The rare kidney disease treatment market is expected to expand at a CAGR of 6% during the forecast period of 2023 to 2030 The global market for rare kidney disease treatment market was valued at US$ 2.5 Bn in 2021. The occurrence of acute renal failure and other kidney illnesses that are relatively uncommon is on the rise dramatically around the world. Rare kidney illnesses have only a limited number of therapeutic options that have been approved. In the global market for the treatment of rare kidney disorders, the introduction of new products as well as the development of more affordable therapies and services are projected to generate profitable opportunities for manufacturers over the next few years, particularly in less developed nations. Due to the high incidence rate and the requirement for efficient renal disease care, nations in Asia, particularly China and India, are seeing an increase in the demand for the treatment of rare kidney illnesses. This demand is rising around the globe. The most important actors are concentrating their efforts on creating new therapeutic options for renal illnesses that are uncommon.

Around 150 distinct conditions are included under the term "rare kidney disease," often known as RKD. It is essential to bring attention to the fact that it is estimated that the prevalence rate of rare kidney disorders in the U.S. and Europe is between 60 and 80 per 100,000 instances of individuals. Along with diabetes and other uncommon genetic kidney disorders, this group of symptoms is thought to be one of the primary causes of renal diseases, despite the fact that its frequency is relatively low. Diabetes is another main cause of renal diseases. Because of the low incidence rate of such conditions and the lack of awareness that exists among the general community, diagnosis is sometimes delayed or performed incorrectly. This, in turn, makes suboptimal treatment management possible, which in turn leads to outcomes that are degraded over the long run. One of the primary reasons that are believed to be responsible for high mortality rates and a severe compromise on the probability of survival of a patient who is afflicted with rare kidney disorders is the fact that the factors described above are some of the key reasons. Because of this rise in mortality, firms active in the pharmaceutical sector have been forced to undertake crucial research endeavors in order to discover novel medications that are capable of effectively treating rare kidney disorders.

An Alarmingly High Prevalence of Long-Term Conditions That Can Result in Kidney Diseases

1 in 10 people around the world is living with chronic kidney disease (CKD), which is a condition that can be fatal if left untreated. Between the years 2015 and 2017, high blood pressure and diabetes were the two leading causes of kidney failure, accounting for 75% of all occurrences. In addition, a study that was conducted and released by the National Renal Foundation in the year 2020 found that approximately 35.0% of the diabetic population that was over the age of 20 will develop chronic kidney disease over the course of time. According to the findings that were published by the National Kidney Foundation Inc., CKD is projected to be the leading cause of death in the United States in the year 2020, surpassing both prostate cancer and breast cancer. People have a tendency to be unaware of the emergency nature of this health issue. Because of CKD, over 80 million adult Americans, or 1 in every 3, are at risk.

The Centers for Disease Control and Prevention (CDC) have projected that in the year 2020, 1 in 3 persons who have diabetes and 1 in 5 adults who have high blood pressure will develop chronic renal disease. According to the American Diabetes Association, there were 30.3 million persons living in the United States who had diabetes in 2015. The Centers for Disease Control and Prevention (CDC) estimates that approximately 118,000 people in the United States began treatment for end-stage renal disease in 2014. It is anticipated that the number of people receiving treatment for this condition will increase as more people become aware of the condition. In addition, the Centers for Disease Control and Prevention (CDC) reports that around 96% of persons who have kidney damage are unaware that they have chronic kidney disease. This highlights the underlying market perspective in the kidney disease market. During the time of the forecast, these factors are anticipated to contribute to the expansion of the market.

