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, Type of Therapy, Key Geographical Regions, Leading Drug Developers and Sales Forecast

Rare Kidney Disease Market Bolstered by $8 Billion Investment and Government Initiatives, with Increasing Awareness Driving Growth

The global rare kidney diseases market is estimated to grow from $3.9 billion in 2024 to $12.1 billion by 2035, representing a CAGR of 11%, during the forecast period 2024-2035.

This research study consists of industry trends, detailed rare kidney diseases market analysis, key market insights, partnerships and collaborations analysis, funding and investments, clinical trial analysis and market impact analysis. The report includes rare diseases market forecast and opportunity analysis.

Rare kidney diseases are a group of conditions, with at least 150 different disorders falling into this category. In Europe, a rare kidney disease is defined as rare when the prevalence is less than 1 in 2000 people and in the US, the disease is designated rare when less than 200,000 people are affected. It is worth mentioning that in 2020, of more than 8,000 individuals diagnosed with rare kidney disease, only 500 received treatment for a rare kidney disease received treatment. Studies have also shown that 12% individuals with kidney failure suffer from a rare kidney disease leading to chronic debilitation and morbidity. Thereby, several initiatives have been taken by stakeholders to promote the development of therapeutics focused on rare kidney diseases.

It is worth mentioning that, since 2019, around USD 8 billion has been invested in companies developing therapies for rare kidney diseases. However, advancements in the diagnosis and treatment of rare kidney diseases have been hampered by several factors, including limited patient population, lack of prognostic biomarkers, clinical heterogeneity, and insufficient model organisms. To address such challenges, several promising advancements have been made in this domain.

For instance, the emergence of precision medicine (genomic sequencing and personalized therapies) has enabled the targeted treatment of rare kidney diseases, drug repurposing involving the identification of new therapeutic targets and leveraging the pharmacological properties for existing drugs which has emerged as a cost-effective strategy for treating rare kidney diseases. It is worth mentioning that the integration of artificial intelligence (AI) and machine learning algorithms to optimize the personalized treatment of rare kidney diseases is one of the prominent examples of technological advancements in the rare kidney diseases market.

Over the past few years, a number of rare kidney disease drugs targeting a myriad of indications have received approval from various regulatory authorities, globally. In 2023, the Food and Drug Administration (FDA) granted approval for Elfabrio (developed by Protalix BioTherapeutics) for the treatment of Fabry Disease. Additionally, the FDA granted approval to Tarpeyo (developed by Calliditas Therapeutics) for the treatment of IgA Nephropathy. Given the lucrative opportunity associated with these drugs, the rare kidney diseases market has gained attention of both private and public investors in the past few years leading to the substantial market growth during the forecast period.

Rare Kidney Diseases Market Share Insights

The market research report presents an in-depth analysis of the various rare kidney disease developers that are involved in the global rare kidney diseases industry, across different segments, as defined below:

• Historical Trend: 2018-2023
• Base Year: 2024
• Forecast Period: 2024-2035
• Market Size Value in 2024: $3.9 Billion
• Growth Rate: CAGR of 11% from 2024 to 2035
• 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
  • GSK
  • Otsuka PharmaceuticalMallinckrodt Pharmaceuticals
  • Novartis
  • Otsuka Pharmaceutical
  • Roche
  • Sanofi
  • Travere Therapeutics
  • Other Players
• Geography
  • North America
  • Europe
  • Asia-Pacific
  • Rest of the World
• Leading Market Players
  • 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
• PowerPoint Presentation (Complimentary)
• Customization Scope: 15% Free Customization
• Excel Data Packs (Complimentary)
  • Market Landscape Analysis
  • Company Competitiveness Analysis
  • Partnerships and Collaborations
  • Funding and Investments
  • Clinical Trial Analysis
  • Clinical Commercial Attractiveness
  • Key Opinion Leaders
  • Market Size and Opportunity Analysis

Rare Kidney Diseases Market Key Segments

Market Share by Target Disease Indication

The global rare kidney diseases market is segmented into various target disease indication, such as Nephrotic Syndrome, Hepatorenal Syndrome, Distal Renal Tubular Acidosis, Fabry Disease, Lupus Nephritis, Atypical Hemolytic Uremic Syndrome, Immunoglobulin A Nephropathy, Autosomal Dominant Polycystic Kidney Disease, Autosomal Recessive Polycystic Kidney Disease, C3 Glomerulopathy, Membranous Nephropathy, Focal Segmental Glomerulosclerosis and others. The Fabry disease occupies the largest share in 2024 and is expected to remain dominant during the forecast period. This can be attributed to the high incidence rate of Fabry disease, affecting about 1 in 15,000 people. Further, Lupus Nephritis segment is estimated to grow at the highest CAGR (>8%) from 2024 to 2035.

