The global transthyretin amyloidosis treatment market is experiencing significant growth, driven by an aging population, increased disease awareness, and advancements in targeted therapies. Valued at USD 6.94 billion in 2024, the market is projected to reach USD 20.35 billion by 2033, reflecting a robust CAGR of 11.60% from 2025 to 2033. Key growth factors include the adoption of RNA-based drugs, the emergence of combination therapies, and a shift towards patient-centric care models. North America currently leads the market, holding a 46.3% share in 2024, fueled by advanced healthcare infrastructure and regulatory support for rare diseases.
Study Assumption Years
- Base Year: 2024
- Historical Years: 2019–2024
- Forecast Years: 2025–2033
Transthyretin Amyloidosis Treatment Market Key Takeaways
- Market Size & Growth: The global ATTR treatment market was valued at USD 6.94 billion in 2024 and is expected to reach USD 20.35 billion by 2033, growing at a CAGR of 11.60%.
- Regional Dominance: North America holds the largest market share at 46.3% in 2024, driven by advanced healthcare systems and early adoption of novel therapies.
- Therapy Segmentation: Targeted therapies dominate with an 85.6% market share in 2024, owing to their effectiveness in addressing disease causality.
- Disease Type: Wild-type amyloidosis accounts for 56.2% of the market in 2024, primarily due to its prevalence among the aging population.
- Distribution Channels: Hospital pharmacies lead with a 48.6% market share in 2024, reflecting their critical role in managing complex treatments.
- Emerging Trends: The market is witnessing a rise in combination therapies and personalized medicine approaches, enhancing treatment efficacy and patient outcomes.
Market Growth Factors
- Rising Prevalence of Transthyretin Amyloidosis Worldwide
The growing number of patients diagnosed with transthyretin amyloidosis is fueling the demand for effective treatments. As aging populations increase across major regions, more cases of both hereditary and wild-type ATTR are being identified. This rise in prevalence is compelling healthcare providers to expand treatment access and encouraging pharmaceutical firms to accelerate new therapy pipelines. With improved screening programs and better disease recognition, patient pools are expected to grow significantly. The resulting demand for specialized medications and therapies is creating robust opportunities in the market. Ultimately, the rising patient base is driving research funding, healthcare investments, and long-term growth of the transthyretin amyloidosis treatment market on a global scale.
- Targeted therapy market share in 2025 for the Effectiveness of Tafamidis and Patisiran
Tafamidis and Patisiran have become breakthrough drugs in the Transthyretin Amyloidosis Treatment Market due to their proven effectiveness. Tafamidis stabilizes transthyretin proteins, preventing harmful deposits, while Patisiran uses RNA interference to reduce abnormal protein production. Clinical studies demonstrate extended survival rates and improved patient quality of life with both drugs. Their FDA and EMA approvals have further enhanced market confidence, encouraging wider physician adoption. With patient demand growing for clinically validated therapies, these drugs have become the backbone of treatment strategies. Their success is influencing ongoing research and setting new standards for the industry’s treatment landscape.
- Innovative drug development for amyloidosis
The rise of innovative drug development is a core driver of the Transthyretin Amyloidosis Treatment Market. Pharmaceutical companies are investing heavily in next-generation therapies that go beyond traditional stabilizers. New approaches include RNA-based drugs, gene silencers, and monoclonal antibodies, designed to halt or reverse amyloid buildup. This surge of innovation aims to address unmet medical needs and improve patient outcomes. Partnerships between biotech firms and research institutions are accelerating clinical advancements. These therapies are expected not only to enhance efficacy but also reduce treatment burden, making innovation a vital force shaping the competitive market landscape in the coming years.
Request for a sample copy of this report:
https://www.imarcgroup.com/transthyretin-amyloidosis-treatment-market/requestsample
Market Segmentation
Breakup by Therapy:
- Targeted Therapy:
- Vyndaqel/Vyndamax: Tafamidis-based drugs that stabilize transthyretin proteins, slowing disease progression.
