Global Cystinosis Market Insights & Epidemiology, 2017-2028 - Analysis of Marketed & Emerging Drugs; Procysbi, Cystadrops, Cystaran, Cystagon, AVR-RD-04, ELX-02, and A0003 - ResearchAndMarkets.com

DUBLIN--()--The "Cystinosis - Market Insights, Epidemiology, and Market Forecast - 2028" drug pipelines has been added to ResearchAndMarkets.com's offering.

This report deliver an in-depth understanding of the disease, historical and forecasted epidemiology as well as the market trends of Cystinosis in the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.

There are three types of cystinosis, depending on when symptoms first appear (known as onset): Infantile (early-onset) cystinosis; later childhood or adolescent (late-onset) cystinosis; and adult cystinosis. About 95% of patients with cystinosis have the infantile/early-onset form, making it the most common variant of this disease, and most patients develop kidney problems. In many cases, the categorization is also done as, nephropathic cystinosis, intermediate cystinosis, and non-nephropathic (or ocular) cystinosis.

According to this research, it was observed that most of the Cystinosis cases are contributed by nephropathic cystinosis, and least is contributed by ocular type.

Cystinosis Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology (Total Prevalent Cases of Cystinosis in the 7MM, Total Diagnosed Prevalent Cases of Cystinosis in the 7MM, and Type-specific Diagnosed Prevalent Cases of Cystinosis in the 7MM) scenario of Cystinosis in the 7MM covering United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017-2028.

According to this research, the total prevalent population of Cystinosis in seven major markets is 2,956 in 2017. These cases are expected to increase with a significant CAGR during the study period (2017-2028).

The total diagnosed prevalent population of Cystinosis in seven major markets was 1,308 in 2017.

Cystinosis Drug Chapters

The mainstay of cystinosis treatment is the cystine-depleting aminothiol cysteamine or mercaptoethylamine. The two oral therapies (cysteamine bitartrate) that are approved in the US and Europe are, Procysbi and Cystagon. In 1994, Cystagon was introduced, and this is currently the most widely used preparation of cysteamine. Later in 1997, the drug got approval in Europe, and in 2014 got approved in Japan.

Procysbi (cysteamine bitartrate) is a delayed-release capsule indicated for the treatment of nephropathic cystinosis. The drug was discovered and developed by Raptor Pharmaceutical (Raptor). In the year 2013, the FDA and EMA approved Procysbi for the treatment of nephropathic cystinosis in adults and children.

Additionally, there are two therapies (Cystadrops and Cystaran) that are marketed for ocular type. Cystadrops is marketed in Europe and Cystaran is marketed in the US. Recently, in 2019, Recordati has filed NDA for Cystadrops in the US. This therapy already got approval in Europe in the year 2017, for the treatment of ocular cystinosis. With the expected launch of Cystadrops in the US, cystaran may face a tough competition.

Due to disease complexity, not many companies are working in this therapeutic area. The few companies that are focusing on this target are Avrobio, Eloxx Pharmaceuticals, and Mylan. AVR-RD-04 is a lentiviral-based gene therapy being developed by Avrobio for Cystinosis patients. Since it's a gene therapy - a one-time treatment - it is the potential to provide a permanent cure for the disease. Hence it will be a major factor for attracting the patient pool.

AVR-RD-04 will face a tough competition with ELX-02 and other approved therapies and generate a major market share. The present treatment option for cystinosis has cystine depleting therapies only, whereas, ELX-02 represents a different mechanism of action (Ribosomal protein modulators) and hence it is expected that it will give a tough competition to procysbi and cystagon.

Cystinosis Market Outlook

According to this research, the total market size of Cystinosis in seven major markets was USD 130.46 million in 2017. The market size of Cystinosis is expected to increase at a significant CAGR during the study period (2017-2028). Among all the seven major markets, the United States accounts for the highest market size of Cystinosis. In 2017, the market size of Cystinosis in the US was found to be USD 86.52 million.

Cystinosis Drugs Uptake

The current market size of Cystinosis is mainly attributed by procysbi and cystagon that are marketed for the treatment of nephropathic cystinosis; cystaran and cystadrops that is marketed for ocular cystinosis. There are several other off-labeled therapies as well that help in the management of cystinosis that includes the use of hormonal therapy and renal cell transplantation. The market size is expected to increase during the forecast period (2019-2028), due to potential therapies AVR-RD-04, ELX-02 and A0003.