Market Share by Type of Molecule

This segment highlights the distribution of the rare kidney diseases market across different types of molecules, such as biologics and small molecules. The small molecules segment occupies the largest share in 2024 and is expected to remain dominant during the forecast period. This can be primarily attributed to the fact that small molecules have low molecular weight and can easily pass through cell membranes to reach intracellular targets, thereby advancing new capabilities to create advanced potential therapies.

Market Share by Type of Biologics

This segment highlights the distribution of the rare kidney diseases market across different types of biologics, such as monoclonal antibodies and proteins. The monoclonal antibodies segment occupies the largest share in 2024 and is expected to remain dominant during the forecast period. This can be primarily attributed to the physiological properties of monoclonal antibodies, including selectivity, specificity, stability, and ability to block specific protein-protein interactions.

Market Share by Route of Administration

The global rare kidney diseases market is categorized into oral, intravenous, and subcutaneous route of administration. Owing to their safety and convenient nature, the segment for oral route of administration is likely to grow at a higher CAGR (11%) as compared to other routes of administration in the coming years.

Market Share by Type of Therapy

The rare kidney diseases market is categorized into monotherapy and combination therapy. Owing to their safety and convenient nature, the segment for combination therapy is likely to grow at a higher CAGR (75%) as compared to monotherapy in the coming years.

Market Share by Geography

This segment highlights the distribution of rare kidney diseases market across key geographical regions, including North America, Europe, Asia-Pacific, and rest of the world. It is worth highlighting that currently North America captures 50% of the current total rare kidney diseases market share. Further, market in Europe is expected to grow at a CAGR of close to 6% during the forecast period. This can be attributed to the fact that European countries, including Germany, France, and Spain have a growing incidence rate for such diseases. Further, several investments and initiatives have been taken by government authorities to promote the rare kidney diseases market.

Rare Kidney Diseases Market Key Insights

The report features an extensive study of the current market landscape, market size and future opportunities associated with developers within the rare kidney disease, during the given forecast period. The market report highlights the efforts of several stakeholders engaged in this rapidly emerging segment of rare kidney diseases industry. Key takeaways of the rare kidney diseases market report are briefly discussed below.

Competitive Landscape of Drug Developers for Rare Kidney Diseases

The current market landscape features the presence of over 115 companies involved in developing various rare kidney disease therapies. In addition, more than 20 rare kidney disease drugs have been commercialized for targeting various indications with more than 115 therapies in the clinical stage of development. Furthermore, the rare kidney disease market features the presence of small, mid-sized and large companies having the required expertise to develop and commercialize therapies.

Rare Kidney Diseases Market Trends: Increasing Partnerships and Collaborations to Foster Progress and Innovation

The rare kidney disease drug developers have forged several partnerships with other stakeholders in order to enhance their drug portfolios. The growing need for rare kidney disease therapies is evident from the rise in partnership activity in the rare kidney disease market. It is worth highlighting that a significant increase has been witnessed in 2023, wherein majority of the deals were focused on product and technology licensing, followed by the instances of stakeholders collaborating for research and development. In November 2023, Cabaletta Bio entered into a technology licensing agreement with Cellares to manufacture CABA-201, for treating Lupus Nephritis. This can be attributed to the incessant efforts of stakeholders to further advance the development of their drugs across different phases of preclinical and clinical development. Such partnerships are expected to drive significant rare kidney diseases market growth during the forecast period.

Key Drivers of the Rare Kidney Diseases Market

The funding raised, government initiatives and the likely future potential of rare kidney disease therapies will primarily drive the rare kidney diseases market. It is interesting to note that over the past few years, development efforts in rare kidney diseases domain have been adequately supported by various government and non-government agencies. Venture capital firms and other key investors have invested around USD 8 billion in the last 5 years in the companies working towards development of therapeutics targeting rare kidney diseases.

