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. It is expected to grow at a CAGR of 11.60% during the forecast period from 2025 to 2033. This growth is driven by rising geriatric populations, increased diagnosis rates, and advancements in targeted and RNA-based therapies.
Study Assumption Years
Base Year: 2024
Historical Year/Period: 2019-2024
Forecast Year/Period: 2025-2033
Transthyretin Amyloidosis Treatment Market Key Takeaways
The global market size was USD 6.94 Billion in 2024.
The CAGR is 11.60% for the forecast period 2025-2033.
North America dominates the market with an over 46.3% market share in 2024.
The United States holds 78.60% share of the North American market.
The wild-type amyloidosis segment accounts for 56.2% market share in 2024.
Targeted therapy leads with 85.6% market share in 2024.
Hospital pharmacies lead distribution with 48.6% share in 2024.
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Market Growth Factors
The growth of the transthyretin amyloidosis treatment market is largely driven by the global aging population. Transthyretin amyloidosis predominantly affects adults over 60 years, and as WHO projects, by 2030 one in every six people worldwide will be 60 or older, doubling to 2.1 billion by 2050. The wild-type form of ATTR, strongly associated with aging, increases the pool of potential patients. Longer lifespans and better healthcare result in more chronic conditions being managed, increasing demand for therapies.
Advancements in targeted and RNA-based therapies are key growth drivers. Targeted therapies constituted 85.6% of the market in 2024, including drugs like patisiran and vutrisiran that silence abnormal gene expression or stabilize transthyretin proteins, slowing disease progression. The approval of drugs such as BridgeBio's Attruby and the widespread use of tafamidis and inotersen reflect innovation and effective management options for patients.
Personalized medicine is transforming treatment strategies, with genetic testing helping distinguish hereditary from wild-type ATTR for tailored therapies. Companies focus on therapies targeting specific mutations or protein misfolding, improving adherence and outcomes. Combination therapies are also gaining traction, combining mechanisms such as TTR stabilizers and RNA-based treatments to halt disease progression and reduce side effects, supported by ongoing clinical trials.
Market Segmentation
By Therapy:
Targeted Therapy: Commands 85.6% market share due to disease-modifying effects through gene silencing or protein stabilization using drugs like patisiran, vutrisiran, and tafamidis, supported by growing adoption and clinical data.
By Type:
ATTR with Polyneuropathy (ATTR-PN): Holds 78.1% market share, driven by hereditary cases, symptoms of nerve damage, and extensive use of therapies targeting genetic mutations such as patisiran and inotersen.
By Disease:
Wild Type Amyloidosis: Largest segment at 56.2% share, prevalent in aging populations. Improved diagnostic tools and RNA-based treatments increase demand, addressing disease progression beyond symptoms.
By Distribution Channel:
Hospital Pharmacies: Lead with 48.6% share, critical for diagnosis and administration of complex ATTR therapies like tafamidis and patisiran, supported by specialized care and institutional purchasing power.
Regional Insights
North America dominates the market with a 46.3% share in 2024. Growth is driven by advanced healthcare infrastructure, high disease awareness, regulatory support, and adoption of innovative therapies. The United States, holding 78.60% of North America's share, benefits from extensive FDA approvals, diagnostic capabilities, reimbursement policies, and clinical trials, fueling significant market expansion.
Recent Developments & News
In October 2024, Alnylam Pharmaceuticals submitted a supplemental New Drug Application (sNDA) to the FDA for vutrisiran, targeting ATTR amyloidosis with cardiomyopathy (ATTR-CM). This drug was previously FDA approved for hereditary ATTR amyloidosis with polyneuropathy.
In September 2024, AstraZeneca's Alexion received FDA fast track designation for ALXN2220, intended for managing transthyretin amyloidosis with cardiomyopathy.
Key Players
Akcea Therapeutics, Inc
Alnylam Pharmaceuticals, Inc.
AstraZeneca
BridgeBio, Inc.
Intellia Therapeutics, Inc
Pfizer Inc
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