Rare Kidney Disorders Market, 2022-2035

Rare Kidney Disorders Market, 2022-2035

The term rare kidney diseases represents around 150 different indications. It is worth highlighting that the prevalence rate of rare kidney disorders (RKD) is estimated to be 60-80 per 100,000 cases / individuals in the US and Europe. Despite their relatively low prevalence, these set of indications are believed to be one of the leading causes of renal diseases, along with diabetes and other rare genetic kidney disorders. The low disease incidence rate of such disorders, coupled to the lack of awareness among the general population, often leads to late or erroneous diagnosis. This, in turn, enables suboptimal therapeutic management, as well as compromised long-term outcomes. The aforementioned factors are some of the key reasons that are believed to be responsible for high mortality rates and severe compromise on chance of survival of a patient suffering with rare kidney disorders. It is worth mentioning that, post the onset of the COVID-19 pandemic, the treatment of patients affected with rare kidney disorders became even more challenging. This can be primarily attributed to the fact that the current treatment options for such indications are heavily reliant on regular patient visits to the hospitals or medical centers. Consequently, during the pandemic, a 30% increase in mortality rate was observed in affected patients. This increase in mortality has compelled players engaged in the pharmaceutical domain to undertake necessary research initiatives in order to identify novel drugs that can efficiently treat rare kidney disorders.

Given the complexity associated with conventional treatment options, such as dialysis, kidney transplant and biopsy, drug developers engaged in this domain have shifted their focus towards the development of drugs that can target a myriad of rare kidney disorders. It is worth highlighting that, since 2020, over seven drugs have received marketing approval for the treatment of rare kidney disorders; these are (in reverse chronological order of approval) Galafold® (Fabry disease, European Medicines Agency (EMA), 2021), Lupkynis™ (Lupus Nephritis (LN), United States Food and Drug Administration (USFDA), 2021), Sibnayal™ (distal Renal Tubular Acidosis (dRTA), EMA, 2021), Tarpeyo® (Immunoglobulin A Nephropathy (IgAN), USFDA, 2021), Benlysta® (LN, USFDA, 2020), Cystadrops® (Cystinosis, USFDA, 2020), and Ultomiris® (Atypical Hemolytic Uremic Syndrome (aHUS), Japan, 2020). Further, more than 90 candidates targeting rare kidney disorders are currently under clinical investigation, while several novel leads are being evaluated in early stages of development. Given the lucrative opportunity associated with these targets, this domain has gained attention of both private and public investors in the past few years. In fact, over USD 4.5 billion has been invested in this domain since 2020. As more candidates are likely to progress towards advanced stages of development, we expect the market to witness aggressive growth in the foreseen future.

The Rare Kidney Disorders Market - Distribution by Target Indications (IgA Nephropathy, Lupus Nephritis, Focal Segmental Glomerular Sclerosis, Membranous Nephropathy, C3 Glomerulopathy and Others), Type of Molecule (Small Molecules and Biologics (Monoclonal Antibody, Hormone, Recombinant Protein, and Others)), Route of Administration (Oral, Intravenous, Subcutaneous, and Others) and Key Geographies (North America, Europe, Asia Pacific and Rest of the World): Industry Trends and Global Forecast, 2022-2035 report features an extensive study of the current market landscape and the likely future potential associated with the rare kidney disorders market, over the next decade. The study also features an in-depth analysis, highlighting the capabilities of various industry stakeholders engaged in this field. Amongst other elements, the report features:

A detailed executive summary of key insights captured during our research. It offers a high-level view on the current state of rare kidney disorders market and its likely evolution in the mid to long term.

A brief introduction to various aspects of rare kidney disorders. It includes a detailed discussion on the historical evolution and factors contributing to the treatment of rare kidney disorders. Further, it highlights various prominent target indications associated with rare kidney disorders, application of omics technology and its future perspectives.

