Table of Content

1
Key Insights

2
Hunter Syndrome Market Overview at a Glance

2.1
Market Share (%) Distribution of Hunter Syndrome in 2017

2.2
Market Share (%) Distribution of Hunter Syndrome in 2030

3
Disease Background and Overview: Hunter Syndrome

3.1
Introduction

3.2
Classification of Mucopolysaccharidosis

3.3
Clinical Characteristics of Hunter Syndrome

3.3.1
Growth

3.3.2 Eyes
3.3.3
Ear, Nose, Throat

3.3.4
Joints/Skeletal

3.3.5
Respiratory

3.3.6
Cardiovascular

3.3.7
Gastrointestinal

3.3.8
Nervous System

3.3.9
Endocrine

3.4 Molecular Genetics
3.4.1
Gene structure

3.4.2
Pathogenic variants

3.4.3
Normal gene product

3.4.4
Abnormal gene product

3.5
Symptoms

3.6
Complications and risk factors

3.7
Diagnosis

3.7.1
Urinary GAG excretion

3.7.2
Enzyme activity

3.7.3
Genetic testing

3.7.4
Preimplantation genetic diagnosis and prenatal testing

3.7.5
Establishing the Diagnosis

3.8
Differential diagnosis

4
Diagnostic Algorithm for Hunter Syndrome

5
Epidemiology and Patient Population

5.1
Key Findings

5.2
7MM Total Diagnosed Prevalent Population of Hunter Syndrome

6
Country-Wise Epidemiology of Hunter Syndrome

6.1
United States

6.1.1
Assumptions and Rationale

6.1.2
Total Diagnosed Prevalent Population of Hunter Syndrome in the United States

6.1.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the United States

6.2
EU5 Countries

6.3
Germany

6.3.1
Assumptions and Rationale

6.3.2
Total Diagnosed Prevalent Population of Hunter Syndrome in Germany

6.3.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Germany

6.4
France

6.4.1
Assumptions and Rationale

6.4.2
Total Diagnosed Prevalent Population of Hunter Syndrome in France

6.4.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in France

6.5
Italy

6.5.1
Assumptions and Rationale

6.5.2
Total Diagnosed Prevalent Population of Hunter Syndrome in Italy

6.5.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Italy

6.6
Spain

6.6.1
Assumptions and Rationale

6.6.2
Total Diagnosed Prevalent Population of Hunter Syndrome in Spain

6.6.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Spain

6.7
United Kingdom

6.7.1
Assumptions and Rationale

6.7.2
Total Diagnosed Prevalent Population of Hunter Syndrome in the United Kingdom

6.7.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the United Kingdom

6.8
Japan

6.8.1
Assumptions and Rationale

6.8.2
Total Diagnosed Prevalent Population of Hunter Syndrome in Japan

6.8.3
Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Japan

7
Treatment

7.1
Symptomatic treatment

7.1.1
Skeletal abnormalities

7.1.2
Cardiovascular system

7.1.3
Respiratory disease and upper airway manifestations

7.1.4
Gastrointestinal system

7.1.5
Central nervous system

7.1.6
Peripheral nervous system

7.2
Specific Treatment

7.2.1
Visual problems

7.2.2
Hearing

7.2.3
Education and Behavior

7.2.4
Anesthetic complications

7.3
Prevention of Primary Manifestations

7.3.1
Enzyme replacement therapy (ERT)

7.3.2
Stem cell transplantation

7.3.3
Gene Therapy

7.4
Prevention of Secondary Complications

8
Recognized Establishments

9
Unmet Needs

10
Marketed Profile

10.1.1
Elaprase (idursulfase): Takeda

10.1.2
Drug Description

10.1.3
Regulatory Milestones

10.1.4
Other Development Activities

10.1.5
Safety and Efficacy

10.1.6
Product Profile

10.2
Emerging Drugs

10.3
Key Cross Competition

10.4
JR-141: JCR Pharmaceuticals

10.4.1
Product Description

10.4.2
Other Development Activities

10.4.3
Clinical Development

10.4.4
Safety and Efficacy

10.4.5
Product Profile

10.5
TAK-609/SHP609/HGT-2310: Takeda

10.5.1
Product Description

10.5.2
Other Development Activities

10.5.3
Clinical Development

10.5.4
Safety and Efficacy

10.5.5
Product Profile

10.6
GC1111 (Hunterase): Green Cross Corporation/GC Pharma

10.6.1
Product Description

10.6.2
Other Development Activities

10.6.3
Clinical Development

10.6.4
Safety and Efficacy

10.6.5
Product Profile

