가족성 아밀로이드 다발신경병증 : 시장 점유율 분석, 업계 동향과 통계, 성장 예측(2026-2031년)

According to Mordor Intelligence, the familial amyloid polyneuropathy market size is expected to grow from USD 3.38 billion in 2025 to USD 3.67 billion in 2026 and is forecast to reach USD 5.56 billion by 2031 at 8.66% CAGR over 2026-2031.

Global Familial Amyloid Polyneuropathy Market Trends and Insights

Expansion of Disease-Modifying Therapy Approvals

The familial amyloid polyneuropathy market broadened between late 2024 and mid-2026 as several therapies reached key regulatory milestones. The FDA approved a supplemental new drug application for vutrisiran, marketed as AMVUTTRA, for ATTR-CM in March 2025. The approval made AMVUTTRA the first medicine approved for both hATTR-PN and ATTR-CM. It also enabled providers to serve both conditions through an established specialty pharmacy infrastructure. The European Commission approved eplontersen, marketed as WAINZUA, for Stage 1 and Stage 2 ATTRv-PN in March 2025. WAINZUA was the only EU-approved medicine for this indication that could be delivered through a monthly self-administered autoinjector at that time. The European Commission also granted marketing authorization for ATTROGY, diflunisal from Purpose Pharma, in July 2025 for adults with Stage 1 or Stage 2 polyneuropathy. The pivotal Phase 3 study showed an 18-point NIS+7 advantage over placebo at 24 months.

Growing RNA-Silencer Adoption

RNA-silencing medicines have become an important treatment mechanism in the familial amyloid polyneuropathy market. These medicines include small interfering RNAs and antisense oligonucleotides that reduce transthyretin production. Their use has reduced the central role once held by liver transplantation for suppressing transthyretin production. AMVUTTRA, a quarterly subcutaneous siRNA, generated USD 2.31 billion in 2025 global net product revenue. This represented a 138% increase from USD 970 million in 2024, following the March 2025 ATTR-CM approval. ONPATTRO revenue declined 32% to USD 172.8 million in 2025 as patients moved to the once-quarterly formulation. Eplontersen generated USD 212 million in 2025 while its EU launch progressed, and an ATTR-CM application was expected in 2026. The shift toward quarterly and monthly dosing is moving care toward home-based administration and is changing distribution needs in the familial amyloid polyneuropathy market.

Ultra-High Treatment Cost and Reimbursement Friction

Treatment cost is a continuing limit on patient access in the familial amyloid polyneuropathy market. Annual list prices ranged from USD 244,000 for acoramidis at launch to USD 464,000 for vutrisiran. European health-technology assessment bodies have often linked reimbursement to post-marketing evidence obligations. This approach can leave diagnosed patients untreated while coverage decisions move through payer systems. It can also give payers greater influence over formulary placement and rebate rates when several branded medicines compete for the same patients. EU orphan-medicine rules and national pricing systems in Japan and Germany can support entry while also placing pressure on net revenue through the product lifecycle. Cost pressure is therefore likely to affect access patterns even where diagnostic capacity is improving.

Other drivers and restraints analyzed in the detailed report include:

1. Earlier Genetic Testing and Multisystem Diagnosis
2. Family Cascade Testing and Specialist Care Capacity
3. Regulatory, Safety-Monitoring, and Vitamin A Management Requirements

For complete list of drivers and restraints, kindly check the Table Of Contents.

Segment Analysis

Treatment accounted for 82.31% of the familial amyloid polyneuropathy market share in 2025. Disease-modifying transthyretin stabilizers and RNA silencers supported this position. TTR gene silencers have become a major source of growth within treatment. AMVUTTRA recorded USD 2.31 billion in 2025 net product revenue, while eplontersen recorded USD 212 million in its launch year. TTR stabilizers retain a substantial installed patient base. Clinical evidence indicates that gene-silencing approaches can reduce circulating transthyretin by 80% to 90%, while oral medicines provide partial stabilization. Prescribing may continue to move toward silencers among clinicians treating progressive polyneuropathy. Gene-editing therapies remained investigational in 2026, and MAGNITUDE-2 resumed enrollment after the FDA lifted its hold.

Diagnosis is forecast to grow at a 10.25% CAGR through 2031. The familial amyloid polyneuropathy market size for diagnosis is supported by reimbursement for hereditary amyloidosis sequencing panels and nuclear scintigraphy. Single-gene TTR sequencing has shown a 15.6% positivity rate in genetics-referred cohorts. This performance creates a reason to use targeted protocols in high-suspicion groups. Bone-avid radiotracer imaging can enable biopsy-free ATTR diagnosis when AL amyloidosis has been excluded. Tissue biopsy and amyloid subtyping remain relevant when imaging results are unclear or when immunohistochemistry is needed. Multisystem assessments that combine genetics and imaging can increase the number of services used in each diagnostic episode. This supports diagnostic revenue growth above the overall rate for the familial amyloid polyneuropathy market.

