Transthyretin Amyloidosis Treatment Market For Pharmaceutical / Biotechnology Companies (Manufacturers) For 2025 - 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, 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.

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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. Advancements in Gene-Silencing Therapies

Gene-silencing therapies, including RNA interference (RNAi) and antisense oligonucleotides (ASOs), have made a remarkable impact on the treatment of ATTR. These therapies work by specifically targeting and decreasing the production of faulty transthyretin proteins in the liver, which helps to prevent additional amyloid deposits. Clinical trials have shown notable improvements in neuropathy and cardiomyopathy outcomes. Patients and doctors are increasingly opting for these therapies because they offer a chance to modify the disease rather than just provide temporary relief. The growing accessibility of these treatments in developed markets, along with ongoing clinical research in emerging markets, is driving their uptake. This understanding of the mechanism is intended to slow disease progression while minimizing organ damage, positioning gene-silencing as a key trend.

2. Growing Geriatric Population

Transthyretin amyloidosis is particularly common among older adults, especially those aged 60 and above. The increasing number of elderly people worldwide is a key factor driving market growth. As people age, they experience various physiological changes and often have other health issues, making them more vulnerable to the cardiac and nerve-related complications associated with ATTR. Healthcare providers, like cardiologists and neurologists, are concentrating on delivering therapies that are suitable for older patients and have good safety records. There's a noticeable trend towards patient-centered care, which includes long-term monitoring and treatments that can be done at home. Insights into the mechanisms of the disease suggest that targeting interventions for older patients and managing their treatment early can lead to fewer hospitalizations and better survival rates, which aligns treatment approaches with the needs of the patient demographic and opens new opportunities for market growth.

3. Technological Advancements in Diagnostics

Recent advancements in diagnostic technologies, including genetic testing, cardiac imaging, and biomarkers, are making it possible to detect ATTR early and accurately. Getting the right diagnosis is key to reducing misdiagnosis and ensuring that patients receive timely interventions with therapies that can change the course of the disease. Patients are eager to access treatments that not only improve survival but also minimize complications. There's a noticeable trend towards incorporating AI and machine learning into diagnostics, which enhances both accuracy and patient stratification. Understanding the molecular profile of amyloidosis is crucial for selecting the right therapies and optimizing outcomes. Better diagnostics are directly impacting how quickly treatments are adopted, which supports market growth by enabling healthcare providers to identify and manage patients sooner.

Market Segmentation

Breakup by Therapy:

• Targeted Therapy:
o Vyndaqel/Vyndamax: Tafamidis-based drugs that stabilize transthyretin proteins, slowing disease progression.
o Onpattro: Patisiran, an RNA interference therapy that reduces transthyretin production.
o Amvuttra: Vutrisiran, a next-generation RNAi therapeutic with improved dosing convenience.
o Tegsedi: Inotersen, an antisense oligonucleotide that inhibits transthyretin synthesis.
o 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:
o Polyneuropathy: Genetic mutations leading to nerve damage.
o Cardiomyopathy: Genetic mutations causing heart muscle involvement.
o 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:

o North America (United States, Canada)
o Asia Pacific (China, Japan, India, South Korea, Australia, Indonesia, Others)
o Europe (Germany, France, United Kingdom, Italy, Spain, Russia, Others)
o Latin America (Brazil, Mexico, Others)
o 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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