Cystinosis Market: Epidemiology, Therapies, Companies, DelveInsight | Horizon Pharma, Recordati S.p.A., Chiesi Farmaceutici, Leadiant Biosciences, Mylan Pharmaceuticals, Avrobio, Eloxx Pharmaceuticals

The Cystinosis Treatment Market is poised to change with increased competition among pharmaceutical companies, potentially leading to improved accessibility, affordability, and innovation in treatment options. Additionally, advancements in gene therapy and precision medicine may reshape treatment paradigms.

Emerging therapies for Cystinosis, including NPI-001 and others, are anticipated to drive growth in the Cystinosis market in the coming years.

DelveInsight has released a new report titled "Cystinosis - Market Insights, Epidemiology, and Market Forecast-2034", providing a comprehensive analysis of the condition, including historical and projected epidemiology as well as market trends across the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Discover about the Cystinosis market report @ https://www.delveinsight.com/report-store/cystinosis-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Some of the key facts of the Cystinosis Market Report:

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The Cystinosis market is expected to evolve as competition among pharmaceutical companies intensifies, potentially improving treatment accessibility, affordability, and innovation. Advances in gene therapy and precision medicine may also transform current treatment approaches.

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Epidemiology for Cystinosis is projected to increase due to enhanced diagnostic capabilities, which promote greater awareness and earlier detection. Additionally, therapies that extend patient survival could contribute to a growing patient population over time.

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Standard treatment primarily involves cysteamine bitartrate therapy, available in immediate- or delayed-release formulations, to reduce cystine accumulation. Approved therapies such as PROCYSBI and CYSTADROPS help prevent cystine buildup and associated complications.

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Nephropathic (infantile) cystinosis, the most common form, is usually diagnosed in infancy and results from CTNS gene mutations, which impair cystine transport in lysosomes. Without timely treatment, nephropathic cystinosis can progress to renal failure, emphasizing the importance of early intervention.

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Emerging treatments, including gene therapies and other targeted approaches, aim to address the underlying genetic cause of the disease, offering the potential for curative outcomes or improved long-term disease management.

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In 2023, the estimated prevalence of Cystinosis was about 1,300 cases in the United States, 1,600 cases across the EU4 and the UK, and around 55 cases in Japan.

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Significant regulatory milestones include the FDA approval of Casgevy Trademark (exagamglogene autotemcel) on December 8, 2023, as the first CRISPR-based gene-editing therapy in the U.S. for sickle cell disease, and CYSTADROPS, approved on August 25, 2020, as the first eye drop formulation for treating corneal cystine crystal deposits in cystinosis patients.

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Key companies developing new therapies for Cystinosis include Horizon Pharma, Recordati S.p.A., Chiesi Farmaceutici, Leadiant Biosciences, Mylan Pharmaceuticals, Avrobio, Eloxx Pharmaceuticals, and others. Promising treatments in development include NPI-001, PROCYSBI (cysteamine bitartrate), CYSTADROPS (cysteamine hydrochloride), among others.

Cystinosis Overview

Cystinosis is a rare genetic disorder characterized by the accumulation of the amino acid cystine within cells throughout the body. While it primarily affects the kidneys and eyes, other organs and tissues can also be involved. The condition is classified into three main forms: nephropathic (infantile), intermediate, and non-nephropathic (ocular). It arises from mutations in the CTNS gene, which encodes a protein responsible for transporting cystine out of cells. When this protein is nonfunctional, cystine builds up in lysosomes, forming crystals that damage cells and tissues. Although cystinosis is typically inherited in an autosomal recessive manner, some cases occur sporadically without a family history. Common signs include poor growth, dehydration, electrolyte imbalances, and frequent urination. Early diagnosis and treatment are crucial to prevent complications and improve quality of life.

Nephropathic cystinosis, the most severe form, usually manifests in infancy and can lead to kidney dysfunction or failure if untreated. Intermediate cystinosis tends to appear in childhood or adolescence, causing milder kidney impairment. Non-nephropathic cystinosis primarily affects the eyes, leading to photophobia, corneal cystine crystals, and progressive vision loss.

