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시장보고서
상품코드
2082911
아밀로이드증 : 시장 인사이트, 역학 및 시장 예측(2036년)Amyloidosis - Market Insight, Epidemiology, and Market Forecast - 2036 |
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DelveInsight
본 아밀로이드증 시장 보고서에서는 표준 치료, 임상 실무, 진화하는 치료 알고리즘 등 현재의 치료 현황에 대한 종합적인 분석을 제공합니다. 또한, 아밀로이드증 환자의 부담 추이, 수익 및 시장 점유율 추이, 정점 시의 환자 점유율 및 치료 도입률 분석을 평가함과 동시에, 세계 각 지역 시장 규모에 대한 상세한 평가 및 성장률 예측(과거 데이터 및 2022년-2036년 예측)을 제시하고 있습니다. 본 보고서는 아밀로이드증 분야에서 주요 미충족 의료 수요를 부각시키고, 경쟁 환경 및 임상 상황을 분석하여 고부가가치 기회를 도출함으로써, 향후 시장 성장 가능성에 대한 명확한 전망을 제시하고 있습니다.
아밀로이드증에 대한 질환 인지도 및 진단율 향상
아밀로이드증의 전반적인 유병률 및 진단률은 이 질환에 대한 인식 제고, 첨단 영상 진단 및 바이오마커 기반 진단법의 보급, 유전자 검사 이용 확대, 그리고 AL형과 ATTR형 아형의 감별 정확도 향상으로 인해 증가하고 있으며, 이는 시장의 지속적인 성장을 뒷받침하고 있습니다.
표적 치료 및 질환 수정 치료 분야의 기회 확대
아밀로이드증 시장은 RNA 침묵제, 단일클론 항체, 형질세포를 표적으로 하는 치료법, 그리고 아밀로이드 생성 감소, 장기 반응 개선, 나아가 장기적인 질병 진행 양상을 변화시킬 가능성을 목표로 하는 새로운 유전자 편집 접근법 등 표적 치료법의 급속한 발전에 힘입어 성장하고 있습니다.
조기 진단과 다직종 협력을 통한 질환 관리에 대한 관심 증가
특히 심장 아밀로이드증 및 신경계 아밀로이드증의 경우, 조기 진단과 다학제적 협력을 통한 질환 관리에 대한 임상적 관심이 높아짐에 따라, ATTR 및 AL 아밀로이드증 환자 집단 모두에서 치료 대상이 되는 환자층이 확대되고 있으며, 치료법의 도입이 가속화되고 있습니다.
아밀로이드증 분야의 새로운 경쟁 구도
차세대 트랜스사이레틴 안정화제, siRNA 치료법, 아밀로이드 침착을 표적으로 하는 단일클론 항체, 그리고 새로운 플라스마세포 표적 약물의 개발이 지속되는 가운데, 아밀로이드증 분야경쟁 구도는 변화하고 있습니다. 넥시그란·지클메란(nex-z), 누클레실란, 안셀라미맙, 기타 임상시험 중인 치료법 등 새로운 파이프라인 자산들은 예측 기간 동안 시장 경쟁을 더욱 심화시킬 것으로 예측됩니다.
아밀로이드증 개요
아밀로이도증은 조직이나 장기에 비정상적인 구조를 띤 단백질(아밀로이드 섬유)이 축적되어 진행성 장기 기능 장애를 일으키는 일련의 희귀질환입니다. 이 질환은 일반적으로 심장, 말초 신경, 신장에 영향을 미치며, 임상 증상은 아밀로이드 단백질의 유형에 따라 달라집니다. 주요 전신성 유형으로는 트랜스사이레틴(TTR) 유전자의 변이로 인해 발생하는 유전성 트랜스사이레틴 아밀로이드증(hATTR), 비정상적인 형질세포에 의한 단일클론 경쇄의 과잉 생산으로 인해 발생하는 AL 아밀로이드증 등이 있습니다.
아밀로이드증의 진단
아밀로이드증의 진단은 임상 양상이 다양하고, 다른 심장·신경·신장 질환과 증상이 겹치기 때문에 종종 지연되는 경향이 있어 어려움이 따릅니다. 초기 평가에는 AL 아밀로이드증 평가를 목적으로 하는 혈청 및 소변의 면역고정법을 이용한 단일 클론성 단백질 검사 및 혈청 유리 경쇄 측정이 포함됩니다. 진단은 콩고 레드 염색을 실시한 조직 생검을 통해 확정되며, 편광 하에서 사과 녹색의 복굴절이 확인됩니다. 아형의 확인을 위해서는 질량 분석법에 기반한 아밀로이드 유형 분석, 유전성 트랜스사이레틴 아밀로이드증(ATTR) 유전자 검사, 그리고 심장 ATTR 아밀로이드증에 대한 심초음파 검사, 심장 MRI, 99mTc-PYP 신티그래피 등의 첨단 영상 진단 기술이 사용되기도 합니다. 비가역적인 장기 손상을 예방하기 위해서는 조기 진단이 매우 중요합니다.
