Transthyretin Amyloidosis Treatment Market - Global Industry Size, Share, Trends, Opportunity, and Forecast, Segmented By Type (ATTR-PN, ATTR-CM), By Therapy (Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax), Supportive Therapy, Pipeline Therapy), By Disease Type (Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type), Wild Type Amyloidosis), By Distribution Channel (Hospital Pharmacies, Specialty Pharmacies, Retail Pharmacies, Online Pharmacies), By Region and Competition, 2021-2031F

Forecast Period	2027-2031
Market Size (2025)	USD 7.78 Billion
CAGR (2026-2031)	14.81%
Fastest Growing Segment	Hospitals Pharmacy
Largest Market	North America
Market Size (2031)	USD 17.82 Billion

Market Overview

The Global Transthyretin Amyloidosis Treatment Market will grow from USD 7.78 Billion in 2025 to USD 17.82 Billion by 2031 at a 14.81% CAGR. The Global Transthyretin Amyloidosis Treatment Market encompasses pharmacological interventions designed to stabilize the transthyretin protein or suppress its hepatic production to prevent amyloid fibril deposition in the heart and peripheral nerves. The market is primarily driven by the increasing geriatric population and the widespread adoption of non-invasive nuclear scintigraphy, which has significantly improved diagnostic rates compared to traditional biopsy methods. According to the European Society of Cardiology, in 2025, approximately 19% of older patients with unexplained hypertrophic cardiomyopathy were identified as having transthyretin amyloid cardiomyopathy, underscoring the expanding addressable patient pool resulting from targeted screening initiatives.

However, one significant challenge impeding market expansion is the high cost of disease-modifying therapies, which creates substantial reimbursement barriers and limits patient access in cost-sensitive healthcare systems. This financial constraint often results in delayed treatment initiation or refusal of coverage, effectively restricting revenue growth despite the rising incidence of diagnosed cases globally.

Key Market Drivers

The introduction and uptake of novel disease-modifying therapies acts as the primary catalyst for the Global Transthyretin Amyloidosis Treatment Market. Clinicians are increasingly prescribing targeted agents that stabilize the transthyretin tetramer or silence its hepatic production, effectively altering the natural history of the disease. This shift is evidenced by the commercial trajectory of established standards of care and the rapid adoption of new market entrants. According to Pfizer Inc., October 2024, in the 'Third Quarter 2024 Earnings', the Vyndaqel family of products achieved global revenues of 1.45 billion USD, marking a 63 percent operational growth driven by robust demand. Furthermore, the market continues to diversify with additional therapeutic options gaining ground; according to AstraZeneca, November 2024, in the 'Year to date and Q3 2024 Results', the recently launched treatment Wainua generated 23 million USD in quarterly revenue, demonstrating immediate traction within the polyneuropathy segment.

Complementing this commercial expansion is a robust clinical pipeline and accelerated research and development investments focused on expanding labeled indications and improving mortality outcomes. Pharmaceutical developers are aggressively pursuing next-generation RNA interference and antisense oligonucleotide therapies to address the larger cardiomyopathy patient population, which was historically difficult to treat. A pivotal development in this domain occurred recently with the release of data supporting the efficacy of gene silencers in cardiac manifestations. According to Alnylam Pharmaceuticals, June 2024, in the 'HELIOS-B Phase 3 Study Results', vutrisiran demonstrated a statistically significant 28 percent reduction in the composite risk of all-cause mortality and recurrent cardiovascular events in patients with ATTR amyloidosis with cardiomyopathy. These clinical advancements are critical for regulatory expansions that will likely significantly increase the addressable market size in the coming years.

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Key Market Challenges

The prohibitive cost of disease-modifying therapies for transthyretin amyloidosis acts as a substantial bottleneck to market expansion by severing the link between improved diagnosis and revenue generation. While screening initiatives successfully identify more patients, the high pricing of these lifelong treatments compels public and private payers to institute rigid reimbursement criteria. These financial barriers frequently manifest as complex prior authorization requirements or coverage denials, which prevents the conversion of the expanding addressable patient pool into a serviceable market. Consequently, manufacturers face a discrepancy where clinical demand rises, but actual product uptake is artificially suppressed by economic gatekeeping.

This disconnect is evidenced by the direct impact of financial toxicity on patient access. According to the Amyloidosis Research Consortium, in 2024, one in five patients with transthyretin amyloidosis reported delaying the initiation of treatment specifically due to cost or insurance coverage barriers. This significant portion of the patient population being forced to postpone or forego therapy demonstrates how affordability issues directly reduce the practical market size and hamper the commercial trajectory of the sector.

Key Market Trends

The advancement of In Vivo CRISPR-Cas9 gene editing therapeutics is fundamentally reshaping the market by moving the treatment paradigm from chronic management toward potential one-time curative interventions. Unlike existing gene silencers that require regular lifelong administration, these next-generation agents utilize lipid nanoparticles to permanently inactivate the TTR gene within hepatocytes, effectively abolishing the production of pathogenic proteins. This transformative potential to halt disease progression after a single dose is substantiated by recent clinical durability data; according to Intellia Therapeutics, November 2024, in the 'Phase 1 Clinical Data Announcement', the investigational agent nexiguran ziclumeran demonstrated a consistent and deep mean serum TTR reduction of 90 percent at month 12 across all treated patients, regardless of their baseline levels.

