![]() |
市場調查報告書
商品編碼
1479824
罕見腎臟病市場:產業趨勢和到2035年的全球預測:依目標疾病,分子類型,生物類型,給藥途徑,治療類型,地區,主要藥物開發商和銷售預測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 |
罕見腎臟病市場規模預計到 2024 年將達到 39 億美元,並在 2024-2035 年預測期間以 11% 的複合年增長率擴張。
罕見腎臟疾病包括至少 150 種疾病。在歐洲,如果一種疾病的盛行率低於 2,000 人中就有 1 人,則被視為罕見疾病;而在美國,如果疾病影響的人數少於 20 萬人,則被視為罕見疾病。值得注意的是,2020 年有 8,000 多人被診斷出患有罕見腎臟疾病,儘管需求驚人,但只有 500 人接受了治療。研究表明,超過12%的腎衰竭患者患有罕見的腎臟疾病,導致慢性虛弱和發病。因此,該領域的各個利益相關者正在做出各種努力來開發針對這些病症的客製化療法。此外,自 2019 年以來,約有 80 億美元投資於致力於開發罕見腎臟疾病治療方法的公司。然而,診斷和治療的進展受到患者群體有限、缺乏預後生物標記、臨床多樣性和模型生物不足等因素的阻礙。為了應對這些課題,該領域取得了顯著進展。
包括基因組定序和個人化治療在內的精準醫學的出現,促進了罕見腎臟疾病的標靶治療。藥物再利用涉及確定新的治療標靶和利用現有藥物的藥理學特性,已成為一種具有成本效益的策略。此外,整合人工智慧(AI)和機器學習演算法來優化個人化治療是一項重大技術進步。最近,多種針對罕見腎臟疾病各種適應症的藥物已獲得世界各地監管機構的批准。2023年,美國食品藥物管理局(FDA)批准Elfabrio(由Protalix BioTherapeutics開發)用於治療法布瑞氏症,Tarpeyo(由Calliditas Therapeutics開發)用於治療IgA腎病變。考慮到與這些藥物相關的廣闊前景,私人和公共投資者都在關注罕見腎病市場,從而在預測期內刺激顯著增長。
該報告調查了全球罕見腎病市場,並依目標疾病、分子類型、生物類型、給藥途徑、治療類型、地區、地區提供了市場概況。
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