市场调查报告书
商品编码
1479824
罕见肾臟病市场:产业趋势和到2035年的全球预测:依目标疾病,分子类型,生物类型,给药途径,治疗类型,地区,主要药物开发商和销售预测Rare Kidney Diseases Market: Industry Trends and Global Forecasts, Till 2035: Distribution by Target Disease Indications, Type of Molecule, Type of Biologics, Route of Administration, Key Geographical Regions, Leading Drug Developers and Sales Forecast |
罕见肾臟病市场规模预计到 2024 年将达到 39 亿美元,并在 2024-2035 年预测期间以 11% 的复合年增长率扩张。
罕见肾臟疾病包括至少 150 种疾病。在欧洲,如果一种疾病的盛行率低于 2,000 人中就有 1 人,则被视为罕见疾病;而在美国,如果疾病影响的人数少于 20 万人,则被视为罕见疾病。值得注意的是,2020 年有 8,000 多人被诊断出患有罕见肾臟疾病,儘管需求惊人,但只有 500 人接受了治疗。研究表明,超过12%的肾衰竭患者患有罕见的肾臟疾病,导致慢性虚弱和发病。因此,该领域的各个利益相关者正在做出各种努力来开发针对这些病症的客製化疗法。此外,自 2019 年以来,约有 80 亿美元投资于致力于开发罕见肾臟疾病治疗方法的公司。然而,诊断和治疗的进展受到患者群体有限、缺乏预后生物标记、临床多样性和模型生物不足等因素的阻碍。为了应对这些课题,该领域取得了显着进展。
包括基因组定序和个人化治疗在内的精准医学的出现,促进了罕见肾臟疾病的标靶治疗。药物再利用涉及确定新的治疗标靶和利用现有药物的药理学特性,已成为一种具有成本效益的策略。此外,整合人工智慧(AI)和机器学习演算法来优化个人化治疗是一项重大技术进步。最近,多种针对罕见肾臟疾病各种适应症的药物已获得世界各地监管机构的批准。2023年,美国食品药物管理局(FDA)批准Elfabrio(由Protalix BioTherapeutics开发)用于治疗法布瑞氏症,Tarpeyo(由Calliditas Therapeutics开发)用于治疗IgA肾病变。考虑到与这些药物相关的广阔前景,私人和公共投资者都在关注罕见肾病市场,从而在预测期内刺激显着增长。
该报告调查了全球罕见肾病市场,并依目标疾病、分子类型、生物类型、给药途径、治疗类型、地区、地区提供了市场概况。
Rare Kidney Diseases Market: Industry Trends and Global Forecasts, Till 2035: Distribution by Target Disease Indications (Atypical Hemolytic Uremic Syndrome, Fabry Disease, Immunoglobulin A Nephropathy, Focal Segmental Glomerulosclerosis, Autosomal Dominant Polycystic Kidney Disease, Lupus Nephritis and Others), Type of Molecule (Small Molecules and Biologics), Type of Biologics (Monoclonal Antibodies and Proteins), Route of Administration (Oral, Intravenous, Subcutaneous, and Others), Type of Therapy (Monotherapy, Combination Therapy), Key Geographical Regions (North America, Europe, Asia-Pacific and Rest of the World), Leading Drug Developers and Sales Forecast
The Rare Kidney Diseases Market is valued at USD 3.9 billion in 2024 growing at a CAGR of 11% during the forecast period 2024-2035.
Rare kidney diseases encompass a spectrum of conditions, accounting to at least 150 distinct disorders. In Europe, a condition is deemed rare if its prevalence falls below 1 in 2000 individuals, while in the US, it's classified as rare if fewer than 200,000 people are affected. Notably, in 2020, out of over 8,000 individuals diagnosed with rare kidney diseases, only 500 received treatment, despite the alarming need. Research reveals that over 12% of individuals with kidney failure are afflicted with rare kidney diseases, resulting in chronic debilitation and morbidity. Consequently, various stakeholders in this domain have taken various initiatives to advance therapeutics tailored for these conditions. Further, since 2019, approximately USD 8 billion has been invested in the companies dedicated to developing therapies for rare kidney diseases. Nonetheless, progress in diagnosis and treatment has been impeded by such factors, such as limited patient populations, absence of prognostic biomarkers, clinical diversity, and inadequate model organisms. In order to cater to these challenges, notable strides have been made in the field.
