Amyloidosis Therapeutics Market Size & Share Analysis - Growth Trends and Forecast (2026 - 2031)

Global Amyloidosis Therapeutics Market Trends and Insights

Regulatory Approvals of Disease-Modifying Therapies

Regulatory activity between 2024 and 2026 has changed treatment choices in the amyloidosis therapeutics market. The FDA approved acoramidis for ATTR-CM in November 2024 after the ATTRibute-CM study reported a 42% reduction in the composite of all-cause mortality and recurrent cardiovascular hospitalizations over 30 months among 632 participants. The FDA then approved vutrisiran for ATTR-CM in March 2025, extending an RNA interference option across cardiomyopathy and polyneuropathy manifestations htm). Its approvals in the United States, Brazil, the European Union, and Canada may shorten the period before local coverage decisions because payers can compare decisions across markets. The traditional FDA approval of daratumumab-hyaluronidase-fihj plus VCd for newly diagnosed AL amyloidosis in November 2025 reinforced an established regimen after extended ANDROMEDA follow-up. These decisions increase the need for manufacturers to show practical value in addition to clinical benefit.

Aging Population and ATTR-CM Identification

ATTR cardiomyopathy mainly affects adults older than 65 years, and the incidence in the United States increased through 2023. Claims-based evidence reported ATTR-CM prevalence of 54.9 per million among older adults in the United States. Autopsy findings have also identified cardiac amyloid deposits in people who were not recognized clinically, indicating that diagnosed prevalence does not capture the full clinical burden. Earlier recognition can add new patients to the treated population without requiring patients to switch therapies. Japan has a reported ATTR-CM prevalence of 100 per million per year, which supports demand as its diagnosis pathway develops. Orphan-drug pathways administered by regulators such as the FDA and EMA also remain important for clinical development in rare diseases.

AI-Enabled Echocardiography and PYP Referral Pathways

Artificial intelligence-supported echocardiography is becoming part of the cardiac amyloidosis referral pathway. A 2025 multicenter study across 18 sites found that an AI model using echocardiographic video differentiated cardiac amyloidosis from phenotypic mimics with an AUROC of 0.93, 85% sensitivity, and 93% specificity.^{[1]“Cardiac Amyloidosis Detection From a Single Echocardiographic Video Clip, A Novel Artificial Intelligence-Based Screening Tool,”} Mayo Clinic reported that its AI echocardiography model received FDA Breakthrough Device designation for screening for amyloid cardiomyopathy.^{[2]Mayo Clinic, “Detecting Cardiac Amyloidosis Early From a Single AI-Enhanced Echocardiographic Video Clip,”} Earlier referral can bring patients into treatment before advanced organ involvement develops. This may support the use of TTR stabilizers and gene silencers in people who previously entered care later. It also places more attention on early efficacy evidence as diagnosis routes become more systematic.

Rising Disease Awareness and Earlier Diagnosis

Education efforts and patient advocacy have supported more awareness of ATTR amyloidosis among clinicians. New specialist centers opened in Tennessee and Lombardy during 2025, extending access beyond established referral locations.^{[3]“IRCCS Ospedale Galeazzi-Sant’Ambrogio Recognized as a Regional Reference Center for the Treatment of Amyloidosis,”} NHS England commissioned the Midlands Amyloidosis Service in Birmingham as a national referral service in 2025.^{[4]University Hospitals Birmingham, “Midlands Amyloidosis Service Brings Specialist Care Closer to Home,”} As community centers gain treatment capability, oral and subcutaneous regimens may be easier to use than infusion-based products. This could alter treatment selection outside academic hospitals. French early-access procedures for rare diseases have also allowed access to certain therapies before formal marketing authorization.

High Treatment and Lifetime Disease-Management Costs

The cost of long-term treatment can restrict access despite regulatory approval. AL amyloidosis care also requires resource-intensive anti-plasma-cell regimens, which can raise total healthcare use. Frequency-adjusted daratumumab regimens have been studied partly to reduce treatment intensity while maintaining efficacy. Patients with coverage can still face repeated prior authorization and the risk of delayed continuation. In Brazil, South Africa, and Gulf Cooperation Council states, the lack of rare-disease funding structures may delay reimbursed access after authorization. These limits can slow revenue realization in the amyloidosis therapeutics market even when the medical need is clear.

