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2020
Hunter Syndrome
Market Insights, Epidemiology, and Market
Forecast- 2030
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Healthcare
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Key Insights
The Report contains:
Research
Analysis
Pipeline
Valuation
Market
Analysis
Research
Analysis
Pipeline
Valuation
Market
Analysis
Epidemiology
Forecast
Research
Analysis
Pipeline
Valuation
Market
Analysis
Epidemiology
Forecast
Market size by
therapies
Pipeline analysis across different
stages of development (Phase
III), different emerging trends,
and comparative analysis of
pipeline products with detailed
clinical profiles, key cross-
competition, launch date along
with product development
activities
Detailed historical and
forecasted market covering
the United States, EU5, and
Japan, from 2017 to 2030
Overview of pathophysiology,
various diagnostic approaches
and treatment algorithm
including detailed chapters for
marketed products and
emerging therapies
Historical as well as forecasted
epidemiology in the United
States, EU5, and Japan from
2017 to 2030
Detailed Market size by therapies, covering
the United States, EU5 (Germany, France,
Italy, Spain, and the United Kingdom), and
Japan, from 2017 to 2030
Research
Analysis
Pipeline
Valuation
Market
Analysis
Epidemiology
Forecast
Market size by
therapies
Insightful Reviews
on Market Drivers &
Market Barriers
Strategic analysis of
Treatment options &
Emerging Therapies
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The report provides an understanding of the
epidemiology and changing market dynamics of Hunter
Syndrome in the seven major pharmaceutical markets
for the period 2017–2030
Report Introduction
DelveInsight’s “Hunter Syndrome -
Market Insights, Epidemiology &
Market Forecast-2030” report
provides an overview of the disease
and market size of Hunter Syndrome
for the seven major pharmaceutical
markets i.e., the United States, EU5
(Germany, France, Italy, Spain, and the
United Kingdom), and Japan. This
report covers the various treatment
practices, and Hunter Syndrome
forecasted epidemiology from 2020 to
2030, segmented by the seven major
markets.
Key Strengths
❖ 11 Year Forecast
❖ 7MM Coverage
❖ Epidemiology Segmentation
❖ Drugs Uptake
❖ Highly Analyzed Market
❖ Key Cross Competition
❖ Market Size by Country
❖ Market Size by Therapy
Key Assessments
❖ Current Treatment Practices
❖ Unmet Needs
❖ Emerging Drug Profiles
❖ Market Attractiveness
✓ The report covers the descriptive overview of Hunter Syndrome,
explaining its causes and currently available therapies.
✓ Comprehensive insight has been provided into the epidemiology of
the Hunter Syndrome and its treatment in the 7MM.
✓ Additionally, an all-inclusive account of both the current and
emerging therapies for Hunter Syndrome is provided, along with
the assessment of new therapies, which will have an impact on the
current treatment landscape.
✓ A detailed review of historical and forecasted Hunter Syndrome
market 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 Hunter Syndrome
market.
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Epidemiology Insights
• According to the National Organization for Rare Disorders, MPS II occurs in approximately 1 in 100,000 to 1 in
170,000 male births.
• As per the study published by Maurizio Scarpa et al., titled, “Mucopolysaccharidosis type II: European
recommendations for the diagnosis and multidisciplinary management of a rare disease” MPS II is one of the
most common mucopolysaccharidoses, with an estimated prevalence of 1 in 140,000-156,000 live births in
Europe.
• According to the study by Shaukat Khan et al., “In Japan, the highest birth prevalence was 0.84 for MPS II,
accounting for 55% of all MPS. MPS I, III, and IV accounted for 15, 16, and 10%, respectively.”
• Recent studies from Germany and the Netherlands report an overall incidence of 1 case in 140,000-330,000
live births, and, more specifically, 1 case per 77,000 male births.
Hunter Syndrome, also known as Mucopolysaccharidosis type II (MPS II), I2S deficiency and Iduronate 2-
sulfatase deficiency. It is an inherited disorder of carbohydrate metabolism that occurs almost exclusively
in males. MPS II is caused by mutations in the IDS gene and is inherited in an X-linked manner. Females
are generally unaffected carriers of this condition.
As per National MPS Society, Hunter syndrome takes its name from Charles Hunter, the professor of
medicine in Manitoba, Canada, who first described two brothers with the disease in 1917. MPS II has a
wide range of symptoms that vary in severity and can be managed and treated with enzyme replacement
therapies. There is no cure for MPS II and it affects 1 in 100,000 to 1 in 170,000 primarily males.
It is characterized by distinctive facial features, a large head, hydrocephalus, enlargement of the liver and
spleen (hepatosplenomegaly), umbilical or inguinal hernia, and hearing loss. Individuals with this condition
may additionally have joint deformities and heart abnormalities involving the valves.
At birth, individuals with MPS II do not display any features of the condition. Between ages 2 and 4, they
develop full lips, large rounded cheeks, a broad nose, and an enlarged tongue (macroglossia). The vocal
cords also gets enlarged, which results in a deep, hoarse voice. Narrowing of the airway causes frequent
upper respiratory infections and short pauses in breathing during sleep (sleep apnea). As the disorder
progresses, individuals need medical assistance to keep their airway open.
Hunter Syndrome – Epidemiology by Gender
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Epidemiology Segmentation-7MM
1
3
2
2
Total Prevalent Cases of Hunter Syndrome
(2017–2030)
Severity Specific Prevalence of Hunter Syndrome [Mild and
Severe Form] (2017–2030)
Diagnosed and Treatable Population of Hunter Syndrome
(2017–2030)
There are two types of MPS II, called the severe and
mild types. While both types affect many different
organs and tissues as described above, people with
severe MPS II also experience a decline in intellectual
function and a more rapid disease progression.