Cystinosis Market Outlook

The primary goal of cystinosis treatment is to reduce cystine accumulation in cells, preventing tissue damage and slowing disease progression. Keeping cystine levels low is essential for managing symptoms, preserving kidney function, and improving the overall quality of life for patients.

The standard therapy for cystinosis involves cysteamine-based medications that deplete cystine. Approved treatments include PROCYSBI (delayed-release cysteamine bitartrate) and CYSTAGON (immediate-release cysteamine bitartrate), which help break down cystine and limit its harmful effects on tissues and organs. Additionally, CYSTADROPS (cysteamine hydrochloride), developed by Recordati S.p.A., targets corneal cystine crystals through localized eye drop therapy, addressing ocular symptoms directly.

A promising emerging approach is CRISPR/Cas9 gene editing, being developed by multiple companies. This therapy aims to correct CTNS gene mutations, restoring the function of the cystinosin protein responsible for cystine transport. By addressing the underlying genetic cause, CRISPR/Cas9 has the potential to provide a curative treatment, enabling normal cystine transport and reducing cellular accumulation. Although still in early development, this gene-editing approach offers significant promise for transforming cystinosis management, potentially improving long-term outcomes and offering a lifelong cure.

Discover how the Cystinosis market is rising in the coming years @ https://www.delveinsight.com/sample-request/cystinosis-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Cystinosis Marketed Drugs

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PROCYSBI (cysteamine bitartrate) : Horizon Pharmaceutical/Chiesi Farmaceutici

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CYSTADROPS (cysteamine hydrochloride) : Recordati S.p.A

Cystinosis Emerging Drugs

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NPI-001 : Nacuity Pharmaceuticals, Inc.

Scope of the Cystinosis Market Report

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Study Period: 2020-2034

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Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]

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Key Cystinosis Companies: Horizon Pharma, Recordati S.p.A., Chiesi Farmaceutici, Leadiant Biosciences, Mylan Pharmaceuticals, Avrobio, Eloxx Pharmaceuticals, Mylan, and others

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Key Cystinosis Therapies: Dazukibart (PF-06823859), and others

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Cystinosis Therapeutic Assessment: Cystinosis current marketed and Cystinosis emerging therapies

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Cystinosis Market Dynamics: Cystinosis market drivers and Cystinosis market barriers

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Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter's five forces, BCG Matrix, Market entry strategies

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Cystinosis Unmet Needs, KOL's views, Analyst's views, Cystinosis Market Access and Reimbursement

To know what's more in our Cystinosis report, visit https://www.delveinsight.com/report-store/cystinosis-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr

Key benefits of the Cystinosis Market Report:

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Cystinosis market report covers a descriptive overview and comprehensive insight of the Cystinosis Epidemiology and Cystinosis market in the 7MM (the United States, EU5 (Germany, Spain, France, Italy, UK) & Japan).

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The Cystinosis market report provides insights into the current and emerging therapies.

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The Cystinosis market report provides a global historical and forecasted market covering drug outreach in 7MM.

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The Cystinosis market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Cystinosis market.

Got queries? Click here to know more about the Cystinosis market Landscape [https://www.delveinsight.com/sample-request/cystinosis-market?utm_source=abnewswire&utm_medium=market&utm_campaign=kpr]

Table of Contents

1. Report Introduction

2. Executive Summary

3. SWOT analysis

4. Cystinosis Patient Share (%) Overview at a Glance

5. Cystinosis Market Overview at a Glance

6. Cystinosis Disease Background and Overview

7. Cystinosis Epidemiology and Patient Population

8. Country-Specific Patient Population of Cystinosis

9. Cystinosis Current Treatment and Medical Practices

10. Unmet Needs

11. Cystinosis Emerging Therapies

12. Cystinosis Market Outlook

13. Country-Wise Cystinosis Market Analysis (2020-2034)

14. Market Access and Reimbursement of Therapies

15. Market drivers

16. Market barriers

17. Appendix

18. Cystinosis Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

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Related Reports:

Cystinosis Pipeline Insights, DelveInsight

"Cystinosis Pipeline Insight, 2024" report by DelveInsight outlines comprehensive insights of present clinical development scenarios and growth prospects across the Cystinosis market. A detailed picture of the Cystinosis pipeline landscape is provided, which includes the disease overview and Cystinosis treatment guidelines.

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.

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