The term rare kidney diseases (RKD) represents around 150 different indications. It is worth highlighting that the prevalence rate of rare kidney diseases 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 disease. The low 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 diseases. It is worth mentioning that, post the onset of the COVID-19 pandemic, the treatment of patients affected with rare kidney diseases 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 diseases.
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 diseases. It is worth highlighting that, since 2020, over seven drugs have received marketing approval for the treatment of rare kidney diseases; 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 diseases 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.
Scope of the Report
The Rare Kidney Disease Market 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 diseases 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 diseases market and its likely evolution in the mid to long term.
- A brief introduction to various aspects of rare kidney diseases. It includes a detailed discussion on the historical evolution and factors contributing to the treatment of rare kidney diseases. Further, it highlights various prominent target indications associated with rare kidney diseases, 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 diseases, 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 diseases, 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 diseases (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 diseases 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 diseases 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 diseases, 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 diseases, 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 diseases 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.
Key Questions Answered
- Who are the leading players engaged in the development of drugs targeting rare kidney diseases?
- 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 diseases?
- 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 diseases?
- How is the current and future market opportunity likely to be distributed across key market segments?
Table of Contents
Companies Mentioned
- AbbVie
- Achillion Pharmaceuticals
- Aduro Biotech
- Advicenne
- Aevitas Therapeutics (A subsidiary of Fortress Biotech)
- Alexion Pharmaceuticals (A subsidiary of AstraZeneca)
- Algomedix
- Alnylam Pharmaceuticals
- Amicus Therapeutics
- Amyndas Pharmaceuticals
- Anelixis Therapeutics (Acquired by Eledon Pharmaceuticals)
- Apellis Pharmaceuticals
- Apple Tree Partners
- Arrowhead Pharmaceuticals
- ARYA Sciences Acquisition
- Asahi Kasei Pharma
- Astellas Pharma
- AstraZeneca
- Aurinia Pharmaceuticals
- AVROBIO
- Beam Therapeutics
- BioCryst Pharmaceuticals
- Biogen
- Boehringer Ingelheim
- Bristol-Myers Squibb
- Calliditas Therapeutics
- ChemoCentryx
- CHIESI Farmaceutici
- Chinook Therapeutics
- Cian Healthcare
- Complexa
- Cormorant Asset Management
- Dicerna Pharmaceuticals
- EcoR1 Capital
- Eledon Pharmaceuticals (Formerly known as Novus Therapeutics)
- Ember Therapeutics
- Evotec
- ExCEEd Orphan
- Foresee Pharmaceuticals
- Frazier Healthcare Partners
- FrostPharma
- Fulcrum Therapeutics
- GENERIUM
- Gilead Sciences
- GlaxoSmithKline
- Goldfinch Bio
- Grifols
- Halozyme Therapeutics
- Horizon Pharma
- Horizon Therapeutics
- Idorsia Pharmaceuticals
- I-Mab Biopharma
- InflamaCORE
- Ionis Pharmaceuticals
- Jiangsu Hengrui Medicine
- Kezar Life Sciences
- Lonza
- Mallinckrodt Pharmaceuticals
- MedImmune
- Monashee Investment Management
- Morehouse School of Medicine
- Morningside
- MorphoSys
- myTomorrows
- Novartis Pharmaceuticals
- Omeros
- Oraxion Therapeutics (A subsidiary of Aten Porus Lifesciences)
- Organon
- Orphan Technologies
- Otsuka Pharmaceutical
- Pfizer
- Pivotal bioVenture Partners
- Protalix Biotherapeutics
- Reata Pharmaceuticals
- Recordati Rare Diseases
- Regulus Pharmaceuticals
- RemeGen Biosciences
- Rigel Pharmaceuticals
- Roche
- ROHTO Pharmaceutical
- Samsara BioCapital
- Sanofi
- Savient Pharmaceuticals
- Silence Therapeutics
- Surveyor Capital
- Swedish Orphan Biovitrum (SOBI)
- Syntimmune
- Takeda
- Travere Therapeutics (Formerly known as Retrophin)
- TwinPharma
- University of Massachusetts Medical School
- University of Pennsylvania
- University of Washington
- Vera Therapeutics
- Vifor Pharma
- Visterra (Acquired by Otsuka Pharmaceutical)
- Zealand Pharma
- ZyVersa Therapeutics (Formerly known as Variant Pharmaceuticals)
Methodology
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