Individuals with the severe form begin to lose basic
functional skills (developmentally regress) between the
ages of 6 and 8. The life expectancy of these individuals
is 10-20 years.
Individuals with mild MPS II also have a shortened
lifespan, but they typically live into adulthood and their
intelligence is not affected. Heart disease and airway
obstruction are major causes of death in people with
both types of MPS II.
Severity specific prevalence of
Hunter Syndrome
Mild Form Severe Form
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Graphical Representation of Hunter Syndrome Epidemiology
Total Prevalent Cases of Hunter Syndrome in the 7MM (2017–2030)
Diagnosed and Treatable Cases of Hunter Syndrome in 7MM (2017–2030)
2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
PopulationinNumbers(N)
Total Prevalent Cases in 7MM
United States France Italy Spain Germany United Kingdom Japan
2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
PopulationinNumbers(N)
Diagnosed and Treatable Cases
Diagnosed Cases Treated Cases
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Market Outlook: Hunter Syndrome
The treatment of MPS II disease is multi-disciplinary and is best treated by a team of specialists, who have
knowledge about the disease, and who can offer supportive and symptomatic care. Care is directed toward
relieving the symptoms of the disease. Enzyme replacement therapy (ERT) is now available for MPS II and
this treatment ameliorates the somatic features of the disorder but does not treat the central nervous
system.
The ERT listed below has been approved by the Food and Drug Administration (FDA) as orphan products in
2006 for the treatment of this condition:
• Idursulfase (Brand name: Elaprase) – It is sponsored by Shire (now Takeda), a company that acquired
the original developer of the drug, TKT Therapeutics, in 2005.
FDA-approved indication: Hunter syndrome (mucopolysaccharidosis II, MPS II). Idursulfase has been
shown to improve walking capacity in these patients.
Idursulfase is still in phase II/III for Mucopolysaccharidosis II (in adolescents, in children) in Spain, USA,
Argentina, Mexico, United Kingdom, Australia, Canada, Colombia and France.
Other treatments of MPS II are symptomatic and supportive. These management interventions commonly
include developmental, occupational, and physical therapy. Hernias and joint contractures may be
corrected by surgery. Tonsillectomy and adenoidectomy (surgical removal of both the tonsils and the
adenoids) can be used to alleviate breathing and swallowing problems. Positive pressure ventilation (CPAP
or tracheostomy) can be used to apply mild air pressure to keep the airway open for proper breathing.
Carpal tunnel release can treat carpal tunnel syndrome. Surgical implantation of a ventricular shunt may
be used to treat possible hydrocephalus. Hearing devices may be prescribed to treat hearing loss.
Developmental, physical, and occupational therapy can be helpful. Genetic counseling is recommended for
families that have a child with MPS II.
Alternatives such as stem cell transplantation (STC) using the umbilical cord blood, peripheral blood
hematopoietic cells or bone marrow have also been used, but they appear to offer limited clinical benefits
in patients with this disease and have been associated with a serious risk of morbidity and mortality. Though
enzyme replacement therapy by idursulfase may improve certain symptoms, it does not cross the blood-
brain barrier (BBB) and will not alleviate neurological symptoms. Therefore, there is a significant unmet
need for a more efficacious therapy which is capable of crossing BBB.
To overcome these challenges researchers have focused on the development of well-tolerated therapies
that can cross the blood-brain barrier. One approach that has been explored is the infusion of ERT into the
cerebrospinal fluid, thereby enabling widespread distribution throughout the CNS. Other areas of research
include the use of pharmacological chaperones, gene therapy, and substrate reduction therapy. Also, the
unmet needs include lack of curative treatment for MPS II, lack of accurate diagnostic tests, etc.
Market Insights
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The Hunter Syndrome market is expected to experience a positive growth in the coming years owing to the
already prescribed product along with the launch of distinctive emerging therapies in the coming years.
Additionally, due to the lack of therapies which effectively stop or slow the disease progression a potential
opportunity exists for the development and launch of novel therapies.
Region-wise Market Size of Hunter Syndrome (2017–2030)
2017
US
DE
FR
IT
ESP
UK
JP
2030
US
DE
FR
IT
ESP
UK
JP
Current treatment in Hunter syndrome
landscape has only one approved
Enzyme Replacement Therapy, Stem
Cell Transplantation and other off-label
therapies
The market size of Hunter
syndrome is expected to
increase at a significant growth
during the study period
[2017–2030]
Expected launch of JR-141 (JCR Pharmaceuticals)
and many others for treating Hunter syndrome shall
fuel the growth of the market
Many clinical trials are evaluating potential
treatments enrolling people with Hunter
syndrome. Hence, increasing R&D activities, and
positive results of these trials shall fuel the
therapeutic market size of Hunter syndrome
The therapies under development are focused
on novel approaches to treat/improve the
disease condition
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Graphical Representation of Hunter Syndrome Market Size in 7MM
Total Market Size of Hunter Syndrome in the 7MM (2017–2030)
Market Size of Hunter Syndrome in the United States (2017–2030)
2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
USD,Million
Total Market Size of Hunter Syndrome (INDICATIVE)
USA UK Germany France Spain Italy Japan
2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030
Market Size of Hunter Syndrome by Upcoming Therapies
(INDICATIVE)
Therapy 1 Therapy 2 Therapy 3 Therapy 4
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Emerging Drugs Analysis
The dynamics of Hunter syndrome market is anticipated to change in the coming years owing to the
improvement in the diagnosis methodologies, raising awareness of the disease, incremental healthcare
spending across the world, and expected the launch of emerging therapies during the forecast period of
2020–2030. Companies like JCR Pharmaceuticals (JR-141), Green Cross Corporation (GC1111B or
Hunterase), Regenxbio Inc. (RGX-121), and others are working toward the development of new treatment
therapies for Hunter syndrome. While some of the companies have recently shifted their focus toward this
indication, whereas others are expected to create a significant influence on the market size during the
forecast period.
