Transthyretin Amyloidosis Treatment Market Size & Share Analysis - Growth Trends And Forecast (2025 - 2030)

Global Transthyretin Amyloidosis Treatment Market Trends and Insights

Regulatory approvals of disease-modifying drugs

Three pivotal approvals in 2024-2025 redefined the transthyretin amyloidosis treatment market. The FDA cleared acoramidis in November 2024 after a Phase 3 trial showed 42% fewer deaths and hospitalizations than placebo^{[1]U.S. Food and Drug Administration, “FDA approves drug for heart disorder caused by transthyretin-mediated amyloidosis,” fda.gov}. Vutrisiran followed in March 2025, reducing all-cause mortality by 36% in 655 patients. Europe approved acoramidis in November 2025, and Japan did so in March 2025, creating near-simultaneous global access. These decisions end the tafamidis monopoly, introduce price competition, and broaden mechanistic diversity for different disease stages.

Intensifying clinical-trial pipeline (RNA silencers, gene editing)

CRISPR innovation accelerated as Intellia’s single-dose NTLA-2001 cut mean serum TTR 91% at day 28 in 72 participants. Alnylam’s nucresiran achieved 96% peak reduction with biannual dosing, ushering in longer-acting regimens. Intellia’s 765-patient MAGNITUDE Phase 3 study marks the first pivotal in-vivo gene-editing trial, while Neurimmune’s ALXN2220 Phase 3 effort introduces amyloid depletion. Pipeline breadth supports precision treatment algorithms matched to disease phenotype, mutation, and stage.

Rising disease awareness via centres & advocacy groups

International registries revealed 18% ATTR-CM prevalence among high-risk heart-failure cohorts, up from earlier estimates. The Mayo Clinic detected cardiac amyloidosis in 1.25% of > 31,000 echocardiograms^{[2]American College of Cardiology, “Efficacy and Safety of Acoramidis,” acc.org}. Consensus guidelines published in 2024 recommend routine screening in bilateral carpal-tunnel patients, where 13.4% had ATTR-CM. BridgeBio’s TRACE-AI network deploys machine-learning tools across health systems, multiplying case-finding beyond specialist referrals. Earlier identification enlarges the treatable population and sustains market growth.

AI-enabled echocardiography screening tools

AI models now detect cardiac amyloidosis with 91.1% AUC on routine ECGs. Pfizer’s hospital-based deployments facilitate large-scale automated image analysis, flagging candidates for confirmatory scans. The PANES-HF study documented novice echocardiography paired with AI yielding 84.6% sensitivity and 91.4% specificity. Multicentre algorithms standardize cardiac scintigraphy interpretation, minimizing inter-reader variability. These advances shorten diagnostic latency from 8.6 years to months, expanding the transthyretin amyloidosis treatment market.

Ultra-high therapy costs

Annual list prices surpass USD 225,000 for tafamidis and USD 244,000 for acoramidis, placing them among the costliest cardiovascular drugs. Health systems adopting value-based frameworks demand real-world outcome data to justify budgets, and insurers impose step-therapy rules before covering premium agents. These economics restrict uptake in lower-income regions and could temper overall market velocity unless negotiated discounts widen access.

Under-diagnosis due to non-specific symptoms

Up to 80% of patients remain unidentified worldwide. Wild-type disease often presents with musculoskeletal or autonomic symptoms that delay suspicion. European centers detect 24% prevalence in high-risk heart-failure clinics compared with 5% in North America, underscoring variable awareness. In 5% of biopsy-confirmed cases, bone-scintigraphy is negative, necessitating invasive biopsy^{[3]Orphanet Journal of Rare Diseases, “Bone scintigraphy limitations in cardiac uptake,” ojrd.com}. Education and AI-driven diagnostics are expected to narrow this gap.

Segment Analysis

By Amyloidosis Type: Cardiac Manifestations Drive Market Leadership

ATTR-CM generated 63.55% of 2024 revenue, underpinned by validated scintigraphy and echocardiography pathways and robust cardiology networks. The transthyretin amyloidosis treatment market size for cardiac phenotypes is projected to progress at double-digit CAGR, reinforced by mortality-lowering data from both stabilizers and silencers. ATTR-PN, although smaller, is on a 29.25% trajectory as vutrisiran and eplontersen win broader neurology indications.

