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Nephropathic Cystinosis Clinical Trial Insight, Pipeline Assessment, Companies and Emerging Drugs by DelveInsight

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Nephropathic Cystinosis Clinical Trial Insight, Pipeline Assessment, Companies and Emerging Drugs by DelveInsight

August 20
18:33 2020
Nephropathic Cystinosis Clinical Trial Insight, Pipeline Assessment, Companies and Emerging Drugs by DelveInsight

“Nephropathic Cystinosis Pipeline Insight, 2020”


(Albany, NY) DelveInsight has launched a new Report on “Nephropathic Cystinosis Pipeline Insight, 2020”.


Nephropathic Cystinosis Pipeline Insight 2020” report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Nephropathic Cystinosis market. A detailed picture of the Nephropathic Cystinosis pipeline landscape is provided, which includes the disease overview and Nephropathic Cystinosis treatment guidelines.

The assessment part of the report embraces in-depth Nephropathic Cystinosis commercial assessment and clinical assessment of the Nephropathic Cystinosis pipeline products from the pre-clinical developmental phase to the marketed phase. In the report, a detailed description of the drug is proffered including mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology, Nephropathic Cystinosis collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.


Key Companies Covered:

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


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“Infantile (Nephropathic Cystinosis) is the most common form of the disease, accounting for 95 % of cases.”

Nephropathic Cystinosis treatment is directed toward the specific symptoms that are apparent in each individual and is best treated by a team of specialists, who have knowledge about the disease, and who can offer supportive and symptomatic care. Care is directed toward relieving the symptoms of the disease. Cystine Depleting Therapies are now available for the Nephropathic Cystinosis treatment.
The ultimate goal of the Nephropathic Cystinosis treatment is to reduce the cystine levels in the body. The treatment approach involves cystine depleting therapies, symptomatic medications, and kidney transplant in severe cases. There are two cystine depleting therapies which are approved by the FDA for the treatment of this disease including Cystagon (Mylan Pharmaceuticals) and Procysbi (Horizon Pharma USA, Inc.). Cystagon (Mylan Pharmaceuticals) is approved for Nephropathic Cystinosis treatment in adults and children. It reacts with cystine to form another amino acid, called cysteine, and a compound called a cysteine-cysteamine salt. The body is able to remove this salt from the cells. The amount of cystine in the organs is therefore reduced, and this limits the damage to these organs.

PROCYSBI (Horizon Pharma USA, Inc.) is the other candidate which is approved for Nephropathic Cystinosis treatment in adult and pediatric patients 2 years of age and older. It participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.

Both of the therapies have received orphan drug designation from the USFDA. Apart from these therapies, certain symptomatic treatment is also advised and consequently happens to occupy a considerable share in the overall treatment landscape. Infants and young children with cystinosis receive fluid and electrolytes, such as sodium and potassium. Vitamin D and phosphate salts are also given to deal with the problems of weaker bones. Diabetes is usually managed with a low-sugar diet, insulin, or other medicines that help control sugar in the blood. In severe cases where none of the therapies is effective, kidney transplant is recommended.


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Key benefits of the Report:

1. Nephropathic Cystinosis market report covers a descriptive overview and comprehensive insight of the Nephropathic Cystinosis epidemiology and Nephropathic Cystinosis market in the 7 MM (United States, EU5 (Germany, Spain, France, Italy, UK) & Japan.)

2. Nephropathic Cystinosis market report provides insights on the current and emerging therapies.

3. Nephropathic Cystinosis market report provides a global historical and forecasted market covering drug outreach in 7 MM.

4. Nephropathic Cystinosis market report offers an edge that will help in developing business strategies by understanding trends shaping and driving the Nephropathic Cystinosis market.


Key Questions

  • What are the current options for Nephropathic Cystinosis treatment?
  • How many companies are developing therapies for the treatment of Nephropathic Cystinosis
  • What are the principal therapies developed by these companies in the industry?
  • How many therapies are developed by each company for the treatment of Nephropathic Cystinosis?
  • How many Nephropathic Cystinosis emerging therapies are in early-stage, mid-stage, and late stage of development for the treatment of Nephropathic Cystinosis?
  • Out of total pipeline products, how many therapies are given as a monotherapy and in combination with other therapies? 
  • What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, and major licensing activities that will impact Nephropathic Cystinosis market? 
  • Which are the dormant and discontinued products and the reasons for the same?
  • What is the unmet need for current therapies for the treatment of Nephropathic Cystinosis?  
  • What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing Nephropathic Cystinosis therapies? 
  • What are the clinical studies going on for Nephropathic Cystinosis and their status?
  • What are the results of the clinical studies and their safety and efficacy? 
  • What are the key designations that have been granted for the emerging therapies for Nephropathic Cystinosis
  • How many patents are granted and pending for the emerging therapies for the treatment of Nephropathic Cystinosis


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Table of contents

1. Report Introduction

2. Nephropathic Cystinosis 

3. Nephropathic Cystinosis Current Treatment Patterns

3.1. Nephropathic Cystinosis Treatment Guidelines

4. Nephropathic Cystinosis – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Nephropathic Cystinosis Late Stage Products (Phase-III)

7. Nephropathic Cystinosis Mid Stage Products (Phase-II)

8. Early Stage Products (Phase-I)

9. Pre-clinical Products and Discovery Stage Products

10. Inactive Products

11. Dormant Products

12. Nephropathic Cystinosis Discontinued Products

13. Nephropathic Cystinosis Product Profiles

14. Nephropathic Cystinosis Key Companies

15. Nephropathic Cystinosis Key Products

16. Dormant and Discontinued Products

17. Nephropathic Cystinosis Unmet Needs

18. Nephropathic Cystinosis Future Perspectives

19. Nephropathic Cystinosis Analyst Review  

20. Appendix

21. Report Methodology


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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 end to end comprehensive solutions to improve their performance.

Media Contact
Company Name: DelveInsight Business Research LLP
Contact Person: Ankit Nigam
Email: Send Email
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City: Albany
State: New York
Country: United States