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Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Market

DelveInsight’s latest report, “Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Market Insights, Epidemiology, and Market Forecast – 2032,” provides an in-depth analysis of the ATTR-CM landscape across the 7MM (the United States, EU4, the United Kingdom, and Japan), offering a comprehensive view of disease epidemiology, emerging treatment paradigms, and evolving market dynamics through 2032.

Intellia Therapeutics has announced encouraging longer-term Phase 1 results for its investigational gene-editing therapy, Nexiguran Ziclumeran (nex-z), in patients with Transthyretin (ATTR) Amyloidosis with Cardiomyopathy, reinforcing optimism within the ATTR-CM treatment landscape. Developed using cutting-edge CRISPR-based in vivo editing technology, the therapy achieved sustained reductions in transthyretin levels along with a favorable safety profile over extended follow-up periods. These findings highlight Nexiguran Ziclumeran’s potential to become the first single-dose, disease-modifying therapy for ATTR-CM, representing a pivotal step forward in precision medicine and the treatment of rare cardiovascular disorders. This breakthrough reflects the growing momentum of transformative gene-editing innovations poised to redefine therapeutic standards for amyloidosis and beyond.

Gain exclusive insights into how gene-editing innovations and next-generation therapies are transforming the ATTR-CM landscape – Transthyretin Amyloid Cardiomyopathy Market Forecast

Understanding ATTR-CM

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) is a rare, life-threatening disease caused by the deposition of misfolded transthyretin (TTR) proteins in the heart, leading to restrictive cardiomyopathy and heart failure. The condition can manifest as either wild-type ATTR-CM, typically associated with aging, or hereditary ATTR-CM, resulting from genetic mutations in the TTR gene.

Despite being underdiagnosed for decades, increased awareness, advanced imaging modalities, and genetic testing have significantly improved early diagnosis and management in recent years.

From Pfizer and Alnylam to Intellia Therapeutics, understand how top players are advancing breakthrough solutions for rare cardiac diseases. Download the complete market report from DelveInsight to stay ahead of industry trends – Transthyretin Amyloid Cardiomyopathy Market Trends and Insights

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Epidemiology

The prevalence of ATTR-CM remains insufficiently defined due to underdiagnosis and delayed detection, largely attributed to its varied clinical presentation and the historical absence of sensitive diagnostic tools. However, advancements in nuclear cardiac imaging—particularly the use of technetium pyrophosphate scans—have enabled non-invasive diagnosis without the need for cardiac biopsy. As a result, more patients are now being accurately identified, contributing to an apparent rise in prevalence. In the United States alone, approximately 5,000 to 7,000 new cases are diagnosed each year. Studies indicate that around 20% of patients with heart failure and increased myocardial wall thickness may have ATTR-CM, highlighting a growing recognition of the disease as survival outcomes improve.

Wild-type ATTR-CM (wATTR-CM) represents the more prevalent form of the condition, typically occurring in older adults, with a notable predominance among men. Research has shown that the occurrence of wATTR deposits increases with age and is frequently associated with other cardiac conditions common in elderly populations, such as aortic stenosis, atrial fibrillation, and heart failure with preserved ejection fraction (HFpEF). Recent community-based studies have also identified a significant prevalence of ATTR-CM in older male patients exhibiting HFpEF and left ventricular hypertrophy, reinforcing its growing clinical relevance.

Leverage DelveInsight’s in-depth data on epidemiology, treatment trends, and pipeline developments to inform business decisions and R&D investments – Transthyretin Amyloid Cardiomyopathy Prevalence

ATTR-CM Market Insights and Outlook

According to DelveInsight’s analysis, the ATTR-CM market across the 7MM is poised for substantial expansion through 2032, driven by an increasing diagnosed patient pool, improved therapeutic awareness, and the emergence of next-generation disease-modifying therapies.

The market growth is further supported by novel drug classes such as TTR stabilizers, silencers, and gene editing therapies that aim to slow or halt disease progression rather than merely alleviating symptoms.

Key pharmaceutical players, including Pfizer, Alnylam Pharmaceuticals, Ionis Pharmaceuticals, Intellia Therapeutics, and Eidos Therapeutics, are leading the competitive landscape with differentiated approaches targeting transthyretin production, aggregation, and stabilization.