Mixed-phenotype cases form a rising clinical subset, prompting combined organ-targeted strategies. THAOS registry evidence shows high-dose tafamidis delivering neurological stability in cardiac-dominant patients, illustrating tailored dosing. Early carpal-tunnel signs, detected in 22% of cardiac patients during routine orthopedic surgery, serve as a cross-phenotype screening trigger. Continuous phenotype refinement sustains stratified demand in the transthyretin amyloidosis treatment market.

Note: Segment shares of all individual segments available upon report purchase

By Therapy: Stabilizers Maintain Dominance Amid Silencer Innovation

Stabilizers retained 53.53% revenue share in 2024, paced by once-daily oral tafamidis and acoramidis, the latter achieving ≥ 90% stabilization. The transthyretin amyloidosis treatment market size attributed to stabilizers is forecast to rise steadily yet cede proportion to silencers as mortality curves favor RNA interference.

Silencers are advancing 30.85% annually, aided by quarterly subcutaneous dosing and dual cardiac-neurologic labeling. Gene editing threatens to redraw boundaries; NTLA-2001’s 91% knockdown after a single infusion positions it as a future one-time cure. Depleter antibodies such as ALXN2220 add further mechanistic variety, ensuring multi-modal regimens will characterize the transthyretin amyloidosis treatment market.

By Disease Type: Wild-Type Prevalence Drives Hereditary Innovation

Wild-type disease, representing 55.63% of 2024 sales, dominates aging Western populations. Prevalence reaches 16.8% among preserved-ejection-fraction heart-failure patients, reinforcing volume growth. Hereditary variants grow 32.87% per year as sequencing panels become routine and ethnic-specific mutations receive targeted outreach. The transthyretin amyloidosis treatment market share for hereditary forms is set to widen with mutation-directed registries and newborn-screening pilots.

Ethnic clustering shapes regional strategies: Val122Ile affects 3.4% of African Americans, Ala97Ser is prevalent in East Asia, and Val50Met remains endemic in Portugal and Sweden. Precision screening plus pipeline mutation-specific agents position hereditary segments for outsized contribution to overall transthyretin amyloidosis treatment industry expansion.

Note: Segment shares of all individual segments available upon report purchase

By Distribution Channel: Hospital Networks Dominate Specialty Growth

Hospital pharmacies controlled 68.13% spending in 2024 owing to infusion infrastructure and cardiology-neurology co-management. Specialty pharmacies, however, are scaling at 29.7% as home-based RNA administration and manufacturer patient-service hubs proliferate. The transthyretin amyloidosis treatment market size captured by specialty channels is set to rise further after the 2025 Medicare Part D redesign limits out-of-pocket liability to USD 2,000, a policy likely to migrate to other advanced economies.

Retail pharmacies stay marginal because prior-authorisation steps and cold-chain complexities deter stocking. As gene-editing therapies reach commercialization, single-dose hospital infusion combined with lifelong hematology oversight will reinforce hospital dominance while enlarging specialty follow-up services.

Geography Analysis

North America retained 47.63% revenue in 2024, sustained by early FDA approvals, Medicare coverage, and > 1,300 dedicated amyloid clinics. Mayo Clinic data show 1.25% amyloidosis prevalence in echocardiography cohorts, underscoring strong screening penetration. The region’s Part D cost cap entering 2025 improves affordability, likely raising prescription volume for premium agents.

Europe benefits from unified regulatory decisions; the November 2025 acoramidis approval synchronized launches across 27 EU states. Diagnostic prevalence is 24% in high-risk heart-failure clinics, higher than other regions. Germany and Italy host robust amyloidosis centers, while the Bayer-BridgeBio alliance ensures commercial reach. Emerging EU markets leverage cross-border treatment programs, widening access without duplicative regulatory delays.

Asia-Pacific posts the fastest CAGR at 18.87% through 2030. Japan approved acoramidis in March 2025, establishing first-in-class access and reimbursement inside a mature cardiovascular market. China’s EPIC-ATTR study evaluates eplontersen among domestic patients, supporting eventual National Reimbursement Drug List inclusion. Australia’s RNA Blueprint underpins regional production, while Korea’s 13.4% prevalence in preserved-ejection-fraction cohorts points to latent demand. These dynamics collectively bolster the transthyretin amyloidosis treatment market across APAC.