Cystinuria Market Report and Forecast 2024-2032

Cystinuria Market Outlook

The cystinuria market size was valued at USD 907.19 million in 2023, driven by increasing investment initiatives for research and development activities. The market size is anticipated to grow at a CAGR of 14.8% during the forecast period of 2024-2032 to achieve a value of USD 3141.8 million by 2032.

Cystinuria Market Overview

Cystinuria, a rare kidney stone disease results in the formation of cystine stones in the kidneys, bladder, and ureters. This condition is inherited from both parents through a gene defect. Symptoms of cystinuria may include blood in the urine, severe pain on one side of the back or side, nausea, vomiting, and pain in the pelvis, groin, or abdomen. Cystinuria is typically asymptomatic when no stones are present; however, symptoms will recur each time stones form in the kidneys, which is common.

The cystinuria market demand is driven by the rising incidence of the genetic condition. The market is further experiencing growth driven by increased regulatory attention and greater awareness of available treatments for this condition. This growth is further supported by expanded healthcare infrastructure investments by the government and private organizations coupled with the rise in inherited disorder prevalence. The collaborations among key players and the growing disposable income of individuals are also collectively contributing to the cystinuria market growth.

However, the lack of knowledge among healthcare professionals and people owing to the rarity of the disease and limited reimbursement in developing economies is a major factor expected to restrain the market growth. However, this lack of knowledge may help the government to develop new programs and launch initiatives to spread awareness about cystinuria. Additionally, the excessive costs associated with research and development, coupled with the scarcity of treatment options, are again expected to hinder the market growth. Stringent drug approval regulations further impede the pace of market growth. Overcoming these challenges will be crucial for sustained and robust development in the cystinuria market to relieve the patients suffering from the condition.

Promising Developments

The market is poised for significant growth driven by some interesting and promising developments in treatments and substantial investments in research for cystinuria. For instance, in January 2024, a new treatment, Advicenne's potassium bicarbonate + potassium citrate in prolonged release (PR) form is progressing through Phase III clinical development, showing potential as a treatment for cystinuria. This advancement exhibits a significant step toward addressing the therapeutic needs of those affected by this rare kidney stone disease, contributing to the rising cystinuria market share. 

Increasing Research Grants 

The market is driven by significant contributions through research grants. Determined researchers and their dedicated efforts backed by grants are highlighting the importance of exploring innovative solutions for this condition. For instance, the generous support from the Kiriwina Investment Company, amounting to USD 120,000 over five years, has enabled the establishment of a groundbreaking mouse model of cystinuria. Dr Malcolm Starkey received two grants to support his group’s research on cystinuria. This model, created through advanced CRISPR/Cas9 gene editing, exhibits the most common mutation causing the disease, providing a potentially strong platform for developing targeted therapies and propelling the cystinuria market demand.

These advancements reflect a growing interest and commitment within the medical community and among investors to dig deeper into understanding cystinuria and innovating treatments. The introduction of novel therapies in clinical development and substantial research funding is expected to drive the cystinuria market forward. As these initiatives progress, they promise to improve patient outcomes and potentially transform the standard of care for individuals battling cystinuria.

Clinical Trials

In December 2023, a clinical trial with Ethical Committee approval was conducted by Viola D’Ambrosio to characterize bone mineral density (BMD) in cystinuria. The study included thirty-nine patients and 40% were women, adult cystinuric patients followed at 3 specialized outpatient clinics in Italy (Rome, Naples, and Verona). The successful completion of the trial showcased a high prevalence of low BMD in cystinuric patients, despite having normal (or moderately impaired) kidney function and being relatively young.

The insights from this clinical trial highlighted the crucial gap of unmet needs in cystinuria. Such results are expected to gain traction among pharmaceutical companies to recognize the impacts of cystinuria on BMD in women along with kidney association. This may result in additional research and development of drugs and treatments to address this issue and boost the cystinuria market during the forecast period.

Cystinuria Market Segmentations

Market Breakup by Disease Type

• Type I cystinuria

• Type II cystinuria

• Type III cystinuria

Market Breakup by Drug Type

• Tiopronin

• Penicillamine

• Others

Market Breakup by Dosage Form

• Solutions

• Tablet

• Injection

• Others

Market Breakup by Route of Administration

• Oral

• Intravenous

• Topical

• Others

Market Breakup by Distribution Channel

• Hospital Pharmacy

• Online Pharmacy

• Retail Pharmacy

Market Breakup by -8MM

• United States

• EU-4 and the United Kingdom 
  • Germany
  • France
  • Italy
  • Spain
  • United Kingdom

• Japan

• China

Cystinuria Market Regional Analysis

The United States is dominating the region market for cystinuria and is likely to lead the global market during the forecast period of 2024-2032. The region is dominating due to the increased prevalence of cystinuria and continued research and development activities to fill a critical gap of unmet medical needs in the market. The combined efforts of researchers and companies to create and establish an appropriate yet effective treatment for patients suffering from this rare disease are poised to foster cystinuria market growth in the forecast period. For instance, In February 2023, Ethicon, a Johnson & Johnson MedTech company, announced the first patient successfully receiving robotic-assisted removal of kidney stones using the MONARCH™ Platform for Urology.

As part of a clinical study, UCI Health, the clinical enterprise of the University of California, Irvine (UCI), used the MONARCH Platform for Urology to complete the first robotically assisted electromagnetic (EM)-guided percutaneous access and mini-percutaneous nephrolithotomy (PCNL) procedure. The clinical study involves a collaboration with co-investigator, Dr. Mihir Desai from the University of Southern California (USC).

Cystinuria Market: Competitor Landscape

The key features of the market report include patent analysis, grants analysis, clinical trials analysis, funding and investment analysis, partnerships, and collaborations analysis by the leading key players. The major companies in the market are as follows:

• Retrophin

• Recordati Rare Diseases

• Mission Therapeutics

• Enterome Bioscience

• Horizon Therapeutics

• Arcturus Therapeutics

• Codexis

• Mito pharmaceuticals

• Abbott

• Bausch Health Companies

• Allena Pharmaceuticals

• Mylan N.V. (now part of Viatris)

• Chiesi Farmaceutici S.p.A.

Please note that this only represents a partial list of companies, and the complete list has been provided in the report.

Key Highlights of the Report