Cystinosis Market to Witness Growth by 2032, Estimates DelveInsight | Companies- AVROBIO, Birch Therapeutics, Nacuity Pharmaceuticals, expected to boost the market

DelveInsight's "Cystinosis Market Insights, Epidemiology, and Market Forecast-2032" report delivers an in-depth understanding of 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 provides current treatment practices, emerging drugs, the market share of the individual therapies, and the current and forecasted Cystinosis market size from 2019 to 2032, segmented by seven major markets. The Report also covers current Cystinosis treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying potential of the Cystinosis market.

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Cystinosis: An Overview

Cystinosis is a rare, multisystem genetic disorder characterized by the accumulation of an amino acid called cystine in different tissues and organs of the body including the kidneys, eyes, muscles, liver, pancreas and brain. Generally, cystinosis is broken down into three different forms known as nephropathic cystinosis, intermediate cystinosis and non-nephropathic (or ocular) cystinosis. The age of onset, symptoms, and severity of cystinosis can vary greatly from one person to another. Nephropathic cystinosis presents in infancy and is the most common and severe form. Early detection and prompt treatment are critical in slowing the development and progression of symptoms associated with cystinosis.

The kidneys and eyes are the two organs most often affected. Individuals with nephropathic or intermediate cystinosis ultimately require a kidney transplant. Non-nephropathic cystinosis only affects the corneas of the eyes. Cystinosis is caused by mutations of the CTNS gene and is inherited as an autosomal recessive disease. At one time, nephropathic cystinosis was fatal at a very young age. However, the development of a medication known as cysteamine (which lowers the levels of cystine in the cells of the body) and improvements in kidney transplantation have transformed cystinosis from a fatal kidney disorder to a chronic, multisystem disorder with a life expectancy well into adulthood and even beyond 50 years of age.

The specific symptoms and severity of cystinosis vary greatly from one person to another based upon several factors including age of onset and whether the disorder is promptly diagnosed and treated. The progression of the disorder can be slowed by early diagnosis and treatment. Eventually, cystinosis can affect all tissues of the body. The age of onset for different symptoms varies greatly.

All types of cystinosis are caused by mutations of the CTNS gene. The disease is inherited in an autosomal recessive fashion. Recessive genetic disorders occur when an individual inherits a mutation in the same gene from each parent. If an individual receives one normal gene and one gene for the disease, the person will be a carrier for the disease, but usually will not show symptoms. Carriers for cystinosis (e.g., parents of affected individuals) never have symptoms of the disease. The risk for two carrier parents to both pass the defective gene and, therefore, have an affected child is 25 percent with each pregnancy.

The risk of having a child who is a carrier like the parents is 50 percent with each pregnancy. The chance for a child to receive normal genes from both parents and be genetically normal for that particular trait is 25 percent. The risk is the same for males and females. Cystinosis affects males and females in equal numbers. The disorder is estimated to occur in 1 in 100,000-200,000 people in the general population. Cystinosis has been reported worldwide, in all ethnic groups.

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Cystinosis Market

The Cystinosis market outlook of the report helps to build a detailed comprehension of the historical, current, and forecasted Cystinosis market trends by analyzing the impact of current Cystinosis therapies on the market and unmet needs, and drivers, barriers, and demand for better technology.

This segment gives a thorough detail of the Cystinosis market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated Cystinosis market data are presented with relevant tables and graphs to give a clear view of the market at first sight.

According to DelveInsight, the Cystinosis market in 7MM is expected to witness a major change in the study period 2019-2032.

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Cystinosis Epidemiology

The Cystinosis epidemiology section provides insights into the historical and current Cystinosis patient pool and forecasted trends for seven individual major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the Cystinosis market report also provides the diagnosed patient pool, trends, and assumptions.

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Cystinosis Drugs Uptake

This section focuses on the uptake rate of the potential Cystinosis drugs recently launched in the Cystinosis market or expected to be launched in 2019-2032. The analysis covers the Cystinosis market uptake by drugs, patient uptake by therapies, and sales of each drug.

Cystinosis Drugs Uptake helps in understanding the drugs with the most rapid uptake and the reasons behind the maximal use of new drugs and allows the comparison of the drugs based on Cystinosis market share and size, which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.

Cystinosis Pipeline Development Activities

The Cystinosis report provides insights into different therapeutic candidates in Phase II, and Phase III stages. It also analyses Cystinosis key players involved in developing targeted therapeutics.

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Cystinosis Emerging Drugs

AVR RD 04: AVROBIO

AVROBIO's investigational gene therapy for cystinosis is being studied in a Phase 1/2 investigational trial sponsored by the University of California, San Diego. The first patient was dosed in October 2019. The single-arm trial is expected to enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis.

NP-3: Nacuity Pharmaceuticals

Nacuity has developed an oral dosage form containing a small molecule antioxidant for the treatment of nephropathic cystinosis. In vitro POC, chronic oral toxicology studies and a Phase 1 clinical trial in healthy volunteers have been successfully completed. Clinical development program is planned. This program has been granted orphan and rare pediatric disease designations; thus, an eligible new drug application would result in Nacuity receiving seven years of U.S. FDA regulatory exclusivity and potentially a priority review voucher.

Cystinosis Therapeutics Assessment

Major key companies such as AVROBIO, Birch Therapeutics, Nacuity Pharmaceuticals, and others, are working proactively in the Cystinosis Therapeutics market to develop novel therapies which will drive the Cystinosis treatment markets in the upcoming years.

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Cystinosis Report Key Insights

1. Cystinosis Patient Population
2. Cystinosis Market Size and Trends
3. Key Cross Competition in the Cystinosis Market
4. Cystinosis Market Dynamics (Key Drivers and Barriers)
5. Cystinosis Market Opportunities
6. Cystinosis Therapeutic Approaches
7. Cystinosis Pipeline Analysis
8. Cystinosis Current Treatment Practices/Algorithm
9. Impact of Emerging Therapies on the Cystinosis Market

Table of Contents

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
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 Therapies
13. Cystinosis Seven Major Market Analysis
14. Attribute Analysis
15. Cystinosis Market Outlook (7 major markets)
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

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About DelveInsight

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