As Cystinuria increasingly impacts populations worldwide and is linked to comorbidities such as diabetes, cardiovascular disease, and certain cancers, there is a rising need for safer and more effective treatment options. DelveInsight reports that the Cystinuria pipeline includes over 5 pharmaceutical and biotech companies actively developing more than 5 therapeutic candidates for the condition. These candidates are at various stages of clinical and preclinical development, reflecting significant innovation and dedication to addressing this important health challenge.
DelveInsight’s “Cystinuria Pipeline Insight 2025” offers a comprehensive strategic analysis of the R&D landscape, including clinical trial progress, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report serves as a vital resource for researchers, healthcare investors, and decision-makers seeking insights into the evolving Cystinuria therapeutics market and the breakthroughs shaping its future.
Explore the Cutting-Edge Landscape of Cystinuria Drug Development
Key Takeaways from the Cystinuria Pipeline Report
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DelveInsight’s Cystinuria pipeline report highlights an active therapeutic landscape with over 5 companies developing more than 5 pipeline candidates for Cystinuria treatment.
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ADV7103 is currently being assessed in the Phase 2/3 CORAL-1 trial, which is enrolling Cystinuria patients across sites in France and Belgium, with primary study completion anticipated in May 2025. In May 2024, the FDA approved tiopronin delayed-release tablets for patients aged 9 years and older who do not respond to standard therapies.
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Cycle Pharmaceuticals, in collaboration with Torrent Pharma Inc., launched VENXXIVA™ (tiopronin) delayed-release tablets in the U.S. in March 2025. This formulation offers flexible dosing and is supported by patient assistance programs under Cycle Vita™. Additionally, in March 2024, ADV7103 received Orphan Drug Designation from the FDA, incentivizing the development of treatments for rare diseases by providing benefits such as market exclusivity upon approval.
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Key Cystinuria players including Revive Therapeutics, Otsuka Pharmaceutical, Eloxx Pharmaceuticals, Travere Therapeutics, Advicenne, and others, are actively exploring new therapies to enhance the Cystinuria treatment landscape. Promising pipeline candidates include Bucillamine and several others.
Cystinuria Overview:
Cystinuria is a hereditary metabolic disorder in which the kidneys excrete excessive amounts of cystine and related amino acids—lysine, arginine, and ornithine—into the urine. Since cystine has low solubility, it can form crystals or kidney stones, although not all individuals with the condition develop stones. The disorder results from mutations in the SLC3A1 or SLC7A9 genes, which impair normal amino acid transport in the kidneys. It is inherited in an autosomal recessive manner, requiring two defective gene copies for symptoms to manifest. Carriers typically remain asymptomatic but may have slightly elevated cystine levels in their urine.
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Cystinuria Pipeline Analysis
The Cystinuria pipeline insights report 2025, provides insights into:
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Provides comprehensive insights into key companies developing therapies in the Cystinuria Market.
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Categorizes Cystinuria therapeutic companies by development stage: early, mid, and late-stage.
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Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.
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Reviews emerging Cystinuria drugs under development based on:
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Stage of development
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Cystinuria Route of administration
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Target receptor
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Monotherapy vs. combination therapy
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Cystinuria Mechanism of action
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Molecular type
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Offers detailed analysis of:
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Company-to-company and company-academia collaborations
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Cystinuria Licensing agreements
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Funding and investment activities supporting future Cystinuria market advancement.
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Cystinuria Emerging Drugs
Bucillamine: Revive Therapeutics
Bucillamine, a cysteine-derived medication long used in Japan and South Korea for rheumatoid arthritis, is now being evaluated in Phase 2 trials for cystinuria. The drug features two thiol groups and has demonstrated greater efficacy than NAC in replenishing glutathione levels, highlighting its potential to protect against lung damage during infections such as influenza.
Cystinuria Pipeline Therapeutic Assessment
Cystinuria Assessment by Product Type
• Mono
• Combination
• Mono/Combination
Cystinuria By Stage
• Late-stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
Cystinuria Assessment by Route of Administration
• Oral
• Parenteral
• Intravenous
• Subcutaneous
• Topical
Cystinuria Assessment by Molecule Type
• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
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Table of Contents
1. Report Introduction
2. Executive Summary
3. Cystinuria Current Treatment Patterns
5. Cystinuria – DelveInsight’s Analytical Perspective
5. Therapeutic Assessment
5. Cystinuria Late-Stage Products (Phase-III)
7. Cystinuria Mid-Stage Products (Phase-II)
8. Early Stage Products (Phase-I)
9. Pre-clinical Products and Discovery Stage Products
10. Inactive Products
11. Dormant Products
12. Cystinuria Discontinued Products
13. Cystinuria Product Profiles
15. Cystinuria Key Companies
15. Cystinuria Key Products
15. Dormant and Discontinued Products
17. Cystinuria Unmet Needs
18. Cystinuria Future Perspectives
19. Cystinuria Analyst Review
20. Appendix
21. Report Methodology
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