Cystinosis Market Overview: Epidemiology, Treatment Landscape, and Key Players - Insights by DelveInsight | Featuring Horizon Pharma, Recordati S.p.A., Chiesi Farmaceutici, Leadiant Biosciences, Mylan Pharmaceuticals, AVROBIO, Eloxx Pharmaceuticals

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

DelveInsight has released a new report titled "Cystinosis - Market Insights, Epidemiology, and Market Forecast - 2034," offering a comprehensive analysis of Cystinosis. The report covers historical and projected epidemiological trends, as well as market dynamics across the United States, the 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=openpr&utm_medium=pressrelease&utm_campaign=kpr

Some of the key facts of the Cystinosis Market Report:

The dynamics of the Cystinosis market are set to evolve with heightened competition among pharmaceutical companies, which could lead to better accessibility, affordability, and innovation in treatment options. Additionally, advancements in gene therapy and precision medicine have the potential to redefine treatment approaches.
Cystinosis epidemiology is anticipated to rise due to improved diagnostic techniques, fostering greater awareness and earlier detection. Moreover, advancements in treatments that extend patient survival could result in a larger patient population over time.
The standard treatment for Cystinosis involves cysteamine bitartrate therapy, which reduces cystine levels through immediate or delayed-release formulations. PROCYSBI and CYSTADROPS are approved treatments that help prevent cystine buildup.
Nephropathic or infantile cystinosis, the most common form, is typically diagnosed during infancy. It is caused by mutations in the CTNS gene, impairing cystine transport in lysosomes. Without proper treatment, nephropathic cystinosis can progress to renal failure, underscoring the importance of early diagnosis and intervention.
Emerging therapies for Cystinosis, including gene therapy and novel targeted treatments, offer hope for patients. These therapies aim to target the root genetic cause of the disease, providing potential curative options or more effective long-term management solutions.
In 2023, the total number of Cystinosis prevalent cases in the United States was estimated to be around 1,300. In the EU4 countries and the UK, the prevalent population was approximately 1,600. Meanwhile, in Japan, the number of prevalent cases was estimated to be around 55 in 2023.
On December 8, 2023, the FDA approved CasgevyTM (exagamglogene autotemcel), the first CRISPR-based gene-editing therapy in the U.S., for treating sickle cell disease.
Approved on August 25, 2020, CYSTADROPS is the first FDA-approved eye drop formulation for treating corneal cystine crystal deposits in individuals with cystinosis.
Key Cystinosis companies such as Horizon Pharma, Recordati S.p.A., Chiesi Farmaceutici, Leadiant Biosciences, Mylan Pharmaceuticals, Avrobio, Eloxx Pharmaceuticals, Mylan, and others are evaluating new drugs for Cystinosis to improve the treatment landscape.
Promising Cystinosis therapies include NPI-001, PROCYSBI (cysteamine bitartrate), CYSTADROPS (cysteamine hydrochloride), and others.

Cystinosis Overview

Cystinosis is a rare genetic metabolic disorder characterized by the accumulation of the amino acid cystine within cells, leading to cellular and tissue damage. While the condition primarily affects the kidneys and eyes, it can also impact other organs and systems throughout the body. There are three main forms of cystinosis: nephropathic (or infantile), which is the most severe and presents in early infancy; intermediate cystinosis, with a later onset and milder symptoms; and non-nephropathic (ocular) cystinosis, which primarily affects the eyes and generally spares the kidneys.

The disorder is caused by mutations in the **CTNS** gene, which encodes a protein responsible for transporting cystine out of lysosomes. Without a functional CTNS protein, cystine accumulates and crystallizes within lysosomes, causing progressive damage to affected cells and tissues. Cystinosis follows an autosomal recessive inheritance pattern, meaning both parents must carry a copy of the mutated gene. However, in rare cases, the disease may appear sporadically without a known family history.

Common symptoms of cystinosis include poor growth, dehydration, frequent urination, and electrolyte imbalances, especially in infants and young children. If left untreated, the condition can lead to severe complications, including kidney failure. Early diagnosis and intervention are essential for slowing disease progression, managing symptoms, and improving the quality of life for individuals with cystinosis.

Cystinosis Market Outlook

The primary goal of cystinosis treatment is to reduce cystine accumulation within cells to prevent tissue damage and slow disease progression. Maintaining low cystine levels is essential for symptom management, kidney preservation, and overall quality of life. Standard therapy involves cystine-depleting drugs such as cysteamine bitartrate. PROCYSBI (delayed-release) and CYSTAGON (immediate-release) are two approved treatments that help break down cystine and reduce its toxic effects. For ocular symptoms, CYSTADROPS-an eye drop formulation-targets corneal cystine crystals directly. In addition to existing therapies, CRISPR/Cas9 gene editing is emerging as a potential breakthrough. This cutting-edge approach aims to correct the underlying CTNS gene mutation, potentially restoring normal cystine transport and offering a curative solution. Though still in development, gene editing represents a promising advancement in the long-term treatment of cystinosis.

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

Cystinosis Marketed Drugs
PROCYSBI (cysteamine bitartrate) : Horizon Pharmaceutical/Chiesi Farmaceutici
CYSTADROPS (cysteamine hydrochloride) : Recordati S.p.A

Cystinosis Emerging Drugs
NPI-001 : Nacuity Pharmaceuticals, Inc.

Scope of the Cystinosis Market Report
Study Period: 2020-2034
Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
Key Cystinosis Companies: Horizon Pharma, Recordati S.p.A., Chiesi Farmaceutici, Leadiant Biosciences, Mylan Pharmaceuticals, Avrobio, Eloxx Pharmaceuticals, Mylan, and others
Key Cystinosis Therapies: Dazukibart (PF-06823859), 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

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

Key benefits of the Cystinosis Market Report:
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).
The Cystinosis market report provides insights into the current and emerging therapies.
The Cystinosis market report provides a global historical and forecasted market covering drug outreach in 7MM.
The Cystinosis market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Cystinosis market.

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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.

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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.