Increased Focus on the Drug Development and Approvals

It is anticipated that the burden of a number of different rare kidney illnesses will dramatically increase all over the world within the next few years. There has been an increase in the prevalence of conditions that affect the kidneys, including Fabry disease, atypical hemolytic uremic syndrome, lupus nephritis, nephropathic cystinosis, renal nutcracker syndrome, polycystic kidney disease (PKD), distal renal tubular acidosis, and IgA nephropathy disease. Because of the difficulties connected with standard treatment choices including dialysis, kidney transplantation, and biopsy, pharmaceutical companies working in this field have moved their focus to the creation of medications that can target a wide variety of rare kidney illnesses. It is important to note that over seven different medications have been granted marketing approval for the treatment of rare kidney diseases since the year 2020. These medications are as follows Galafold® (Fabry disease, European Medicines Agency (EMA), 2021), LupkynisTM (Lupus Nephritis (LN), United States Food and Drug Administration (USFDA), 2021), SibnayalTM (distal Renal Tubular Acidosis). In addition, there are over 90 potential treatments for rare kidney illnesses that are currently undergoing clinical testing, and other innovative leads are presently being investigated in the preliminary stages of drug development. In the recent past, both private and public investors have shown interest in this market due to the potentially profitable possibility connected with these targets. In point of fact, more than 4.5 billion US dollars have been invested in this sector from the year 2020. We anticipate that the market will see rapid expansion in the foreseeable future as a result of the fact that a greater number of candidates are anticipated to advance to more advanced phases of development.

There are a number of rare kidney disorders that are not currently being addressed, which may result in major health problems in the future. It is believed that roughly 1 in 40,000 males and approximately 1 in 20,000 women in the U.S. are afflicted with the Fabry disease, as stated by the World Health Organization (WHO). According to the statistics provided by the National Organization for Rare Disorders, Inc., the incidence rate of the atypical hemolytic uremic syndrome in the general population of the country is two people for every one million.

The major companies in this market are focusing on the design and development of innovative treatments for rare kidney diseases. For instance, Reata Pharmaceuticals is currently testing a new medication for polycystic kidney disease called bardoxolone methyl in phase 3 of the clinical trial process. Migalastat Monotherapy is the only medicine developed by Amicus Therapeutics that is currently in the phase 3 testing phase for the treatment of Fabry disease.

Strong Product Pipeline Drives the Growth in the Enzyme Replacement Therapy Market

The enzyme replacement treatment sector accounted for a considerable part of the market in 2021. This may be broken down into medication classes. Products that provide enzyme replacement therapy are most commonly utilized for the treatment of Fabry disease. Galafold, Replagal, and Fabrazyme are some of the products that fall within this category.

It is anticipated that during the course of the forecast, the segment would be driven by an increase in the utilization of enzyme replacement therapy in the treatment of Fabry disease. The presence of a robust enzyme replacement product pipeline is yet another important element that is driving growth in the industry. The introduction of enzyme replacement therapy goods in territories that have not yet been exploited is a primary focus of many companies. This is anticipated to contribute to the segment's expansion in the not-too-distant future.

Expanded Treatment Options Increasing Awareness of the Fabry Disease Segment

The Fabry disease segment is expected to account for a significant portion of the market over the projection period. This is based on the disease's indication. The growth in the number of patients diagnosed with Fabry disease and the increased use of innovative treatments, such as chaperone therapy, are the primary drivers of this market sector. The segment is projected to be propelled by intensive research and development efforts, a robust product pipeline, and a rise in the utilization of substrate reduction therapies and enzyme replacement therapies.

Galafold, Replagal, and Fabrazyme are the medications that have been approved for the treatment of Fabry disease. It is anticipated that further products currently in phase 3 will be marketed in the very near future. In addition, there has been a recent uptick in the number of persons who have been diagnosed with Fabry disease, which is expected to drive up demand for novel treatment approaches.

Favorable Reimbursement Policies Fostering Growth in the Hospital Pharmacies Market Segment

It is anticipated that the hospital pharmacies segment would account for a considerable part of the market throughout the projection period in terms of the distribution channel. As a result of the more favorable payment policies, hospitals are increasingly being given preference for the treatment of uncommon urinary disorders. As a result, it is predicted that the segment would expand at a high pace throughout the course of the projection year. It is anticipated that over the next few years, the market will be propelled by an increase in knowledge of rare kidney disorders and novel treatment choices, as well as a trend toward the usage of new and improved pharmaceuticals by both patients and clinicians.

Market Insights

In 2021, the diagnostic segment was responsible for the largest percentage of the revenue share. The rise in the number of blood tests, urine tests, and other imaging procedures is mostly responsible for the expansion of the market. In the market for renal diseases, the diagnosis sector held the greatest share of the market in 2021 with a revenue share of 55%.  In 2021, a significant portion of the market was held by the hospital's segment, and it is predicted that this same segment would record the greatest CAGR in the market throughout the course of the forecast year. The hospital sector led the global renal diseases market with a revenue share of 48% in 2021.

