Fabry Disease - Market Insights, Epidemiology and Market Forecast-2028
DelveInsight’s ‘Fabry Disease - Market Insights, Epidemiology and Market Forecast-2028’ report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of Fabry Disease in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom), and Japan.
The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Fabry Disease from 2017 to 2028 segmented by seven major markets. The Report also covers current treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market.
• The United States
• EU5 (Germany, France, Italy, Spain and the United Kingdom)
Study Period: 2017-2028
Fabry Disease - Disease Understanding and Treatment Algorithm
Fabry disease (FD) is a devastating, progressive, inherited condition caused by lysosomal dysfunction linked to chromosome X and caused by mutations in the GLA gene located in chromosome Xq22.1 and which encodes the alpha-galactosidase A (AGA) enzyme. It is a multi-systemic and a life-threatening condition which is one of the most common lysosomal storage disease after the Gaucher Disease. It is characterized by specific neurological, cutaneous, renal, cardiovascular, cochleo-vestibular and cerebrovascular manifestations.
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 deficiency or absent activity of enzyme alpha-galactosidase A. Alpha-galactosidase A (AGA) enzyme hydrolyses the terminal α -galactosyl moieties from glycolipids and glycoproteins. Absent or deficient activity of lysosomal exoglycohydrolase agalactosidase A (a-D-galactoside galactohydrolase),results in progressive accumulation of globotriaosylceramide (Gb3 or GL-3; also known as ceramidetrihexoside or CTH) and related glycosphingolipids (galabiosylceramide) within lysosomes which are ubiquitous subcellular organelles, in a variety of cell types, including capillary endothelial cells, renal (podocytes, tubular cells, glomerular endothelial, mesangial and intersticial cells), cardiac (cardiomyocytes and fibroblasts) and nerve cells.
The DelveInsight Fabry Disease market report gives a thorough understanding of the Fabry Disease by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for Fabry Disease in the US, Europe, and Japan.
Fabry Disease Epidemiology
The Fabry Disease epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the DelveInsight report also provides the diagnosed and treatable patient pool and their trends along with assumptions undertaken.
The disease epidemiology covered in the report is segmented by diagnosed prevalent population, gender-specific diagnosed prevalent population and phenotype-specific diagnosed prevalent population of Fabry Disease.
The DelveInsight report also provides the epidemiology trends observed in the 7MM during the study period, along with the assumptions undertaken. The calculated data are presented with relevant tables and graphs to give a clear view of the epidemiology at first sight.
According to DelveInsight, the prevalent population of Fabry Disease was estimated to be 16,800 [7MM] in 2018. United States accounts for the highest Fabry Disease cases, followed by EU5 (Germany, France, Italy, Spain & UK) and Japan. Among the EU5 countries UK had the highest prevalent patient population of Fabry Disease, followed by France.
Fabry Disease Drug Chapters
This segment of the Fabry Disease report encloses the detailed analysis of marketed drugs and late stage (Phase-III) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases.
Currently, the effective management of Fabry disease requires a multidisciplinary approach. Comprehensive therapy includes intravenously administered enzyme replacement therapy (ERT) or Oral Chaperone Therapy, conventional medical treatment, and adjunct therapies, and may include lifestyle modifications and prophylactic medications. Fabrazyme (Sanofi-Genzyme); Galafold (Amicus Therapeutics) and Replagal (Shire) are the approved therapies for the treatment of Fabry disease. All these therapies are available in Europe and Japan however Replagal is withdrawn from the market in the US by the Shire in 2012. Galafold (migalastat) is a first-in-class chaperone therapy approved in the European Union as a monotherapy for Fabry disease in patients with amenable mutations. It got approved in May 2016 in Europe being developed by Amicus Therapeutics. Detailed chapters for all of these drugs have been covered in the report.
Fabry Disease Market Outlook
The Fabry Disease market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology.
This segment gives a through detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria’s, 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 calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight.
According to DelveInsight, the global market of Fabry Disease was estimated to be USD 1,211.3 million in 2018. The United States accounts for the largest market size of Fabry Disease, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain), and Japan.
The dynamics of Fabry Disease market is anticipated to change in the coming years owing to the expected launch of emerging therapies during the forecasted period 2019-2028. Several companies are working robustly on many new therapies in Europe with novel mechanism of action; including Lucerastat (Idorsia Pharmaceuticals); Pegunigalsidase alfa (Protalix Biotherapeutics); Venglustat (Sanofi Genzyme) and AVR-RD-01 (Avrobio). The United States accounts for the largest market size of Fabry Disease, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain), and Japan
Fabry Disease Drugs Uptake
This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers market uptake by drugs; patient uptake by therapies and sales of each drug.
This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allows 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.
The market size of Fabry Disease includes the drug-uptake for individual therapies, which is further segmented on the basis of market size and market share by Line of therapy and Route of Administration.
To counter current unmet needs of the market and to provide better treatment options for Fabry Disease, several companies are working robustly on developing new therapies with novel mechanism of action; including Lucerastat (Idorsia Pharmaceuticals); Pegunigalsidase alfa (Protalix Biotherapeutics); Venglustat (Sanofi Genzyme) and AVR-RD-01 (Avrobio) which are expected to enter Fabry Disease market by 2028.
Lucerastat is a small molecule called an “iminosugar,” developed by Idorsia Pharmaceuticals for the treatment of Fabry Disease. It funtions as a substrate reduction therapy. The company is currently leading a pivotal Phase III study, which is being designed to assess the effects of lucerastat on neuropathic pain and gastrointestinal symptoms, as well as safety and tolerability, in patients with Fabry disease.
Pegunigalsidase alfa (PRX-102) is a novel, PEGylated, chemically modified, α-galactosidase A enzyme replacement therapy developed by Protalix BioTherapeutics. It is currently in Phase III stage of development for the treatment of Fabry Disease.
Fabry Disease Report Insights
• Patient Population
• Therapeutic Approaches
• Pipeline Analysis
• Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies
Fabry Disease Report Key Strengths
• 10 Year Forecast
• 7MM Coverage
• Epidemiology Segmentation
• Drugs Uptake
• Highly Analyzed Market
• Key Cross Competition
Fabry Disease Report Assessment
• Current Treatment Practices
• Unmet Needs
• Detailed Pipeline Product Profiles
• Market Attractiveness
• Market Drivers and Barriers
• This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving the Fabry Disease market.
• Organize sales and marketing efforts by identifying the best opportunities for Fabry Disease market.
• To understand the future market competition in the Fabry Disease market.
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