JR-141 (JCR Pharmaceuticals) is a blood-brain-barrier penetrating therapeutic enzyme for Hunter syndrome
currently undergoing one Phase III (Japan) and one Phase II (Brazil) clinical trials. In March 2018, JR-141 was
designated under the SAKIGAKE Designation System in Japan by the Ministry of Health, Labour and Welfare,
and is planning to apply for marketing approval in 2020. It has also received Orphan Drug Designation from
both the EMA and the FDA.
GC1111B or Hunterase developed by Green Cross is the world’s second Hunter syndrome drug
(recombinant iduronate-2-sulfatase) and is a biobetter of Shire’s Elaprase used to treat Hunter syndrome.
The USFDA had also awarded Green Cross, the official Orphan Drug Designation for Hunterase back in
2013. It has already been approved in South Korea and marketed in eight countries in South America and
North Africa. It is currently undergoing a phase II study to determine the safety and efficacy in the United
States. However, there is another Phase III clinical trial going on in Korea. If once approved, it will open a
new paradigm for patients suffering from Hunter Syndrome, offering a distinct therapeutic alternative with
improved efficacy and safety.
Apart from these, Denali Therapeutics’ is developing DNL310 (Iduronate 2-sulfatase) which is a
recombinant enzyme engineered to cross the blood-brain barrier (Denali’s Enzyme Transport Vehicle (ETV)
Technology) and to replace the Iduronate enzyme and treat neuropathic and systemic forms of the disease.
The company will start its Phase I/II trial by June 2020. However, pre-clinical studies with DNL310 in a
relevant disease model demonstrated increased brain uptake and a more robust reduction of downstream
markers of disease in the central nervous system compared to standard ERT. It has also been granted
orphan drug product and rare pediatric disease designation by the USFDA.
Additionally, there are many other potential therapies under trial for MPS II which have shown promising
results during the early phase clinical trials. For example, Sangamo Therapeutics has received an Orphan
Drug Designation and Fast Track designation from the FDA for SB-913 (Phase I/II) as a Genome Editing
Treatment for MPS II. RGX-121 (Regenxbio) has received orphan drug product, rare pediatric disease, and
Fast Track designation from the FDA.
Drug Uptake
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Table of Content to View
1. Report Introduction
2. Hunter Syndrome Market Overview at a Glance
2.1. 7 Major Market Size (%) of Hunter syndrome in 2017
2.2. 7 Major Market Size (%) of Hunter syndrome in 2030
3. Disease Background and Overview: Hunter syndrome
3.1. Introduction
3.2. Causes and Risk Factors
3.3. Pathophysiology
3.4. Signs and Symptoms
3.5. Diagnosis
4. Epidemiology and Patient Population
4.1. Key Findings
4.2. Total Prevalent Population of Hunter syndrome in 7MM [2017–2030]
4.3. Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030]
4.4. Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030]
5. Epidemiology of Hunter syndrome by Countries (2017–2030)
5.1. United States- Epidemiology (2017–2030)
5.1.1. Assumptions and Rationale
5.1.2. Total Prevalent Population of Hunter syndrome in the US [2017–2030]
5.1.3. Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030]
5.1.4. Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030]
5.2. EU-5 - Epidemiology (2017–2030)
5.2.1. Assumptions and Rationale
5.3. Germany-Epidemiology (2017–2030)
5.3.1. Total Prevalent Population of Hunter syndrome in Germany [2017–2030]
5.3.2. Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030]
5.3.3. Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030]
5.4. France-Epidemiology (2017–2030)
5.4.1. Total Prevalent Population of Hunter syndrome in France [2017–2030]
5.4.2. Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030]
5.4.3. Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030]
5.5. Italy-Epidemiology (2017–2030)
5.5.1. Total Prevalent Population of Hunter syndrome in Italy [2017–2030]
5.5.2. Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030]
5.5.3. Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030]
5.6. Spain-Epidemiology (2017–2030)
5.6.1. Total Prevalent Population of Hunter syndrome in Spain [2017–2030]
5.6.2. Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030]
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5.6.3. Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030]
5.7. United Kingdom-Epidemiology (2017–2030)
5.7.1. Total Prevalent Population of Hunter syndrome in the UK [2017–2030]
5.7.2. Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030]
5.7.3. Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030]
5.8. Japan-Epidemiology (2017–2030)
5.8.1. Total Prevalent Population of Hunter syndrome in Japan [2017–2030]
5.8.2. Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030]
5.8.3. Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030]
