Transthyretin Amyloidosis Treatment Market Forecast: What Will It Be Worth by 2033?

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, 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

1. Technological Advancements in RNA-Based Therapies

The ATTR treatment landscape is being transformed by RNA-based therapies, such as patisiran and vutrisiran, which target the root cause of the disease by silencing faulty gene expressions. These therapies have demonstrated significant efficacy in reducing disease progression and improving patient quality of life. For instance, vutrisiran has shown a 28% reduction in all-cause mortality and cardiovascular events among patients with transthyretin amyloid cardiomyopathy . The success of these treatments is driving increased investment and interest in RNA-based approaches, not only for ATTR but also for other genetic and rare diseases.

2. Regulatory Support and Orphan Drug Designations

Regulatory agencies like the U.S. FDA and the European Medicines Agency are providing significant support for the development of ATTR treatments through orphan drug designations and priority review pathways. These incentives encourage pharmaceutical companies to invest in research and development of novel therapies. For example, BridgeBio's Attruby (acoramidis) received FDA approval in November 2024 for the treatment of transthyretin amyloid cardiomyopathy, highlighting the regulatory momentum in this space . Such support accelerates the availability of innovative treatments to patients and fosters a competitive market environment.

3. Increasing Disease Awareness and Early Diagnosis

Enhanced awareness of ATTR among healthcare providers and patients is leading to earlier diagnosis and treatment initiation. Advancements in diagnostic tools, including genetic testing and imaging techniques, are facilitating the identification of both hereditary and wild-type forms of the disease. This early detection is crucial, as timely intervention can significantly improve patient outcomes. Moreover, educational initiatives and advocacy efforts are contributing to a better understanding of the disease, encouraging more individuals to seek medical attention and increasing the demand for effective therapies.

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 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 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.

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