(Albany, USA) DelveInsight’s Fabry Disease Market Insights report includes a comprehensive understanding of current treatment practices, Fabry disease emerging drugs, market share of individual therapies, and current and forecasted market size from 2019 to 2032, segmented into 7MM [the United States, the EU5 (the United Kingdom, Italy, Spain, France, and Germany), Japan].
Some facts from the Fabry Disease Market Report
- As per DelveInsight analysis, the Fabry disease market size in the 7MM was approximately USD 1,200 million in 2021.
- As per the estimates, the total Fabry disease prevalent cases in the 7MM were approximately 13K in 2021.
- Globally, leading Fabry disease companies such as Protalix Biotherapeutics, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, GREENOVATION BIOTECH GMBH, Shire, Takeda, Amicus Therapeutics, Protalix, uniQure, Codexis, MP6 Therapeutics, CellGenTech, and others are developing novel Fabry disease drugs that can be available in the Fabry disease market in the coming years.
- The promising Fabry disease therapies in the pipeline include PRX-102 (Pegunigalsidase Alfa), Venglustat, ST-920, FLT190, 4D-310, Lucerastat, Moss-aGal, and others.
- The increasing prevalence of the disease, along with the promising emerging pipeline therapies with new mechanisms of action, will fuel the Fabry disease market during the forecast period.
Discover which therapies are expected to grab the major Fabry disease market share @ Fabry Disease Market Forecast
Fabry Disease Overview
Fabry disease is an inherited lysosomal storage disorder caused by a nonfunctional or partially functional enzyme called alpha-galactosidase A (-Gal A). Reduced -Gal A activity in lysosomes leads to the accumulation of enzyme substrates (Gb3 and lyso-Gb3), which causes cellular damage in tissues throughout the body.
Despite being X-linked, heterozygous women may experience all of the signs and symptoms seen in men; however, when compared to hemizygous males, the signs and symptoms of Fabry disease in women typically appear later and with less severity.
Fabry disease symptoms include pain that spreads throughout the body (called a Fabry crisis), gastrointestinal complications, headaches, impaired sweating, vertigo, and hearing loss. Several tests, such as enzyme assay and newborn screening, are used for Fabry disease diagnosis.
Fabry Disease Epidemiology Segmentation
DelveInsight estimates that there were approximately 13K prevalent cases of Fabry disease in the 7MM in 2021.
Among the EU5 countries, Germany had the highest number of Fabry disease prevalent cases in 2021.
The Fabry disease market report proffers epidemiological analysis for the study period 2019–2032 in the 7MM segmented into:
- Prevalent Cases of Fabry Disease
- Gender-specific Cases of Fabry Disease
- Age-specific Cases of Fabry Disease
- Phenotype-specific cases of Fabry Disease
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Fabry Disease Treatment Market
Fabry disease treatment includes enzyme replacement therapy (ERT), oral chaperone therapy, and adjunctive treatment such as ACE inhibitors or angiotensin receptor blockers, antiplatelet drugs, and analgesics. Studies have shown ERT to delay, but not always prevent, some clinical complications of FD. Currently, effective Fabry disease management necessitates a multidisciplinary approach that includes intravenous ERT or chaperone therapy, as well as adjunct therapies such as lifestyle modifications and prophylactic medications.
ERT, which must be administered intravenously, has improved patient outcomes. However, intravenous administration of the enzyme can be difficult due to the need for repeated calculations. Lifelong, bimonthly infusions may result in poor venous access, possibly necessitating subcutaneous ports, which may carry the risk of bacterial infection and complicate future hemodialysis access. Furthermore, administering recurrent IV infusions in the pediatric population can be difficult. Infusion reactions, which can be fatal, are typically mediated by anti-drug antibody (ADA) responses and neutralization; these have been shown to inhibit the ERT and can correlate with poor outcomes. Although ERT is generally well tolerated, there is still room for improvement; chaperone therapy has been implemented to address these limitations.
Currently, there are only two therapies approved for Fabry disease treatment in the US market: Galafold (Amicus Therapeutics) and Fabrazyme (Sanofi-Genzyme). In the European and Japanese Fabry disease treatment market, Replagal (Takeda/Shire), Fabrazyme (Sanofi-Genzyme), and Galafold (Amicus Therapeutics) are currently the only approved therapies for Fabry disease patients.
