DelveInsight’s “Cystinosis Market Insights, Epidemiology, and Market Forecast 2032” report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Cystinosis market size, share, trends, and growth opportunities in the seven major markets (7MM) (i.e., the United States, EU4 (Germany, Spain, Italy, France), the United Kingdom and Japan).
The Cystinosis market report covers emerging drugs, current treatment practices, market share of individual therapies, and current & forecasted market size from 2019 to 2032. It also evaluates the current treatment practice/algorithm, key drivers & barriers impacting the market growth, and unmet medical needs to curate the best of the opportunities and assess the underlying potential of the market.
Cystinosis: An Overview
Cystinosis is a disease caused by an abnormal buildup of a particular amino acid called cystine. It is a rare but serious disease with a lifelong impact and can affect many parts of the body. There are three types of cystinosis, depending on when symptoms first appear (known as onset): infantile (early-onset) cystinosis, late childhood or adolescent (late-onset) cystinosis, and adult cystinosis.
Cystinosis is a recessive genetic disorder, and patients must receive an abnormal copy of the cystinosin gene from each parent to develop the disease. In people with cystinosis, a buildup of cystine can lead to the formation of crystals.
Cystinosis is caused by bi-allelic mutations in the CTNS gene (17p13.2) encoding cystinosin, a lysosomal cystine-proton co-transporter. Consequently, cystine accumulates in the lysosomes of affected cells and forms crystals in low lysosomal pH.
Cystinosis Market Key Facts
The total prevalent cases of Cystinosis in the United States were found to be approximately 1,330 in 2021. These prevalent cases are expected to increase during the study period (2022-2032).
The total diagnosed prevalent population of Cystinosis in the EU5 was observed to be approximately 659 in 2021. Whereas, in Japan, the diagnosed prevalent cases were estimated to be approximately 23 cases in 2021.
Among 7MM, the United States has the highest diagnosed prevalent cases of cystinosis, with approximately 665 cases in 2021. In EU-5, the UK has the highest number of cases, i.e., 224, followed by Germany, with 182 cases. While Italy has the least number of cases, with 43 in 2021.
In 2021, there were approximately 609 cases of Infantile nephropathic cystinosis, 33 cases of Juvenile nephropathic cystinosis, and 23 cases of Adult-onset (Ocular or non-nephropathic cystinosis) in the US.
Japan had approximately 23 diagnosed prevalent cases of cystinosis in 2021. These cases are expected to decrease during the study period. Out of the total diagnosed cases, 21 cases account for infantile nephropathic cystinosis, 1 case of Juvenile nephropathic cystinosis, and 1 case of Adult-onset in 2021.
Cystinosis Market
The market outlook section of the report helps to build a detailed comprehension of the historical, current and forecasted Cystinosis market size by analyzing the impact of current and emerging pipeline therapies. It also thoroughly assesses the market drivers & barriers, unmet needs, and emerging technologies set to impact the market dynamics.
The report gives complete detail of the Cystinosis market trend for each marketed drug and mid & late-stage pipeline therapies by evaluating their impact based on the annual cost of therapy, their Mechanism of Action (MOA), Route of Administration (ROA), molecule types, competition with other therapies, brand value, and their impact on the market.
Cystinosis Epidemiology Assessment
The epidemiology section provides insights into the historical, current, and forecasted epidemiology trends in the seven major countries (7MM) from 2019 to 2032. It helps to recognize the causes of current and forecasted epidemiology trends by exploring numerous studies and research. The epidemiology section also provides a detailed analysis of diagnosed and prevalent patient pool, future trends, and views of key opinion leaders.
The Report Covers the Cystinosis Epidemiology, Segmented as –
Total Prevalent cases of Cystinosis in the 7MM [2019–2032]
Diagnosed Prevalent cases of Cystinosis in the 7MM [2019–2032]
Type-specific Diagnosed Prevalent cases of Cystinosis in the 7MM [2019–2032]
Cystinosis Drugs Uptake and Pipeline Development Activities
The drug uptake section focuses on the uptake rate of potential drugs recently launched in the Cystinosis market or expected to be launched during the study period. The analysis covers the 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 analyses recent developments such as collaborations, acquisitions, mergers, licensing patent details, and other information for emerging therapies.
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Cystinosis Therapeutics Analysis
Several major pharma and biotech companies are developing therapies for Cystinosis. Currently, AVROBIO is leading the therapeutics market with its Cystinosis drug candidates in the most advanced stage of clinical development.
The Leading Players in the Cystinosis Therapeutics Market Include:
AVROBIO
Birch Therapeutics
Chiesi Farmaceutici
Eloxx Pharmaceuticals
Horizon Pharma
Leadiant Biosciences
Mylan Pharmaceuticals
Nacuity Pharmaceuticals
Recordati S.p.A.
And Many Others
Cystinosis Emerging and Marketed Drugs Covered in the Report Include:
A0003: Mylan
AVR-RD-04 (CTNS-RD-04): Avrobio
Cystagon (cysteamine bitartrate): Mylan Pharmaceuticals
ELX-02: Eloxx Pharmaceuticals
NP-3: Nacuity Pharmaceuticals
Procysbi (cysteamine bitartrate): Horizon Pharma/Chiesi Farmaceutici
The Report Covers the In-depth Assessment of the Emerging Drugs & Key Companies. Download the Sample Report to Learn More @
Table of Content (TOC)
1. Key Insights
2. Executive Summary
3. Cystinosis Competitive Intelligence Analysis
4. Cystinosis Market Overview at a Glance
5. Cystinosis Disease Background and Overview
6. Cystinosis Patient Journey
7. Cystinosis Epidemiology and Patient Population (In the US, EU5, and Japan)
8. Cystinosis Treatment Algorithm, Current Treatment, and Medical Practices
9. Cystinosis Unmet Needs
10. Key Endpoints of Cystinosis Treatment
11. Cystinosis Marketed Products
12. Cystinosis Emerging Drugs and Latest Therapeutic Advances
13. Cystinosis Seven Major Market Analysis
14. Attribute Analysis
15. Cystinosis Market Outlook (In US, EU5, and Japan)
16. Cystinosis Access and Reimbursement Overview
17. KOL Views on the Cystinosis Market
18. Cystinosis Market Drivers
19. Cystinosis Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
*The Table of Contents (TOC) is not exhaustive; the final content may vary. Refer to the sample report for the complete table of contents.
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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.
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