Rare Kidney Diseases Market Size, Share & Trends Analysis Report By Target Indication, By Type of Molecule (Small Molecules, Biologics, By Route of administration (Oral, Intravenous, Subcutaneous, Others), By End-User, By Distribution, By Region, And By Segment Forecasts, 2025-2034

Report Id: 1414 Pages: 190 Last Updated: 06 March 2025 Format: PDF / PPT / Excel / Power BI
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Global Rare Kidney Diseases Market Size is valued at USD 2.9 Billion in 2024 and is predicted to reach USD 8.1 Billion by the year 2034 at a 11.0% CAGR during the forecast period for 2025-2034.

Rare Kidney Diseases Market

Key Industry Insights & Findings from the Report:

  • The emergence of the rare kidney disease market is credited to the improved understanding and identification of these conditions, resulting in higher diagnosis rates.
  • The increasing incidences and prevalence of kidney diseases are expected to drive industry growth.
  • North America dominated the market and accounted for a global revenue share in 2024.
  • One of the significant concerns restraining industry growth is the slow drug approvals and complex disease mechanisms.

Kidney diseases have the potential to intensify over time, eventually leading to kidney failure, which is when the kidneys completely stop working. In addition, having one of these diseases can put you at a higher risk for developing another condition, such as cardiovascular disease. At least 150 distinct conditions are classified as rare kidney diseases, and the majority are inherited. The diagnosis and treatment of rare kidney diseases as a group may present overlapping difficulties, even though individual cases of these diseases present unique obstacles. Diagnosis, management, and treatment of inherited kidney disorders are complicated, especially when multisystem complications are present. These diseases can result in numerous physical and mental impairments, lowering life expectancy and reducing the quality of life. In the last two years, the COVID-19 infection severely affected the human lifestyle and approach towards chronic disease treatment. This led to delayed detection of rare kidney diseases and became responsible for the increase in the number of patients.

Several important factors are responsible for the growth of the rare kidney disease market. It is anticipated that the increasing use of next-generation sequencing techniques will improve diagnostic accuracy for rare kidney diseases, aid in elucidating the molecular mechanism of disease, facilitate genetic counselling, and provide opportunities for carrier testing. Renal replacement therapy advancements and increased accessibility allow for more prolonged patient survival, but frequently at the expense of their quality of life. FDA approvals, Government initiatives and funding also contribute to expanding the market for such complicated diseases. In 2021, IgA nephropathy was rare kidney disease, and the FDA approved the first medication to reduce urine protein. The FDA had approved Tarpeyo (budesonide) delayed-release capsules to treat adults with primary immunoglobulin A (IgA) nephropathy who are at risk of rapid disease progression by lowering proteinuria (increased protein levels in the urine).

Multiple factors, including unresolved genetic defects, a lack of biomarkers to track disease progression, heterogeneous clinical phenotypes, and outdated diagnostic classifications that do not reflect underlying pathophysiological mechanisms, have impacted therapeutic advancements in rare kidney diseases. Slow drug approvals and complex studies will also restrain the market's growth.

Market Segmentation

The Rare Kidney Diseases market is segmented into target indications, type of molecule, and route of administration. Target indication segment includes IgA Nephropathy, Lupus Nephritis, Focal Segmental Glomerular Sclerosis, Membranous Nephropathy, C3 Glomerulopathy and others. Another segment is a type of molecule, which comprises Small Molecules and biologics. The subsegment biologics include Monoclonal Antibody, Hormone, Recombinant Protein, and Others. The last segment is the route of administration which has Oral, Intravenous, Subcutaneous, and other routes as subsegments.

According to regional analysis, North America will lead the global Rare Kidney Diseases market during the forecast period due to rapid clinical advancement breakthroughs and research for treatments of kidney diseases.

Recent Developments:

  • In Aug 2023, Novartis successfully finalized its acquisition of Chinook Therapeutics, a clinical-stage biopharmaceutical business based in the United States. Chinook Therapeutics specializes in the research and production of precision medicines specifically designed for the treatment of kidney ailments. The agreement comprised a $3.2 billion initial payment, with a potential total purchase value of up to $3.5 billion.
  • In March 2024, Travere Therapeutics, Inc. submitted a sNDA to the U.S. FDA to convert the current accelerated approval of FILSPARI® (sparsentan) in IgA nephropathy (IgAN) to full approval. In February 2023, the FDA granted expedited approval to FILSPARI as the initial and exclusive non-immunosuppressive therapy specifically designed to address glomerular damage in the kidney and decrease proteinuria in people with primary IgAN who might be at risk of rapid disease progression.

