RARE KIDNEY DISEASES MARKET
As per Roots Analysis, the global rare kidney diseases market size is valued at USD 2.2 billion in 2024 and is projected to reach USD 2.8 billion in the current year and USD 11. 6 billion by 2035, growing at a CAGR of 15.2% during the forecast period.
The opportunity for rare kidney diseases market has been distributed across the following segments:
Target Disease Indications
- Atypical Hemolytic Uremic Syndrome
- Fabry Disease
- Focal Segmental Glomerulosclerosis
- Immunoglobulin A Nephropathy
- Lupus Nephritis
- Others
Type of Molecule
- Biologics
- Small Molecules
Type of Biologic
- Enzymes
- Hormones
- Monoclonal Antibodies
- Proteins
Route of Administration
- Intravenous
- Oral
- Subcutaneous
Type of Therapy
- Monotherapy
- Combination Therapy
Key Geographical Regions
- North America
- Europe
- Asia-Pacific
RARE KIDNEY DISEASES MARKET: GROWTH AND TRENDS
Kidney diseases include a wide array of conditions that hinder the natural ability of the kidney to efficiently filter waste and excess fluid from the blood. These diseases also compromise the overall function of the kidneys to regulate blood pressure and maintain the overall fluid balance in the body. Notably, in the early stages, kidney diseases often present no symptoms, making the diagnosis challenging. However, as waste accumulates in the body, patients may experience symptoms, such as high blood pressure, anemia, poor nutritional health and nerve damage. According to the US Centers for Disease Control and Prevention, approximately 35.5 million people in the US were estimated to be affected by kidney diseases, in 2024, accounting for nearly 14% of the overall population. A subset of these conditions, rare kidney diseases, affect a smaller fraction of individuals worldwide. Under the Orphan Drug Act, a disease is classified as rare in the US if it affects less than 200,000 people. However, in the European Union, rare diseases are defined as those impacting around 5 per 10,000 Europeans.
Rare kidney diseases primarily affect the kidneys' filtering units (known as glomeruli), leading to the spillage of protein components and blood into the urine. These conditions can either be congenital, acquired or inherited, making it difficult to identify specific biomarkers for diagnosis and treatment. Without timely intervention, rare kidney diseases often lead to kidney failure, necessitating dialysis or transplantation. It is important to mention that kidney failure due to a rare chronic kidney disease has been observed in at least 3 out of 5 children and 1 in 9 adults suffering from kidney diseases. To address these challenges, significant advancements have been made in diagnosing and treating rare kidney diseases. Precision medicines, including genomic sequencing and personalized therapies, have enabled the targeted treatment of these diseases. Additionally, the integration of big data and machine learning has helped several drug developers and researchers by improving patient recruitment for clinical trials and identifying accurate urinary biomarkers for tailored treatment.

Moreover, substantial investment from both public and private sectors accelerates research and development in this domain. Presently, drug developers have secured over USD 12 billion in funding for rare kidney research and treatment development. Further, with a growing focus on efficient diagnosis and treatment, the market for rare kidney disease drugs / therapies is poised for robust growth, presenting significant opportunities for stakeholders in the coming years.
RARE KIDNEY DISEASES MARKET: KEY INSIGHTS
The report delves into the current state of the rare kidney diseases market and identifies potential growth opportunities within the industry. Some key findings from the report include:
- Over 70% of the rare kidney drugs / therapies are in clinical stages of development; notably, most of the drugs / therapies are being investigated for the treatment of immunoglobulin A nephropathy and lupus nephritis.
- The rising interest in this domain is evident from the wide array of partnerships established between various stakeholders in the recent past; in fact, 65% of the total deals were inked in the last three years.
- Several investors, having realized the opportunity within this domain, have invested significant capital across various funding rounds; 45% of the investments in this domain were made through secondary offerings.
- Nearly 40% of the drugs targeting rare kidney diseases are currently in Phase II of clinical development; notably, around 60% of the trials have been sponsored by industry players.