6. Treatments & Medical Practices
6.1. Treatment Algorithm
6.2. Treatment Guidelines
7. Marketed Products
7.1. Elaprase: Shire
7.1.1. Drug description
7.1.2. Regulatory milestones
7.1.3. Generic Availability
7.1.4. Safety and efficacy
7.1.5. Product Profile
8. Emerging Therapies
8.1. Key Cross Competition
8.2. JR-141: JCR Pharmaceuticals
8.2.1. Product Description
8.2.2. Regulatory Milestones
8.2.3. Clinical Development
8.2.4. Clinical Pipeline Activity
8.2.5. Ongoing Trials Information
8.2.6. Safety and efficacy
8.2.7. Product Profile
8.3. GC1111B or Hunterase: Green Cross Corporation
8.3.1. Product Description
8.3.2. Regulatory Milestones
8.3.3. Clinical Development
8.3.4. Clinical Pipeline Activity
8.3.5. Ongoing Trials Information
8.3.6. Safety and efficacy
8.3.7. Product Profile
8.4. RGX-121: Regenxbio Inc.
8.4.1. Product Description
8.4.2. Regulatory Milestones
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8.4.3. Clinical Development
8.4.4. Clinical Pipeline Activity
8.4.5. Ongoing Trials Information
8.4.6. Safety and efficacy
8.4.7. Product Profile
List to be continued in report…
9. Hunter syndrome Market Size
9.1. Key Findings
9.2. Total 7MM Hunter syndrome Market Analysis
9.3. Overview of Total Hunter syndrome Market
9.4. Market size of Hunter syndrome Market by 7MM (2017–2030)
10. 7MM: Country-Wise Market Analysis
10.1. United States Market Size
10.1.1. Total Market Size of Hunter syndrome
10.1.2. Market Size of Hunter syndrome by Therapies
10.2. Germany Market Size
10.2.1. Total Market Size of Hunter syndrome
10.2.2. Market Size of Hunter syndrome by Therapies
10.3. France Market Size
10.3.1. Total Market Size of Hunter syndrome
10.3.2. Market Size of Hunter syndrome by Therapies
10.4. Italy Market Size
10.4.1. Total Market Size of Hunter syndrome
10.4.2. Market Size of Hunter syndrome by Therapies
10.5. Spain Market Size
10.5.1. Total Market Size of Hunter syndrome
10.5.2. Market Size of Hunter syndrome by Therapies
10.6. United Kingdom Market Size
10.6.1. Total Market Size of Hunter syndrome
10.6.2. Market Size of Hunter syndrome by Therapies
10.7. Japan Market Size
10.7.1. Total Market Size of Hunter syndrome
10.7.2. Market Size of Hunter syndrome by Therapies
11. Market Drivers
12. Market Barriers
13. Report Methodology
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13.1. Sources Used
14. DelveInsight Capabilities
15. Disclaimer
16. About DelveInsight
*Table of Contents is not exhaustive; will be provided in the final report
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List of Tables
Table 1 Total Prevalent Population of Hunter syndrome in 7MM [2017–2030]
Table 2 Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030]
Table 3 Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030]
Table 4 Total Prevalent Population of Hunter syndrome in the US [2017–2030]
Table 5 Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030]
Table 6 Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030]
Table 7 Total Prevalent Population of Hunter syndrome in Germany [2017–2030]
Table 8 Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030]
Table 9 Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030]
Table 10 Total Prevalent Population of Hunter syndrome in France [2017–2030]
Table 11 Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030]
Table 12 Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030]
Table 13 Total Prevalent Population of Hunter syndrome in Italy [2017–2030]
Table 14 Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030]
Table 15 Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030]
Table 16 Total Prevalent Population of Hunter syndrome in Spain [2017–2030]
Table 17 Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030]
Table 18 Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030]
Table 19 Total Prevalent Population of Hunter syndrome in the UK [2017–2030]
Table 20 Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030]
Table 21 Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030]
Table 22 Total Prevalent Population of Hunter syndrome in Japan [2017–2030]
Table 23 Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030]
Table 24 Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030]
Table 25 JR-141, Clinical Trials by Recruitment status, 2020
Table 26 JR-141, Clinical Trials by Zone, 2020
Table 27 GC1111B, Clinical Trials by Recruitment status, 2020
Table 28 GC1111B, Clinical Trials by Zone, 2020
Table 29 RGX-121, Clinical Trials by Recruitment status, 2020
Table 30 RGX-121, Clinical Trials by Zone, 2020
Table 31 Market size of Hunter syndrome Market by 7MM (2017–2030)
Table 32 Region-wise Market size of Hunter syndrome, USD Million (2017–2030)
Table 33 7MM-Market Size by Therapy in USD, Million (2017–2030)
Table 34 United States Market Size of Hunter syndrome in USD, Million (2017–2030)
Table 35 United States Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Table 36 Germany Market Size of Hunter syndrome in USD, Million (2017–2030)
Table 37 Germany Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Table 38 France Market Size of Hunter syndrome in USD, Million (2017–2030)
Table 39 France Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Table 40 Italy Market Size of Hunter syndrome in USD, Million (2017–2030)
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Table 41 Italy Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Table 42 Spain Market Size of Hunter syndrome in USD, Million (2017–2030)
Table 43 Spain Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Table 44 United Kingdom Market Size of Hunter syndrome in USD, Million (2017–2030)
Table 45 United Kingdom Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Table 46 Japan Market Size of Hunter syndrome in USD, Million (2017–2030)
Table 47 Japan Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
*List of Table is not exhaustive; will be provided in the final report
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List of Figures
Figure 1 Total Prevalent Population of Hunter syndrome in 7MM [2017–2030]
Figure 2 Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030]
Figure 3 Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030]
Figure 4 Total Prevalent Population of Hunter syndrome in the US [2017–2030]
Figure 5 Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030]
Figure 6 Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030]
Figure 7 Total Prevalent Population of Hunter syndrome in Germany [2017–2030]
Figure 8 Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030]
Figure 9 Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030]
Figure 10 Total Prevalent Population of Hunter syndrome in France [2017–2030]
Figure 11 Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030]
Figure 12 Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030]
Figure 13 Total Prevalent Population of Hunter syndrome in Italy [2017–2030]
Figure 14 Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030]
Figure 15 Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030]
Figure 16 Total Prevalent Population of Hunter syndrome in Spain [2017–2030]
Figure 17 Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030]
Figure 18 Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030]
Figure 19 Total Prevalent Population of Hunter syndrome in the UK [2017–2030]
Figure 20 Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030]
Figure 21 Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030]
Figure 22 Total Prevalent Population of Hunter syndrome in Japan [2017–2030]
Figure 23 Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030]
Figure 24 Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030]
Figure 25 Unmet Needs
Figure 26 Market size of Hunter syndrome Market by 7MM (2017–2030)
Figure 27 Region-wise Market size of Hunter syndrome, USD Million (2017–2030)
Figure 28 7MM-Market Size by Therapy in USD, Million (2017–2030)
Figure 29 United States Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 30 United States Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Figure 31 Germany Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 32 Germany Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Figure 33 France Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 34 France Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Figure 35 Italy Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 36 Italy Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Figure 37 Spain Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 38 Spain Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Figure 39 United Kingdom Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 40 United Kingdom Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
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Figure 41 Japan Market Size of Hunter syndrome in USD, Million (2017–2030)
Figure 42 Japan Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
Figure 43 Market Drivers
Figure 44 Market Barriers
*List of Figures is not exhaustive; will be provide in the final report
D e l v e I n s i g h t | P a g e | 18
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Appendix
Report Methodology
DelveInsight is a premier source for pharmaceutical and biotechnology sector analysis, delivering
exclusive, trusted commercial insight into industry performance through its proprietary platform.