Payer Prior Authorization and Site-of-Care Fragmentation

United States insurers use prior authorization requirements for major ATTR therapies. Policies can require proof of diagnosis, cardiac imaging, New York Heart Association class, and documentation concerning combination therapy. Twelve-month approval periods add recurring administrative work for community practices. This burden is pronounced where new regional treatment programs are intended to expand access. Reimbursement can also differ between infusion centers and physician offices, even when the clinical choice favors subcutaneous delivery. In Germany, benefit assessments for vutrisiran illustrate how pricing and reimbursement review can slow a commercial launch after marketing authorization.

*Our forecasts treat driver/restraint impacts as directional, not additive. The impact forecasts reflect baseline growth, mix effects, and variable interactions.

Segment Analysis

By Amyloidosis Type: ATTR Volumes Rise While AL Maintains Revenue Leadership

AL amyloidosis accounted for 42.57% of the amyloidosis therapeutics market share in 2025. Its revenue position reflects long-standing clinical infrastructure for plasma-cell-directed treatment and high therapy cost per patient. The November 2025 traditional approval of daratumumab-hyaluronidase-fihj plus VCd established a durable standard for newly diagnosed AL amyloidosis. The extended ANDROMEDA follow-up reported a hazard ratio of 0.62 for overall survival. This treatment pathway supports continued demand for multi-agent anti-plasma-cell regimens.

ATTR amyloidosis is projected to record the fastest growth at 8.89% CAGR through 2031. More approved therapies and improved detection of older adults with unexplained heart failure support this expansion. AA amyloidosis remains the smallest revenue category and is primarily addressed by controlling its underlying inflammatory disease. IL-6 targeted biologics may indirectly affect care in this group. ATTR is likely to add patients faster, while AL retains higher revenue per patient because its treatment can include layered drug regimens. Regulatory requirements for orphan therapies continue to influence trial design and post-marketing monitoring across types.

By Therapy Class: TTR Stabilizers Lead While Gene Silencers Gain Ground

TTR stabilizers held 38.93% of the amyloidosis therapeutics market share in 2025. Oral tafamidis and acoramidis support this position in the ATTR-CM population. Stabilizers remain familiar to prescribers and fit routine outpatient use. Gene silencers are forecast to expand at 9.23% CAGR through 2031. Vutrisiran’s ATTR-CM approval and eplontersen’s established use in hereditary ATTR polyneuropathy have broadened the clinical role of this therapy class.

Amyloid depleters have yet to establish an approved role in AL amyloidosis. Prothena discontinued birtamimab in May 2025 after the Phase 3 AFFIRM-AL trial did not meet its primary endpoint in Mayo Stage IV AL amyloidosis. Attention has consequently shifted toward ATTR-focused antibodies, including ALXN2220 and coramitug. Anti-plasma-cell and chemotherapy regimens remain important in AL treatment. Formulary decisions made around stabilizer-first care are likely to be reassessed as cardiovascular outcome evidence for silencers accumulates. The amyloidosis therapeutics industry is thus balancing established oral treatment with newer approaches that reduce TTR production.

By Drug Modality: Small Molecules Lead While RNA Interference Grows Fastest

Small molecules held 52.91% of revenue in 2025. This leadership is supported by the oral use of tafamidis and acoramidis in a large ATTR-CM treated population. RNA interference is forecast to grow at 9.76% CAGR through 2031. In HELIOS-B, vutrisiran reduced the risk of all-cause mortality and recurrent cardiovascular events by 28% in the overall population and 33% in the monotherapy subgroup. These data provide a basis for differentiated use in ATTR-CM.

Genetic confirmation is increasingly used to guide modality selection for hereditary disease. Patients with pathogenic TTR variants and early neurological involvement may be considered for silencers. Plasma-derived and recombinant therapies remain supportive options for selected AL amyloidosis patients. Monoclonal antibodies include anti-plasma-cell therapies in AL disease and amyloid-clearing candidates in ATTR disease. Their future commercial role depends on Phase 3 outcomes. A 2026 review noted that GalNAc-conjugated siRNA platforms, including vutrisiran and eplontersen, allow subcutaneous administration and reduce dependence on infusion facilities.

