With changing lifestyles and bad eating habits, healthcare systems witness an increase in rare kidney diseases that are difficult to treat. It is worth noting that 60-80 per 100,000 cases / individuals in the United States and Europe suffer from rare kidney disorders, including Focal Segmental Glomerular Sclerosis, C3 Glomerulopathy, Lupus Nephritis, IgA Nephropathy and Membranous Nephropathy. These diseases are primarily due to genetic abnormalities that create difficulties in diagnosing and treating patients. Though the rate of disease is relatively lower in comparison to other chronic disorders, lack of awareness and late diagnosis may result in severe consequences. The above-mentioned factors have become the key reason for the high mortality rate due to rare kidney disorders.
Further, the complexity associated with the treatment options available for kidney disorders, such as kidney transplant, dialysis, and biopsy, has given the attention of key players in the domain to develop advanced therapeutic drugs that can treat these rare kidney disorders. Given the growing efforts of rare kidney diseases market players, since 2020, more than seven drugs have been approved by the FDA for the treatment of patients namely 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). Despite the lucrative opportunities rising in this industry, rare kidney disease market players are fraught with several challenges, which are listed below.
Rare Kidney Diseases Market: Competitive Landscape
Presently, the rare kidney disease market landscape features several industrial and non-industrial players that hold the required expertise to develop novel drugs for treating kidney diseases. It is worth noting here that over 90 candidates that are targeting rare kidney diseases are currently being evaluated under different clinical stages of development. Several drug targets are being evaluated in the early clinical trial stages of development. Driven by the rising opportunities to develop advanced therapeutic drugs to treat rare kidney diseases, the market has catered to the attention of various private and public stakeholders within the past few years. Notably, since 2020, more than USD 4.5 billion in capital investment has been made in the rare kidney disease market. As the pipeline of drug candidates progresses towards the development stages, it is expected that the field will witness a trajectory in the upcoming years.
Challenges Need to Address in the Rare Kidney Diseases Market
Despite the growing drug candidates pipeline that remarks growth of the market, several challenges hinder progress in the upcoming years. One of the promising challenges is limited awareness among individuals and healthcare professionals about kidney diseases and the severe consequences of late diagnoses. Further, the lack of particular symptoms related to diseases also contributed to the difficulty in the identification and diagnosis of rare kidney diseases. Other examples of challenges associated with rare kidney disease market are listed below:
▪ Limited Pool of Patients
There is a limited pool of patients suffering from rare kidney diseases, which makes it challenging for healthcare experts and pharmaceutical companies to diagnose, research, and develop advanced therapeutic solutions. Additionally, the limited availability of patient populations represents several formidable challenges, such as financial viability for research and limited market size. The restrictions may result in high treatment costs for the patients and affect the research process. To overcome these challenges, pharmaceutical companies are required to make concerted efforts to support the research for novel therapeutic drugs to treat rare kidney disorders.
▪ Regulatory Challenges
The limited data available about rare kidney disorders and small patient pools may create challenges for market leaders in meeting regulatory guidelines. Moreover, obtaining approval from regulatory authorities for new treatment is a complex and lengthy process—the process requires accurate documentation of clinical research to ensure safety and efficacy. The absence of standard treatment guidelines for rare kidney diseases further makes it challenging for healthcare providers to determine the effective treatment that can address the individual requirements of patients.
▪ High Initial Investment
Research on new therapeutic drugs to treat patients with rare kidney diseases requires a high initial investment. The high initial investment due to limited market size can create challenges specifically for the small industrial players. In order to mitigate the high initial investment concern, rare kidney disease market players have to make collaborative efforts to accelerate the research on finding advanced therapeutic drugs.
Recent Developments in the Rare Kidney Diseases Market
It is worth noting here that the rare kidney disease market is witnessing several development and collaboration activities that are driving growth in this industry. Further, advances in imaging technologies, including biomarker identification, contributed to the accurate and early diagnosis of rare kidney diseases. Examples of the key developments that have taken place in this field are listed below:
⬝ In June 2023, Novartis announced the acquisition of Chinook Therapeutics by making a considerable investment of USD 3.5 billion with the aim of boosting late-stage drug development that is lined up for the treatment of rare kidney diseases.
⬝ In May 2023, Chinook Therapeutics and Ionis Pharmaceuticals entered into a collaboration agreement to discover, develop, and market the antisense oligonucleotide therapy used for patients suffering from chronic kidney diseases.
⬝ In February 2023, Travere Therapeutics announced the approval of Filspari drug by the FDA to treat IgA nephropathy, a form of autoimmune disease that attacks the kidneys and leads to organ failure.
Concluding Remarks
Overall, the rare kidney disease market witnessed slow growth due to a lack of standardization and regulatory guidelines. The right advocacy about rare kidney diseases can encourage the development of treatment for these types of diseases. Moreover, several government bodies are offering incentives such as funding support and orphan drug designation to support the development of treatments for rare kidney diseases. As the drug pipeline turns towards successful approval of drugs to treat rare kidney diseases, it will be possible to mitigate the risk of mortality. Driven by the ongoing awareness and rising interest of the key players in the field, the rare kidney diseases market is likely to grow at a progressive rate in the upcoming years.
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