Complete Report Scope:

• By Modality
  • Treatment
    • TTR Stabilizers
    • TTR Gene Silencers
    • Gene-Editing Therapies
    • Supportive Care
  • Diagnosis
    • TTR Single-Gene Sequencing
    • Hereditary Amyloidosis Next-Generation Sequencing Panels
    • Targeted Variant Testing
    • Tissue Biopsy and Amyloid Subtyping
    • Nuclear Scintigraphy and Cardiac Imaging
    • Others
• By Disease Stage
  • Stage 1, Ambulation Without Assistance
  • Stage 2, Ambulation With Assistance
  • Stage 3, Wheelchair-Dependent Disease
  • Presymptomatic Pathogenic TTR-Variant Carriers
• By Distribution Channel
  • Hospital Pharmacies
  • Retail Pharmacies
  • Online and Digital Fulfillment
  • Other Distribution Channels
• By Geography
  • North America
    • United States
    • Canada
    • Mexico
  • Europe
    • Germany
    • United Kingdom
    • France
    • Italy
    • Spain
    • Rest of Europe
  • Asia-Pacific
    • China
    • Japan
    • India
    • Australia
    • South Korea
    • Rest of Asia-Pacific
  • Middle East and Africa
    • GCC
    • South Africa
    • Rest of Middle East and Africa
  • South America
    • Brazil
    • Argentina
    • Rest of South America

Geography Analysis

North America held 45.61% of the familial amyloid polyneuropathy market share in 2025. The United States has been the primary approval and launch location for major transthyretin-targeting medicines. The March 2025 FDA approval of vutrisiran for ATTR-CM expanded prescribing beyond neurology into cardiology. This added a larger patient group to specialty pharmacy systems already serving hATTR-PN. Canada and Mexico contribute smaller revenue shares. Their uptake is affected by formulary review schedules and specialist center availability in major metropolitan areas. North American competition also reflects the substantial commercial role of branded therapy.

Europe has concentrated endemic clusters in Portugal and Sweden and a developed clinical research base. Its adoption patterns vary because health-technology assessments and reimbursement decisions are made country by country. Eplontersen received European Commission approval for Stage 1 and Stage 2 ATTRv-PN in March 2025. Germany, France, Spain, Italy, and the United Kingdom remain major revenue pools for the familial amyloid polyneuropathy market. EU orphan-medicine designations can support access but are followed by price negotiations and evidence obligations. Portugal's approach to family monitoring supports earlier identification within endemic clusters. The region combines strong clinical capacity with uneven access to high-cost medicines.

Asia-Pacific is forecast to grow at an 11.25% CAGR through 2031. Japan's multidisciplinary care pathways coordinate genetic testing, neurological assessment, and cardiac imaging through regional referral networks. China's approval of eplontersen in 2025 expanded access to an RNA-silencing option in a large rare-disease population. South Korea and Australia have supporting conditions through RNAi research activity and an orphan-drug access pathway, respectively. Brazil also remains important because of its endemic Val30Met cluster. ANVISA approved BEYONTTRA, acoramidis, for ATTR-CM in Brazil in May 2026. BridgeBio planned commercialization with Biopas in the second half of 2026.

1. Alnylam Pharmaceuticals
2. Ambry Genetics
3. Arcturus Therapeutics Holdings Inc.
4. ARUP Laboratories
5. AstraZeneca
6. Attralus, Inc.
7. BridgeBio Pharma, Inc.
8. Corino Therapeutics, Inc.
9. Intellia Therapeutics, Inc.
10. Invitae
11. Ionis Pharmaceuticals
12. LabCorp
13. Life Molecular Imaging GmbH
14. Neurimmune AG
15. Novo Nordisk
16. Pfizer
17. Proclara Biosciences, Inc.
18. Prothena Corporation plc
19. Regeneron Pharmaceuticals
20. Silence Therapeutics plc
21. SOM BIOTECH, S.L.
22. Takeda Pharmaceuticals
23. YolTech Therapeutics Co., Ltd.

Additional Benefits:

