Fabry Disease Epidemiology Forecast

DelveInsight’s ‘Fabry Disease—Epidemiology Forecast—2032’ 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: 2019–2032

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 (a Fabry crisis), gastrointestinal complications, headaches, impaired sweating, vertigo, and hearing impairment.

 

It was once thought that Fabry disease only affected males. Females were considered 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.

Fabry Disease: Epidemiology

The Fabry Disease epidemiology division provides insights into the historical and current patient pool and 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 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 total prevalent population of Fabry Disease in the 7MM countries was estimated to be more than 13,000 cases in 2020.
  • As per the estimates, the United States had the highest prevalent population of Fabry Disease in 2022. Among the EU5 countries, the Germany had the highest prevalent population of Fabry Disease with approximately 1,150 cases, followed by the UK in 2020. On the other hand, Spain had the lowest, nearly 650 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

  • 11-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 treatments by identifying the overall scenario of the indications.

Key Questions Answered

  • What major factors will drive the change in the patient population in Fabry Disease during the forecast period (2019–2032)?
  • What are the key findings of the Fabry Disease epidemiology across 7MM, and which country will have the highest number of patients during the forecast period (2019–2032)?
  • What would be the total number of patients with Fabry Disease across the 7MM during the forecast period (2019–2032)?
  • Among the EU5 countries, which country will have the highest number of patients during the forecast period (2019–2032)?
  • At what CAGR is the patient population expected to grow in the 7MM forecast period (2019–2032)?
  • 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 the data presented in the report and showcases disease trends over an 11-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

1. Key Insights

2. Report Introduction

3. Executive Summary of Fabry Disease

4. Disease Background and Overview

4.1. Introduction

4.2. Classification

4.3. Signs and Symptoms

4.4. Etiology

4.5. Inheritance Pattern

4.6. Genetics

4.7. Molecular Basis

4.8. Pathogenesis

4.9. Diagnosis

4.9.1. European Fabry Working Group (EFWG): Diagnostic criteria

4.9.2. Challenges in Diagnosis

4.10. Treatment and Management of Fabry Disease

4.10.1. Guidelines for the Management of Fabry Disease

4.10.2. Croatian Society: Recommendation for the treatment of Fabry Disease

4.10.3. Treatment Practices

4.10.4. Consensus recommendations for diagnosis, management, and treatment of Fabry disease in pediatric patients

5. Epidemiology and Patient Population

5.1. Key Findings

5.2. 7MM Total Prevalence of Fabry Disease

5.3. Epidemiology of Fabry Disease

5.4. The United States

5.4.1. Prevalence of Fabry Disease in the United States

5.4.2. Gender-specific cases of Fabry Disease in the United States

5.4.3. Phenotype-specific cases of Fabry Disease in the United States

5.4.4. Age-specific cases of Fabry Disease in the United States

5.5. EU5

5.5.1. Total Prevalence of Fabry Disease in EU5

5.5.2. Germany

5.5.2.1. Gender-specific cases of Fabry Disease in Germany

5.5.2.2. Phenotype-specific cases of Fabry Disease in Germany

5.5.2.3. Age-specific cases of Fabry Disease in Germany

5.5.3. France

5.5.3.1. Gender-specific cases of Fabry Disease in France

5.5.3.2. Phenotype-specific cases of Fabry Disease in France

5.5.3.3. Age-specific cases of Fabry Disease in France

5.5.4. Italy

5.5.4.1. Gender-specific cases of Fabry Disease in Italy

5.5.4.2. Phenotype-specific cases of Fabry Disease in Italy

5.5.4.3. Age-specific cases of Fabry Disease in Italy

5.5.5. Spain

5.5.5.1. Gender-specific cases of Fabry Disease in Spain

5.5.5.2. Phenotype-specific cases of Fabry Disease in Spain

5.5.5.3. Age-specific cases of Fabry Disease in Spain

5.5.6. The United Kingdom

5.5.6.1. Gender-specific cases of Fabry Disease in the United Kingdom

5.5.6.2. Phenotype-specific cases of Fabry Disease in the United Kingdom

5.5.6.3. Age-specific cases of Fabry Disease in the United Kingdom

5.5.7. Japan

5.5.7.1. Prevalence of Fabry Disease in Japan

5.5.7.2. Gender-specific cases of Fabry Disease in Japan

5.5.7.3. Phenotype-specific cases of Fabry Disease in Japan

5.5.7.4. Age-specific cases of Fabry Disease in Japan

6. Patient Journey

7. KOL Views

8. Unmet Needs

9. Appendix

9.1. Bibliography

9.2. Report Methodology

10. DelveInsight Capabilities

11. Disclaimer

12. About DelveInsight

List of Tables

Table 1: Summary of Fabry Disease, Epidemiology, and Key Events (2019–2032)

Table 2: Pathophysiologic Findings in Fabry Disease

Table 3: Proposed Assessments for Fabry Disease

Table 4: Clinical Evaluation and Monitoring of Adult Patients With Fabry Disease

Table 5: Clinical evaluation and Monitoring of Pediatric Patients With Fabry Disease

Table 6: Diagnostic criteria of European Fabry Working Group (EFWG)

Table 7: Gold-standard Methods to Diagnose Fabry Disease

Table 8: Fabry disease: Common Misdiagnoses

Table 9: Current Guidelines for Instituting Enzyme Replacement Therapy in Fabry Disease Patients

