As per the National Fabry Disease Foundation, Fabry disease is a rare genetic disorder which is caused by the deficiency of defective gene, i.e., alpha-galactosidase-A gene (GLA gene) which leads to the deficiency or absent activity of enzyme alpha-galactosidase A. Absent or deficient activity of lysosomal exoglycohydrolase, alpha-galactosidase A result in progressive accumulation of globotriaosylceramide and related glycosphingolipids (galabiosylceramide) within lysosomes.
The primary disease process starts in infancy, or even as early as in the fetal stage of development. In FD, lysosomal storage and cellular dysfunction are believed to trigger a cascade of events including cellular death, compromised energy metabolism, small vessel injury, K(Ca) channel dysfunction in endothelial cells, oxidative stress, impaired autophagosome maturation, tissue ischemia and, importantly, development of irreversible cardiac and renal tissue fibrosis.
The Fabry Disease market report covers emerging drugs, current treatment practices, Fabry Disease market share of the individual therapies, current and forecasted Fabry Disease Market Size from 2017 to 2030 segmented by seven major markets.
The report also provides detailed current Fabry Disease treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assesses the underlying potential of the market.
Fabry Disease Market Key Facts
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As per DelveInsight estimates, the total prevalent population of Fabry disease (FD) in the 7MM was found to be 16,691 in 2017. Among all the countries, the estimates show a higher prevalent population of Fabry disease (FD) in the United States with 7,785 cases in 2017.
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Among the EU5 countries, the UK had the highest prevalent population of Fabry disease with 1,661 cases, followed by Italy which had the prevalent population of 1,551 cases in 2017. On the other hand, Spain had the lowest prevalent population.
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Females are more prone to FD than males in the United States. This trend is expected to increase during the study period and as per DelveInsight’s analyst’s assessments, in 2017, there were 3,270 cases of Fabry Disease among males and 4,515 cases among females.
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Key Benefits of Fabry Disease Market Report
Fabry Disease market report provides an in-depth analysis of Fabry Disease Market Size, Share, Trend, Epidemiology and Market Forecast till 2030, in 7 major market i.e. EU5 (Germany, Italy, Spain, France and the UK), Japan, and the United States.
The Fabry Disease market report will help in developing business strategies by understanding the Fabry Disease Market trends & developments, key players and future market competition that will shape and drive the Fabry Disease market in the upcoming years.
The Fabry Disease market report covers Fabry Disease current treatment practices, emerging drugs, market share of the individual therapies in 7 MM.
The report provides a detailed assessment of the Fabry Disease market in terms of market drivers & barriers, Unmet Needs, market opportunities, patient population, comparative analysis of pipeline products with detailed clinical profiles, and other factors.
The Fabry Disease market outlook section of the report helps to build the detailed comprehension of the historic, current and forecasted Fabry Disease market trends by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand for better technology.
The report gives a thorough detail of Fabry Disease market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on the annual cost of therapy, inclusion and exclusion criteria, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders.
The Fabry Disease epidemiology section covers insights about historical and current Fabry Disease patient pool and forecasted trends for every seven major countries (i.e. the United States, EU5 countries (Germany, Spain, Italy, France, and the United Kingdom), and Japan from 2017 to 2030.
It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. The epidemiology section also provides the diagnosed patient pool and their trends along with assumptions undertaken.
Fabry Disease Drugs Uptake and Key Market Players
Fabry Disease Drugs Uptake section focuses on the rate of uptake of the potential drugs recently launched in the Fabry Disease market or expected to get launched in the market during the study period. The analysis covers Fabry Disease market uptake by drugs; patient uptake by therapies; and sales of each drug.
Drugs Uptake section helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.
Some of the emerging therapies in the Fabry Disease market include Lucerastat, Pegunigalsidase alfa, AVR-RD-01, FLT190, Moss-aGal, RVX000222 and others.
Key players in the Fabry Disease market include Idorsia Pharmaceuticals, Protalix Biotherapeutics, Avrobio, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, GREENOVATION BIOTECH GMBH, Resverlogix Corp, and others.
Table of Contents
1. Key Insights
2. Executive Summary
3. Fabry Disease Competitive Intelligence Analysis
4. Fabry Disease Market Overview at a Glance
5. Fabry Disease Disease Background and Overview
6. Fabry Disease Patient Journey
7. Fabry Disease Epidemiology and Patient Population
8. Fabry Disease Treatment Algorithm, Current Treatment, and Medical Practices
9. Fabry Disease Unmet Needs
10. Key Endpoints of Fabry Disease Treatment
11. Fabry Disease Marketed Products
12. Fabry Disease Emerging Therapies
13. Fabry Disease Seven Major Market Analysis
14. Attribute Analysis
15. Fabry Disease Market Outlook (7 major markets)
16. Fabry Disease Access and Reimbursement Overview
17. KOL Views on the Fabry Disease Market.
18. Fabry Disease Market Drivers
19. Fabry Disease Market Barriers
20. Appendix
21. DelveInsight Capabilities
22. Disclaimer
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