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Fabry Disease - Market Insight, Epidemiology And Market Forecast - 2032

Fabry Disease - Market Insight, Epidemiology And Market Forecast - 2032

  • May 2022
  • 181 pages
  • ID: 6276114
  • Format: PDF
  • Delve Insight


Table of Contents

‘Fabry Disease—Market Insights, Epidemiology, and Market Forecast—2032’ report deliver an in-depth understanding of the historical and 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 Fabry Disease market report provides current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted Fabry Disease market Size from 2019 to 2032, segmented by seven major markets. The report also covers the current Fabry Disease treatment practice/algorithm, market drivers, market barriers, and unmet medical needs to curate the best opportunities and assesses the underlying market potential.

Geographies Covered
• The United States
• EU5 (Germany, France, Italy, Spain, and the United Kingdom)
• Japan

Study Period: 2019–2032

Fabry Disease: Disease Understanding and Treatment Algorithm

Fabry Disease Overview
Fabry disease is an inherited lysosomal storage disease caused by a nonfunctional or only partially functional enzyme, alpha-galactosidase A (?-gal A). Decreased activity of alpha-gal A in lysosomes results in the accumulation of enzyme substrates (Gb3 and lyso-Gb3) which cause cellular damage in tissues throughout the body.
Symptoms include pain that spreads through the body (a Fabry crisis), gastrointestinal complications, headaches, impaired sweating, vertigo, and hearing impairment.
It was once thought that Fabry disease only affected males, and females were thought of as only “carriers.” However, now it is known that both men and women can get Fabry disease, although it may manifest differently depending on gender. Despite being X-linked, heterozygous women may experience all the signs and symptoms of Fabry disease seen in men; although compared with hemizygous males, signs and symptoms of Fabry disease in women typically emerge at an older age and with less severity.
Enzyme replacement therapy (ERT) with agalsidase alfa (Replagal) or agalsidase beta (Fabrazyme) has been the standard of care since 2001. Fabrazyme is the only enzyme replacement medication for Fabry disease that has been approved by the FDA and has long-term efficacy and safety data. It received accelerated approval from the FDA in 2003. The FDA converted the enzyme replacement treatment authorization to a full, regular approval 18 years later. Replagal, developed by the Shire, received approval from the European Commission as an Orphan Drug in August 2001 and in February 2007 in Japan with the marketing rights with Dainippon Sumitomo Pharma. It is currently approved for the treatment of FD in 38 countries but not in the US. Also, Agalsidase Beta BS is the Fabrazyme biosimilar developed by JCR Pharmaceuticals and GlaxoSmithKline. It has been available in Japan since 2018. It has a pricing advantage over Fabrazyme and Replagal, the only Japan-originated therapeutic.
Though ERT has several benefits, it also has serious limitations. These include an inconvenient dosing schedule every 2 weeks causing variation in enzyme levels, risk of infusion-related reactions and infections, and the possibility of developing antibodies against treatment.
Galafold has been the recent entrant in the FD market in the last 15 years. The therapy gained significant market share from the existing treatments as it alleviated the current unmet needs. Also, as per NICE, it represents better value for money and may also provide more clinical benefits. Galafold is only eligible to patients with amenable mutations (35–50% of the overall Fabry disease population); therefore, one-size-fits-all treatment does not hold for Fabry disease.
Several new therapies are under research, including new forms of ERT, substrate reduction therapy, mRNA therapy, and gene therapy, to address the current challenges in the treatment.

Fabry Disease Diagnosis and Treatment
It covers the details of conventional and current medical therapies and diagnosis available in the Fabry Disease market to treat the condition. It also provides the country-wise treatment guidelines and algorithms across the United States, Europe, and Japan.
The Fabry Disease market report gives a thorough understanding of Fabry Disease by including disease definition, symptoms, causes, pathophysiology, and diagnosis. It also provides the treatment algorithms and treatment guidelines for Fabry Disease in the US, Europe, and Japan.

Fabry Disease Epidemiology
The Fabry Disease epidemiology division provides insights into the historical and current patient pool along with the forecasted trend for every seven major countries. It helps recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of the report also provides the prevalent patient pool, trends, and assumptions undertaken.