The advent of precision medicine, encompassing genomic sequencing and personalized therapies, has facilitated targeted interventions for rare kidney diseases. Drug repurposing, involving the identification of new therapeutic targets and harnessing the pharmacological properties of existing drugs, has emerged as a cost-effective strategy. Additionally, the integration of artificial intelligence (AI) and machine learning algorithms in order to optimize personalized treatment stands out as a prominent technological advancement. Recently, several drugs targeting various indications of rare kidney diseases have garnered approval from regulatory authorities worldwide. In 2023, the Food and Drug Administration (FDA) sanctioned Elfabrio (developed by Protalix BioTherapeutics) for Fabry Disease and Tarpeyo (developed by Calliditas Therapeutics) for IgA Nephropathy. Given the promising prospects associated with these drugs, both private and public investors have turned their attention towards the rare kidney diseases market, fueling the substantial growth during the forecast period.
Key Market Segments
Type of Target Disease Indication
Atypical Hemolytic Uremic Syndrome
Fabry Disease
Immunoglobulin A Nephropathy
Focal Segmental Glomerulosclerosis
Autosomal Dominant Polycystic Kidney Disease
Lupus Nephritis
Others
Type of Molecule
Small Molecules
Biologics
Type of Biologic
Monoclonal Antibodies
Proteins
Route of Administration
Oral
Intravenous
Subcutaneous
Others
Type of Therapy
Monotherapy
Combination Therapy
Leading Drug Developers
Alexion Pharmaceuticals
Amicus Therapeutics
Aurinia Pharmaceuticals
Otsuka PharmaceuticalMallinckrodt Pharmaceuticals
Novartis
Otsuka Pharmaceutical
Roche
Sanofi
Travere Therapeutics
Other Players
Geography
North America
Europe
Asia-Pacific
Rest of the World
Research Coverage:
A comprehensive examination of the current market landscape pertaining to rare kidney diseases, encompassing various parameters, including the year of establishment, company size (based on the employee count), location of headquarters, phase of development (discovery to marketed), route of administration (oral, intravenous, subcutaneous, and others), type of molecule (biologics and small molecules), subtype of biologics (monoclonal antibodies, proteins, cell and gene therapies, RNA, biosimilars, stem cells, and live biotherapeutic products), dosing frequency (single dose, multiple dose, combination), type of therapy (monotherapy and combination therapy), target patient population (children, adults, and elderly patients), and target indication (Lupus Nephritis, Immunoglobulin A, Nephropathy, Focal Segmental Glomerulosclerosis, Membranous Nephropathy, Fabry Disease, Autosomal Polycystic Kidney Disease, Nephrotic Syndrome, Alport Syndrome, Atypical Hemolytic Uremic Syndrome, and C3 Glomerulopathy).
Detailed profiles of leading companies (shortlisted based on the robustness of their drug portfolios) operating in the rare kidney diseases market. Each profile features the company overview (year of establishment, employee count, location of headquarters, and leadership team), financial information (if available), rare kidney disease portfolio specifics, recent advancements, and an insightful projection for the future.
Brief summaries of notable companies (shortlisted based on their robust drug portfolios) operating in the rare kidney diseases market. Each profile highlights a concise overview of the company (year of establishment, employee count, location of headquarters, and leadership team) and financial information (if available).
A comprehensive analysis of company competitiveness among key developers in the rare kidney diseases domain, considering various pertinent parameters. These include supplier robustness (evaluated by years of experience and company size), company competitiveness (assessed by the most advanced drug development phase, quantity of drugs developed, route of administration, and molecule type), and the breadth of indications targeted by each drug.
An exhaustive examination of the collaborations formed among stakeholders involved in this sector since 2019, encompassing licensing agreements, research and development collaborations, mergers and acquisitions, product development partnerships, commercialization agreements, distribution partnerships, joint ventures, supply agreements, service agreements, technology utilization agreements, process development agreements, and other pertinent arrangements.
