Global Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Treatment Market Size, Share, Trends and Forecasts 2031

Key Findings

• ATTR-CM (a cardiac manifestation of transthyretin amyloidosis) is increasingly diagnosed thanks to improved imaging, genetic testing, and biomarker screening, boosting the addressable patient population.
• The treatment landscape includes transthyretin stabilizers, gene-silencing therapies (RNAi, antisense), and emerging novel modalities such as CRISPR and monoclonal antibodies.
• The market is dominated by established players (e.g. Pfizer’s tafamidis) while newer entrants (e.g. Alnylam’s vutrisiran/Amvuttra) seek market share via superior efficacy or dosing convenience.
• Regions with advanced healthcare infrastructure (North America, Europe) lead current adoption; emerging markets in Asia-Pacific are attracting investment and regulatory focus.
• Strategic alliances, licensing deals, and co-development partnerships are accelerating pipeline advancement and geographic expansion.
• Challenges around reimbursement, long-term safety, diagnosis uptake, and high therapy cost persist as key constraints.

ATTR-CM Market Size and Forecast

The broader transthyretin amyloidosis treatment market was valued at USD 6,644.3 million in 2024 and is expected to reach USD 16,526.5 million by 2030 (CAGR ≈ 13.9%). Within that, the ATTR-CM segment is a key growth driver: it generated USD 4.7 billion in 2023 and is projected to lead segment expansion.  According to a projection, the ATTR-CM treatment submarket could be worth USD 19.37 billion by 2032 from a base of USD 2.91 billion in 2025. Another source forecasts the global ATTR-CM market could reach USD 35.93 billion by 2029 at a high CAGR of ~28.2 %. Given these multiple estimates, a conservative forecast for 2031 would place the ATTR-CM segment in the USD 14–25 billion range, assuming continued pipeline success, regulatory approvals, and market uptake.

Market Overview

ATTR-CM (transthyretin amyloid cardiomyopathy) is characterized by misfolded transthyretin protein depositing in the heart, causing restrictive cardiomyopathy, heart failure, and morbidity. Two main forms exist: wild-type and hereditary (variant) ATTR. Diagnosis is increasingly enabled by non-invasive cardiac scintigraphy (e.g., 99mTc-PYP), cardiac MRI, genetic testing, and biomarker assays (NT-proBNP, troponin). Current standards include stabilization of TTR (e.g. tafamidis) and newer gene-silencing medications (RNAi, antisense) that reduce production of transthyretin at its source. The market is transitioning to more potent and combinatory therapies. While North America holds the dominant share due to high diagnosis rates and payer acceptance, Europe is growing steadily given regulatory alignment, and Asia-Pacific is emerging as both a trial region and commercialization target. Manufacturers and biotech firms are investing in local partnerships, regulatory submissions, and awareness programs to boost diagnosis and accessibility across geographies.

Future Outlook

Over the next decade, ATTR-CM therapy will evolve toward more potent, safer, and convenient regimens. Next-generation gene editing (e.g. CRISPR-based TTR knockout) and monoclonal antibodies may complement existing RNAi and stabilizer approaches. Combination regimens (e.g. stabilizers + gene-silencers) may optimize outcomes. Diagnostics will continue to improve (blood-based assays, AI-enhanced imaging), enabling earlier detection and treatment. Market access and payer reimbursement strategies will mature, with outcome-based pricing models and value-based contracts. Manufacturing scale-up, especially for RNAi and antisense products, will become critical. Expansion in emerging markets will depend on lowering entry barriers, local clinical validation, and increasing physician awareness. As the addressable patient base broadens and therapeutic options diversify, ATTR-CM treatment is expected to become a mainstream cardiology specialty rather than a niche rare disease.

ATTR-CM Market Trends

• Shifting Dominance to Gene-Silencing Therapies
  While stabilizers (e.g. tafamidis) have been the backbone of ATTR-CM therapy, RNAi and antisense agents (e.g. vutrisiran/Amvuttra) are rapidly gaining traction due to deeper impact on disease biology and improved endpoints.
  These newer therapies are able to reduce transthyretin production, potentially halting further deposition.
  Their favorable clinical trial results (e.g. reduced mortality, cardiovascular events) are strengthening adoption. 
  Longer dosing intervals or subcutaneous administration (versus daily oral) provide convenience advantages.
  As their long-term safety and efficacy data mature, gene-silencing agents may become first-line standards.
  Future trends may see integration with novel modalities like antisense + stabilizer or combined with immunotherapy approaches.

