Cystinosis Treatment Market 2034: Clinical Trials, EMA, PDMA, FDA Approvals, Medication, Revenue, Statistics, Therapies, Prevalence, Companies by DelveInsight

The Cystinosis market is expected to surge due to the disease's increasing prevalence and awareness during the forecast period. Furthermore, launching various multiple-stage Cystinosis pipeline products will significantly revolutionize the Cystinosis market dynamics.
(Albany, USA) DelveInsight's "Cystinosis Market Insights, Epidemiology, and Market Forecast-2034 report offers 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.

The Cystinosis market report covers emerging drugs, current treatment practices, market share of the individual therapies, and current & forecasted market size from 2020 to 2034. It also evaluates the current treatment practice/algorithm, market drivers & barriers, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.

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Some of the key facts of the Cystinosis Market Report:

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The Cystinosis market size is anticipated to grow with a significant CAGR during the study period (2020-2034)

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In 2023, the total number of prevalent Cystinosis cases in the United States was estimated to be around 1,300.

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In 2023, the total prevalent population of Cystinosis in the EU4 countries and the UK was estimated at approximately 1,600 cases, while Japan had around 55 prevalent cases during the same year.

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In 2023, the United States reported the highest number of diagnosed prevalent cases of cystinosis, with around 680 cases. Within the EU4 and the UK, the UK had the most cases, followed by Germany, France, and Spain, while Italy had the fewest.

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In the 7MM, there were an estimated 1,250 cases of infantile nephropathic cystinosis, 65 cases of juvenile nephropathic cystinosis, and 45 cases of adult-onset (ocular or non-nephropathic) cystinosis.

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Key Cystinosis Companies: Horizon Pharmaceutical/Chiesi Farmaceutici, Recordati S.p.A, Nacuity Pharmaceuticals, Inc., Nacuity Pharmaceuticals, Avrobio, and others

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Key Cystinosis Therapies: PROCYSBI (cysteamine bitartrate), CYSTADROPS (cysteamine hydrochloride), NPI-001, N-Acetylcysteine Amide, RP103, AVR-RD-04 (CTNS-RD-04), and others

Cystinosis Overview

Cystinosis is a rare genetic disorder characterized by the accumulation of cystine, an amino acid, within cells. This buildup occurs because the lysosomes, which are responsible for breaking down and recycling cellular materials, cannot properly transport cystine out of the cell. Over time, the excessive cystine forms crystals that can damage various organs and tissues, particularly the kidneys and eyes.

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Cystinosis Market

The dynamics of the Cystinosis market are anticipated to change in the coming years owing to the expected launch of emerging therapies and others during the forecasted period 2020-2034.

"There is only one upcoming therapy AVR-RD04 (Gene therapy) till date in the Cystinosis pipeline, to assess the long-term safety and durability of AVR-RD-04 treatment in participants who received a single dose administration of lentiviral gene therapy and will further decrease the market share of both the drugs."

Cystinosis Epidemiology

The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2020 to 2034. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides a detailed analysis of the diagnosed patient pool and future trends.

Cystinosis Epidemiology Segmentation:

The Cystinosis market report proffers epidemiological analysis for the study period 2020-2034 in the 7MM segmented into:

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Total Prevalence of Cystinosis

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Prevalent Cases of Cystinosis by severity

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Gender-specific Prevalence of Cystinosis

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Diagnosed Cases of Episodic and Chronic Cystinosis

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Cystinosis Drugs Uptake and Pipeline Development Activities

The drugs uptake section focuses on the rate of uptake of the potential drugs recently launched in the Cystinosis market or expected to get launched during the study period. The analysis covers Cystinosis market uptake by drugs, patient uptake by therapies, and sales of each drug.

Moreover, the therapeutics assessment section helps understand the drugs with the most rapid uptake and the reasons behind the maximal use of the drugs. Additionally, it compares the drugs based on market share.

The report also covers the Cystinosis Pipeline Development Activities. It provides valuable insights about different therapeutic candidates in various stages and the key companies involved in developing targeted therapeutics. It also analyzes recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.

Cystinosis Therapies and Key Companies

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PROCYSBI (cysteamine bitartrate): Horizon Pharmaceutical/Chiesi Farmaceutici

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

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NPI-001 : Nacuity Pharmaceuticals, Inc.

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N-Acetylcysteine Amide: Nacuity Pharmaceuticals

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RP103: Horizon Pharma USA

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AVR-RD-04 (CTNS-RD-04): Avrobio

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Cystinosis Market Drivers

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Advancement in technology

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Development of Biomarkers

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Gene therapy can improve the treatment

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Development of a new delivery system

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New symptomatic findings

Cystinosis Market Barriers

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Limited treatment option is available

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Lack of emerging pipeline

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Lack of disease understanding

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Challenges with the current therapies

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 Pharmaceutical/Chiesi Farmaceutici, Recordati S.p.A, Nacuity Pharmaceuticals, Inc., Nacuity Pharmaceuticals, Avrobio, and others

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Key Cystinosis Therapies: PROCYSBI (cysteamine bitartrate), CYSTADROPS (cysteamine hydrochloride), NPI-001, N-Acetylcysteine Amide, RP103, AVR-RD-04 (CTNS-RD-04), 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

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Table of Contents

1. Cystinosis Market Report Introduction

2. Executive Summary for Cystinosis

3. SWOT analysis of Cystinosis

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. Cystinosis Unmet Needs

11. Cystinosis Emerging Therapies

12. Cystinosis Market Outlook

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

14. Cystinosis Market Access and Reimbursement of Therapies

15. Cystinosis Market Drivers

16. Cystinosis Market Barriers

17. Cystinosis Appendix

18. Cystinosis Report Methodology

19. DelveInsight Capabilities

20. Disclaimer

21. About DelveInsight

About DelveInsight

DelveInsight is a leading Healthcare 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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