A detailed overview of the current market landscape of more than 127 drug candidates that are either approved or currently under development for the treatment of various rare kidney disorders, based on several relevant parameters, such as phase of development (approved, registration, phase III, phase II, phase I, pre-clinical and discovery), target molecule (complement component 5 (C5), complement component 3 (C3), a proliferation-inducing ligand (APRIL), B lymphocyte stimulator (BLyS), Complement Factor B, and others), route of administration (oral, intravenous, subcutaneous, and others), and target indication (complement 3 glomerulopathy (C3G), focal segmental glomerular sclerosis (FSGS), IgA nephropathy (IgAN), lupus nephritis (LN), membranous nephropathy (MN)), type of molecule (biologics and small molecules), and type of biologic (monoclonal antibody (mAb), hormone, recombinant protein, enzyme replacement therapy (ERT), gene therapy, small interfering ribonucleic acid (siRNA) molecule and others). In addition, it presents details of the companies engaged in the development of drugs targeting rare kidney disorders, based on several relevant parameters, such as year of establishment, company size (in terms of number of employees), location of headquarters, and most active players.

Elaborate profiles of approved and clinical stage drugs indicated for rare kidney disorders (phase II / III or above). Each profile features an overview of the drug developer, clinical trial information related to the drug, clinical trial results, and estimated sales revenues (if available).

A detailed competitiveness analysis of companies engaged in the development of rare kidney disorders targeting drugs, based on their developer strength (in terms of years of experience), portfolio strength (in terms of number of drugs developed), and portfolio diversity (in terms of phase of development, target indication, and drug designation).

An in-depth analysis of the various collaborations and partnerships that have been inked by players engaged in the development of drugs targeting various rare kidney disorders since 2017, based on several relevant parameters, such as year of partnership, type of partnership, focus area, type of partner and regional distribution of partnerships.

An analysis of the funding and investments made within the domain, during the period 2016-2022, based on several relevant parameters, such as year of funding, type of funding (seed financing, venture capital financing, debt financing, grants, IPOs and other offerings), leading players (in terms of amount invested) and key investors (in terms of number of funding instances).

A detailed clinical trial analysis of ongoing clinical studies for the evaluation of various drug candidates targeting rare kidney disorders, based on several relevant parameters, such as trial registration year, current trial status, phase of development, key target indications, patient age, study design and key geographical regions.

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.

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 indication.

One of the key objectives of this report was to evaluate the current market size and the future potential associated with rare kidney disorders market, over the coming years. We have developed an informed estimate on the likely evolution of the market in the short to mid-term and long term, for the period 2022-2035. Additionally, the report features the likely distribution of the current and forecasted opportunity across relevant parameters, such as [A] target indications (complement 3 glomerulopathy (C3G), FSGS, IgAN, LN, membranous nephropathy (MN)), [B] type of molecule (small molecules and biologics (monoclonal antibody, hormone, recombinant protein, and others)),[C] route of administration (oral, intravenous, subcutaneous, and others) [D] key geographical regions (North America, Europe, Asia-Pacific, and Rest of the World (RoW)). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.

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

MARKET SEGMENTATIONS

Rare Kidney Disorders Market: Market Segmentations

Market Segments Details

Forecast Period 2022 - 2035

Target Indications IgA Nephropathy

Lupus Nephritis

Focal Segmental Glomerular Sclerosis

Membranous Nephropathy

C3 Glomerulopathy

Other Disorders

Type of Molecule Small Molecules

Biologics

Route of Administration Oral

Intravenous

Subcutaneous

Other Route of Administration

Key Geographical Regions North America

Europe

Asia-Pacific

Rest of the World

Source: Roots Analysis

RESEARCH METHODOLOGY

The data presented in this report has been gathered via secondary research. For all our projects, we have conducted interviews / surveys with various experts in this domain (academia, industry, medical practice and other associations) in order to solicit their opinions on emerging trends in the market. This is primarily useful for us to draw out our own opinion on how the market will evolve across different regions and technology segments. Wherever possible, the available data has been checked for accuracy from multiple sources of information.

The secondary sources of information include

Annual reports

Investor presentations

SEC filings

Industry databases

News releases from company websites

Government policy documents

Industry analysts’ views

While the focus has been on forecasting the market till 2035, the report also provides our independent view on various technological and non-commercial trends emerging in the industry. This opinion is solely based on our knowledge, research and understanding of the relevant market gathered from various secondary and primary sources of information.

KEY QUESTIONS ANSWERED

Who are the leading players engaged in the development of drugs targeting rare kidney disorders?

Which are the key drugs being developed across early and late stages of development?

Who are the key players providing services related to kidney care?

Which are the key indications targeted by drugs indicated for rare kidney disorders?

What type of partnership models are commonly adopted by stakeholders engaged in this domain?

Who are the key investors in the domain?