• Broader Geographic Penetration & Diagnosis Expansion
  ATTR-CM has been historically underdiagnosed; recent efforts in awareness, imaging adoption, and biomarker screening are increasing case identification, especially in developed markets.
  Asia-Pacific, Latin America, and Middle East & Africa present high growth potential as healthcare systems adopt advanced cardiology diagnostics.
  Local clinical trial sites and real-world registries are being developed to validate efficacy across ethnicities and comorbid populations.
  Partnerships with local companies and regulatory agencies are helping to overcome market-entry barriers.
  Some markets may introduce screening guidelines for older patients with heart failure with preserved ejection fraction (HFpEF) to find ATTR-CM cases early.
  Growth in penetration directly expands the treated population and reinforces market scale.

• Outcome-Based Pricing and Reimbursement Innovation
  Given the high cost of therapy and chronic nature of treatment, payers and governments are pushing for value-based contracts tied to endpoints (e.g. hospitalization reduction, mortality benefit).
  Companies are negotiating risk-sharing agreements and performance guarantees.
  Health technology assessments (HTA) increasingly demand long-term real-world data to justify coverage.
  Pricing strategies may include tiered pricing by region, patient subgroup, or therapy line.
  Market access will be tied to demonstrating cost-effectiveness in heart failure populations.
  This trend will influence which therapies succeed based not only on efficacy but also on economic models and policy.

• Emergence of Combination and Adjunctive Therapies
  Rather than monotherapy, combining stabilizers, gene-silencers, and possibly antibody or small-molecule agents may increase effectiveness in halting disease progression.
  Adjunctive therapies targeting cardiac remodeling, neurohormonal modulation, or anti-fibrosis may complement TTR-targeted approaches.
  Clinical trials combining therapies (e.g. tafamidis + vutrisiran) are likely to test synergistic benefits.
  Such combinations may tailor therapy to disease stage and patient phenotypes (wild-type vs variant).
  Regulatory pathways for combination regimens will become a differentiator.
  Over time, the standard of care may shift to multi-mechanism regimens in ATTR-CM.

• Adoption of Digital Tools, AI & Real-World Evidence Integration
  Digital platforms (remote monitoring, wearable sensors) will support patient follow-up, adherence tracking, and event detection (e.g. arrhythmias, heart failure exacerbations).
  AI will assist in imaging interpretation (cardiac MRI, PET, echocardiography) to refine diagnosis and progression monitoring.
  Real-world evidence (RWE) registries will validate long-term safety, outcomes, and cost-effectiveness across broader populations.
  Data from electronic health records and claims will inform payer decisions and therapy value.
  Integration of digital endpoints may become part of reimbursement models.
  These technologies will support adoption, monitoring, and market differentiation.

• Regulatory and Safety Emphasis on Long-Term Outcome Data
  Regulatory agencies will increasingly demand long-term outcomes such as survival, cardiovascular events, and quality-of-life improvements.
  Safety monitoring for off-target effects, liver toxicity, immunogenicity, and long-term exposure will be critical.
  Post-marketing surveillance, registries, and risk mitigation plans will be mandatory, especially for novel gene-based therapies.
  Harmonization of regulatory standards across regions will facilitate global launches.
  Approvals may depend on surrogate biomarkers (e.g. TTR reduction, imaging changes), but regulators may insist on confirmatory endpoint trials.
  Safety/efficacy data transparency will be key to clinician and payer confidence.

Market Growth Drivers

• Elevated Prevalence and Underdiagnosis of ATTR-CM
  The actual prevalence of ATTR-CM is higher than historically recognized, particularly among elderly patients with heart failure with preserved ejection fraction (HFpEF).
  Increased awareness, screening, and non-invasive diagnostic adoption are converting a large undiagnosed reservoir into treated patients.
  The aging global population further increases incidence and prevalence.
  Many patients with unexplained cardiomyopathy or aortic stenosis are being reclassified to ATTR-CM.
  This expansion of the diagnosed base underpins sustained growth.
  As diagnosis gaps shrink, more patients will become eligible for therapy.