- The rare kidney disease market is likely to grow at an annualized rate (CAGR) of ~15%, till 2035; the majority of the market share (~70%) is expected to be captured by North America, by 2035.
- Currently, the monoclonal antibodies dominate the market; notably, oral route of administration is anticipated to capture the majority share in the market by 2035.
RARE KIDNEY DISEASES MARKET: KEY SEGMENTS
Lupus Nephritis is the Fastest Growing Segment in the Rare Kidney Diseases Market
Based on the type of target disease indication, the global rare kidney disease market is segmented into atypical hemolytic uremic syndrome, Fabry disease, focal segmental glomerulosclerosis, immunoglobulin A nephropathy, lupus nephritis, and others. Currently, the majority share of the rare kidney disease market is captured by lupus nephritis, due to the increasing awareness regarding the adversity of the disease among both patient population and healthcare professionals.
Small Molecules are Likely to Hold the Largest Share of the Rare Kidney Diseases Market During the Forecast Period
Based on the type of molecule, the global market is segmented into small molecules and biologics. Amongst these types, the small molecules segment occupies the largest share of the current overall market. However, it is important to note that biologics are expected to emerge as a promising alternative that can target underlying biological pathways associated with rare kidney disease, therefore, capturing a larger market share in the coming years.
Rare Kidney Diseases Market for Proteins is Likely to Grow at a Relatively Faster Pace During the Forecast Period
Based on the type of biologic, the global market value is segmented across enzymes, hormones, monoclonal antibodies, and proteins. Notably, the monoclonal antibodies segment dominates the current market. Moreover, this trend is unlikely to change in the foreseen future owing to their proven precision in treating rare kidney diseases.
Oral Route of Administration Holds the Largest Share of the Rare Kidney Diseases Market
Based on the route of administration, the global market is segmented across intravenous, oral and subcutaneous routes of administration. Currently, the oral route is expected to hold the majority rare kidney disease market share owing to its non-invasive nature and increased patient compliance as compared to other routes. However, it is worth mentioning that this trend is expected to change during the forecast period, with the subcutaneous route of administration capturing a larger share of the overall market in the coming years.
By Type of Therapy, Monotherapy Segment is Likely to Dominate the Market During the Forecast Period
Based on the type of therapy, the global market for rare kidney diseases is segmented across monotherapy and combination therapy. At present, the monotherapy segment holds the maximum share in the overall market. However, the market for combination therapy is likely to grow at a relatively higher CAGR during the forecast period.
North America Accounts for the Largest Share of the Market
Based on key geographical regions, the market is segmented into North America, Europe, and Asia-Pacific. In the current scenario, North America is likely to capture the largest market share. However, the market in Asia-Pacific is expected to grow at a faster pace during the forecast period owing to the fact that various Asian countries, such as China, Japan, Korea, and India are reporting increasing incidence rates of rare kidney diseases which necessitates more effective treatment management systems in this region.
Example Players in the Rare Kidney Diseases Market
- Alexion Pharmaceuticals
- Amicus Therapeutics
- Apellis Pharmaceuticals
- Aurinia Pharmaceuticals
- GlaxoSmithKline
- Novartis
- Roche
- Travere Therapeutics
- Vera Therapeutics
RARE KIDNEY DISEASES MARKET: RESEARCH COVERAGE
The report on Rare Kidney Diseases Market features insights into various sections, including:
- Market Sizing and Opportunity Analysis: An in-depth analysis of the rare kidney diseases market, focusing on key market segments, including [A] type of disease indication, [B] type of molecule, [C] type of biologic, [D] route of administration, [E] type of therapy, [F] key geographical regions, and [G] key players.
- Market Impact Analysis: A thorough analysis of various factors, such as drivers, restraints, opportunities, and existing challenges that are likely to impact market growth.