DelveInsight’s reports are continuously updated and revised. The following research methodology
is followed for all databases and reports.
Coverage
The objective of updating DelveInsight coverage is to ensure that it represents the most up-to-
date vision of the industry possible. The DelveInsight is a fully integrated solution for
comprehensive intelligence on various pharmaceutical products, both in the market and in the
pipeline, across the globe.
Research Design
Secondary Research
The secondary research on internal and external sources is being carried out to source qualitative
and quantitative information relating to each market. The secondary research sources that are
typically referred to include, but are not limited to:
• Company websites, annual reports, financial reports, broker reports, investor
presentations, and SEC Filings Industry trade journals and other literature.
• Internal and external proprietary databases.
• National government documents, statistical databases, and market reports.
• News articles, press releases, and web-casts specific to the companies operating in the
market.
Primary Research
DelveInsight conducts primary research by interacting with the healthcare professionals, doctors,
industry’s experts, and industry participants and commentators in order to validate its data and
analysis. The discussion provides first-hand information on the market size, market trends, growth
trends, competitive landscape, and future outlook, for example.
• It helps in validating and strengthening the secondary research findings.
• It further develops the Analysis Team’s expertise and market understanding.
• An interactive one-stop, fully integrated business intelligence (BI) platform, catering the
need of the global pharmaceuticals market, across all the value chains.
• The comprehensive integration allows for the seamless comparison of the market
dynamics, helps strategic planning, and decision making to stay competitive in the global
pharmaceuticals industry.
• We help our clients to find answers relevant to business decision-making, including
potential market opportunities, competitor assessments, and business environmental
dynamics enhances companies to plan development, trials or process-based research on
their competitor focus.
D e l v e I n s i g h t | P a g e | 19
DelveInsight
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• Forecast Methodology
• DelveInsight Reports are prepared considering different epidemiology and market factors,
keeping past and current trends in focus. Our forecasting models are prepared based on
the Hybrid approach, where we use both top to bottom and bottom to top approaches
and analyze different factors and parameters to forecast the numbers.
•
• We develop a deep understanding by doing systemic literature research for
Epidemiological data around different geographies. We prepare the forecast model based
on different data sources, considering past and current trends from different factors such
as improvements in survival and disease therapy, unmet need, the risk of disease by gender
and age group, population size, diagnose trends, treatment factors, and others.
•
• Our Market Forecast model consider factors such as unmet medical need, drug competitor
factor, patient compliance, product price, marketed drugs calibration based on actual sales
and their impact in different geographies, emerging drugs and their impact, novel agents
analysis based on their results, therapeutic potential, regulatory success, launch dates,
etc., market challenges, drivers and barriers, and other factors.
•
• Our major sources are company annual reports, scientific literature, epidemiology studies
conducted by different agencies, Pub-Med Articles, the country-specific government
released population sources, and others. To keep up with current market trends, we try to
take doctors, KOLs, and industry experts’ opinion through secondary as well as primary
research to come up with the actual market size.
•
• Expert Panel Validation
• DelveInsight uses a panel of experts to cross verify research and forecast methodologies
and drive its analytical content. The panel comprises of marketing managers, product
specialists, international sales managers from pharmaceutical companies; academics from
research universities and consultants from professional services companies.
D e l v e I n s i g h t | P a g e | 20
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DelveInsight Capabilities
Our Consulting Services collaborate with Biotech & Pharmaceutical companies, Venture Capitalist,
R&D units, and API manufacturers to help them in critical business decisions, building commercial
excellence and growing businesses in an increasingly competitive and challenging environment.
DelveInsight delivers strategic CI Inputs to clients for monitoring competition and staying ahead via
our expertise in gathering industry intelligence, leading to better decision-making.
Our in-depth insights help market research and business development teams of global bio-Pharma
companies in understanding the market, identifying and targeting the untapped opportunities.
DelveInsight’s forecasting through epidemiology- and unit-based methodologies help create
flexible and robust models to accommodate key inputs for effective market forecasting.
DelveInsight’s CI team has supported many global clients by delivering CI projects with high-quality
insights, helping improve their business processes, combining primary and secondary research
methods and validating information to provide un-biased insights.