Key Topics Covered

1. Key Insights

2. Cystinosis Market Overview at a Glance

2.1. Market Share (%) Distribution of Cystinosis in 2017

2.2. Market Share (%) Distribution of Cystinosis in 2028

3. Cystinosis: Disease Background and Overview

3.1. Introduction

3.2. Classification of Cystinosis

3.2.1. Nephropathic cystinosis

3.2.2. Intermediate cystinosis/Late-Onset/Juvenile cystinosis

3.2.3. Non-nephropathic or ocular cystinosis

3.3. Cause of Cystinosis

3.4. Pathophysiology of Cystinosis

3.4.1. Cellular events in cystinosis related to cystine accumulation

3.4.2. Cellular events in cystinosis likely unrelated to cystine accumulation

3.5. Clinical description and complications

3.6. Genetic Basis of Cystinosis

3.7. Functional Characteristics of CTNS Mutations: Genotype-Phenotype Correlations

3.8. Diagnostics and Biomarker Follow-up

3.8.1. Differential Diagnosis and Biomarker Follow-up

4. Epidemiology and Patient Population

4.1. Key Findings

4.2. 7MM Total Prevalent Patient Population of Cystinosis

4.3. 7MM Total Diagnosed Prevalent Patient Population of Cystinosis

5. Country Wise-Epidemiology of Cystinosis

5.1. United States Epidemiology

5.1.1. Assumptions and Rationale

5.1.2. Total Prevalent Cases of Cystinosis in the United States

5.1.3. Total Diagnosed Prevalent Cases of Cystinosis in the United States

5.1.4. Type-Specific Diagnosed Prevalence of Cystinosis in the United States

5.2. EU5 Epidemiology

5.3. Japan Epidemiology

6. Treatment and Management of Cystinosis

6.1. Symptomatic treatment

6.2. Cystine-depleting therapy

6.3. Therapeutic monitoring and Prognosis

6.4. Treatment Guidelines for Cystinosis

6.4.1. The European Reference Network for Rare Kidney Diseases

7. Unmet Needs

8. Marketed Drugs

8.1. Procysbi (cysteamine bitartrate): Horizon Pharma

8.1.1. Drug Description

8.1.2. Regulatory Milestones

8.1.3. Other Development Activities

8.1.4. Advantages and Disadvantages

8.1.5. Clinical Development

8.1.6. Clinical Trials Information

8.1.7. Safety and Efficacy

8.1.8. Product Profile

8.2. Cystadrops (cysteamine hydrochloride): Recordati S.p.A.

8.3. Cystaran (cysteamine hydrochloride): Leadiant Biosciences

8.4. Cystagon (cysteamine bitartrate): Mylan Pharmaceuticals

9. Emerging Drugs

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

9.1.1. Product Description

9.1.2. Other Developmental Activities

9.1.3. Clinical Development

9.1.4. Clinical trial information

9.1.5. Safety and Efficacy

9.1.6. Product Profile

9.2. ELX-02: Eloxx Pharmaceuticals

9.3. A0003: Mylan

10. Cystinosis: 7 Major Market Analysis

10.1.1. Key Findings

10.1.2. Market Size of Cystinosis in the 7MM

10.1.3. Market Size of Cystinosis by Therapies in the 7MM

11. Market Outlook: 7MM

11.1. United States Market Size

11.1.1. The total Market size of Cystinosis

11.1.2. Cystinosis Market by Current Treatment and Emerging Therapies

11.2. EU5

11.3. Japan

12. Market Drivers

13. Market Barriers

14. SWOT Analysis

15. KOL Insights

Companies Mentioned

  • Horizon Pharma/Raptor Pharmaceuticals
  • Mylan Pharmaceuticals/Recordati
  • Leadiant Biosciences
  • Recordati S.p.A.
  • Avrobio
  • Eloxx Pharmaceuticals
  • Mylan EPD

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Contacts

ResearchAndMarkets.com
Laura Wood, Senior Press Manager
press@researchandmarkets.com
For E.S.T Office Hours Call 1-917-300-0470
For U.S./CAN Toll Free Call 1-800-526-8630
For GMT Office Hours Call +353-1-416-8900