Fabry disease – Market outlook, Epidemiology, Competitive Landscape, and Market Forecast Report – 2020 To 2030

 Fabry disease is a rare inherited disorder of glycosphingolipid (fat) metabolism resulting from the absent or markedly deficient activity of the lysosomal enzyme, α-galactosidase A (α-Gal A). This disorder belongs to a group of diseases known as lysosomal storage disorders. This enzymatic deficiency is caused by alterations (mutations) in the α-galactosidase A (GLA) gene that instructs cells to make the α-galactosidase A (α-Gal A) enzyme.

 

Etiology-

Fabry disease is caused by alterations (mutations) in the alpha-galactosidase A (GLA) gene located on the X-chromosome. This enzyme is active in lysosomes, which are structures that serve as recycling centers within cells. Alpha-galactosidase A normally breaks down a fatty substance called globotriaosylceramide. Mutations in the GLA gene alter the structure and function of the enzyme, preventing it from breaking down this substance effectively.

 

Epidemiology-

According to Thelansis disease modeling data, results of literature reviews, surveys, and registry analyses, the prevalence in white male populations has been linked to Fabry disease in a wide range, approximately 1:17,000 to 1:117,000. Classic Fabry disease mutations are seen in approximately 1:22,000 to 1:40,000 males, and atypical presentations are associated with about 1:1000 to 1:3000 males and 1:6000 to 1:40,000 females.

 

The competitive landscape of Fabry disease includes country-specific approved as well as pipeline therapies. Any asset/product-specific designation or review and Accelerated Approval are being tracked and supplemented with analyst commentary.

KOLs insights of Fabry disease across 8 MM market from the center of Excellence/ Public/ Private hospitals participated in the study. Insights around current treatment landscape, epidemiology, clinical characteristics, future treatment paradigm, and Unmet needs.

Fabry disease Market Forecast: Patient Based Forecast Model (MS. Excel Based Automated Dashboard) which Data Inputs with sourcing, Market Event, and Product Event, Country specific Forecast Model, Market uptake and patient share uptake, Attribute Analysis, Analog Analysis, Disease burden, and pricing scenario, Summary, and Insights.

Read more: Fabry disease – Market outlook, Epidemiology, Market Forecast, and Competitive Landscape Report – 2020 To 2030

S. No    Asset               Company                                 Stage

1          Moss-aGal        Greenovation Biotech GmbH     Phase 1

2          4D-310 4D Molecular Therapeutics        Phase 2

3          ST-920 Sangamo Therapeutics  Phase 2

4          RVX000222      Resverlogix Corp           Phase 2

5          AVR-RD-01       AvroBio Phase 2

6          PRX-102           Protalix Phase 2

7          FLT190 Freeline Therapeutics    Phase 2

8          Lucerastat        Idorsia Pharmaceuticals Ltd.      Phase 1

9          pegunigalsidase alfa      Protalix Phase 3

10        REPLAGAL       Shire    Phase 3

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