1
2
3
4
5
6
Our subject matter experts help deliver high-quality insights, supporting clients in key decisions like
prioritizing indications, analyzing clinical trials, and analyzing the patents and publications.
Our in-house conference/congress intelligence experts gather information from various websites
of congresses and analyze the data to map the KOLs attending the congresses, for effective
planning and providing an un-biased analysis.
Competitive Intelligence
Market/BD
Assessment Studies
Forecasting
Primary CI
R&D
Analytics
Conference/Congress
Intelligence
D e l v e I n s i g h t | P a g e | 21
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Disclaimer
All Rights Reserved.
No part of this publication may be reproduced, stored in a retrieval
system, or transmitted in any form by any means, electronic,
mechanical, photocopying, recording or otherwise, without the prior
permission of the publisher, DelveInsight.
The facts of this report were believed to be correct at the time of
publication but cannot be guaranteed. Please note that the findings,
conclusions, and recommendations that DelveInsight delivers will be
based on information gathered in good faith from both primary and
secondary sources, whose accuracy we were not always in a position
to guarantee.
As such DelveInsight can accept no liability whatsoever for the actions taken based on any
information that may subsequently prove to be incorrect.
About DelveInsight
DelveInsight is a Business Consultant company and serves as a Knowledge partner across the value
chain of the Pharmaceutical Industry. With the use of proprietary databases and analytical models,
DelveInsight provides cutting-edge market and pipeline analysis and API intelligence across all
therapy areas to the Pharma and biotech sector, helping clients to quantify market events and
evaluate their impact on the valuation of products, portfolios, and companies. Additional
information is available at www.delveinsight.com.
For further information or queries, please contact the Sales Team, DelveInsight
at salessupport@delveinsight.com or info@delveinsight.com
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Hunter syndrome market size, market share and market forecast 2030

  • 1. 2020 Hunter Syndrome Market Insights, Epidemiology, and Market Forecast- 2030
  • 2. D e l v e I n s i g h t | P a g e | 1 DelveInsight Copyright Healthcare Market Research Key Insights The Report contains: Research Analysis Pipeline Valuation Market Analysis Research Analysis Pipeline Valuation Market Analysis Epidemiology Forecast Research Analysis Pipeline Valuation Market Analysis Epidemiology Forecast Market size by therapies Pipeline analysis across different stages of development (Phase III), different emerging trends, and comparative analysis of pipeline products with detailed clinical profiles, key cross- competition, launch date along with product development activities Detailed historical and forecasted market covering the United States, EU5, and Japan, from 2017 to 2030 Overview of pathophysiology, various diagnostic approaches and treatment algorithm including detailed chapters for marketed products and emerging therapies Historical as well as forecasted epidemiology in the United States, EU5, and Japan from 2017 to 2030 Detailed Market size by therapies, covering the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan, from 2017 to 2030 Research Analysis Pipeline Valuation Market Analysis Epidemiology Forecast Market size by therapies Insightful Reviews on Market Drivers & Market Barriers Strategic analysis of Treatment options & Emerging Therapies
  • 3. D e l v e I n s i g h t | P a g e | 2 DelveInsight Copyright Healthcare Market Research The report provides an understanding of the epidemiology and changing market dynamics of Hunter Syndrome in the seven major pharmaceutical markets for the period 2017–2030 Report Introduction DelveInsight’s “Hunter Syndrome - Market Insights, Epidemiology & Market Forecast-2030” report provides an overview of the disease and market size of Hunter Syndrome for the seven major pharmaceutical markets i.e., the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom), and Japan. This report covers the various treatment practices, and Hunter Syndrome forecasted epidemiology from 2020 to 2030, segmented by the seven major markets. Key Strengths ❖ 11 Year Forecast ❖ 7MM Coverage ❖ Epidemiology Segmentation ❖ Drugs Uptake ❖ Highly Analyzed Market ❖ Key Cross Competition ❖ Market Size by Country ❖ Market Size by Therapy Key Assessments ❖ Current Treatment Practices ❖ Unmet Needs ❖ Emerging Drug Profiles ❖ Market Attractiveness ✓ The report covers the descriptive overview of Hunter Syndrome, explaining its causes and currently available therapies. ✓ Comprehensive insight has been provided into the epidemiology of the Hunter Syndrome and its treatment in the 7MM. ✓ Additionally, an all-inclusive account of both the current and emerging therapies for Hunter Syndrome is provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape. ✓ A detailed review of historical and forecasted Hunter Syndrome market 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 Hunter Syndrome market.