10.7
DNL310: Denali Therapeutics

10.7.1
Product Description

10.7.2
Other Development Activities

10.7.3
Clinical Development

10.7.4
Product Profile

10.8
RGX-121: Regenxbio

10.8.1
Product Description

10.8.2
Other Development Activities

10.8.3
Clinical Development

10.8.4
Safety and Efficacy

10.8.5
Product Profile

10.9
SB-913: Sangamo Therapeutics

10.9.1
Product Description

10.9.2
Other Development Activities

10.9.3
Clinical Development

10.9.4
Safety and Efficacy

10.9.5
Product Profile

11
Hunter Syndrome: 7 Major Market Analysis

11.1
Key Findings

11.2
Market Size of Hunter Syndrome in the 7MM

11.3
The United States Market Outlook

11.4
United States Market Size

11.4.1
Total Market size of Hunter Syndrome

11.4.2
Market Size by Therapies

11.5
EU-5 Countries: Market Outlook

11.6
Germany

11.6.1
Total Market size of Hunter Syndrome in Germany

11.6.2
Market Size by Therapies

11.7
France

11.7.1
Total Market Size of Hunter Syndrome

11.7.2
Market Size by Therapies

11.8
Italy

11.8.1
Total Market Size of Hunter Syndrome

11.8.2
Market Size by Therapies

11.9
Spain

11.9.1
Total Market Size of Hunter Syndrome

11.9.2
Market Size by Therapies

11.1
United Kingdom

11.10.1
Total Market Size of Hunter Syndrome

11.10.2
Market Size by Therapies

11.11
Japan: Market Outlook

11.12
Japan market Size

11.12.1
Total Market Size of Hunter Syndrome

11.12.2
Market Size by Therapies

12
KOL Reviews

13
Case Reports

13.1
Diagnosis, Quality of Life, and Treatment of Patients with Hunter syndrome in the French Healthcare System: A Retrospective Observational Study (France)

13.2
Idursulfase Enzyme Replacement Therapy in an Adult Patient with Severe Hunter Syndrome having a Novel Mutation of Iduronate-2-sulfatase gene (Japan)

14
Market Drivers

15
Market Barriers

16
SWOT Analysis

17
Appendix

17.1
Bibliography

17.2
Report Methodology

18
DelveInsight Capabilities

19
Disclaimer

20
About DelveInsight



List of Figures


Figure 1: Symptoms of Hunter Syndrome

Figure 2: Diagnostic Algorithm for Hunter Syndrome

Figure 3: Total Diagnosed Prevalent Population of Hunter Syndrome in the 7MM (2017–2030)

Figure 4: Total Diagnosed Prevalent Population of Hunter Syndrome in the United States (2017–2030)

Figure 5: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the United States (2017–2030)

Figure 6: Total Diagnosed Prevalent Population of Hunter Syndrome in Germany (2017–2030)

Figure 7: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Germany (2017–2030)

Figure 8: Total Diagnosed Prevalent Population of Hunter Syndrome in France (2017–2030)

Figure 9: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in France (2017–2030)

Figure 10: Total Diagnosed Prevalent Population of Hunter Syndrome in Italy (2017–2030)

Figure 11: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Italy (2017–2030)

Figure 12: Total Diagnosed Prevalent Population of Hunter Syndrome in Spain (2017–2030)

Figure 13: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Spain (2017–2030)

Figure 14: Total Diagnosed Prevalent Population of Hunter Syndrome in the United Kingdom (2017–2030)

Figure 15: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the United Kingdom (2017–2030)

Figure 16: Total Diagnosed Prevalent Population of Hunter Syndrome in Japan (2017–2030)

Figure 17: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Japan (2017–2030)

Figure 18: Lysosomal recombinant iduronate-2-sulfatase transport

Figure 19: Hematopoietic stem Cell Therapy Protocol in Hunter Syndrome Patients

Figure 20: Unmet Needs of Hunter Syndrome

Figure 21: 7 Major Market Size of Hunter Syndrome in USD Million (2017–2030)

Figure 22: Market Size of Hunter Syndrome in the United States, USD Millions (2017–2030)

Figure 23: Market size of Hunter Syndrome by therapies in the United States, in USD Million (2017–2030)

Figure 24: Market Size of Hunter Syndrome in Germany, USD Million (2017–2030)