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Fabry Disease Pipeline Therapies and Key Companies
- PRX-102 (Pegunigalsidase Alfa): Protalix Biotherapeutics
- Venglustat: Sanofi Genzyme
- ST-920: Sangamo Therapeutics
- FLT190: Freeline Therapeutics
- 4D-310: 4D Molecular Therapeutics
- Lucerastat: Idorsia Pharmaceuticals
- Moss-aGal: GREENOVATION BIOTECH GMBH
Learn more about the FDA-approved drugs for Fabry disease treatment @ https://www.delveinsight.com/sample-request/fabry-disease-market
Fabry Disease Market Dynamics
To counter the current unmet needs of the Fabry disease market and to provide better treatment options, several companies are working aggressively to develop new therapies with a novel mechanism of action. In addition, many therapies are being studied in various stages of clinical trials. Pegylated forms of –GAL, gene therapy, and substrate reduction therapy are examples of these.
Moreover, the development of a potential biomarker for tracking and predicting disease severity and potential progression, as well as the efficacy of any therapeutic intervention, contributes to the growth of the Fabry disease market.
Furthermore, combining new oral therapy approaches can alleviate the relative lack of enzyme replacement therapy efficacy, which is due in part to antibody formation. Moreover, due to patients’ poor quality of life with Fabry disease, there is significant patient willingness toward expensive therapies in the Fabry disease market.
Additionally, in Fabry disease, considerable progress has been made in terms of understanding disease pathology, diagnosis, and treatment effects. In several countries, newborn screening for Fabry disease has been implemented, allowing for the early detection of individuals with Fabry mutations.
However, certain factors are impacting the growth of the Fabry disease market. Enzyme and chaperone therapies are costly and invasive, requiring biweekly infusions, thereby slowing the Fabry disease market growth.
Moreover, ERT has not demonstrated dramatic improvements in all organ systems, with benefits primarily seen in kidney disease and reduced left ventricular hypertrophy. Only 35-50% of Fabry disease patients with amenable GLA mutations are candidates for migalastat therapy. Due to difficulties with standardized disease activity/therapy monitoring, patients develop neutralizing antibodies to ERT, contributing to reduced therapy efficacy.
Furthermore, gene therapy is expensive; additionally, the healthcare system in most countries, including the United States, is not designed to handle large one-time payments. Compared to small molecules, gene therapies require more time to approve. Moreover, approval guidelines for gene therapies are not as well defined as for small molecules or monoclonal antibodies and thus hindering the Fabry disease market.
Scope of the Fabry Disease Market Report
- Study Period: 2019–2032
- Coverage: 7MM [The United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan]
- Market Size in 2021: USD 1,200 Million
- Key Fabry Disease Companies: Protalix Biotherapeutics, Sanofi Genzyme, Sangamo Therapeutics, Freeline Therapeutics, 4D Molecular Therapeutics, Idorsia Pharmaceuticals, GREENOVATION BIOTECH GMBH, Shire, Takeda, Amicus Therapeutics, Protalix, uniQure, Codexis, MP6 Therapeutics, CellGenTech, and others
- Key Fabry Disease Therapies: PRX-102 (Pegunigalsidase Alfa), Venglustat, ST-920, FLT190, 4D-310, Lucerastat, Moss-aGal, and others
- Therapeutic Assessment: Fabry Disease current marketed and emerging therapies
- Fabry Disease Market Dynamics: Fabry Disease market drivers and barriers
- Competitive Intelligence Analysis: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies
- Unmet Needs, KOL’s views, Analyst’s views, Fabry Disease Market Access and Reimbursement
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Table of Contents
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1. |
Fabry Disease Market Key Insights |
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2. |
Fabry Disease Market Report Introduction |
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3. |
Fabry Disease Market Overview at a Glance |
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4. |
Fabry Disease Market Executive Summary |
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5. |
Fabry Disease Background and Overview |
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6. |
Fabry Disease Treatment and Management |
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7. |
Fabry Disease Epidemiology and Patient Population |
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8. |
Fabry Disease Patient Journey |
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9. |
Fabry Disease Marketed Drugs |
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10. |
Fabry Disease Emerging Drugs |
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11. |
Seven Major Fabry Disease Market Analysis |
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12. |
Fabry Disease Market Outlook |
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13. |
Potential of Current and Emerging Therapies |
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14. |
Fabry Disease KOL Views |
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15. |
Fabry Disease Market Drivers |
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16. |
Fabry Disease Market Barriers |
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17. |
Fabry Disease Unmet Needs |
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18. |
SWOT Analysis |
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19. |
Appendix |
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20. |
DelveInsight Capabilities |
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21. |
Disclaimer |
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22. |
About DelveInsight |
About DelveInsight
DelveInsight is a leading Life Science market research and business consulting company recognized for its off-the-shelf syndicated market research reports and customized solutions to firms in the healthcare sector.
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