아밀로이드증의 치료
아밀로이드증의 치료는 아밀로이드 형성 단백질의 생성을 억제하고, 질환의 진행을 늦추며, 장기와 관련된 합병증을 관리하는 데 중점을 두고 있습니다. 유전성 트랜스사이레틴 아밀로이드증의 경우, 승인된 치료법으로는 VYNDAMAX와 같은 TTR 안정화제 및 ONPATTRO, TEGSEDI, AMVUTTRA, WAINUA와 같은 유전자 침묵제가 포함됩니다. 이 약제들은 비정상적인 트랜스사이레틴 생성을 억제하여, 신경학적 및 심장 기능 저하를 늦추는 데 도움이 됩니다.
AL 아밀로이드증의 치료는 주로 다발성 골수종에 사용되는 것과 동일한 항형질세포 요법을 통해 기저가 되는 형질세포 클론을 표적으로 삼습니다. 현재의 표준 치료에는 DARZALEX FASPRO를 기반으로 한 치료 요법이 포함되어 있으며, 적격한 환자의 경우 자가 조혈모세포 이식이 시행되기도 합니다. 심장, 신장 및 기타 장기의 합병증을 관리하기 위해서는 여전히 지지 요법이 필수적이며, 단일클론 항체나 CAR T세포 치료법 등 새로운 치료법들이 현재 임상시험 중입니다.
아밀로이드증의 역학 분석 및 예측에 관한 주요 연구 결과
아밀로이드증의 치료 양상은 최근 몇 년간 현저하게 변화하고 있으며, ATTR 및 AL 두 가지 아형 모두에서 주로 지지 요법 위주에서 표적화된 작용기전에 기반한 치료로 전환되고 있습니다. 현재의 치료 전략은 아밀로이드 단백질의 생성을 억제하고, 질병의 진행을 늦추며, 장기 기능을 유지하고, 생존율을 높이는 데 중점을 두고 있습니다.
ATTR 아밀로이드증의 경우, 타파미디스, 파티실란, 이노텔센, 에프론텔센, 부토리실란 등의 RNA 침묵화제와 같은 질환 수정 요법이 트랜스사이레틴(TTR) 수치를 안정화하거나 감소시킴으로써 치료 방식을 완전히 바꿔 놓았습니다. 현재 이 분야에서는 차세대 RNA 표적 치료법 및 유전자 편집 치료법으로 전환되고 있으며, 넥시그란·지클메란(nex-z) 등의 임상시험용 약물이 더욱 강력하고 지속적인 TTR 억제를 목표로 하고 있습니다.
AL 아밀로이드증의 경우, 치료는 기존의 화학요법에서 보다 표적을 좁힌 형질세포 중심 치료 요법으로 발전하고 있습니다. DARZALEX FASPRO를 기반으로 한 병용 요법은 1차 치료에서 중요한 핵심 치료법으로 자리 잡고 있습니다. 한편, BCMA를 표적으로 하는 CAR-T 요법, 이중 특이성 항체 및 삼중 특이성 항체, 피브리릴을 표적으로 하는 단일클론 항체와 같은 새로운 치료법들이 치료 선택지를 확대하고 정밀 의학으로의 전환을 뒷받침하고 있습니다.
이러한 진전에도 불구하고, 진단 지연, 돌이킬 수 없는 장기 손상, 그리고 아밀로이드 침착을 완전히 제거할 수 있는 근치적 치료법의 부재로 인해 여전히 해결되지 않은 의료적 요구가 많이 남아 있습니다. 그 결과, 아밀로이드 생성을 억제하고 아밀로이드를 능동적으로 제거하며 장기 기능을 회복시키는 것을 결합한 접근법에 대한 관심이 높아지고 있습니다.
전반적으로, 질환에 대한 인지도 제고, 조기 진단, 표적 치료법 접근성 확대, 그리고 후기 개발 단계에 있는 파이프라인의 강화로 인해, 주요 7개국 전체의 아밀로이드증 시장이 꾸준히 성장할 것으로 예측됩니다. 시장 규모 면에서는 미국이 여전히 가장 크며, 그 뒤를 유럽과 일본이 잇고 있습니다.