By Formulation: Tablets Lead While Prefilled Syringes Build Momentum

Tablets held 51.47% of revenue in 2025. Tafamidis and acoramidis account for much of this share because both serve the ATTR-CM population through oral regimens. This formulation suits community prescribing and regular outpatient follow-up. Prefilled syringes are forecast to grow at 10.15% CAGR through 2031. Vutrisiran’s quarterly 25 mg subcutaneous prefilled syringe received approvals in several markets, including Canada, in December 2025.

Prefilled syringes can support care at home and reduce visits for infusion administration. Capsules, vials, and infusion products remain relevant in AL amyloidosis, where bortezomib combinations and daratumumab-based treatment are used. Manufacturers must consider how formulation affects care setting and reimbursement coding. These factors can influence the margin and service model associated with each therapy. The amyloidosis therapeutics market is moving toward a more varied delivery mix rather than a single preferred format. Clinical suitability will remain the main determinant for patients requiring complex regimens.

By Route of Administration: Subcutaneous Delivery Is the Fastest-Growing Route

Oral administration held 52.13% of revenue in 2025. The result is consistent with extensive use of tablet-formulated TTR stabilizers. Oral therapy remains convenient for many ATTR-CM patients. Subcutaneous delivery is forecast to grow at 10.94% CAGR through 2031. Vutrisiran and eplontersen support this route through treatments delivered at extended intervals.

Intravenous administration remains relevant for patisiran and for daratumumab-based AL regimens. Some ATTR patients also continue to receive care through infusion settings. More delivery choices create competition among specialty pharmacies, home infusion providers, and ambulatory sites. The clinical benefits and monitoring needs of a therapy will guide the appropriate site. Current ATTR therapies do not have FDA Risk Evaluation and Mitigation Strategy requirements. However, post-marketing safety surveillance can still affect future labels and route-specific prescribing guidance.

By End User: Specialty Centers Lead While Ambulatory Settings Expand

Hospitals accounted for 48.42% of revenue in 2025. These centers have historically concentrated diagnostic expertise, trials, and prescribing of high-cost therapies. Their multidisciplinary model is important for patients with cardiac, neurological, renal, and hematological complications. Ambulatory surgical centers are forecast to expand at 11.52% CAGR through 2031. Oral and subcutaneous treatments allow more management outside high-acuity inpatient settings.

The Chattanooga Heart Institute’s new dedicated center shows that community prescribing capacity is expanding beyond historic academic hubs. Home care is also becoming more relevant for prefilled syringe administration. Alnylam Assist includes support intended to reduce out-of-pocket costs for eligible patients. Specialty centers may increasingly differentiate through trials, advanced imaging, and multidisciplinary coordination. Routine dispensing and treatment monitoring can shift to ambulatory and home settings. This change widens potential access in the amyloidosis therapeutics market without replacing specialist oversight.

Geography Analysis

North America held 39.55% of the amyloidosis therapeutics market share in 2025. The United States combines active FDA decisions, established specialty pharmacy networks, and broad commercial coverage for vutrisiran. Among Medicare-eligible people, ATTR-CM incidence was 44.3 per 100,000 person-years in 2021 and had increased over time. This suggests that the identified disease remains below the potential prevalence level. Health Canada approved vutrisiran for ATTR-CM in December 2025, supporting Canadian uptake in 2026.

Europe added several important approvals during 2025. The European Commission authorized vutrisiran for ATTR-CM in June 2025. Germany’s benefit evaluations for acoramidis and vutrisiran provide reference points for reimbursement negotiations. NHS England’s Midlands Amyloidosis Service extended access beyond London’s National Amyloidosis Center.

Asia-Pacific is expected to record a 12.29% CAGR through 2031, the fastest rate among regions. Japan’s ATTR-CM prevalence of 100 per million per year offers a large patient base as identification improves. Acoramidis received Japanese approval in March 2025, giving Japan access to both major TTR stabilizer classes. China has established a precedent for ATTR treatment through tafamidis, while AstraZeneca is studying eplontersen in Chinese ATTR-CM patients. Brazil approved vutrisiran for ATTR-CM in March 2025, but access elsewhere remains limited by specialist capacity and patient costs.