Table 10: Adjunctive Therapies and Preventive Measures

Table 11: Implications for Recommendations

Table 12: Treatment Goals for Fabry Disease Patients Associated With Other Conditions

Table 13: Effect of Enzyme Replacement Therapy on Signs and Symptoms of Fabry Disease

Table 14: Enzyme Replacement Therapy

Table 15:  Recommended Analgesic Drugs for Supportive Treatment of Chronic Neuropathic Pain

Table 16: Recommended Analgesic Drugs for Supportive Treatment of Acute Pain in Fabry Disease

Table 17: Prevalence of Fabry Disease in the 7MM (2019–2032)

Table 18: Prevalence of Fabry Disease in the United States (2019–2032)

Table 19: Gender-specific cases of Fabry Disease in the United States (2019–2032)

Table 20: Phenotype-specific cases of Fabry Disease in the United States (2019–2032)

Table 21: Age-specific cases of Fabry Disease in the United States (2019–2032)

Table 22: Prevalence of Fabry Disease in EU5 (2019–2032)

Table 23: Gender-specific cases of Fabry Disease in Germany (2019–2032)

Table 24: Phenotype-specific cases of Fabry Disease in Germany (2019–2032)

Table 25: Age-specific cases of Fabry Disease in Germany (2019–2032)

Table 26: Gender-specific cases of Fabry Disease in France (2019–2032)

Table 27: Phenotype-specific cases of Fabry Disease in France (2019–2032)

Table 28: Age-specific cases of Fabry Disease in France (2019–2032)

Table 29: Gender-specific cases of Fabry Disease in Italy (2019–2032)

Table 30: Phenotype-specific cases of Fabry Disease in Italy (2019–2032)

Table 31: Age-specific cases of Fabry Disease in Italy (2019–2032)

Table 32: Gender-specific cases of Fabry Disease in Spain (2019–2032)

Table 33: Phenotype-specific cases of Fabry Disease in Spain (2019–2032)

Table 34: Age-specific cases of Fabry Disease in Spain (2019–2032)

Table 35: Gender-specific cases of Fabry Disease in the United Kingdom (2019–2032)

Table 36: Phenotype-specific cases of Fabry Disease in the United Kingdom (2019–2032)

Table 37: Age-specific cases of Fabry Disease in the United Kingdom (2019–2032)

Table 38: Prevalence of Fabry Disease in Japan (2019–2032)

Table 39: Gender-specific cases of Fabry Disease in Japan (2019–2032)

Table 40: Phenotype-specific cases of Fabry Disease in Japan (2019–2032)

Table 41: Age-specific cases of Fabry Disease in Japan (2019–2032)

List of Figures

Figure 1: Characteristics of Type 1 Classic and Type 2 Later-onset Fabry Disease

Figure 2: Symptoms of Fabry Disease in Adults, Childhood, and Adolescence

Figure 3: Role of a-galactosidase A in GL-3 Catabolism

Figure 4: Inheritance Pattern of Fabry Disease

Figure 5: α-galactosidase A gene Mutations

Figure 6: Genetic and Molecular Basis of Fabry Disease

Figure 7: Diagnosis Algorithm for Fabry Disease

Figure 8: Treatment Algorithm

Figure 9: Treatment Algorithm for Fabry Disease in Pediatric Patients

Figure 10: Treatment Algorithm for Fabry Disease in Pediatric Patients

Figure 11: Total Prevalent Population of Fabry Disease in 7MM (2019–2032)

Figure 12: Prevalence of Fabry Disease in the United States (2019–2032)

Figure 13: Gender-specific cases of Fabry Disease in the United States (2019–2032)

Figure 14: Phenotype-specific cases of Fabry Disease in the United States (2019–2032)

Figure 15: Age-specific cases of Fabry Disease in the United States (2019–2032)

Figure 16: Total Prevalent Population of Fabry Disease in EU5 (2019–2032)

Figure 17: Gender-specific cases of Fabry Disease in Germany (2019–2032)

Figure 18: Phenotype-specific cases of Fabry Disease in Germany (2019–2032)

Figure 19: Age-specific cases of Fabry Disease in Germany (2019–2032)

Figure 20: Gender-specific cases of Fabry Disease in France (2019–2032)

Figure 21: Phenotype-specific cases of Fabry Disease in France (2019–2032)

Figure 22: Age-specific cases of Fabry Disease in France (2019–2032)

Figure 23: Gender-specific cases of Fabry Disease in Italy (2019–2032)

Figure 24: Phenotype-specific cases of Fabry Disease in Italy (2019–2032)

Figure 25: Age-specific cases of Fabry Disease in Italy (2019–2032)

Figure 26: Gender-specific cases of Fabry Disease in Spain (2019–2032)

Figure 27: Phenotype-specific cases of Fabry Disease in Spain (2019–2032)

Figure 28: Age-specific cases of Fabry Disease in Spain (2019–2032)

Figure 29: Gender-specific cases of Fabry Disease in the United Kingdom (2019–2032)

Figure 30: Phenotype-specific cases of Fabry Disease in the United Kingdom (2019–2032)

Figure 31: Age-specific cases of Fabry Disease in the United Kingdom (2019–2032)

Figure 32: Prevalence of Fabry Disease in Japan (2019–2032)

Figure 33: Gender-specific cases of Fabry Disease in Japan (2019–2032)

Figure 34: Phenotype-specific cases of Fabry Disease in Japan (2019–2032)

Figure 35: Age-specific cases of Fabry Disease in Japan (2019–2032)

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