Also, the increasing awareness and understanding of rare kidney diseases have strengthened this domain with several events being conducted by stakeholders. For instance, in September 2023, the American Kidney Fund (AKF) conducted a general awareness campaign for C3 Glomerulopathy and IgA Nephropathy, which was sponsored by Novartis. Such initiatives will increase the awareness amongst the public, improve the diagnosis and treatment rates, driving the rare kidney disease market.

Rare Kidney Diseases Market Size: Drugs Targeting Fabry Disease Hold the Largest Market Share

The global market opportunity for rare kidney diseases is estimated to be more than $3.9 billion in 2024 which is anticipated to grow at CAGR of 11% during the forecast period 2024-2035. The market growth is primarily fueled by increasing incidence of Fabry diseases, encouraging the stakeholders to develop drugs with higher efficacy rates.

North America Holds the Largest Share of the Rare Kidney Diseases Market

In 2024, North America is anticipated to capture close to 50% market share of the overall rare kidney diseases market. In recent years, research activity for rare kidney diseases has witnessed a tremendous increase in North America. Further, it is worth highlighting that, market in Asia-Pacific is likely to grow at a CAGR of 8.5% during the forecast period 2024-2035.

Leading Drug Developers in the Rare Kidney Diseases Market

Examples of key developers involved in the rare kidney diseases industry (which have also been captured in this market report, arranged in alphabetical order) include 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 and Takeda. This market report includes an easily searchable excel database of all the developers involved in the development of rare kidney diseases therapies.

Recent Developments in the Rare Kidney Diseases Market

Several recent developments have taken place in the market, some of which have been outlined below.

• In October 2023, Advicenne Pharma entered into a distribution agreement with Avanzanite Bioscience to distribute Sibnayal® / ADV7103 in the European countries, including Ireland, Cyprus and Malta.
• In June 2023, Everest Medicines entered into a partnership with Guangdong Academy of Medical Sciences with an aim to advance its renal disease research, scientific innovation and translational studies.
• In May 2023, Cabaletta Bio raised USD 100 million through secondary offering, which the company plans to utilize for the development of its product candidate, CABA-201.

Rare Kidney Diseases Market Report Coverage

The rare kidney diseases market report presents an in-depth analysis, highlighting the capabilities of various stakeholders engaged in this industry, across different segments. Amongst other elements, the market report includes:

• An outline of the systematic research methodology adopted to conduct the study on rare kidney diseases market, providing insights on the various assumptions, methodologies, and quality control measures employed to ensure accuracy and reliability of the findings.
• An overview of economic, strategic, technological, and institutional factors that impact the overall rare kidney diseases industry.
• An executive summary of the insights captured during the research. It offers a high-level view on the current landscape of rare kidney diseases and its likely evolution in the mid-long term.
• A general overview on the key historical events related to kidney diseases, factors contributing to kidney diseases, types of rare kidney diseases, and the challenges associated with rare kidney diseases diagnosis and treatments, along with information on the selection of therapeutic targets, technological advancements and the recent developments in the rare kidney diseases domain.
• A detailed overview of the current market landscape of rare kidney diseases, along with information on several relevant parameters, such as year of establishment, company size (in terms of employee count), location of headquarters, phase of development (discovery, preclinical, Pre-IND, clinical and marketed), route of administration (oral, intravenous, subcutaneous, and others), type of molecule (biologic and small molecule), type of biologic (monoclonal antibody, protein, cell and gene therapy, RNA, biosimilar, stem cells, and live biotherapeutic product), dosing frequency (single dose, multiple dose, and both), type of therapy (monotherapies and combination therapies), target patient segment (children, adults and elderly patients), 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, C3 Glomerulopathy).
• Elaborate profiles of prominent companies (shortlisted based on drug portfolio strength) engaged in rare kidney diseases market. Each profile features a brief overview of the company (including information on year of establishment, number of employees, location of headquarters and leadership team), financial information (if available), details related to rare kidney disease portfolio, recent developments and an informed future outlook.
• Short profiles of prominent companies (shortlisted based on drug portfolio strength) engaged in rare kidney diseases market. Each profile features a brief overview of the company (including information on year of establishment, number of employees, location of headquarters and leadership team) and financial information (if available).
• An insightful company competitiveness analysis of prominent developers in the rare kidney diseases industry, based on several relevant parameters, such as supplier strength (in terms of years of experience and company size), company competitiveness (in terms of highest phase of development of drug, number of drugs developed, route of administration, type of molecule) and the number of indications targeted by drug.
• A detailed analysis of the partnerships established between stakeholders engaged in this industry, since 2019, covering licensing agreement, research and development agreement, merger and acquisition, product development and commercialization agreement, commercialization agreement, distribution agreement, joint venture, supply agreement, service agreement, technology utilization agreement, process development agreement, and other relevant agreements.
• An analysis of the funding and investments that have been made into companies developing rare kidney disease therapies, which include venture capital financing, capital raised from IPOs and subsequent offerings, grants, private placement, equity and debt financing. It includes a detailed analysis of the funding instances that have taken place during the period 2019 to 2023, highlighting the growing interest of venture capital (VC) community and other strategic investors in this market.
• An in-depth analysis of completed and ongoing clinical trials related to rare kidney diseases therapies, based on several relevant parameters, such as trial registration year, trial status, trial phase, study design (type of masking, type of intervention model, primary purpose), type of sponsor, most active industry, and non-industry players (in terms of number of trials), geography, and enrolled patient population.
• A detailed analysis of the clinical and commercial attractiveness for drugs targeting rare kidney disorders, based on several relevant parameters, such as target patient population, dosing frequency and dose strength.
• An insightful analysis, highlighting the key opinion leaders (KOLs) investigating clinical trials related to rare kidney diseases, based on several relevant parameters, such as type of KOL, type of organization, target disease indication, industry affiliated organization, non-industry affiliated organization, geographical location of KOLs. In addition, the chapter highlights the most prominent KOLs, based on the proprietary and third-party scoring criteria.
• A case study highlighting the companies engaged in offering kidney care services, along with information on their year of establishment, company size (in terms of number of employees), location of headquarters and target indications.
• An in-depth analysis of the factors that can impact the growth of rare kidney diseases market. It also features identification and analysis of key drivers, potential restraints, emerging opportunities, and existing challenges.
• A detailed estimate of the current market size, current opportunity for the rare kidney diseases therapies, and the future growth potential of the rare kidney diseases market over the next decade. Based on multiple parameters, such as likely adoption trends and through primary validations, the analyst has provided an informed estimate on the market evolution during the forecast period 2024-2035. The report also features the likely distribution of the current and forecasted opportunity within the rare kidney diseases industry. Further, in order to account for future uncertainties and to add robustness to the model, the analyst has provided three forecast scenarios, namely conservative, base, and optimistic scenarios, representing different tracks of the rare kidney diseases market growth.
• 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 across type of molecule, such as small molecules and biologics.
• Detailed projections of the current and future rare kidney diseases market across type of biologics, such as monoclonal antibodies and proteins.
• Detailed projections of the current and future rare kidney disease market across type of route of administration, such as oral, intravenous, subcutaneous, and others.
• Detailed projections of the current and future rare kidney diseases market across type of therapy, such as monotherapy and combination therapy.
• Detailed projections of the current and future rare kidney diseases industry across key geographical regions, such as North America, Europe, Asia-Pacific and rest of the world.
• Detailed projections of the current and future rare kidney disease market across the sales of forecasted drugs.
• Detailed 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.

One of the key objectives of rare kidney diseases market report was to estimate the current market size, opportunity and the future growth potential of the developers in the market, over the forecast period. Based on multiple parameters, likely adoption trends and through primary validations, the analyst has provided an informed estimate on the market evolution during the forecast period 2024-2035. The market report also features the likely distribution of the current and forecasted opportunity for developers in the market across various segments, such as 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), and leading drug developers.

In order to account for future uncertainties associated with some of the key parameters and to add robustness to the model, the analyst has provided three market forecast scenarios, namely conservative, base, and optimistic scenarios, representing different tracks of the industry’s evolution.

The opinions and insights presented in the market report were influenced by discussions held with stakeholders in the industry. The report features detailed transcripts of interviews held with the following industry stakeholders:

• President and Chief Executive Officer, Mid-sized Company, Israel
• Associate BD Director, Large Company, China

Further, all actual figures have been sourced and analyzed from publicly available information forums and primary research discussions. Financial figures mentioned in this market report are in USD, unless otherwise specified.

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• 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.