전반적으로, 아밀로이드증의 치료 방식은 대증 요법이나 단백질 안정화 요법에서 지속적 유전자 침묵화, 면역 요법, 그리고 유전자 편집 전략으로 진화하고 있습니다. 현재, 표적 생물학적 제제와 RNA 기반 치료법이 시장 성장을 주도하고 있는 한편, 차세대 파이프라인 신약들이 향후 치료법의 확대를 이끌고 있습니다.
DelveInsight's 'Amyloidosis- Market Insights, Epidemiology and Market Forecast - 2036' report delivers an in-depth understanding of amyloidosis, historical and forecasted epidemiology, as well as the amyloidosis market trends in the United States, EU4 (Germany, Spain, Italy, and France), and the United Kingdom, and Japan.
The amyloidosis market report delivers a comprehensive analysis of the current treatment landscape, including standards of care, clinical practices, and evolving therapeutic algorithms. It evaluates amyloidosis patient burden trends, revenue & market share dynamics, peak patient share & therapy uptake analysis, and provides an in-depth market size assessment, and growth rate projections (Historical & Forecast 2022-2036) across global regions. The report highlights key unmet medical needs in amyloidosis and maps the competitive and clinical landscape to uncover high-value opportunities, providing a clear outlook on future market growth potential.
Key Factors Driving the Amyloidosis Market
Rising Amyloidosis Disease Recognition and Diagnosis
The overall prevalence and diagnosis rates of Amyloidosis are increasing due to improved disease awareness, wider availability of advanced imaging and biomarker-based diagnostics, growing use of genetic testing, and better differentiation between AL and ATTR subtypes, supporting continued market expansion.
Growing Opportunities in Targeted and Disease-Modifying Therapies
The amyloidosis market is being driven by rapid advances in targeted therapies, including RNA-silencing agents, monoclonal antibodies, plasma cell-directed therapies, and emerging gene-editing approaches aimed at reducing amyloid production, improving organ response, and potentially altering long-term disease progression.
Increasing Focus on Early Diagnosis and Multidisciplinary Disease Management
Increasing clinical focus on earlier diagnosis and multidisciplinary disease management, particularly in cardiac and neurologic amyloidosis, is expanding treatment eligibility and accelerating therapeutic adoption across both ATTR and AL amyloidosis populations.
Emerging Competitive Landscape in Amyloidosis
The competitive landscape of amyloidosis is evolving with continued development of next-generation transthyretin stabilizers, siRNA therapies, monoclonal antibodies targeting amyloid deposits, and novel plasma cell-targeted agents. Emerging pipeline assets such as nexiguran ziclumeran (nex-z), nucresiran, anselamimab, and other investigational therapies are expected to further strengthen market competition over the forecast period.
Amyloidosis Overview
Amyloidosis is a group of rare disorders caused by the accumulation of misfolded proteins (amyloid fibrils) in tissues and organs, leading to progressive organ dysfunction. The disease commonly affects the heart, peripheral nerves, and kidneys, with clinical manifestations varying by amyloid protein type. Major systemic forms include Hereditary Transthyretin Amyloidosis (hATTR), caused by mutations in the transthyretin (TTR) gene, and AL Amyloidosis, which results from excess monoclonal light-chain production by abnormal plasma cells.
Amyloidosis Diagnosis
Diagnosis of Amyloidosis is often delayed and challenging because of its heterogeneous clinical presentation and overlap with other cardiac, neurologic, and renal disorders. Initial evaluation includes testing for monoclonal proteins using serum and urine immunofixation and serum free light-chain assays to assess for AL Amyloidosis. Diagnosis is confirmed through tissue biopsy with Congo red staining, demonstrating apple-green birefringence under polarized light. Subtype identification may involve mass spectrometry-based amyloid typing, genetic testing for Hereditary Transthyretin Amyloidosis, and advanced imaging techniques such as echocardiography, cardiac MRI, and 99mTc-PYP scintigraphy for cardiac ATTR amyloidosis. Early diagnosis is critical to prevent irreversible organ damage.
Amyloidosis Treatment
Treatment of Amyloidosis focuses on reducing the production of amyloid-forming proteins, slowing disease progression, and managing organ-related complications. In Hereditary Transthyretin Amyloidosis, approved therapies include TTR stabilizers such as VYNDAMAX and gene-silencing agents including ONPATTRO, TEGSEDI, AMVUTTRA, and WAINUA, which reduce abnormal transthyretin production and help delay neurological and cardiac decline.
