Transthyretin Amyloidosis Treatment Market Size, Share & Growth Forecast 2025–2033

The global transthyretin amyloidosis treatment market was valued at USD 6.94 Billion in 2024 and is projected to reach USD 20.35 Billion by 2033, growing at a CAGR of 11.60% during 2025-2033. Growth is driven by advancements in novel therapies, increased diagnosis rates, rising geriatric populations, adoption of RNA-based drugs, emergence of combination therapies, and patient-centric care models.

STUDY ASSUMPTION YEARS

• Base Year: 2024
• Historical Years: 2019-2024
• Forecast Year/Period: 2025-2033

TRANSTHYRETIN AMYLOIDOSIS TREATMENT MARKET KEY TAKEAWAYS

• Current Market Size: USD 6.94 Billion in 2024
• CAGR: 11.60% (2025-2033)
• Forecast Period: 2025-2033
• North America dominates with a 46.3% market share in 2024.
• The United States holds 78.60% market share in North America, driven by FDA approvals and advanced healthcare infrastructure.
• The wild-type amyloidosis segment accounts for 56.2% market share due to aging populations.
• Targeted therapies lead the market with 85.6% share in 2024.
• Hospital pharmacies are the primary distribution channel with 48.6% market share.

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MARKET GROWTH FACTORS

The market for treating transthyretin amyloidosis is really taking off, thanks to exciting advancements in targeted therapies like tafamidis, patisiran, and vutrisiran. These treatments tackle the root causes of the disease by either stabilizing transthyretin proteins or silencing the expression of problematic genes. Pharmaceutical companies are pouring resources into research and development, spurred on by regulatory perks like orphan drug designations and priority reviews, which are helping to fuel a strong pipeline of new therapies.

As diagnosis rates rise and the elderly population grows, the market is set to expand even further. The World Health Organization estimates that by 2030, one in six people globally will be 60 or older, and this number is expected to double to 2.1 billion by 2050. This demographic shift is leading to a higher prevalence of wild-type transthyretin amyloidosis, especially among older adults, which in turn increases the demand for effective treatments.

The adoption of RNA-based therapies and combination treatments is also playing a significant role in market growth. For instance, drugs like vutrisiran have shown a 28% reduction in deaths and cardiovascular events. The rise of personalized medicine, made possible by genetic testing for hereditary ATTR, is paving the way for customized treatment plans that improve patient outcomes. Combination therapies that pair TTR stabilizers with RNA-based drugs are gaining popularity, offering synergistic benefits for managing the disease.

MARKET SEGMENTATION

By Therapy:

• Targeted Therapy: Accounts for 85.6% market share in 2024, including innovative RNA drugs (patisiran, vutrisiran) and transthyretin stabilizers (tafamidis). These therapies slow disease progression by silencing gene expression or stabilizing proteins.
• Supportive Therapy
• Pipeline Therapy

By Type:

• ATTR with Polyneuropathy (ATTR-PN): Leading segment with 78.1% share, characterized by progressive nerve damage impacting quality of life. Innovative therapies target genetic mutations responsible for ATTR-PN.
• ATTR with Cardiomyopathy (ATTR-CM)

By Disease:

• Hereditary Transthyretin Amyloidosis: Polyneuropathy, Cardiomyopathy, Mixed Type
• Wild Type Amyloidosis: Largest share at 56.2% due to high prevalence in aging populations and improved diagnostic tools increasing treatment demand.

By Distribution Channel:

• Hospital Pharmacies: Lead with 48.6% share due to hospitals' role in diagnosis and initiation of treatment requiring advanced therapies.
• Specialty Pharmacies
• Retail Pharmacies
• Online Pharmacies

REGIONAL INSIGHTS

North America leads with a significant market share of 46.3% in 2024, driven by advanced healthcare infrastructure, widespread adoption of innovative therapies, and robust regulatory frameworks. The United States commands 78.60% market share within the region, boosted by improved diagnostics, FDA approvals (e.g., Attruby by BridgeBio), favorable reimbursement policies, and a high prevalence of both wild-type and hereditary ATTR cases. This region remains a key driver for global market growth.

RECENT DEVELOPMENTS & NEWS

• In October 2024, Alnylam Pharmaceuticals, Inc. submitted a supplemental New Drug Application (sNDA) to the U.S. FDA for vutrisiran (AMVUTTRA), targeting ATTR amyloidosis with cardiomyopathy. The drug was previously FDA-approved for polyneuropathy in hereditary ATTR.
• In September 2024, AstraZeneca's rare disease division, Alexion, received FDA fast track designation for ALXN2220, intended for transthyretin amyloidosis with cardiomyopathy.

KEY PLAYERS

• Akcea Therapeutics, Inc
• Alnylam Pharmaceuticals, Inc.
• AstraZeneca
• BridgeBio, Inc.
• Intellia Therapeutics, Inc
• Pfizer Inc

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