- Market Landscape: A comprehensive evaluation of rare kidney diseases drugs / therapies developers, based on several relevant parameters, such as [A] stage of development, [B] target indication, [C] route of administration, [D] type of molecule, [E] type of biologic, [F] dosing frequency, [G] target patient population, and [H] type of therapy.
- Rare Kidney Diseases Drug / Therapies Developer Landscape: The report features a list of leading players engaged in the rare kidney diseases domain, along with analyses based on [A] year of establishment, [B] company size, [C] location of headquarters, and [D] most active players (in terms of number of drugs / therapies developed).
- Company Competitiveness Analysis: An insightful competitiveness analysis of the companies engaged in the rare kidney diseases domain, based on various relevant parameters, such as [A] company strength, [B] portfolio strength, and [C] pipeline diversity.
- Company Profiles: In-depth profiles of key industry players offering rare kidney drugs / therapies across various geographies, focusing on [A] company overviews, [B] drug portfolio, [C] financial information (if available), [D] recent developments and [E] an informed future outlook.
- Partnerships and Collaborations: A detailed analysis of partnerships inked between stakeholders in the rare kidney diseases market, since 2019, based on several relevant parameters, such as [A] year of partnership, [B] type of partnership, [C] target disease indication, [D] type of partner, [E] most active players (in terms of number of partnerships), and [F] geography.
- Funding and Investments Analysis: A detailed analysis of the various funding and investments raised in the rare kidney diseases domain, based on several relevant parameters, such as [A] year of funding, [B] type of funding, [C] amount of funding, [D] target disease indication, [E] geography, and [F] most active players (in terms of number of funding instances).
- Clinical Trial Analysis: An in-depth analysis of completed / ongoing clinical trials related to rare kidney diseases drugs / therapies, based on various relevant parameters, such as [A] trial registration year, [B] trial status, [C] number of patients enrolled, [D] trial phase, [E] study design (type of trial masking, type of intervention model, trial purpose and design allocation), [F] type of sponsor, [G] patient gender, [H] most active sponsor / collaborator (leading industry and non-industry players based on the number of registered trials), and [I] geography.
- Clinical Commercial Attractiveness: A detailed analysis of the clinical and commercial attractiveness for phase III drugs / therapies targeting rare kidney diseases, based on several parameters, such as [A] type of molecule, [B] dosing frequency, [C] route of administration, [D] number of patients enrolled, [E] company size, [F] patient population, and [G] location of clinical trials.
- KOL Analysis: An insightful analysis, highlighting the key opinion leaders investigating the clinical trials related to rare kidney diseases, based on several parameters, such as [A] type of KOL, [B] qualification, [C] type of organization, [D] geographical location of KOLs, [E] target disease indication, and [F] most prominent KOLs (based on our proprietary and third-party scoring criteria).
- Case Study: An insightful case study highlighting the developers engaged in offering kidney care services, along with information on [A] year of establishment, [B] company size, [C] location of headquarters, [D] target indications, [E] benefits offered by the kidney care companies, and [F] recent developments in the domain.
KEY QUESTIONS ANSWERED IN THIS REPORT
- How many companies are currently engaged in this market?
- Which are the leading companies in this market?
- What factors are likely to influence the evolution of this market?
- What is the current and future market size?
- What is the CAGR of this market?
- How is the current and future market opportunity likely to be distributed across key market segments?
- What is the patent filing activity trend in the market?
REASONS TO BUY THIS REPORT
- The report provides a comprehensive market analysis, offering detailed revenue projections of the overall market and its specific sub-segments. This information is valuable to both established market leaders and emerging entrants.
- Stakeholders can leverage the report to gain a deeper understanding of the competitive dynamics within the market. By analyzing the competitive landscape, businesses can make informed decisions to optimize their market positioning and develop effective go-to-market strategies.
- The report offers stakeholders a comprehensive overview of the market, including key drivers, barriers, opportunities, and challenges. This information empowers stakeholders to stay abreast of market trends and make data-driven decisions to capitalize on growth prospects.
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