• The market estimate (ME) sheet in Excel format
• 3 months of analyst support

TABLE OF CONTENTS

1 Introduction

• 1.1 Study Assumptions and Market Definition
• 1.2 Scope of the Study

2 Research Methodology

3 Executive Summary

4 Market Landscape

• 4.1 Market Overview
• 4.2 Market Drivers
  • 4.2.1 Earlier Genetic Testing and Multisystem Diagnosis
  • 4.2.2 Expansion of Disease-Modifying Therapy Approvals
  • 4.2.3 Growing RNA-Silencer Adoption
  • 4.2.4 Expansion of Specialist Amyloidosis Centers
  • 4.2.5 Family Cascade Testing in Endemic Variant Clusters
  • 4.2.6 Monthly and Quarterly Self-Administration Shifting the Site of Care
• 4.3 Market Restraints
  • 4.3.1 Ultra-High Treatment Cost and Reimbursement Friction
  • 4.3.2 Regulatory, Safety-Monitoring and Vitamin A Management Requirements
  • 4.3.3 Genotype and Phenotype Heterogeneity Complicating Endpoint Design
  • 4.3.4 Limited Infusion, Genetic-Counseling and Rare-Disease Laboratory Capacity
• 4.4 Value Chain Analysis
• 4.5 Regulatory Landscape
• 4.6 Technological Outlook
• 4.7 Porter's Five Forces Analysis
  • 4.7.1 Threat of New Entrants
  • 4.7.2 Bargaining Power of Suppliers
  • 4.7.3 Bargaining Power of Buyers
  • 4.7.4 Threat of Substitutes
  • 4.7.5 Competitive Rivalry

5 Market Size & Growth Forecasts (Value, USD)

• 5.1 By Modality
  • 5.1.1 Treatment
    • 5.1.1.1 TTR Stabilizers
    • 5.1.1.2 TTR Gene Silencers
    • 5.1.1.3 Gene-Editing Therapies
    • 5.1.1.4 Supportive Care
  • 5.1.2 Diagnosis
    • 5.1.2.1 TTR Single-Gene Sequencing
    • 5.1.2.2 Hereditary Amyloidosis Next-Generation Sequencing Panels
    • 5.1.2.3 Targeted Variant Testing
    • 5.1.2.4 Tissue Biopsy and Amyloid Subtyping
    • 5.1.2.5 Nuclear Scintigraphy and Cardiac Imaging
    • 5.1.2.6 Others
• 5.2 By Disease Stage
  • 5.2.1 Stage 1, Ambulation Without Assistance
  • 5.2.2 Stage 2, Ambulation With Assistance
  • 5.2.3 Stage 3, Wheelchair-Dependent Disease
  • 5.2.4 Presymptomatic Pathogenic TTR-Variant Carriers
• 5.3 By Distribution Channel
  • 5.3.1 Hospital Pharmacies
  • 5.3.2 Retail Pharmacies
  • 5.3.3 Online and Digital Fulfillment
  • 5.3.4 Other Distribution Channels
• 5.4 By Geography
  • 5.4.1 North America
    • 5.4.1.1 United States
    • 5.4.1.2 Canada
    • 5.4.1.3 Mexico
  • 5.4.2 Europe
    • 5.4.2.1 Germany
    • 5.4.2.2 United Kingdom
    • 5.4.2.3 France
    • 5.4.2.4 Italy
    • 5.4.2.5 Spain
    • 5.4.2.6 Rest of Europe
  • 5.4.3 Asia-Pacific
    • 5.4.3.1 China
    • 5.4.3.2 Japan
    • 5.4.3.3 India
    • 5.4.3.4 Australia
    • 5.4.3.5 South Korea
    • 5.4.3.6 Rest of Asia-Pacific
  • 5.4.4 Middle East and Africa
    • 5.4.4.1 GCC
    • 5.4.4.2 South Africa
    • 5.4.4.3 Rest of Middle East and Africa
  • 5.4.5 South America
    • 5.4.5.1 Brazil
    • 5.4.5.2 Argentina
    • 5.4.5.3 Rest of South America

6 Competitive Landscape

• 6.1 Market Concentration
• 6.2 Market Share Analysis
• 6.3 Company Profiles (includes Global Level Overview, Market Level Overview, Core Segments, Financials as available, Strategic Information, Market Rank/Share, Products and Services, Recent Developments)
  • 6.3.1 Alnylam Pharmaceuticals, Inc.
  • 6.3.2 Ambry Genetics
  • 6.3.3 Arcturus Therapeutics Holdings Inc.
  • 6.3.4 ARUP Laboratories
  • 6.3.5 AstraZeneca PLC
  • 6.3.6 Attralus, Inc.
  • 6.3.7 BridgeBio Pharma, Inc.
  • 6.3.8 Corino Therapeutics, Inc.
  • 6.3.9 Intellia Therapeutics, Inc.
  • 6.3.10 Invitae Corporation
  • 6.3.11 Ionis Pharmaceuticals, Inc.
  • 6.3.12 Labcorp
  • 6.3.13 Life Molecular Imaging GmbH
  • 6.3.14 Neurimmune AG
  • 6.3.15 Novo Nordisk A/S
  • 6.3.16 Pfizer Inc.
  • 6.3.17 Proclara Biosciences, Inc.
  • 6.3.18 Prothena Corporation plc
  • 6.3.19 Regeneron Pharmaceuticals, Inc.
  • 6.3.20 Silence Therapeutics plc
  • 6.3.21 SOM BIOTECH, S.L.
  • 6.3.22 Takeda Pharmaceutical Company Limited
  • 6.3.23 YolTech Therapeutics Co., Ltd.

7 Market Opportunities and Future Outlook

• 7.1 White-Space and Unmet-Need Assessment