- Onpattro: Patisiran, an RNA interference therapy that reduces transthyretin production.
- Amvuttra: Vutrisiran, a next-generation RNAi therapeutic with improved dosing convenience.
- Tegsedi: Inotersen, an antisense oligonucleotide that inhibits transthyretin synthesis.
- Wainua: Eplontersen, a transthyretin-directed antisense oligonucleotide developed for hereditary ATTR.
- Supportive Therapy: Treatments aimed at managing symptoms and improving quality of life.
- Pipeline Therapy: Emerging therapies under clinical development targeting various aspects of ATTR pathology.
Breakup by Type:
- ATTR with Polyneuropathy (ATTR-PN): Characterized by nerve damage leading to sensory and motor impairments; accounts for 78.1% market share in 2024.
- ATTR with Cardiomyopathy (ATTR-CM): Involves heart muscle dysfunction; expected to grow due to increased awareness and improved diagnostics.
Breakup by Disease:
- Hereditary Transthyretin Amyloidosis:
- Polyneuropathy: Genetic mutations leading to nerve damage.
- Cardiomyopathy: Genetic mutations causing heart muscle involvement.
- Mixed Type: Combination of neurological and cardiac symptoms.
- Wild Type Amyloidosis: Non-hereditary form predominantly affecting the elderly; accounts for 56.2% market share in 2024.
Breakup by Distribution Channel:
- Hospital Pharmacies: Primary distribution channel with 48.6% market share in 2024, due to the complexity of treatments and the need for specialized care.
- Specialty Pharmacies: Focus on dispensing high-cost, high-complexity medications.
- Retail Pharmacies: Provide accessibility for maintenance medications and follow-up prescriptions.
- Online Pharmacies: Emerging channel offering convenience and broader reach.
Breakup by Region:
-
- North America (United States, Canada)
- Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, Others)
- Europe (Germany, France, United Kingdom, Italy, Spain, Russia, Others)
- Latin America (Brazil, Mexico, Others)
- Middle East and Africa
Regional Insights
North America leads the ATTR treatment market, holding a 46.3% share in 2024. This dominance is attributed to advanced healthcare infrastructure, early adoption of innovative therapies, and supportive regulatory frameworks. The United States, in particular, accounts for 78.6% of the North American market, driven by high disease awareness, availability of approved treatments like tafamidis and patisiran, and favorable reimbursement policies
Recent Developments & News
In October 2024, Alnylam Pharmaceuticals submitted a supplemental New Drug Application to the U.S. FDA for vutrisiran (Amvuttra) to treat ATTR amyloidosis with cardiomyopathy, expanding its therapeutic indications. Additionally, in September 2024, AstraZeneca’s rare disease division, Alexion, received fast-track designation from the FDA for ALXN2220, a treatment aimed at managing transthyretin amyloidosis with cardiomyopathy. These developments underscore the ongoing innovation and regulatory support in the ATTR treatment landscape.
Key Players
Akcea Therapeutics, Inc, Alnylam Pharmaceuticals, Inc., AstraZeneca, BridgeBio, Inc., Intellia Therapeutics, Inc, Pfizer Inc, etc.
Ask Analyst for Customization:
https://www.imarcgroup.com/request?type=report&id=27702&flag=C
If you require any specific information that is not covered currently within the scope of the report, we will provide the same as a part of the customization.
About Us:
IMARC Group is a global management consulting firm that helps the world’s most ambitious changemakers to create a lasting impact. The company provides a comprehensive suite of market entry and expansion services. IMARC offerings include a thorough market assessment, feasibility studies, company incorporation assistance, factory setup support, regulatory approvals and licensing navigation, branding, marketing and sales strategies, competitive landscape, and benchmarking analyses, pricing and cost research, and procurement research.
Contact Us:
IMARC Group
134 N 4th St. Brooklyn, NY 11249, USA
Email: sales@imarcgroup.com
Tel No:(D) +91 120 433 0800
United States: +1-631-791-1145
