fabry disease epidemiology forecast insight

DelveInsight’s ‘Fabry Disease—Epidemiology Forecast—2034’ report delivers an in-depth understanding of the historical and forecasted epidemiology of Fabry Disease in the United States, EU5 (Germany, Spain, Italy, France, and the United Kingdom), and Japan.

Geographies Covered

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

Study Period: 2021-2034

Fabry Disease: Disease Understanding

Fabry Disease Overview

Fabry disease is an inherited lysosomal storage disease caused by a nonfunctional or only partially functional enzyme, alpha-galactosidase A (α-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 (called 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 in 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 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. Besides, Agalsidase Beta BS, a Fabrazyme biosimilar developed by JCR Pharmaceuticals and GlaxoSmithKline, has been available in the Japanese market since 2018. It has a pricing advantage over Fabrazyme and Replagal, and is 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 latest 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, however, 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: Epidemiology

The Fabry Disease epidemiology division provides insights about 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 diagnosed patient pool and their trends, along with 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 in the 7MM covering the United States, EU5 countries (Germany, France, Italy, Spain, and the United Kingdom), and Japan from 2021 to 2034.

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 total prevalent population of Fabry Disease in the 7MM countries was estimated to be 13,050 cases in 2020.
• As per the estimates, the United States had the highest prevalent population of Fabry Disease in 2020. Among the EU5 countries, the UK had the highest prevalent population of Fabry Disease with 1,113 cases, followed by Germany in 2020. On the other hand, Spain had the lowest prevalent population of 673 cases in 2020.

Scope of the Report

• Fabry Disease report covers a detailed overview explaining its causes, symptoms, classification, pathophysiology, diagnosis, and treatment patterns.
• Fabry Disease Epidemiology Report and Model provide an overview of the risk factors and global trends of Fabry Disease in the seven major markets (7MM: US, France, Germany, Italy, Spain, UK, and Japan).
• The report provides insight into the historical and forecasted patient pool of Fabry Disease in seven major markets covering the United States, EU5 (Germany, Spain, France, Italy, UK), and Japan.
• The report helps recognize the growth opportunities in the 7MM concerning the patient population.
• The report assesses the disease risk and burden and highlights the unmet needs of Fabry Disease.
• The report provides the segmentation of the Fabry Disease epidemiology by prevalent cases of Fabry Disease in the 7MM.
• The report provides the segmentation of the Fabry Disease epidemiology by gender-specific cases of Fabry Disease in the 7MM.
• The report provides the segmentation of the Fabry Disease epidemiology by phenotype-specific cases of Fabry Disease in the 7MM.
• The report provides the Fabry Disease epidemiology segmentation by age-specific Fabry Disease cases in the 7MM.

Report Highlights

• 10-year Forecast of Fabry Disease epidemiology
• 7MM Coverage
• Prevalent Cases of Fabry Disease
• Gender-specific Cases of Fabry Disease
• Phenotype-specific Cases of Fabry Disease
• Age-specific Cases of Fabry Disease

KOL Views

We interview KOLs and obtain SME’s opinions through primary research to fill the data gaps and validate our secondary research. The opinion helps understand the total patient population and current treatment pattern. This will support the clients in potential upcoming novel treatment by identifying the overall scenario of the indications.

Key Questions Answered

• What are the major factors that will drive the change in the patient population in Fabry Disease during the forecast period (2021-2034)?
• What are the key findings pertaining to the Fabry Disease epidemiology across 7MM, and which country will have the highest number of patients during the forecast period (2021-2034)?
• What would be the total number of patients with Fabry Disease across the 7MM during the forecast period (2021-2034)?
• Among the EU5 countries, which country will have the highest number of patients during the forecast period (2021-2034)?
• At what CAGR is the patient population expected to grow in the 7MM forecast period (2021-2034)?
• What are the disease risk, burdens, and unmet needs of Fabry Disease?
• What are the currently available treatments for Fabry Disease?

Reasons to buy

Fabry Disease Epidemiology report will allow the user to:

• Develop business strategies by understanding the trends shaping and driving the global Fabry Disease market.
• Quantify patient populations in the global Fabry Disease market to improve product design, pricing, and launch plans.
• Organize sales and marketing efforts by identifying the age groups and sex that present the best opportunities for Fabry Disease therapeutics in each market covered.
• Understand the magnitude of the Fabry Disease population by its prevalent cases.
• Understand the magnitude of the Fabry Disease population by its gender-specific cases.
• Understand the magnitude of the Fabry Disease population by its phenotype-specific cases.
• Understand the magnitude of the Fabry Disease population by its age-specific cases.
• The Fabry Disease epidemiology report and model were written and developed by Masters and PhD level epidemiologists.
• The Fabry Disease Epidemiology Model developed by DelveInsight is easy to navigate, interactive with dashboards, and epidemiology based on transparent and consistent methodologies. Moreover, the model supports data presented in the report and showcases disease trends over a 10-year forecast period using reputable sources.

Key Assessments

• Patient Segmentation
• Disease Risk and Burden
• Risk of disease by the segmentation
• Factors driving growth in a specific patient population