• Clinical Evidence of Mortality and Morbidity Benefit
  Newer therapies are showing statistically significant reductions in cardiovascular events, mortality, and hospitalizations.
  Improved functional metrics (e.g. 6-minute walk test) and quality-of-life gains support adoption.
  Treatments that more directly modify disease biology rather than just symptom relief are giving confidence to clinicians and payers.
  Growth is driven by regulators and payers granting differentiation to therapies with strong outcome data.
  This evidence base magnifies willingness to adopt newer agents over older ones.

• Innovation in Delivery, Dosing, and Safety Profiles
  Therapies with less frequent dosing (e.g. quarterly or biannual) and non-oral routes (e.g. subcutaneous) improve patient convenience and compliance.
  Improved delivery mechanisms, better safety margins, and reduced side effects will help broaden uptake.
  Therapies that avoid liver toxicity, immunogenicity, or off-target effects gain competitive advantage.
  Enhancements in manufacturing and formulation lower cost and increase scalability.
  Innovation that enhances therapeutic index directly supports broader adoption.

• Growing Investment & Pipeline Diversification
  Biotech and pharmaceutical companies are heavily investing in ATTR-CM as a high-value niche with expanding indication potential.
  Pipeline diversification—including monoclonal antibodies, antisense, CRISPR, and TTR degraders—de-risks the portfolio.
  Venture capital and public R&D funding support accelerated trials and regulatory submissions.
  Collaborations and licensing deals help smaller firms scale development.
  This investment environment fuels a continuous flow of novel therapies into clinical testing.

• Favorable Regulatory and Reimbursement Policies
  Regulators are open to accelerated pathways for therapies addressing unmet needs in rare cardiomyopathies.
  Breakthrough, priority review, and orphan designations support faster market entry.
  Payers are increasingly receptive to outcome-based contracts in chronic diseases.
  Health systems may support biomarker screening programs to enable earlier diagnosis.
  These policies lower entry barriers, manage risk, and facilitate adoption across regions.

• Synergy with the Broader Heart Failure & Cardiovascular Market
  ATTR-CM therapies intersect with heart failure treatment paradigms, enabling cross-therapeutic synergies.
  Patients on standard heart failure medications may also benefit from ATTR-targeted therapy, increasing overlap.
  Cardiologists managing HFpEF and cardiomyopathy cases will increasingly incorporate ATTR-CM diagnostics and therapy.
  Shared clinical infrastructure (imaging, biomarkers) accelerates adoption.
  This alignment with large cardiovascular markets enhances visibility and uptake.

Challenges in the Market

• High Cost and Pricing Pressure
  ATTR-CM treatments are high-cost biologics or gene therapies requiring premium pricing.
  Payers may resist full reimbursement absent strong long-term data, limiting access.
  Regional disparities in healthcare budgets make pricing more contentious in emerging markets.
  Balancing cost-effectiveness while maintaining innovation incentives is a delicate challenge.
  Therapy affordability and pricing negotiations will significantly influence market penetration.

• Diagnosis Gaps and Under-Recognition
  Many ATTR-CM cases remain undiagnosed due to overlap with other cardiomyopathies, low disease awareness, and limited access to advanced diagnostics.
  In many markets, imaging, biomarker, or nuclear scan infrastructure is insufficient.
  Delayed diagnosis reduces eligibility for early intervention, limiting patient pool.
  Educating cardiologists, internists, and primary care physicians is essential.
  Without improved detection, market potential cannot be fully realized.

• Long-Term Safety, Durability & Unknowns
  As many therapies are recently approved or in late-stage trials, long-term safety and durability data are limited.
  Risks like off-target effects, immunogenicity, hepatic or renal toxicity need monitoring.
  Durability of benefit, need for retreatment, or resistance may emerge over time.
  Uncertainties around lifetime therapy use may deter payers.
  Post-market surveillance, registries, and real-world evidence must bridge these gaps.

