Rare Kidney Diseases Market: Industry Trends and Global Forecasts, Till 2035: Distribution by Target Disease Indications
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 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 SegmentsType 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
GSK
Otsuka Pharmaceutical
Mallinckrodt 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
GSK
Mallinckrodt Pharmaceuticals
Synlogic
Takeda