  • 4. D e l v e I n s i g h t | P a g e | 3 DelveInsight Copyright Healthcare Market Research Epidemiology Insights • According to the National Organization for Rare Disorders, MPS II occurs in approximately 1 in 100,000 to 1 in 170,000 male births. • As per the study published by Maurizio Scarpa et al., titled, “Mucopolysaccharidosis type II: European recommendations for the diagnosis and multidisciplinary management of a rare disease” MPS II is one of the most common mucopolysaccharidoses, with an estimated prevalence of 1 in 140,000-156,000 live births in Europe. • According to the study by Shaukat Khan et al., “In Japan, the highest birth prevalence was 0.84 for MPS II, accounting for 55% of all MPS. MPS I, III, and IV accounted for 15, 16, and 10%, respectively.” • Recent studies from Germany and the Netherlands report an overall incidence of 1 case in 140,000-330,000 live births, and, more specifically, 1 case per 77,000 male births. Hunter Syndrome, also known as Mucopolysaccharidosis type II (MPS II), I2S deficiency and Iduronate 2- sulfatase deficiency. It is an inherited disorder of carbohydrate metabolism that occurs almost exclusively in males. MPS II is caused by mutations in the IDS gene and is inherited in an X-linked manner. Females are generally unaffected carriers of this condition. As per National MPS Society, Hunter syndrome takes its name from Charles Hunter, the professor of medicine in Manitoba, Canada, who first described two brothers with the disease in 1917. MPS II has a wide range of symptoms that vary in severity and can be managed and treated with enzyme replacement therapies. There is no cure for MPS II and it affects 1 in 100,000 to 1 in 170,000 primarily males. It is characterized by distinctive facial features, a large head, hydrocephalus, enlargement of the liver and spleen (hepatosplenomegaly), umbilical or inguinal hernia, and hearing loss. Individuals with this condition may additionally have joint deformities and heart abnormalities involving the valves. At birth, individuals with MPS II do not display any features of the condition. Between ages 2 and 4, they develop full lips, large rounded cheeks, a broad nose, and an enlarged tongue (macroglossia). The vocal cords also gets enlarged, which results in a deep, hoarse voice. Narrowing of the airway causes frequent upper respiratory infections and short pauses in breathing during sleep (sleep apnea). As the disorder progresses, individuals need medical assistance to keep their airway open. Hunter Syndrome – Epidemiology by Gender
  • 5. D e l v e I n s i g h t | P a g e | 4 DelveInsight Copyright Healthcare Market Research Epidemiology Segmentation-7MM 1 3 2 2 Total Prevalent Cases of Hunter Syndrome (2017–2030) Severity Specific Prevalence of Hunter Syndrome [Mild and Severe Form] (2017–2030) Diagnosed and Treatable Population of Hunter Syndrome (2017–2030) There are two types of MPS II, called the severe and mild types. While both types affect many different organs and tissues as described above, people with severe MPS II also experience a decline in intellectual function and a more rapid disease progression. Individuals with the severe form begin to lose basic functional skills (developmentally regress) between the ages of 6 and 8. The life expectancy of these individuals is 10-20 years. Individuals with mild MPS II also have a shortened lifespan, but they typically live into adulthood and their intelligence is not affected. Heart disease and airway obstruction are major causes of death in people with both types of MPS II. Severity specific prevalence of Hunter Syndrome Mild Form Severe Form
  • 6. D e l v e I n s i g h t | P a g e | 5 DelveInsight Copyright Healthcare Market Research Graphical Representation of Hunter Syndrome Epidemiology Total Prevalent Cases of Hunter Syndrome in the 7MM (2017–2030) Diagnosed and Treatable Cases of Hunter Syndrome in 7MM (2017–2030) 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 PopulationinNumbers(N) Total Prevalent Cases in 7MM United States France Italy Spain Germany United Kingdom Japan 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 PopulationinNumbers(N) Diagnosed and Treatable Cases Diagnosed Cases Treated Cases
  • 7. D e l v e I n s i g h t | P a g e | 6 DelveInsight Copyright Healthcare Market Research Market Outlook: Hunter Syndrome The treatment of MPS II disease is multi-disciplinary and is best treated by a team of specialists, who have knowledge about the disease, and who can offer supportive and symptomatic care. Care is directed toward relieving the symptoms of the disease. Enzyme replacement therapy (ERT) is now available for MPS II and this treatment ameliorates the somatic features of the disorder but does not treat the central nervous system. The ERT listed below has been approved by the Food and Drug Administration (FDA) as orphan products in 2006 for the treatment of this condition: • Idursulfase (Brand name: Elaprase) – It is sponsored by Shire (now Takeda), a company that acquired the original developer of the drug, TKT Therapeutics, in 2005. FDA-approved indication: Hunter syndrome (mucopolysaccharidosis II, MPS II). Idursulfase has been shown to improve walking capacity in these patients. Idursulfase is still in phase II/III for Mucopolysaccharidosis II (in adolescents, in children) in Spain, USA, Argentina, Mexico, United Kingdom, Australia, Canada, Colombia and France. Other treatments of MPS II are symptomatic and supportive. These management interventions commonly include developmental, occupational, and physical therapy. Hernias and joint contractures may be corrected by surgery. Tonsillectomy and adenoidectomy (surgical removal of both the tonsils and the adenoids) can be used to alleviate breathing and swallowing problems. Positive pressure ventilation (CPAP or tracheostomy) can be used to apply mild air pressure to keep the airway open for proper breathing. Carpal tunnel release can treat carpal tunnel syndrome. Surgical implantation of a ventricular shunt may be used to treat possible hydrocephalus. Hearing devices may be prescribed to treat hearing loss. Developmental, physical, and occupational therapy can be helpful. Genetic counseling is recommended for families that have a child with MPS II. Alternatives such as stem cell transplantation (STC) using the umbilical cord blood, peripheral blood hematopoietic cells or bone marrow have also been used, but they appear to offer limited clinical benefits in patients with this disease and have been associated with a serious risk of morbidity and mortality. Though enzyme replacement therapy by idursulfase may improve certain symptoms, it does not cross the blood- brain barrier (BBB) and will not alleviate neurological symptoms. Therefore, there is a significant unmet need for a more efficacious therapy which is capable of crossing BBB. To overcome these challenges researchers have focused on the development of well-tolerated therapies that can cross the blood-brain barrier. One approach that has been explored is the infusion of ERT into the cerebrospinal fluid, thereby enabling widespread distribution throughout the CNS. Other areas of research include the use of pharmacological chaperones, gene therapy, and substrate reduction therapy. Also, the unmet needs include lack of curative treatment for MPS II, lack of accurate diagnostic tests, etc. Market Insights