Figure 25: Market Size of Hunter Syndrome by therapies in Germany, in USD Million (2017–2030)

Figure 26: Market Size of Hunter Syndrome in France, USD Million (2017–2030)

Figure 27: Market Size of Hunter Syndrome by therapies in France, in USD Million (2017–2030)

Figure 28: Market Size of Hunter Syndrome in Italy, USD Million (2017–2030)

Figure 29: Market Size of Hunter Syndrome by therapies in Italy, in USD Million (2017–2030)

Figure 30: Market Size of Hunter Syndrome in Spain, USD Million (2017–2030)

Figure 31: Market Size of Hunter Syndrome by therapies in Spain, in USD Million (2017–2030)

Figure 32: Market Size of Hunter Syndrome in the United Kingdom, USD Million (2017–2030)

Figure 33: Market Size of Hunter Syndrome by therapies in the United Kingdom, in USD Million (2017–2030)

Figure 34: Market Size of Hunter Syndrome in Japan, USD Million (2017–2030)

Figure 35: Market Size of Hunter Syndrome by therapies in Japan, in USD Million (2017–2030)

Figure 36: Market Drivers

Figure 37: Market Barriers


List of Tables


Table 1: Types of Mucopolysaccharidosis

Table 2: Frequency of the different types of variants reported for the IDS gene

Table 3: Molecular Genetic Testing Used in Hunter Syndrome

Table 4: Total Diagnosed Prevalent Population of Hunter Syndrome in the 7MM (2017–2030)

Table 5: Total Diagnosed Prevalent Population of Hunter Syndrome in the United States (2017–2030)

Table 6: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the United States (2017–2030)

Table 7: Type-specific Diagnosed Prevalence of Hunter Syndrome in Germany (2017–2030)

Table 8: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Germany (2017–2030)

Table 9: Total Diagnosed Prevalent Population of Hunter Syndrome in France (2017–2030)

Table 10: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in France (2017–2030)

Table 11: Total Diagnosed Prevalent Population of Hunter Syndrome in Italy (2017–2030)

Table 12: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Italy (2017–2030)

Table 13: Total Diagnosed Prevalent Population of Hunter Syndrome in Spain (2017–2030)

Table 14: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Spain (2017–2030)

Table 15: Total Diagnosed Prevalent Population of Hunter Syndrome in the United Kingdom (2017–2030)

Table 16: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the United Kingdom (2017–2030)

Table 17: Total Diagnosed Prevalent Population Prevalence of Hunter Syndrome in Japan (2017–2030)

Table 18: Severity-based Diagnosed Prevalent Population of Hunter Syndrome in Japan (2017–2030)

Table 19: Suggested evaluations for patients with Hunter syndrome

Table 20: Comparison of emerging drugs under development

Table 21: Clinical Trial Description

Table 22: Clinical Trial Description

Table 23: Clinical Trial Description

Table 24: Clinical Trial Description

Table 25: Clinical Trial Description

Table 26: Clinical Trial Description

Table 27: 7 Major Market Size of Hunter Syndrome in USD Million (2017–2030)

Table 28: Market Size of Hunter Syndrome in the United States, in USD Million (2017–2030)

Table 29: Market size of Hunter Syndrome by therapies in the United States, in USD Million (2017–2030)

Table 30: Market Size of Hunter Syndrome in Germany, in USD Million (2017–2030)

Table 31: Market size of Hunter Syndrome by therapies in Germany, in USD Million (2017–2030)

Table 32: Market Size of Hunter Syndrome in France, in USD Million (2017–2030)

Table 33: Market size of Hunter Syndrome by therapies in France, in USD Million (2017–2030)

Table 34: Market Size of Hunter Syndrome in Italy, in USD Million (2017–2030)

Table 35: Market size of Hunter Syndrome by therapies in Italy, in USD Million (2017–2030)

Table 36: Market Size of Hunter Syndrome in Spain, in USD Million (2017–2030)

Table 37: Market size of Hunter Syndrome by therapies in Spain, in USD Million (2017–2030)

Table 38: Market Size of Hunter Syndrome in the United Kingdom, in USD Million (2017–2030)

Table 39: Market size of Hunter Syndrome by therapies in the United Kingdom, in USD Million (2017–2030)

Table 40: Market Size of Hunter Syndrome in Japan, in USD Million (2017–2030)

Table 41: Market size of Hunter Syndrome by therapies in Japan, in USD Million (2017–2030)