Key Findings
The disease epidemiology covered in the report provides historical and forecasted Fabry Disease epidemiology segmented as the prevalent cases of Fabry Disease, Gender-specific Cases of Fabry Disease, Phenotype-specific Cases of Fabry Disease and Age-specific Cases of Fabry Disease. The report includes the prevalent scenario of Fabry Disease symptoms in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2019 to 2032.

Country-wise Fabry Disease Epidemiology
The epidemiology segment also provides the Fabry Disease epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan.
The prevalent population of Fabry Disease in the 7MM countries was estimated to be more than 13,000 cases in 2020.

Fabry Disease Drug Chapters
The drug chapter segment of the Fabry Disease report encloses the detailed analysis of Fabry Disease marketed drugs and late stage (Phase-III, Phase-II/III, Phase-II, and Phase-I/II) pipeline drugs. It also helps understand the Fabry Disease 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.
Fabrazyme (agalsidase beta), developed by Sanofi-Genzyme, is an enzyme replacement therapy administered by intravenous infusion once every 2 weeks. It is the first specific treatment approved for FD, receiving FDA approval in April 2003 and is currently approved in more than 75 countries, including Europe (August 2004) and Japan (January 2004). Fabrazyme has witnessed sales growth in many countries, especially in the EU region, driven by an increase in the number of patients opting for treatment.
Replagal, also known as agalsidase alfa, is a recombinant formulation of human ?-galactosidase A (?-GAL A) developed by Shire (now a part of Takeda). The drug is a long-term enzyme replacement therapy for the treatment of FD. It was first approved by the European Commission in the European Union in August 2001 and then approved in Japan in February 2007, where Dainippon Sumitomo Pharma holds its marketing rights.
Galafold (migalastat) is an iminosugar that functions as a first-in-class pharmacologic chaperone and is approved as the first line of therapy for treating adults with a confirmed diagnosis of FD with an amenable GLA variant. It is the first oral drug approved and the first new drug given the green light in more than 15 years (using the accelerated pathway in August 2018 by FDA). Currently, it is being used to treat patients with any one of 348 GLA variants and is only effective for specific genetic mutations, estimated to be beneficial in 35–50% of patients.
Products detail in the report…

Fabry Disease Emerging Drugs
PRX-102 (Pegunigalsidase Alfa) (Protalix Biotherapeutics) is an experimental enzyme replacement therapy (ERT) under development by Protalix Biotherapeutics. It is being developed using Protalix’s plant-based ProCellEx platform, a plant cell culture expressed, and a chemically modified version of the recombinant ?-GAL A protein. As per the company’s expectation, PRX-102 will provide better clinical efficacy and an improved safety profile than currently available treatments. The company has completed three Phase III studies (BALANCE, BRIGHT, and BRIDGE). The final results of the BRIGHT study are expected in Q4 of 2021, while the final outcomes of the BALANCE study will be out in Q1 of 2022. In May 2020, the companies filed an application with the US FDA seeking the accelerated approval of PRX-102, given at a dose of 1 mg/kg every other week, to treat adults with Fabry. After granting it priority review, the agency rejected the application in April 2021 due to issues with facility inspections and manufacturing processes, partially caused by travel restrictions during the COVID-19 pandemic. The decision was not associated with concerns about the therapy’s safety or effectiveness shown in clinical trials. Protalix, following a Type A meeting with the FDA, is preparing for resubmission in the second half of 2022, alongside a submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) planned for the first half of 2022.
Venglustat (Sanofi Genzyme) is an orally administered small molecule being developed for several rare diseases. It is an inhibitor of an enzyme called glucosylceramide synthase (GCS) and modifies the enzyme substrates. GCS turns its substrate, ceramide, into glucosylceramide (GL-1) during lipid metabolism, a series of biochemical reactions that degrade and generate lipids. GL-1 acts as a substrate to other enzymes and is turned into globosides, a subclass of lipids where Gb3 belongs. The molecule is developed by Sanofi Genzyme and is in the Phase III stage of development for FD. The company expects to file a submission by 2025+.
Products detail in the report…