• Complex Regulatory and Approval Pathways Across Regions
  Regulatory requirements differ by country, especially concerning surrogate endpoint acceptance vs clinical outcomes.
  Global harmonization is limited, delaying multi-region launches.
  Some jurisdictions still require invasive endpoints (e.g. biopsy or long-term mortality) before approval.
  Navigating divergent regulations adds cost and complexity to global expansion.
  Companies must tailor clinical programs and regulatory strategies regionally.

• Manufacturing, Supply Chain & Scalability Constraints
  Producing biologics or gene-silencing therapies at scale with consistent quality is challenging and expensive.
  Cold chain, distribution, and supply logistics must maintain strict stability.
  Regional manufacturing hubs may be required to reduce lead times and costs.
  Limited capacity or process yield variability could bottleneck commercialization.
  Ensuring reliable supply while controlling cost is key to market success.

• Competition and Therapeutic Differentiation
  Multiple therapies (stabilizers, gene-silencers, antibodies) will compete for overlapping patient populations.
  Differentiators such as dosing frequency, safety, efficacy, convenience, and price will decide winners.
  Therapies entering later will need strong clinical and economic advantages to displace incumbents.
  Trials must prove superiority or non-inferiority to gain adoption.
  Market fragmentation can lead to competitive pricing pressures and consolidation.

ATTR-CM Market Segmentation

By Therapy Type

• Transthyretin Stabilizers (e.g. tafamidis, acoramidis)

• Gene-Silencing Therapies (RNAi, antisense oligonucleotides)

• Monoclonal Antibodies / Degraders

• Gene Editing / CRISPR Therapies

• Combination Therapies

By Disease Form

• Wild-Type ATTR-CM

• Variant (Hereditary) ATTR-CM

By End User / Setting

• Hospitals & Cardiology Specialty Clinics

• Academic Medical Centers & Research Hospitals

• Outpatient / Ambulatory Care Settings

By Route & Dosing Regimen

• Oral Daily

• Subcutaneous or Intramuscular Long-Interval

• Intravenous

By Region

• North America

• Europe

• Asia-Pacific

• Latin America

• Middle East & Africa

Leading Key Players

• Pfizer Inc. (tafamidis – Vyndaqel / Vyndamax)

• Alnylam Pharmaceuticals (vutrisiran / Amvuttra)

• BridgeBio Pharma / Ionis / Akcea (acoramidis)

• Novartis / Ionis (gene therapies / antisense programs)

• other biotech firms developing monoclonal antibodies, CRISPR-based solutions, or degraders

Recent Developments

• Alnylam secured FDA approval in March 2025 for its drug Amvuttra (vutrisiran) in ATTR-CM, expanding beyond its prior indication in neuropathy.

• Clinical trial results showed vutrisiran reduced mortality and cardiovascular events by ~28 % vs placebo, enhancing its market positioning.

• Pfizer’s tafamidis remains a key anchor in the market, with strong sales and clinical follow-up studies.

• BridgeBio’s acoramidis (Attruby) is being developed as a competitor stabilizer with differentiated profile and may capture share.

• Pipeline programs in monoclonal antibodies, TTR degraders, and gene editing approaches are under development to expand therapeutic options.

• Strategic partnerships and licensing deals are being formed to accelerate development and regional penetration.

This Market Report Will Answer the Following Questions

• What is the projected CAGR and market size for the ATTR-CM segment through 2031?

• Which therapy classes (stabilizers, RNAi, antisense, antibodies, gene editing) will dominate and why?

• How are diagnostic and patient identification innovations influencing addressable populations?

• What are the regulatory, reimbursement, and pricing challenges unique to ATTR-CM?

• Who are the key players and how are their strategies and pipeline differentiating?

• How will combination regimens and next-generation therapies evolve in this market?

• In which regions is growth likely to be fastest, and what are the access barriers?

• How will long-term safety, durability, and real-world evidence impact adoption?

• What role will outcome-based pricing, digital tools, and real-world data play in market uptake?

• What are the major risks and uncertainties that could slow market growth, and how can they be mitigated?