Which regions have emerged as the key hubs for conducting clinical studies focused on rare kidney disorders?

How is the current and future market opportunity likely to be distributed across key market segments?

CHAPTER OUTLINES

Chapter 2 is an executive summary of key insights captured during our research. It offers a high-level view on the current state of rare kidney disorders market and its likely evolution in the mid to long term.

Chapter 3 provides a brief introduction to various aspects of rare kidney disorders. It includes a detailed discussion on the historical evolution and factors contributing to the treatment of rare kidney disorders. Further, it highlights various prominent target indications associated with rare kidney disorders, application of omics technology and its future perspectives.

Chapter 4 provides a detailed overview of the current market landscape of more than 127 drug candidates that are either approved or currently under development for the treatment of various rare kidney disorders, based on several relevant parameters, such as phase of development ( approved, registration, phase III, phase II, phase I, pre-clinical and discovery), target molecule (complement component 5 ( C5), complement component 3 (C3), a proliferation-inducing ligand (APRIL), B lymphocyte stimulator (BLyS), Complement Factor B, and others), route of administration (oral, intravenous, subcutaneous, and others), and target indication (complement 3 glomerulopathy (C3G), FSGS, IgAN, LN, membranous nephropathy (MN)), type of molecule (biologics and small molecules), and type of biologic (monoclonal antibody (mAb), hormone, recombinant protein, enzyme replacement therapy (ERT), gene therapy, small interfering ribonucleic acid (siRNA) molecule and others). In addition, it presents details of the companies engaged in the development of drugs targeting rare kidney disorders, based on several relevant parameters, such as year of establishment, company size (in terms of number of employees), location of headquarters, and most active players.

Chapter 5 provides detailed profiles of approved and clinical stage drugs indicated for rare kidney disorders (phase II / III or above). Each profile features an overview of the drug developer, clinical trial information related to the drug, clinical trial results, and estimated sales revenues (if available).

Chapter 6 features a detailed competitiveness analysis of companies engaged in the development of rare kidney disorders targeting drugs, based on their developer strength (in terms of years of experience), portfolio strength (in terms of number of drugs developed), and portfolio diversity (in terms of phase of development, target indication, and drug designation).

Chapter 7 features an elaborate analysis of the various collaborations and partnerships that have been inked by players engaged in the development of drugs targeting various rare kidney disorders since 2017. It includes details of deals that were / are focused on the rare kidney disorders, which were analyzed on the basis of year of partnership, type of partnership, focus area, type of partner and regional distribution of partnerships. It also consists of a schematic representation showcasing the players that have established the maximum number of alliances related to the rare kidney disorders. Furthermore, we have provided a world map representation of all the deals inked in this field, highlighting those that have been established within and across different continents.

Chapter 8 presents detailed analysis of the funding and investments made within the domain, during the period 2016-2022, based on several relevant parameters, such as year of funding, type of funding (seed financing, venture capital financing, debt financing, grants, IPOs and other offerings), leading players (in terms of amount invested) and key investors (in terms of number of funding instances).

Chapter 9 presents a detailed clinical trial analysis of ongoing clinical studies for the evaluation of various drug candidates targeting rare kidney disorders, based on several relevant parameters, such as trial registration year, current trial status, phase of development, key target indications, patient age, study design and key geographical regions.

Chapter 10 features an 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

Chapter 11 presents an elaborate market forecast analysis, highlighting the future potential of the market till the year 2035. It also includes future sales projections of rare kidney disorders that are either marketed or in advanced stages of clinical development (phase II/III and above). Additionally, the report features the likely distribution of the current and forecasted opportunity across relevant parameters, such as [A] target indications (complement 3 glomerulopathy (C3G), FSGS, IgAN, LN, membranous nephropathy (MN)), [B] type of molecule (small molecules and biologics (monoclonal antibody, hormone, recombinant protein, and others)),[C] route of administration (oral, intravenous, subcutaneous, and others) [D] key geographical regions (North America, Europe, Asia-Pacific, and Rest of the World (RoW)). In order to account for future uncertainties and to add robustness to our model, we have provided three market forecast scenarios, namely conservative, base and optimistic scenarios, representing different tracks of the industry’s growth.

Chapter 12 presents 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 indication.

Chapter 13 is an appendix, which provides tabulated data and numbers for all the figures included in the report.

Chapter 14 is an appendix, which contains a list of companies and organizations mentioned in the report.