|Fabry Disease Market to Boost at a CAGR of 7.65% by 2032 | DelveInsight|
|By: PR Newswire Association LLC. - 04 Oct 2022||Back to overview list
LAS VEGAS, Oct. 4, 2022 /PRNewswire/ -- The overall Fabry disease market is likely to surge owing to the increasing prevalence of the disease along with the promising emerging pipeline therapies with new mechanisms of action during the forecast period.
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].
Key Takeaways from the Fabry Disease Market Report
Discover which therapies are expected to grab the major Fabry disease market share @Fabry Disease Market Report
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:
Download the report to understand which factors are driving Fabry disease epidemiology trends @Fabry Disease Epidemiological Insights
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.
To know more about Fabry disease medication, visit @Fabry Disease Treatment Market
Fabry Disease Pipeline Therapies and Key Companies
Learn more about the FDA-approved drugs for Fabry disease treatment @Drugs for Fabry Disease Treatment
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
Discover more about drugs for Fabry disease in development @Fabry Disease Clinical Trials
Table of Contents
Fabry Disease Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the Fabry disease epidemiology trends.
Fabry Disease Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key Fabry disease companies, including 4D Molecular Therapeutics, Idorsia Pharmaceuticals, GREENOVATION BIOTECH GMBH, Shire, among others.
Gaucher Disease Market Insight, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key Gaucher disease companies, including AVROBIO, Sanofi, Yuhan, MP6 Therapeutics, Gain Therapeutics, among others.
Gaucher Disease Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key Gaucher disease companies, including AVROBIO, Sanofi, Yuhan, MP6 Therapeutics, Gain Therapeutics, among others.
Gaucher's Disease Type 1 Market Insights, Epidemiology, and Market Forecast – 2032 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, market share of the individual therapies, and key Gaucher's disease type 1 companies, including Graphite Bio, AVROBIO, ILIAS Biologics, among others.
Gaucher's Disease Type 1 Pipeline Insight – 2022 report provides comprehensive insights about the pipeline landscape, including clinical and non-clinical stage products, and the key Gaucher's disease type 1 companies, including Graphite Bio, AVROBIO, ILIAS Biologics, among others.
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SOURCE DelveInsight Business Research, LLP
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