Fabry Disease Pipeline, Prevalent Population, Market Analysis and Drug Report

September 23 22:30 2020
Fabry Disease Pipeline, Prevalent Population, Market Analysis and Drug Report

(Albany, US) DelveInsight has launched a new report on Fabry Disease Pipeline


“Fabry Disease Pipeline Insight, 2020” report by DelveInsight outlays comprehensive insights of present clinical development scenario and growth prospects across the Fabry Disease market. A detailed picture of the Fabry Disease pipeline landscape is provided, which includes the disease overview and Fabry Disease treatment guidelines. The assessment part of the report embraces in-depth Fabry Disease commercial assessment and clinical assessment of the Fabry Disease 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, Fabry Disease collaborations, licensing, mergers and acquisition, funding, designations, and other product-related details.

Fabry disease (FD) is a devastating, progressive, inherited condition caused by lysosomal dysfunction linked to chromosome X and mutations in the GLA gene located in chromosome Xq22.1 which encodes the alpha-galactosidase A (AGA) enzyme. It is a multi-systemic and life-threatening condition which is one of the most common lysosomal storage diseases after the Gaucher Disease. It is characterized by specific neurological, cutaneous, renal, cardiovascular, cochleovestibular and cerebrovascular manifestations.


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Fabry Disease Pipeline

Several companies are working robustly on many new therapies, such as Lucerastat (Idorsia Pharmaceuticals), Pegunigalsidase alfa (Protalix Biotherapeutics), AVR-RD-01 (Avrobio), Venglustat (Sanofi Genzyme), ST-920 (Sangamo Therapeutics), 4D-310 (4D Molecular Therapeutics) , RVX000222 (Resverlogix Corp), and others. Mid-stage pipeline is crowded, with several potential therapies with imminent attention of big pharmaceutical companies for this market space.

Lucerastat (Idorsia Pharmaceuticals) is an orally bioavailable small-molecule and low molecular weight iminosugar which inhibits glucosylceramide synthase and has the potential to provide substrate reduction therapy for Fabry disease. Idorsia Pharmaceuticals is currently conducting pivotal Phase III study for the treatment of FD. The trial would recruit up to 108 patients at sites across the U.S., Australia, Canada, and Europe with study results to be expected in June 2021.

Besides, in phase I study, lucerastat was well-tolerated at all doses and did not cause any serious adverse events. Furthermore, Lucerastat has been granted orphan Drug designation in the US and in Europe for the treatment of Fabry disease. Pegunigalsidase alfa (Protalix Biotherapeutics) is an investigational, plant cell culture-expressed, and chemically modified stabilized version of the recombinant α-Galactosidase-A enzyme. Protein sub-units are covalently bound via chemical cross-linking using short PEG moieties, resulting in a molecule with unique pharmacokinetic parameters. In clinical studies, PRX–102 has been observed to have a circulatory half-life of approximately 80 hours. In addition, Phase I/II dose-ranging studies showed high correlation between two Fabry disease biomarkers, supporting potential effectiveness of pegunigalsidase alfa in treating Fabry disease.


Marketed drugs included in the report

  • Fabrazyme: Genzyme
  • Replagal: Shire/ Takeda
  • Galafold: Amicus Therapeutics


Emerging therapies included in the report

  • Lucerastat: Idorsia Pharmaceuticals
  • 4D-310: 4D Molecular Therapeutics
  • Pegunigalsidase alfa: Protalix Biotherapeutics
  • AVR-RD-01: Avrobio
  • Venglustat: Sanofi Genzyme
  • ST-920: Sangamo Therapeutics
  • FLT190: Freeline Therapeutics
  • RVX000222: Resverlogix Corp


Fabry Disease Market

As per DelveInsight assessments, the market size of Fabry disease (FD) in the 7MM was found to USD 833.74 Million in 2017, for the study period (2017-2030). Among 7MM, United States accounts for the highest market size of Fabry disease in comparison with EU5 and Japan. In EU5, the market size of FD in UK is the highest in 2017, while Spain had the lowest market size of Fabry disease. However, the market size of FD in Japan was found to lowest in 2017, considering all the 7MM.


Fabry Disease Report Scope

  • The Fabry Disease report provides an overview of therapeutic pipeline activity and therapeutic assessment of the products by development stage, product type, route of administration, molecule type, and MOA type for Fabry Disease across the complete product development cycle, including all clinical and nonclinical stages.
  • It comprises of detailed profiles of Fabry Disease therapeutic products with key coverage of developmental activities, including technology, collaborations, licensing, mergers and acquisition, funding, designations and other product-related details
  • Detailed Fabry Disease research and development progress and trial details, results wherever available, are also included in the pipeline study.
  • Coverage of dormant and discontinued pipeline projects along with the reasons if available across Fabry Disease.


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Table of content

1. Report Introduction

2. Fabry Disease 

3. Fabry Disease Current Treatment Patterns

4. Fabry Disease – DelveInsight’s Analytical Perspective

5. Therapeutic Assessment

6. Fabry Disease Late Stage Products (Phase-III)

7. Fabry Disease 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. Fabry Disease Discontinued Products

13. Fabry Disease Product Profiles

14. Fabry Disease Key Companies

15. Fabry Disease Key Products

16. Dormant and Discontinued Products

17. Fabry Disease Unmet Needs

18. Fabry Disease Future Perspectives

19. Fabry Disease Analyst Review  

20. Appendix

21. Report Methodology


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