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DelveInsight’s “Cystinosis Market Insights, Epidemiology, and Market Forecast – 2034” report delivers an in-depth understanding of the Cystinosis, historical and forecasted epidemiology and the Cystinosis market trends in the United States, EU4 (Germany, France, Italy, and Spain) and the United Kingdom, and Japan.

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Key Takeaways from the Cystinosis Market Report

• In 2024, DelveInsight estimated approximately 1,700 prevalent cases of cystinosis in the US, accounting for a substantial share of the total cases across the 7MM. Despite its rarity, this concentration highlights the significant clinical burden of cystinosis and its long-term, multisystem impact on affected patients.
• Across EU4 and the UK, infantile nephropathic cystinosis accounts for most type-specific cases, comprising 94% of the total in 2024, supported by early identification of Fanconi syndrome and timely diagnosis within structured pediatric nephrology referral systems.
• In 2024, DelveInsight estimated that the EU4 and the UK together accounted for a notable share of diagnosed prevalent cystinosis cases, with the UK representing the largest national burden at approximately 250 cases. In contrast, Spain reported the lowest number within this region, with approximately 65 diagnosed individuals, highlighting marked variation across European markets.
• In 2024, DelveInsight estimated that total prevalent cases of cystinosis in Japan exceeded 100 cases, representing a comparatively smaller yet clinically significant patient population within the 7MM and underscoring the persistent burden of this rare, lifelong multisystem disorder.
• The leading Cystinosis Companies such as Amgen, Chiesi Farmaceutici, Recordati Rare Diseases, Viatris, Leadiant Biosciences, Thiogenesis Therapeutics, Novartis and others.
• Promising Cystinosis Therapies such as TTI-0102, DFT383 and others.

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Cystinosis Epidemiology Segmentation in the 7MM

• Total Prevalent Cases of Cystinosis
• Total Diagnosed Prevalent Cases of Cystinosis
• Type-specific Diagnosed Prevalent Cases of Cystinosis

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Cystinosis Marketed Drugs

• PROCYSBI (cysteamine bitartrate): Amgen/Chiesi Farmaceutici

PROCYSBI is a delayed-release oral cysteamine formulation approved for nephropathic cystinosis in pediatric and adult patients aged 1 year and older. It provides sustained cysteamine exposure for long-term control of intracellular cystine levels, with weight-based dosing titrated to white blood cell cystine concentrations. PROCYSBI is available as delayed-release capsules (25 mg, 75 mg) and oral granules (75 mg, 300 mg) and is approved for use in the US and Europe.

• CYSTADROPS (cysteamine hydrochloride): Recordati Rare Diseases/Viatris

CYSTADROPS, developed by Recordati Rare Diseases, is a viscous gel eye drop containing cysteamine hydrochloride that reduces corneal cystine crystal accumulation by converting cystine into more soluble compounds. Its prolonged ocular residence enables four-times-daily dosing, improving convenience and patient adherence.

• CYSTAGON/NICYSTAGON (cysteamine bitartrate): Viatris

CYSTAGON is an oral capsule originally developed by Mylan Pharmaceuticals, now Viatris, containing cysteamine bitartrate equivalent to 50 mg or 150 mg of cysteamine; in Japan, it is marketed as NICYSTAGON and is indicated for the treatment of nephropathic cystinosis in pediatric and adult patients.

Cystinosis Emerging Drugs

• DFT383: Novartis

DFT383 is an investigational gene therapy for nephropathic cystinosis designed to deliver a functional CTNS gene and address lysosomal cystine accumulation. Also known as AVR-RD-04 or CTNS-RD-04, the program was acquired from AVROBIO and is being evaluated in pediatric patients in a Phase I/II setting, following earlier adult studies. References by Papillon Therapeutics reflect shared scientific lineage and platform validation, not development ownership.