In AL Amyloidosis, treatment primarily targets the underlying plasma cell clone using anti-plasma cell regimens similar to those used in multiple myeloma. Current standard therapy includes a DARZALEX FASPRO-based regimen, while eligible patients may undergo autologous stem cell transplantation. Supportive care remains essential for managing cardiac, renal, and other organ complications, and emerging therapies such as monoclonal antibodies and CAR T-cell approaches are under clinical investigation.
Amyloidosis Unmet Needs
The section "unmet needs of amyloidosis" outlines the critical gaps between the current state of patient care, diagnosis, and the ideal & effective management of the disease. It highlights the obstacles experienced by patients, clinicians, and researchers and identifies potential solutions for future progress.
Key Findings from Amyloidosis Epidemiological Analysis and Forecast
Amyloidosis Drug Analysis & Competitive Landscape
The Amyloidosis drug chapter provides a detailed, market-focused review of the approved therapies and emerging pipeline across Phase I-III clinical trials. It covers the mechanism of action, clinical trial data, regulatory approvals, patents, collaborations, and strategic partnerships for each therapy, along with their advantages, limitations, and recent developments. This section offers critical insights into the Amyloidosis treatment landscape, supporting market assessment, competitive analysis, and growth forecasting for the Amyloidosis therapeutics market.
Approved Therapies for Amyloidosis
Vutrisiran (AMVUTTRA): Alnylam Pharmaceuticals
AMVUTTRA is a subcutaneously administered transthyretin-directed siRNA (RNAi) therapy developed by Alnylam Pharmaceuticals for the treatment of ATTR amyloidosis, including Hereditary Transthyretin Amyloidosis and wild-type ATTR amyloidosis. The therapy received FDA approval in June 2022 for adults with hATTR amyloidosis with polyneuropathy and subsequently received a positive EU opinion for adults with stage 1 or stage 2 polyneuropathy.
Daratumumab and hyaluronidase-fihj (DARZALEX FASPRO): Johnson & Johnson
DARZALEX FASPRO is the only CD38-directed antibody approved to be given subcutaneously to treat patients with multiple myeloma and now AL amyloidosis. DARZALEX FASPRO is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), which is part of Halozyme's ENHANZE drug delivery technology.
Amyloidosis Pipeline Analysis
Nexiguran ziclumeran (nex-z, NTLA-2001): Intellia Therapeutics and Regeneron
NTLA-2001 is an investigational in vivo CRISPR-based gene-editing therapy being developed by Intellia Therapeutics in collaboration with Regeneron Pharmaceuticals as a one-time treatment for ATTR amyloidosis. The therapy is designed to inactivate the TTR gene and is currently in Phase III development. NTLA-2001 has received Orphan Drug Designation (ODD), Regenerative Medicine Advanced Therapy (RMAT) designation from the US FDA, and ODD from the European Commission.
NXC-201: Immix Biopharma
NXC-201 is a sterically optimized BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy. NXC-201 is being studied for the treatment of patients with r/r AL amyloidosis, with the potential to expand into autoimmune indications. NXC-201 has been awarded ODD and regenerative medicine advanced therapy designation in the US by the FDA and ODD in Europe by the European Medicines Agency in r/r AL Amyloidosis.
Amyloidosis Key Players, Market Leaders, and Emerging Companies
Amyloidosis Drug Updates
Drug Class Insights
The amyloidosis treatment landscape has evolved markedly in recent years, shifting from largely supportive care to targeted, mechanism-based therapies across both ATTR and AL subtypes. Current strategies focus on reducing amyloid protein production, slowing disease progression, preserving organ function, and improving survival outcomes.
In ATTR amyloidosis, disease-modifying therapies such as tafamidis and RNA-silencing agents, including patisiran, inotersen, eplontersen, and vutrisiran, have transformed management by stabilizing or reducing transthyretin levels. The field is now moving toward next-generation RNA-targeting approaches and gene-editing therapies, with investigational agents such as Nexiguran Ziclumeran (nex-z) aiming for deeper and potentially durable TTR suppression.
In AL amyloidosis, treatment has progressed from conventional chemotherapy to more targeted plasma cell-directed regimens. DARZALEX FASPRO-based combinations have become a key backbone in first-line therapy, while emerging modalities such as BCMA-targeted CAR T therapies, bispecific and trispecific antibodies, and fibril-directed monoclonal antibodies are expanding treatment options and reinforcing the shift toward precision medicine.