Delve deeper into evolving diagnostics, emerging therapies, and the expanding patient base fueling the ATTR-CM market through 2032 – Transthyretin Amyloid Cardiomyopathy Companies and FDA Approvals

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Pipeline Developments: Intellia’s Breakthrough in Gene Editing

Adding to the optimism surrounding the ATTR-CM space, Intellia Therapeutics recently presented positive longer-term Phase 1 data for its investigational gene-editing therapy, Nexiguran Ziclumeran (nex-z), in patients with ATTR Amyloidosis with Cardiomyopathy.

The therapy, developed using CRISPR-based in vivo editing technology, demonstrated sustained transthyretin reduction and favorable safety outcomes over extended follow-up periods. The data reinforce Nexiguran Ziclumeran’s potential as the first single-dose, potentially curative therapy for patients suffering from ATTR-CM, marking a significant step forward in precision medicine and rare disease treatment.

This milestone underscores a broader industry trend toward transformative genetic therapies, which could redefine treatment standards for amyloidosis and related disorders.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Market Dynamics

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Market Drivers

1. Advancements in Diagnostic Technologies The emergence of non-invasive imaging techniques, particularly nuclear cardiac imaging using technetium pyrophosphate (PYP) scans, has significantly improved the early detection and accurate diagnosis of ATTR-CM. This has led to greater disease awareness and identification, contributing to market growth.
2. Growing Disease Awareness Among Physicians and Patients Increased clinical understanding of ATTR-CM’s pathophysiology and symptom overlap with other cardiac disorders has resulted in better screening and earlier diagnosis, expanding the patient pool eligible for treatment.
3. Expanding Therapeutic Pipeline and Novel Drug Approvals The development and commercialization of disease-modifying therapies such as tafamidis (Vyndaqel/Vyndamax), patisiran, vutrisiran, and eplontersen have transformed disease management. Ongoing research on next-generation TTR stabilizers and gene-silencing therapies continues to propel market expansion.
4. Improved Survival and Growing Prevalence With improved treatment outcomes and prolonged survival, the number of patients living with ATTR-CM is rising, thereby increasing the demand for continuous medical management and innovative therapies.
5. Increased Screening in At-Risk Populations Routine screening among elderly populations and patients with heart failure with preserved ejection fraction (HFpEF) or aortic stenosis is leading to a surge in newly diagnosed cases, fueling the overall market demand.

Transthyretin Amyloid Cardiomyopathy (ATTR-CM) Market Barriers

1. High Cost of Treatment and Limited Accessibility ATTR-CM therapies, particularly novel gene-silencing and stabilizing drugs, are extremely expensive. The high treatment costs and limited reimbursement policies in several regions restrict patient access and hinder widespread adoption.
2. Underdiagnosis and Misdiagnosis Despite technological progress, ATTR-CM remains underdiagnosed due to its overlapping symptoms with other cardiac conditions. Many patients still face delays in diagnosis, leading to missed treatment opportunities.
3. Limited Availability of Diagnostic Infrastructure Advanced imaging modalities and specialized diagnostic centers are not uniformly available across all regions, especially in developing countries, limiting early detection and disease management.
4. Regulatory and Clinical Challenges Stringent regulatory requirements, coupled with the need for large, long-term clinical trials to prove efficacy and safety, can slow down the introduction of new therapies into the market.
5. Competition from Off-Label and Supportive Therapies Off-label use of existing cardiovascular drugs and supportive therapies may compete with specialized ATTR-CM treatments, particularly in cost-sensitive markets, limiting the uptake of approved disease-specific drugs.

ATTR-CM Competitive Landscape and Future Outlook

With multiple late-stage candidates in development and breakthrough innovations like Intellia’s Nexiguran Ziclumeran, the ATTR-CM market is on the brink of transformation. As therapeutic modalities evolve from chronic management to potential one-time curative options, the competitive environment will intensify, fostering both scientific progress and patient-centric outcomes.

DelveInsight projects significant market growth through 2032, as emerging therapies gain approval and expand access globally. The shift toward precision medicine, combined with a better understanding of amyloid pathophysiology, positions the ATTR-CM space as one of the most promising in the rare cardiovascular disease segment.

Access detailed competitive intelligence and clinical insights in DelveInsight’s comprehensive report – ATTR-CM Clinical Trials and Drugs Analysis

About DelveInsight

DelveInsight is a leading Business Consulting and Market Research firm focused exclusively on the life sciences sector. The company provides comprehensive market intelligence, competitive insights, and strategic consulting to support pharmaceutical, biotech, and medical device companies worldwide in making informed decisions.

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