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

Cystinosis emerging therapies, such as NPI-001, and others, are expected to boost the Cystinosis Market in the upcoming years.

DelveInsight has launched a new report on "Cystinosis - Market Insights, Epidemiology, and Market Forecast-2034" that delivers an in-depth understanding of the Cystinosis, historical and forecasted epidemiology as well as the Cystinosis market trends in the United States, EU5 (Germany, Spain, Italy, France, and 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 disorder marked by the buildup of the amino acid cystine in cells throughout the body. It mainly impacts the kidneys and eyes, though other organs and tissues can also be affected. There are three primary forms of cystinosis: nephropathic (or infantile), intermediate, and non-nephropathic (or ocular). This condition results from mutations in the CTNS gene, which encodes a protein responsible for transporting cystine out of cells. Without functional CTNS protein, cystine accumulates in lysosomes, forming crystals that damage cells and tissues. Although cystinosis follows an autosomal recessive inheritance pattern, some cases may arise sporadically without a family history. Common symptoms include poor growth, dehydration, electrolyte imbalances, and frequent urination. Early detection and treatment are vital for preventing complications and enhancing the quality of life for those affected by cystinosis.

Nephropathic cystinosis, the most severe type, typically appears in infancy and can lead to kidney dysfunction and failure if not treated. Intermediate cystinosis often presents in childhood or adolescence and is associated with milder kidney impairment compared to the nephropathic form. Non-nephropathic cystinosis mainly affects the eyes, causing symptoms like photophobia, corneal crystals, and progressive vision loss.

Cystinosis Market Outlook

The main treatment objective for cystinosis is to reduce the buildup of cystine within cells, preventing tissue damage and slowing the progression of the disease. Maintaining low cystine levels is crucial for managing symptoms, preserving kidney function, and enhancing the overall quality of life for cystinosis patients.

The current standard treatment for cystinosis involves cystine-depleting medications such as cysteamine bitartrate. Two approved drugs for this are PROCYSBI (delayed-release cysteamine bitartrate) and CYSTAGON (immediate-release cysteamine bitartrate), which work by breaking down cystine and minimizing its harmful effects on tissues and organs. Additionally, CYSTADROPS (cysteamine hydrochloride), developed by Recordati S.p.A., provides targeted treatment for corneal cystine crystals, addressing ocular symptoms through localized therapy in the form of eye drops.

An emerging therapy for cystinosis is CRISPR/Cas9 gene editing, currently being developed by several companies. This innovative approach aims to correct mutations in the CTNS gene, restoring the function of the cystinosin protein responsible for transporting cystine. By targeting the genetic cause of cystinosis, CRISPR/Cas9 gene editing holds the potential for a curative treatment. This therapy involves introducing CRISPR components into target cells to precisely edit the CTNS gene, allowing for normal cystine transport and reducing its accumulation. Although still in the early stages, CRISPR/Cas9 gene editing offers promising prospects for transforming cystinosis treatment, potentially leading to better outcomes and even a lifelong cure.

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.