Simultaneously, the commercialization of next-generation high-affinity TTR stabilizers is establishing a new efficacy benchmark by achieving near-complete stabilization of the tetramer compared to first-generation options. These novel small molecules are engineered to maximize binding occupancy in the blood, thereby significantly reducing the release of toxic monomers that drive amyloid deposition in cardiac tissue. This shift toward superior stabilization was validated by recent major regulatory successes; according to BridgeBio Pharma, November 2024, in the 'FDA Approval Announcement for Attruby', the pivotal Phase 3 ATTRibute-CM trial data supporting the approval showed that acoramidis achieved a 42 percent reduction in the composite risk of all-cause mortality and recurrent cardiovascular-related hospitalization events at month 30 relative to placebo.

Segmental Insights

The hospital pharmacy segment represents the fastest-growing category in the global transthyretin amyloidosis treatment market, driven by the critical need for medically supervised administration of complex therapies. Many approved treatments, particularly intravenous infusions, require strict clinical oversight to manage potential adverse events, making hospitals the essential environment for patient care. Additionally, as hospitals house the specialized diagnostic infrastructure required to identify this often-underdiagnosed condition, they naturally serve as the primary point of prescription and dispensing. This consolidation of diagnosis and treatment within clinical facilities fundamentally supports the rapid expansion of the hospital pharmacy sector.

Regional Insights

North America holds the leading position in the Global Transthyretin Amyloidosis Treatment Market, driven by the strong presence of key pharmaceutical developers and advanced diagnostic infrastructure. The region benefits from high disease awareness, which improves patient identification rates compared to other areas. Additionally, the US Food and Drug Administration provides a supportive regulatory environment that accelerates the approval of new therapies. Comprehensive reimbursement policies for rare diseases further sustain market dominance by ensuring wider patient access to essential treatments, solidifying the region's status as a central hub for therapeutic commercialization.

Recent Developments

• In November 2025, Novo Nordisk advanced its investigational therapy, coramitug, into a Phase 3 clinical trial known as CLEOPATTRA for the treatment of transthyretin amyloidosis with cardiomyopathy. The initiation of this late-stage study followed the presentation of Phase 2 data which demonstrated that the amyloid-depleting antibody significantly reduced levels of N-terminal pro-brain type natriuretic peptide, a marker of cardiac stress. The company aims to evaluate the efficacy of the drug in reducing the risk of cardiovascular death and clinical worsening in patients. This move underscored the company's strategic expansion into cardiovascular disease treatments.
• In March 2025, AstraZeneca and Ionis Pharmaceuticals obtained approval from the European Commission for Wainzua (eplontersen) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy. The approval followed a positive opinion from the Committee for Medicinal Products for Human Use and was supported by results from the NEURO-TTRansform Phase 3 trial. The companies emphasized that this approval offers a new self-administered, monthly RNA-targeted treatment option for patients in Europe. This development further solidified the collaboration's presence in the global market for amyloidosis therapies.
• In November 2024, BridgeBio Pharma received approval from the U.S. Food and Drug Administration for acoramidis, marketed as Attruby, for the treatment of adults with transthyretin amyloid cardiomyopathy. The approval was based on data from the ATTRibute-CM Phase 3 trial, which showed that the oral small molecule stabilizer significantly reduced the risk of cardiovascular death and cardiovascular-related hospitalization. The company noted that this product is the first and only approved near-complete transthyretin stabilizer. This regulatory milestone marked a significant expansion in the available therapeutic options for patients suffering from this progressive cardiovascular condition.
• In June 2024, Alnylam Pharmaceuticals announced positive topline results from the HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic for the treatment of transthyretin amyloidosis with cardiomyopathy. The study successfully met its primary endpoint, demonstrating a statistically significant reduction in the composite of all-cause mortality and recurrent cardiovascular events in both the overall and monotherapy populations compared to placebo. The company highlighted that the results showed consistent benefits across key secondary endpoints, including disease progression measures. These findings provided the clinical basis for the company to submit a supplemental New Drug Application to regulatory authorities to expand the drug's label.

Key Market Players

• Alnylam Pharmaceuticals
• Bridgebio Pharma
• Pfizer
• Ionis Pharmaceuticals
• Astrazeneca
• Alexion Pharmaceutical
• Proliferan
• Enobio Pharma
• Moderna
• Intellia Therapeutics
• Regeneron Pharmaceuticals

By Type	By Therapy	By Disease Type	By Distribution Channel	By Region
ATTR-PN ATTR-CM	Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax) Supportive Therapy Pipeline Therapy	Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type) Wild Type Amyloidosis	Hospital Pharmacies Specialty Pharmacies Retail Pharmacies Online Pharmacies	North America Europe Asia Pacific South America Middle East & Africa

Report Scope:

In this report, the Global Transthyretin Amyloidosis Treatment Market has been segmented into the following categories, in addition to the industry trends which have also been detailed below:

• Transthyretin Amyloidosis Treatment Market, By Type:

• ATTR-PN
• ATTR-CM

• Transthyretin Amyloidosis Treatment Market, By Therapy:

• Targeted Therapy (Onpattro, Inotersen, Vyndaqel/Vyndamax)
• Supportive Therapy
• Pipeline Therapy

• Transthyretin Amyloidosis Treatment Market, By Disease Type:

• Hereditary Transthyretin Amyloidosis (Polyneuropathy, Cardiomyopathy, Mixed Type)
• Wild Type Amyloidosis

• Transthyretin Amyloidosis Treatment Market, By Distribution Channel:

• Hospital Pharmacies
• Specialty Pharmacies
• Retail Pharmacies
• Online Pharmacies

• Transthyretin Amyloidosis Treatment Market, By Region:

• North America
• United States
• Canada
• Mexico
• Europe
• France
• United Kingdom
• Italy
• Germany
• Spain
• Asia Pacific
• China
• India
• Japan
• Australia
• South Korea
• South America
• Brazil
• Argentina
• Colombia
• Middle East & Africa
• South Africa
• Saudi Arabia
• UAE