An assessment of funding and investments directed towards companies focusing on rare kidney disease treatments, spanning venture capital investments, funds raised through initial public offerings (IPOs) and subsequent offerings, grants, private placements, as well as equity and debt financing. This analysis delves into funding occurrences from 2019 to 2023, shedding light on the increasing attention from the venture capital community and other strategic investors within this market.
A comprehensive examination of both active and ongoing clinical trials concerning the therapies for rare kidney diseases, focusing on various pertinent factors including the year of trial registration, trial status, trial phase, study design (masking type, intervention model type, and primary purpose), type of sponsor, the most active industry and non-industry players (based on trial count), geographical distribution, and the enrolled patient populations.
An extensive assessment of the clinical and commercial demand of medications aimed at treating rare kidney disorders, considering multiple pertinent factors such as the size of the target patient population, dosing frequency, and the strength of doses.
A thorough examination, accentuating the key opinion leaders (KOLs) engaged in investigating clinical trials associated with rare kidney diseases, considering various pertinent factors including type of KOL, affiliated organization, targeted disease indication, industry and non-industry affiliations, as well as the geographical distribution of KOLs. Moreover, the section emphasizes the most active KOLs, evaluated through both our proprietary and third-party scoring methodologies.
A case study showcasing enterprises providing kidney care services, including details such as their year of establishment, company size (measured by employee count), location of headquarters, and focus on specific medical conditions.
A comprehensive examination of recent expansions of the rare kidney diseases market, encompassing the identification and evaluation of primary catalysts, potential obstacles, emerging prospects, and prevailing hurdles.
A detailed assessment of the present market size, current opportunities within rare kidney diseases therapies, and the prospective growth trajectory of the rare kidney diseases market over the next decade. Drawing on various parameters including anticipated adoption trends and validated primary data, we've formulated an informed projection of market evolution till 2035. Additionally, the report outlines the expected distribution of both current and forecasted opportunities within the rare kidney diseases sector. To accommodate the potential future uncertainties and enhance the robustness of our model, we've presented three forecast scenarios: conservative, base, and optimistic, each representing distinct trajectories for growth within the rare kidney diseases market.
A detailed analysis on the factors influencing the growth of the Detailed projections of the current and future market for rare kidney disease therapeutics across types of target indications, such as Atypical Hemolytic Uremic Syndrome, Fabry Disease, Immunoglobulin A Nephropathy, Focal Segmental Glomerulosclerosis, Autosomal Dominant Polycystic Kidney Disease, Lupus Nephritis and others.
Detailed projections of the current and future rare kidney disease market based on type of molecule, such as small molecules and biologics.
Comprehensive projections of the current and future rare kidney diseases market based on the type of biologics, such as monoclonal antibodies and proteins.
Detailed projections of the current and future rare kidney disease market across the types of routes of administration, such as oral, intravenous, subcutaneous, and others.
Detailed predictions of the current and future rare kidney diseases market across types of therapy, such as monotherapy and combination therapy.
Comprehensive projections of the current and future rare kidney diseases industry across key geographical regions, such as North America, Europe, Asia-Pacific and the rest of the world.
Detailed projections of the current and future rare kidney disease market across the sales of forecasted drugs.
Comprehensive projections of the current and future revenues from the sales of around 20 top-selling drugs developed by the leading players in the rare kidney diseases market, including Alexion Pharmaceuticals, Amicus Therapeutics, Aurinia Pharmaceuticals, GSK, Otsuka Pharmaceutical and other players.
Key Benefits of Buying this Report
The report offers market leaders and newcomers valuable insights into revenue estimations for both the overall market and its sub-segments.
Stakeholders can utilize the report to enhance their understanding of the competitive landscape, allowing for improved business positioning and more effective go-to-market strategies.
The report provides stakeholders with a pulse on the Rare Kidney Diseases Market, furnishing them with essential information on significant market drivers, barriers, opportunities, and challenges.
Leading Market Companies
Alexion Pharmaceuticals
Chinook Therapeutics
Horizon Therapeutics
Jiangsu Hengrui Pharmaceuticals
Novartis
Omeros
Roche
Sanofi
Travere Therapeutics
ZyVersa Therapeutics
Advicenne
Apellis Pharmaceuticals
argenx
Astellas Pharma
Boehringer Ingelheim
Calliditas Therapeutics
Mallinckrodt Pharmaceuticals
Synlogic
Takeda