead to further innovations in transthyretin amyloidosis treatment, reinforcing North America’s position as the market leader. As more therapies reach the market and patient awareness continues to grow, North America is expected to maintain its dominant role in the treatment landscape of transthyretin amyloidosis.

Competitive Landscape:

Some of the prominent market players operating in the global transthyretin amyloidosis treatment market are Alnylam Pharmaceuticals, Inc., Pfizer Inc., BridgeBio Pharma, Inc., Ionis Pharmaceuticals, Inc., and Teva Pharmaceutical Industries Ltd. Companies are exploring markets by expansion, new investment, the introduction of new services, and collaboration as their preferred strategies. Players are exploring new geography through expansion and acquisition to gain a competitive advantage through joint synergy.

Key Companies:

• Alnylam Pharmaceuticals, Inc.
• Pfizer Inc.
• BridgeBio Pharma, Inc.
• Ionis Pharmaceuticals, Inc.
• Teva Pharmaceutical Industries Ltd.
• AstraZeneca
• Akcea Therapeutics
• Intellia Therapeutics, Inc.
• SOM BIOTECH
• Oncopeptides AB

Key Questions Answered by Transthyretin Amyloidosis Treatment Market Report

• Global transthyretin amyloidosis treatment market forecasts from 2025-2030
• Regional market forecasts from 2025-2030 covering Asia-Pacific, North America, Europe, Middle East & Africa, and Latin America
• Country-level forecasts from 2025-2030 covering 15 major countries from the regions as mentioned above
• Transthyretin amyloidosis treatment submarket forecasts from 2025-2030 covering the market by type, therapy, disease, distribution channel, and geography
• Various industry models such as SWOT analysis, Value Chain Analysis about the market
• Analysis of the key factors driving and restraining the growth of the global, regional, and country-level transthyretin amyloidosis treatment markets from 2025-2030
• Competitive Landscape and market positioning of top 10 players operating in the market