Competitive Landscape

Some major key players in the Rare Kidney Diseases Market:

  • GlaxoSmithKline,
  • Recordati Rare Diseases,
  • Amicus Therapeutics,
  • Calliditas Therapeutics,
  • Alexion Pharmaceuticals (A subsidiary of AstraZeneca),
  • Travere Therapeutics,
  • Aurinia Pharmaceutical,
  • Advicenne,
  • Protalix Biotherapeutics,
  • AstraZeneca,
  • Chinook Therapeutics,
  • Reata Pharmaceuticals,
  • Roche,
  • Novartis Pharmaceuticals,
  • Merk & co.,
  • Apellis Pharmaceuticals,
  • Omeros Corporation,
  • Lonza,
  • Other Prominent Players.

Rare Kidney Diseases Market Report Scope

Report Attribute Specifications
Market size value in 2024 USD 2.9 Billion
Revenue forecast in 2034 USD 8.1 Billion
Growth rate CAGR CAGR of 11.0 % from 2025 to 2034
Quantitative units Representation of revenue in US$ Million and CAGR from 2025 to 2034
Historic Year 2021 to 2024
Forecast Year 2025-2034
Report coverage The forecast of revenue, the position of the company, the competitive market structure, growth prospects, and trends
Segments covered Target Indication, Type of Molecule, Route of administration
Regional scope North America; Europe; Asia Pacific; Latin America; Middle East & Africa
Country scope U.S.; Canada; U.K.; Germany; China; India; Japan; Brazil; Mexico ;The UK; France; Italy; Spain; China; Japan; India; South Korea; South East Asia; South Korea; South East Asia
Competitive Landscape GlaxoSmithKline, Recordati Rare Diseases, Amicus Therapeutics, Calliditas Therapeutics, Alexion Pharmaceuticals (A subsidiary of AstraZeneca), Travere Therapeutics, Aurinia Pharmaceutical, Advicenne, Protalix Biotherapeutics, AstraZeneca, Chinook Therapeutics, Reata Pharmaceuticals, Roche, Novartis Pharmaceuticals, Merk & co., Apellis Pharmaceuticals, Omeros Corporation, Lonza, and Other Prominent Players.
Customization scope Free customization report with the procurement of the report, Modifications to the regional and segment scope. Particular Geographic competitive landscape.
Pricing and available payment methods Explore pricing alternatives that are customized to your particular study requirements.

Market Segmentation:

Global Rare Kidney Diseases Market, by Target Indication

  • IgA Nephropathy
  • Lupus Nephritis

  • Focal Segmental Glomerular Sclerosis

  • Membranous Nephropathy

  • C3 Glomerulopathy

  • Cystinosis

  • Dense Deposit Disease

  • Distal Renal Tubular Acidosis

  • Fabry Disease

  • Refractory Gout

  • Atypical Hemolytic Uremic Syndrome

  • Others

Global Rare Kidney Diseases Market, by Type Of Molecule

  • Small Molecules
  • Biologics
    • Monoclonal Antibody
    • Hormone
    • Recombinant Protein
    • Others

 Global Rare Kidney Diseases Market, by Route Of Administration

  • Oral
  • Intravenous
  • Subcutaneous
  • Others

 Global Rare Kidney Diseases Market, by Region

  • North America
  • Europe
  • Asia Pacific
  • Latin America
  • Middle East & Africa

 North America Rare Kidney Diseases Market, by Country

  • U.S.
  • Canada

 Europe Rare Kidney Diseases Market, by Country

  • Germany
  • France
  • Italy
  • Spain
  • Russia
  • Rest of Europe

 Asia Pacific Rare Kidney Diseases Market, by Country

  • India
  • China
  • Japan
  • South Korea
  • Australia & New Zealand

 Latin America Rare Kidney Diseases Market, by Country

  • Brazil
  • Mexico
  • Rest of Latin America

Middle East & Africa Rare Kidney Diseases Market, by Country, 2022-2030 (Value US$ Mn)

  • GCC Countries
  • South Africa
  • Rest of Middle East & Africa

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Research Design and Approach

This study employed a multi-step, mixed-method research approach that integrates:

  • Secondary research
  • Primary research
  • Data triangulation
  • Hybrid top-down and bottom-up modelling
  • Forecasting and scenario analysis

This approach ensures a balanced and validated understanding of both macro- and micro-level market factors influencing the market.