North America Remains as the Global Leader, APAC Promises Significant Opportunities

According to projections made for the market for treatments for rare kidney diseases, North America held the greatest share in 2021, amounting to 45%. The rise in the incidence rate of Fabry disease, atypical hemolytic uremic syndrome, lupus nephritis, nephropathic cystinosis, and other kidney disorders, as well as the region's supremacy, are all possible explanations for this phenomenon. It is anticipated that favorable funding by governments and charitable trusts for healthcare research, along with an increase in the acceptance of technologically advanced products, a surge in healthcare expenditures, early access to advanced technologies, and early access to advanced technologies are going to accelerate market in North America during the forecast period. Because of factors such as the existence of large players and an increase in the number of new product approvals from the U.S. FDA, the market in the region was dominated by the United States. During the time covered by this prediction, Asia Pacific is predicted to be the region with the highest CAGR for the treatment of rare kidney diseases. It is expected that the area would experience considerable market expansion between the years 2023 and 2030 as a result of the vast patient population in countries such as India and China as well as the rise in awareness regarding rare urinary disorders.

Market Consolidation to Intensify Competition During the Forecast Period

The market for treatments of rare kidney disease is very competitive and concentrated, with only a handful of major companies dominating the industry. The majority of corporations are making large investments in research and development, with the primary objective being the creation of novel treatments for rare kidney disorders. Many successful businesses today have implemented growth strategies such as increasing the breadth of their product offerings and engaging in mergers and acquisitions. Product launches and approvals are key strategies that are often adopted by firms in order to expand their global footprints and product portfolios in order to meet the growing demand from consumers. The market participants in the renal disease industry have adopted the collaborative approach in order to increase their client base all over the world. The leading companies in the market for the treatment of rare kidney diseases are as follows: Calliditas Therapeutics AB, GSK plc., Aurinia Pharmaceuticals, Inc., Amicus Therapeutics, Inc., Shire (Takeda Pharmaceutical Company Limited), Sanofi, Advicenne, Alexion Pharmaceuticals, Inc., Horizon Therapeutics plc, Otsuka Pharmaceutical Co., Ltd., and Reata Pharmaceuticals, Inc.

Historical & Forecast Period

This study report represents analysis of each segment from 2022 to 2032 considering 2023 as the base year. Compounded Annual Growth Rate (CAGR) for each of the respective segments estimated for the forecast period of 2024 to 2032.

The current report comprises of quantitative market estimations for each micro market for every geographical region and qualitative market analysis such as micro and macro environment analysis, market trends, competitive intelligence, segment analysis, porters five force model, top winning strategies, top investment markets, emerging trends and technological analysis, case studies, strategic conclusions and recommendations and other key market insights.

Research Methodology

The complete research study was conducted in three phases, namely: secondary research, primary research, and expert panel review. key data point that enables the estimation of Rare Kidney Disease Treatment market are as follows:

• Research and development budgets of manufacturers and government spending
• Revenues of key companies in the market segment
• Number of end users and consumption volume, price and value.
• Geographical revenues generate by countries considered in the report
• Micro and macro environment factors that are currently influencing the Rare Kidney Disease Treatment market and their expected impact during the forecast period.

Market forecast was performed through proprietary software that analyzes various qualitative and quantitative factors. Growth rate and CAGR were estimated through intensive secondary and primary research. Data triangulation across various data points provides accuracy across various analyzed market segments in the report. Application of both top down and bottom-up approach for validation of market estimation assures logical, methodical and mathematical consistency of the quantitative data.

Key questions answered in this report

• What are the key micro and macro environmental factors that are impacting the growth of Rare Kidney Disease Treatment market?
• What are the key investment pockets with respect to product segments and geographies currently and during the forecast period?
• Estimated forecast and market projections up to 2032.
• Which segment accounts for the fastest CAGR during the forecast period?
• Which market segment holds a larger market share and why?
• Are low and middle-income economies investing in the Rare Kidney Disease Treatment market?
• Which is the largest regional market for Rare Kidney Disease Treatment market?
• What are the market trends and dynamics in emerging markets such as Asia Pacific, Latin America, and Middle East & Africa?
• Which are the key trends driving Rare Kidney Disease Treatment market growth?
• Who are the key competitors and what are their key strategies to enhance their market presence in the Rare Kidney Disease Treatment market worldwide?