Fabry Disease Market Size in the 7MM was approximately USD 1,300 million in 2022, estimated DelveInsight

Fabry Disease Market

DELHI, DELHI, INDIA, June 25, 2024 /EINPresswire.com/ -- DelveInsight's “Fabry Disease Market Insights, Epidemiology and Market Forecast– 2032” report delivers an in-depth understanding of the Fabry Disease, historical and forecasted epidemiology as well as the Fabry Disease market trends in the United States, EU4 (Germany, Spain, Italy, and France) and the United Kingdom, and Japan.

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Key Takeaways from the Fabry Disease Market Report
• June 2024;- Sanofi- A Randomized, Double-blind, Placebo-controlled, 12-month Phase 3 Study to Evaluate the Effect of Venglustat on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease Who Are Treatment-naïve or Untreated for at Least 6 Months. This is a 12-month, parallel treatment, Phase 3, double-blind, randomized, placebo controlled study to evaluate the effect of venglustat on neuropathic and abdominal pain symptoms of Fabry disease in participants ≥16 years of age with Fabry disease who are treatment-naïve or untreated for at least 6 months.
• There were 15,290 diagnosed prevalent cases of Fabry Disease estimated to have occurred in the 7MM in 2022 of which 8,355 of the accounted cases were estimated to be from the US alone and these cases are anticipated to increase during the forecast period.
• The diagnosed prevalent cases of Fabry Disease were further divided into Phenotype-specific cases. The Phenotype-specific diagnosed prevalent cases of Fabry Disease are categorized into Classic and Late-onset with 6,939, and 8,351 cases respectively in the 7MM in 2022 which will further increase in 2032.
• The age-specific cases of Fabry Disease were categorized into less than 10 years, 10–19 years, 20–29 years, 30-39 years, 40-49 years, and 50+ years, with 343, 498, 204, 204, 229 and 212 cases in Japan in 2022.
• The leading Fabry Disease Companies such as Genzyme, Shire, Takeda, Protalix Biotherapeutics, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, Greenovation Biotech, GmBH, and others.
• Promising Fabry Disease Therapies such as AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, 4D-310, AMT-191, AL01211, Pegunigalsidase Alfa, and others.

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Fabry Disease Epidemiology Segmentation in the 7MM
• Fabry Disease Diagnosed Prevalent cases
• Fabry Disease Gender-specific Diagnosed Prevalent cases
• Fabry Disease Age-specific Diagnosed Prevalent Cases
• Fabry Disease Phenotype-specific Diagnosed Prevalent Cases

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Fabry Disease Marketed Drugs
• ELFABRIO (PRX-102): Chiesi and Protalix Biotherapeutics
PRX-102 (pegunigalsidase alfa), a PEGylated enzyme replacement therapy (ERT) to treat Fabry disease, is a plant cell culture-expressed, and chemically modified stabilized recombinant version of the a-Galactosidase-A enzyme. It is a PEGylated enzyme replacement therapy which means that it has been modified with a polymer called polyethylene glycol (PEG). These modifications make the drug more stable and help it to last longer in the body. Elfabrio works by replacing the missing alpha-galactosidase A enzyme, which is responsible for breaking down Gb3. The active substance in Elfabrio, pegunigalsidase alfa, is a copy of the human enzyme, produced by a method known as ‘recombinant DNA technology.

Fabry Disease Emerging Drugs
• Venglustat: Sanofi Genzyme
Venglustat (also known as ibiglustat) is an orally administered small molecule being developed by Sanofi Genzyme to treat Fabry disease. It is an inhibitor of an enzyme called glucosylceramide synthase (GCS) and modifies the enzyme substrates. When Venglustat inhibits GCS, it prevents the synthesis of GL-1, thereby reducing the substrate of the following reactions that lead to the formation of Gb3 and its accumulation in the absence of a-galactosidase A. Due to this mechanism of action Venglustat is called substrate reduction therapy (Sanofi, 2021). This molecule is currently in the Phase III stage of clinical development for Fabry disease. The company also expects to file a submission by 2025.

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Fabry Disease Market Outlook
Fabry disease treatment consists of enzyme replacement therapy (ERT), oral chaperone therapy, and adjunctive treatments including ACE inhibitors or angiotensin receptor blockers, antiplatelet drugs, and analgesics. Studies have shown that ERT can delay, but not always prevent, some of the clinical complications of FD. Currently, the effective management of Fabry disease requires a multidisciplinary approach with comprehensive therapy of intravenously administered ERT or chaperone therapy and adjunct therapies, including lifestyle modifications and prophylactic medications.

Fabry Disease Drug Market
ST-920 (isaralgagene civaparvovecis) is an innovative gene therapy being developed by Sangamo Therapeutics to treat Fabry disease. It utilizes a sophisticated approach involving an adeno-associated virus (AAV) vector carrying a specialized GLA gene construct. The GLA gene construct is driven by a proprietary liver-specific promoter developed by Sangamo. The primary objective of this gene therapy is to empower the patient’s liver to produce a consistent and prolonged supply of the a-Gal A enzyme. The company is investigating this gene therapy in Phase I/II STAAR clinical trials in patients with Fabry disease (Sangamo Therapeutics, 2021c).

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Scope of the Fabry Disease Market Report
• Coverage- 7MM
• Fabry Disease Companies- Genzyme, Shire, Takeda, Protalix Biotherapeutics, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, Greenovation Biotech, GmBH, and others.
• Fabry Disease Therapies- AGALSIDASE BETA (GZ419828), Acetaminophen, Diphenhydramine, 4D-310, AMT-191, AL01211, Pegunigalsidase Alfa, and others.
• Fabry Disease Market Dynamics: Fabry Disease Market Drivers and Barriers
• Fabry Disease Market Access and Reimbursement, Unmet Needs and Future Perspectives

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Table of Content
1. Key Insights
2. Fabry Disease Market Report Introduction
3. Fabry Disease Market Overview At A Glance
4. Fabry Disease Epidemiology And Market Forecast Methodology
5. Key Events
6. Executive Summary Of Fabry Disease
7. Fabry Disease Market Disease Background And Overview
8. Fabry Disease Epidemiology And Patient Population
9. Fabry Disease Patient Journey
10. Fabry Disease Marketed Drugs
11. Fabry Disease Emerging Drugs
12. Fabry Disease: Seven Major Market Analysis
13. Key Opinion Leaders’ Views
14. Fabry Disease Swot Analysis
15. Fabry Disease Unmet Needs
16. Fabry Disease Market Access And Reimbursement
17. Appendix
18. Delveinsight Capabilities
19. Disclaimer

About DelveInsight
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DelveInsight
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