Secondary Research

Secondary research for this study involved the collection, review, and analysis of publicly available and paid data sources to build the initial fact base, understand historical market behaviour, identify data gaps, and refine the hypotheses for primary research.

Sources Consulted

Secondary data for the market study was gathered from multiple credible sources, including:

  • Government databases, regulatory bodies, and public institutions
  • International organizations (WHO, OECD, IMF, World Bank, etc.)
  • Commercial and paid databases
  • Industry associations, trade publications, and technical journals
  • Company annual reports, investor presentations, press releases, and SEC filings
  • Academic research papers, patents, and scientific literature
  • Previous market research publications and syndicated reports

These sources were used to compile historical data, market volumes/prices, industry trends, technological developments, and competitive insights.

Secondary Research

Primary Research

Primary research was conducted to validate secondary data, understand real-time market dynamics, capture price points and adoption trends, and verify the assumptions used in the market modelling.

Stakeholders Interviewed

Primary interviews for this study involved:

  • Manufacturers and suppliers in the market value chain
  • Distributors, channel partners, and integrators
  • End-users / customers (e.g., hospitals, labs, enterprises, consumers, etc., depending on the market)
  • Industry experts, technology specialists, consultants, and regulatory professionals
  • Senior executives (CEOs, CTOs, VPs, Directors) and product managers

Interview Process

Interviews were conducted via:

  • Structured and semi-structured questionnaires
  • Telephonic and video interactions
  • Email correspondences
  • Expert consultation sessions

Primary insights were incorporated into demand modelling, pricing analysis, technology evaluation, and market share estimation.

Data Processing, Normalization, and Validation

All collected data were processed and normalized to ensure consistency and comparability across regions and time frames.

The data validation process included:

  • Standardization of units (currency conversions, volume units, inflation adjustments)
  • Cross-verification of data points across multiple secondary sources
  • Normalization of inconsistent datasets
  • Identification and resolution of data gaps
  • Outlier detection and removal through algorithmic and manual checks
  • Plausibility and coherence checks across segments and geographies

This ensured that the dataset used for modelling was clean, robust, and reliable.

Market Size Estimation and Data Triangulation

Bottom-Up Approach

The bottom-up approach involved aggregating segment-level data, such as:

  • Company revenues
  • Product-level sales
  • Installed base/usage volumes
  • Adoption and penetration rates
  • Pricing analysis

This method was primarily used when detailed micro-level market data were available.

Bottom Up Approach

Top-Down Approach

The top-down approach used macro-level indicators:

  • Parent market benchmarks
  • Global/regional industry trends
  • Economic indicators (GDP, demographics, spending patterns)
  • Penetration and usage ratios

This approach was used for segments where granular data were limited or inconsistent.

Hybrid Triangulation Approach

To ensure accuracy, a triangulated hybrid model was used. This included:

  • Reconciling top-down and bottom-up estimates
  • Cross-checking revenues, volumes, and pricing assumptions
  • Incorporating expert insights to validate segment splits and adoption rates

This multi-angle validation yielded the final market size.

Forecasting Framework and Scenario Modelling

Market forecasts were developed using a combination of time-series modelling, adoption curve analysis, and driver-based forecasting tools.

Forecasting Methods

  • Time-series modelling
  • S-curve and diffusion models (for emerging technologies)
  • Driver-based forecasting (GDP, disposable income, adoption rates, regulatory changes)
  • Price elasticity models
  • Market maturity and lifecycle-based projections

Scenario Analysis

Given inherent uncertainties, three scenarios were constructed:

  • Base-Case Scenario: Expected trajectory under current conditions
  • Optimistic Scenario: High adoption, favourable regulation, strong economic tailwinds
  • Conservative Scenario: Slow adoption, regulatory delays, economic constraints

Sensitivity testing was conducted on key variables, including pricing, demand elasticity, and regional adoption.

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Frequently Asked Questions

GlaxoSmithKline, Recordati Rare Diseases, Amicus Therapeutics, Calliditas Therapeutics, Alexion Pharmaceuticals

Rare Kidney Diseases Market is expected to grow at a 11.0% CAGR during the forecast period for 2025-2034.

Rare Kidney Diseases Market Size is valued at USD 2.9 Billion in 2024 and is predicted to reach USD 8.1 Billion by the year 2034

Target Indication, Type of Molecule and Route of administration are the key segments of the Rare Kidney Diseases Market.

North American region is leading the Rare Kidney Diseases Market.
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