Fabry Disease Market Outlook
The Fabry Disease market outlook of the report builds a detailed comprehension of the historical, current, and forecasted Fabry Disease market trends by analyzing the impact of current therapies on the market, unmet needs, and demand for better technology.
This segment gives a thorough detail of the 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’s, mechanism of action, compliance rate, growing need for 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 the analyst, the Fabry Disease market in the 7MM is expected to change in the study period 2019–2032.
ERT has changed the outcome of patients; however, the enzyme must be delivered intravenously, which can be challenging as it requires repeated calculations. Lifelong, bimonthly infusions can lead to poor venous access, potentially requiring subcutaneous ports, which can risk bacterial infection and complicate future hemodialysis access. Furthermore, the administration of recurrent IV infusions in the pediatric population can be challenging. Infusion reactions can be life-threatening and are usually mediated by anti-drug antibody (ADA) responses and neutralizing; these have shown an inhibitory effect on the ERT and can correlate with poor outcomes. Although ERT is generally tolerated well, there is room for improvement, and to overcome these limitations, chaperone therapy has been introduced.

Key Findings
This section includes a glimpse of the Fabry Disease market in the 7MM. The market size of Fabry Disease in the seven major markets was nearly USD 1,200 million in 2020.

The United States: Market Outlook
This section provides the total Fabry Disease market size and market size by therapies of Fabry Disease in the United States.
The United States accounts for the highest market size of Fabry Disease compared to the EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.
FD is highly underdiagnosed, and a significant unmet need remains among patients. The treatment choices for people living with FD in the US have been limited. It is a condition associated with morbidity that substantially impacts survival and day-to-day function. If left untreated, there is a high likelihood that the disease will progress from a less severe to a more serious one. Compared to the general population, FD causes a substantial reduction in life expectancy - by an average of 15 years in female patients and 20 years in male patients.
The need for adjunctive therapies to improve outcomes in patients with FD cannot be overlooked, and standards of care for patients with chronic kidney disease (CKD) or hypertrophic cardiomyopathy must be provided. Angiotensin-converting enzyme inhibitors or angiotensin receptor blockers have been shown to decrease the progression of kidney disease in FD. Given the low risk of Vitamin D therapy, it is recommended that Vitamin D levels be checked and replete as needed. FD is a risk factor for stroke; statin therapy should be considered. Further supportive therapies can be provided based on the comorbidities.
To counter the market’s current unmet needs and provide better treatment options, several companies are working aggressively to develop new therapies with a novel mechanism of action.

EU-5 Countries: Market Outlook
The total Fabry Disease market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are also mentioned.

Japan Market Outlook
The total Fabry Disease market size and market size by therapies of Fabry Disease in Japan are also mentioned.

Fabry Disease Drugs Uptake
This section focuses on the rate of uptake of the potential drugs recently launched or expected to get launched in the market during the study period 2019–2032. The analysis covers Fabry Disease 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 based on market share and size, which again will be useful in investigating factors important in the market uptake and in making financial and regulatory decisions.

Fabry Disease Pipeline Development Activities
The report provides insights into different therapeutic candidates in Phase III, Phase II/III, Phase II, and Phase I/II stage. It also analyses Fabry Disease’s key players involved in developing targeted therapeutics.
Major players include Protalix Biotherapeutics, Sanofi Genzyme, Idorsia Pharmaceuticals, Avrobio, Sangamo Therapeutics, 4D Molecular Therapeutics, and Freeline Therapeutics, whose key products are expected to get launched in the US market by 20XX.

Pipeline Development Activities
The report covers collaborations, acquisitions, mergers, licensing, and patent details for Fabry Disease emerging therapies.
KOL Views
To keep up with current market trends, we take KOLs and SMEs’ opinions working in the Fabry Disease domain through primary research to fill the data gaps and validate our secondary research. Their opinion helps understand and validate current and emerging therapies treatment patterns or Fabry Disease market trends. This will support the clients in potential upcoming novel treatments by identifying the overall scenario of the market and the unmet needs.

Competitive Intelligence Analysis
We perform a Competitive and Market Intelligence analysis of the Fabry Disease market by using various Competitive Intelligence tools: SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.