• TTI-0102: Thiogenesis Therapeutics

TTI-0102, developed by Thiogenesis Therapeutics, is an oral cysteamine prodrug designed to overcome limitations of traditional thiol therapies. Leveraging the 505(b)(2) pathway, it builds on existing cysteamine safety data, has shown good tolerability at high doses, preserves cystine depletion and antioxidant activity, and qualifies as a New Chemical Entity with five years of exclusivity. The program is advancing toward a pivotal Phase III trial, with IND submission expected in the second half of 2026.

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Cystinosis Treatment Market

The cystinosis treatment landscape is largely defined by long-standing cysteamine-based therapies rather than next-generation, curative, or fully disease-modifying interventions. Systemic control relies on PROCYSBI and CYSTAGON/NICYSTAGON (cysteamine bitartrate), while ocular involvement is addressed with CYSTADROPS and CYSTARAN (cysteamine hydrochloride). Although these agents reduce cystine accumulation and have improved survival, their clinical impact is constrained by high dosing frequency, tolerability issues such as gastrointestinal effects and odor, formulation limitations, and adherence challenges, often resulting in incomplete and variable long-term disease control. This dependence on burdensome, decades-old therapies highlights persistent gaps in efficacy, convenience, and quality-of-life outcomes for patients with this lifelong multisystem disorder.

Cystinosis Companies

Amgen, Chiesi Farmaceutici, Recordati Rare Diseases, Viatris, Leadiant Biosciences, Thiogenesis Therapeutics, Novartis, and others.

Cystinosis Market Outlook

The Cystinosis market is entering a period of focused evolution as improved survival and deeper understanding of disease biology shift attention from childhood renal preservation to lifelong multisystem burden. While cysteamine-based therapies have transformed early outcomes, current care remains centered on chronic cystine depletion rather than correction of the underlying CTNS defect, leaving extrarenal complications to increasingly dominate adult disease. Real-world use of systemic and ocular cysteamine continues to be limited by high treatment burden, tolerability issues, and long-term adherence challenges, resulting in variable disease control over time. As the adult cystinosis population grows, gaps in durable efficacy, convenience, and quality-of-life outcomes are becoming more clinically visible and strategically important. Looking ahead, cystinosis is moving toward a more biology-driven phase, with innovation increasingly focused on reducing lifelong treatment burden and achieving broader, more durable multisystem control. These shifts position the disease at a clear inflection point, where emerging science has the potential to meaningfully redefine long-term care and patient outcomes.

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Scope of the Cystinosis Market Report

• Coverage- 7MM
• Study Period- 2020-2034
• Cystinosis Companies- Amgen, Chiesi Farmaceutici, Recordati Rare Diseases, Viatris, Leadiant Biosciences, Thiogenesis Therapeutics, Novartis and others.
• Cystinosis Therapies- TTI-0102, DFT383 and others.
• Cystinosis Therapeutic Assessment: Cystinosis Current marketed and Cystinosis Emerging Therapies
• Cystinosis Market Dynamics: Cystinosis market drivers and Cystinosis market barriers
• Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
• Cystinosis Unmet Needs, KOL’s views, Analyst’s views, Cystinosis Market Access and Reimbursement

Table of Contents

1 Key Insights

2 Report Introduction

3 PNH Market Overview at a Glance

4 Epidemiology and Market Forecast Methodology

5 Executive Summary of PNH

6 Key Events

7 Disease Background and Overview

8 Treatment and Management

9 Epidemiology and Patient Population

10 Patient Journey

11 Marketed Therapies

12 Emerging Therapies

13 PNH: 7 Major Market Analysis

14 KOL Views

15 SWOT Analysis

16 Unmet Needs

17 Reimbursement Scenario in PNH

18 Appendix

19 DelveInsight Capabilities

20 Disclaimer

About Us

DelveInsight is a leading healthcare-focused market research and consulting firm that provides clients with high-quality market intelligence and analysis to support informed business decisions. With a team of experienced industry experts and a deep understanding of the life sciences and healthcare sectors, we offer customized research solutions and insights to clients across the globe. Connect with us to get high-quality, accurate, and real-time intelligence to stay ahead of the growth curve.

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