Cystinuria Pipeline 2025: Groundbreaking Clinical Advancements by 5+ Global Leaders - DelveInsight | Featuring Revive Therapeutics, Otsuka Pharmaceutical, Eloxx Pharmaceuticals, Travere Therapeutics, Advicenne

With Cystinuria reaching epidemic proportions globally and contributing significantly to comorbid conditions such as diabetes, cardiovascular disease, and certain cancers, there is a growing demand for safer, more effective treatment options. According to DelveInsight, the Cystinuria pipeline comprises 5+ pharmaceutical and biotech companies actively developing 5+ therapeutic candidates targeting Cystinuria. These therapies span various stages of clinical and non-clinical development, underscoring the intense innovation and commitment to addressing one of the most pressing public health challenges of our time.
DelveInsight's "Cystinuria Pipeline Insight 2025" report provides a detailed and strategic evaluation of the ongoing R&D landscape. It covers clinical trial progression, emerging therapies, mechanisms of action, competitive positioning, and key company initiatives. The report serves as a crucial resource for stakeholders-including researchers, healthcare investors, and decision-makers, seeking insights into the evolving Cystinuria Therapeutics Market and the breakthroughs shaping its future trajectory.

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Key Takeaways from the Cystinuria Pipeline Report

DelveInsight's Cystinuria pipeline report depicts a robust space with 5+ active players working to develop 5+ pipeline therapies for Cystinuria treatment.
ADV7103 is currently undergoing evaluation in the Phase 2/3 CORAL-1 study, which is enrolling patients with cystinuria across centers in France and Belgium. The primary completion of these studies is expected in May 2025.
In May 2024, the FDA approved tiopronin delayed-release tablets for the treatment of cystinuria in patients aged 9 years and older who are not responsive to standard measures.
Cycle Pharmaceuticals, in partnership with Torrent Pharma Inc., announced the U.S. launch of VENXXIVATM (tiopronin) delayed-release tablets in March 2025. This formulation offers dosing flexibility and is supported by patient assistance programs through Cycle VitaTM.
In March 2024, the FDA granted Orphan Drug Designation to ADV7103 for the treatment of cystinuria. This designation is intended to encourage the development of treatments for rare diseases by providing benefits such as market exclusivity upon approval.
Key Cystinuria companies such as Revive Therapeutics, Otsuka Pharmaceutical, Eloxx Pharmaceuticals, Travere Therapeutics, Advicenne, and others are evaluating new drugs for Cystinuria to improve the treatment landscape.
Promising Cystinuria pipeline therapies in various stages of development include Bucillamine, and others.

Cystinuria Overview:

Cystinuria is a genetic metabolic disorder where the kidneys excrete too much cystine and similar amino acids-lysine, arginine, and ornithine-into the urine. Because cystine doesn't dissolve well, it can form crystals or stones in the kidneys, bladder, or urinary tract, though not all affected individuals develop stones. The condition is caused by mutations in the SLC3A1 or SLC7A9 genes, which disrupt normal amino acid transport in the kidneys. It follows an autosomal recessive inheritance pattern, meaning a person must inherit two faulty copies of the gene to show symptoms. Carriers usually do not have symptoms but may have mildly elevated cystine levels in their urine.

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Cystinuria Pipeline Analysis
The Cystinuria pipeline insights report 2025, provides insights into:
Provides comprehensive insights into key companies developing therapies in the Cystinuria Market.

Categorizes Cystinuria therapeutic companies by development stage: early, mid, and late-stage.

Highlights major companies involved in targeted therapy development, including both active and inactive (paused/discontinued) projects.

Reviews emerging Cystinuria drugs under development based on:

Stage of development

Cystinuria Route of administration

Target receptor

Monotherapy vs. combination therapy

Cystinuria Mechanism of action

Molecular type

Offers detailed analysis of:

Company-to-company and company-academia collaborations

Cystinuria Licensing agreements

Funding and investment activities supporting future Cystinuria market advancement.

Unlock key insights into emerging Cystinuria therapies and market strategies here: https://www.delveinsight.com/report-store/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

Cystinuria Emerging Drugs

Bucillamine: Revive Therapeutics

Bucillamine, a cysteine-based drug used for decades in Japan and South Korea to treat rheumatoid arthritis, is currently in Phase 2 trials for cystinuria. It contains two thiol groups and is significantly more effective than NAC at restoring glutathione levels, giving it strong potential to prevent lung damage during infections like 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

Download sample pages to get an in-depth assessment of the emerging Cystinuria therapies and key Cystinuria companies: https://www.delveinsight.com/sample-request/cystinuria-pipeline-insight?utm_source=openpr&utm_medium=pressrelease&utm_campaign=kpr

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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Contact Info:
Kritika Rehani
Assistant Manager, Marketing & Branding
krehani@delveinsight.com
info@delveinsight.com
+15599557579

About DelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing comprehensive end-to-end solutions to improve their performance.

This release was published on openPR.