Despite these advances, major unmet needs persist due to late diagnosis, irreversible organ damage, and the absence of curative therapies that can fully eliminate amyloid deposits. As a result, increasing focus is being placed on approaches that combine suppression of amyloid production with active amyloid clearance and organ recovery.
Overall, improving disease awareness, earlier diagnosis, expanding access to targeted therapies, and a strengthening late-stage pipeline are expected to support steady growth of the amyloidosis market across the 7MM, with the US remaining the dominant market, followed by Europe and Japan.
Drug Class/Insights into Leading Emerging and Marketed Therapies in Amyloidosis (2022-2036 Forecast)
The Amyloidosis market comprises targeted small molecules, biologics, RNA-based therapies, and emerging cell and gene therapies aimed at reducing amyloid production, stabilizing precursor proteins, suppressing abnormal plasma cells, or enhancing amyloid clearance across ATTR and AL amyloidosis.
Overall, the amyloidosis treatment landscape is evolving from supportive and protein-stabilizing approaches toward durable gene-silencing, immunotherapy, and gene-editing strategies, with targeted biologics and RNA-based therapies currently driving market growth while next-generation pipeline agents shape future therapeutic expansion.
Amyloidosis Drug Uptake
This section focuses on the uptake rate of potential drugs expected to be launched in the market during the forecast period (2026-2036). The analysis covers the Amyloidosis drug's uptake, performance at peak, factors affecting performance during prime years of growth, patient uptake by therapy, and anticipated sales generated by each drug.
Drug uptake in Amyloidosis is increasing steadily due to improving diagnosis rates, growing disease awareness, and expanding use of targeted therapies across ATTR and AL amyloidosis. In ATTR amyloidosis, RNA-based therapies such as vutrisiran, patisiran, inotersen, and eplontersen are witnessing strong uptake because of their robust efficacy in reducing transthyretin production and slowing disease progression, while tafamidis continues to maintain significant use in ATTR cardiomyopathy.
In AL Amyloidosis, uptake of DARZALEX FASPRO-based regimens has increased substantially following its approval as the first FDA-approved therapy specifically indicated for AL amyloidosis. Emerging therapies, including BCMA-targeted therapies, bispecific antibodies, and gene-editing approaches, are expected to further support future market growth.
Amyloidosis Therapies Price Scenario & Trends
Pricing and analogue assessment of amyloidosis therapies highlights evolving price dynamics structures. This section summarizes the cost of approved treatments, the closest and most appropriate analogue selection for emerging therapies, and the understanding of how pricing influences market access, adherence, and long-term uptake.
Further details are provided in the final report....
Industry Experts and Physician Views for Amyloidosis
To keep up with Amyloidosis market trends, we take Key Opinion Leaders (KOLs) and Subject Matter Experts (SMEs) opinions working in the domain through primary research to fill the data gaps and validate our secondary research. Industry experts were contacted for insights on the emerging amyloidosis therapies, evolving treatment landscape, patient adherence to conventional therapies, therapy switching trends, drug adoption and uptake, accessibility challenges, and epidemiology and real-world prescription patterns in amyloidosis, including MD, PhD, Instructor, Postdoctoral Researcher, Professor, Researcher, and others.
DelveInsight's analysts connected with 10+ KOLs to gather insights at the country level. Centers such as the Columbia University Irving Medical Center, Universite Paris-Saclay, Bicetre Hospital, Mayo Clinic, National Amyloidosis Centre, etc., were contacted. Their opinion helps understand and validate current and emerging Amyloidosis therapies, highlight unmet medical needs, provide epidemiological context, and support strategic decisions for market access, therapy adoption, and pipeline prioritization in amyloidosis.
Qualitative Analysis: SWOT and Conjoint Analysis
We perform qualitative and market Intelligence analysis using various approaches, such as SWOT analysis and conjoint analysis.
In the SWOT analysis of amyloidosis, strengths, weaknesses, opportunities, and threats in terms of disease diagnosis, patient awareness, patient burden, competitive landscape, cost-effectiveness, and geographical accessibility of therapies are provided.
Conjoint analysis analyzes emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. Scoring is given based on these parameters to analyze the effectiveness of therapy.
The team of analysts analyzes promising emerging therapies based on relevant attributes such as safety, efficacy, frequency of administration, route of administration, and order of entry. In efficacy, the trial's primary and secondary outcome measures are evaluated, whereas the therapies' safety is evaluated, wherein the acceptability, tolerability, and adverse events are mainly observed. In addition, the scoring is also based on the route of administration, order of entry, probability of success, and the addressable patient pool for each therapy. According to these parameters, the final weightage score and the ranking of the emerging therapies are decided.
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