Scope of the Report
• The report covers the descriptive overview of Fabry Disease, explaining its causes, signs and symptoms, pathophysiology, and currently available therapies.
• Comprehensive insight has been provided into the Fabry Disease epidemiology and treatment in the 7MM.
• Additionally, an all-inclusive account of both the current and emerging therapies for Fabry Disease is provided, along with the assessment of new therapies, which will impact the current treatment landscape.
• A detailed review of the Fabry Disease market, historical and forecasted, is included in the report, covering drug outreach in the 7MM.
• The report provides an edge while developing business strategies by understanding trends shaping and driving the global Fabry Disease market.

Report Highlights
• In the coming years, the Fabry Disease market is set to change due to the rising awareness of the disease and incremental healthcare spending across the world, which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.
• The companies and academics are working to assess challenges and seek opportunities that could influence Fabry Disease R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.
• Major players are involved in developing therapies for Fabry Disease. The launch of emerging therapies will significantly impact the Fabry Disease market.
• A better understanding of disease pathogenesis will also contribute to developing novel therapeutics for Fabry Disease.
• Our in-depth analysis of the pipeline assets across different stages of development (Phase III and Phase II), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross-competitor, launch date along with product development activities will support the clients in the decision-making process regarding their therapeutic portfolio by identifying the overall scenario of the research and development activities.

Fabry Disease Report Insights
• Patient Population
• Therapeutic Approaches
• Fabry Disease Pipeline Analysis
• Fabry Disease Market Size and Trends
• Market Opportunities
• Impact of upcoming Therapies

Fabry Disease Report Key Strengths
• 11-years Forecast
• 7MM Coverage
• Fabry Disease Epidemiology Segmentation
• Key Competitors
• Highly Analyzed Market

• Drugs Uptake

Fabry Disease Report Assessment
• Current Treatment Practices
• Unmet Needs
• Pipeline Product Profiles
• Market Attractiveness

Key Questions

Market Insights:
• What was the Fabry Disease Market share (%) distribution in 2019, and what would it look like in 2032?
• What would be the Fabry Disease total market Size and market Size by therapies across the 7MM during the forecast period (2019–2032)?
• What are the key findings pertaining to the market across 7MM, and which country will have the largest Fabry Disease market Size during the forecast period (2019–2032)?
• At what CAGR is the Fabry Disease market expected to grow in the 7MM forecast period (2019–2032)?
• What would be the Fabry Disease market outlook across the 7MM during the forecast period (2019–2032)?
• What would be the Fabry Disease market growth till 2032, and what will be the resultant market Size in the year 2032?
• How would the future opportunities affect the market dynamics and subsequent analysis of the associated trends?

Epidemiology Insights:
• What are the disease risk, burden, and unmet needs of Fabry Disease?
• What is the historical Fabry Disease patient pool in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What would be the forecasted patient pool of Fabry Disease in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan?
• What will be the growth opportunities in the 7MM concerning the patient population pertaining to Fabry Disease?
• Out of all 7MM countries, which country would have the highest prevalence of Fabry Disease during the forecast period (2019–2032)?
• At what CAGR is the population expected to grow in the 7MM forecast period (2019–2032)?

Current Treatment Scenario, Marketed Drugs, and Emerging Therapies:
• What are the current options for the treatment of Fabry Disease?
• What are the current treatment guidelines for treating Fabry Disease in the US, Europe, and Japan?
• What are the Fabry Disease marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?
• How many companies are developing therapies for the treatment of Fabry Disease?
• How many therapies are developed by each company for the treatment of Fabry Disease?
• How many emerging therapies are in the mid-stage and late stages of development for the treatment of Fabry Disease?
• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Fabry Disease therapies?
• What are the recent novel therapies, targets, mechanisms of action, and technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Fabry Disease and its status?
• What key designations have been granted for the emerging therapies for Fabry Disease?
• What are the global historical and forecasted markets for Fabry Disease?

Reasons to buy
• The report will help in developing business strategies by understanding trends shaping and driving the Fabry Disease market
• To understand the future market competition in the Fabry Disease market and an Insightful review of the key market drivers and barriers.
• Organize sales and marketing efforts by identifying the best opportunities for Fabry Disease in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom), and Japan.
• Identifying strong upcoming players in the market will help devise strategies that will help get ahead of competitors.
• Organize sales and marketing efforts by identifying the best opportunities for the Fabry Disease market.
• To understand the future market competition in the Fabry Disease market.

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