  • 8. D e l v e I n s i g h t | P a g e | 7 DelveInsight Copyright Healthcare Market Research The Hunter Syndrome market is expected to experience a positive growth in the coming years owing to the already prescribed product along with the launch of distinctive emerging therapies in the coming years. Additionally, due to the lack of therapies which effectively stop or slow the disease progression a potential opportunity exists for the development and launch of novel therapies. Region-wise Market Size of Hunter Syndrome (2017–2030) 2017 US DE FR IT ESP UK JP 2030 US DE FR IT ESP UK JP Current treatment in Hunter syndrome landscape has only one approved Enzyme Replacement Therapy, Stem Cell Transplantation and other off-label therapies The market size of Hunter syndrome is expected to increase at a significant growth during the study period [2017–2030] Expected launch of JR-141 (JCR Pharmaceuticals) and many others for treating Hunter syndrome shall fuel the growth of the market Many clinical trials are evaluating potential treatments enrolling people with Hunter syndrome. Hence, increasing R&D activities, and positive results of these trials shall fuel the therapeutic market size of Hunter syndrome The therapies under development are focused on novel approaches to treat/improve the disease condition
  • 9. D e l v e I n s i g h t | P a g e | 8 DelveInsight Copyright Healthcare Market Research Graphical Representation of Hunter Syndrome Market Size in 7MM Total Market Size of Hunter Syndrome in the 7MM (2017–2030) Market Size of Hunter Syndrome in the United States (2017–2030) 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 USD,Million Total Market Size of Hunter Syndrome (INDICATIVE) USA UK Germany France Spain Italy Japan 2017 2018 2019 2020 2021 2022 2023 2024 2025 2026 2027 2028 2029 2030 Market Size of Hunter Syndrome by Upcoming Therapies (INDICATIVE) Therapy 1 Therapy 2 Therapy 3 Therapy 4
  • 10. D e l v e I n s i g h t | P a g e | 9 DelveInsight Copyright Healthcare Market Research Emerging Drugs Analysis The dynamics of Hunter syndrome market is anticipated to change in the coming years owing to the improvement in the diagnosis methodologies, raising awareness of the disease, incremental healthcare spending across the world, and expected the launch of emerging therapies during the forecast period of 2020–2030. Companies like JCR Pharmaceuticals (JR-141), Green Cross Corporation (GC1111B or Hunterase), Regenxbio Inc. (RGX-121), and others are working toward the development of new treatment therapies for Hunter syndrome. While some of the companies have recently shifted their focus toward this indication, whereas others are expected to create a significant influence on the market size during the forecast period. JR-141 (JCR Pharmaceuticals) is a blood-brain-barrier penetrating therapeutic enzyme for Hunter syndrome currently undergoing one Phase III (Japan) and one Phase II (Brazil) clinical trials. In March 2018, JR-141 was designated under the SAKIGAKE Designation System in Japan by the Ministry of Health, Labour and Welfare, and is planning to apply for marketing approval in 2020. It has also received Orphan Drug Designation from both the EMA and the FDA. GC1111B or Hunterase developed by Green Cross is the world’s second Hunter syndrome drug (recombinant iduronate-2-sulfatase) and is a biobetter of Shire’s Elaprase used to treat Hunter syndrome. The USFDA had also awarded Green Cross, the official Orphan Drug Designation for Hunterase back in 2013. It has already been approved in South Korea and marketed in eight countries in South America and North Africa. It is currently undergoing a phase II study to determine the safety and efficacy in the United States. However, there is another Phase III clinical trial going on in Korea. If once approved, it will open a new paradigm for patients suffering from Hunter Syndrome, offering a distinct therapeutic alternative with improved efficacy and safety. Apart from these, Denali Therapeutics’ is developing DNL310 (Iduronate 2-sulfatase) which is a recombinant enzyme engineered to cross the blood-brain barrier (Denali’s Enzyme Transport Vehicle (ETV) Technology) and to replace the Iduronate enzyme and treat neuropathic and systemic forms of the disease. The company will start its Phase I/II trial by June 2020. However, pre-clinical studies with DNL310 in a relevant disease model demonstrated increased brain uptake and a more robust reduction of downstream markers of disease in the central nervous system compared to standard ERT. It has also been granted orphan drug product and rare pediatric disease designation by the USFDA. Additionally, there are many other potential therapies under trial for MPS II which have shown promising results during the early phase clinical trials. For example, Sangamo Therapeutics has received an Orphan Drug Designation and Fast Track designation from the FDA for SB-913 (Phase I/II) as a Genome Editing Treatment for MPS II. RGX-121 (Regenxbio) has received orphan drug product, rare pediatric disease, and Fast Track designation from the FDA. Drug Uptake
  • 11. D e l v e I n s i g h t | P a g e | 10 DelveInsight Copyright Healthcare Market Research Table of Content to View 1. Report Introduction 2. Hunter Syndrome Market Overview at a Glance 2.1. 7 Major Market Size (%) of Hunter syndrome in 2017 2.2. 7 Major Market Size (%) of Hunter syndrome in 2030 3. Disease Background and Overview: Hunter syndrome 3.1. Introduction 3.2. Causes and Risk Factors 3.3. Pathophysiology 3.4. Signs and Symptoms 3.5. Diagnosis 4. Epidemiology and Patient Population 4.1. Key Findings 4.2. Total Prevalent Population of Hunter syndrome in 7MM [2017–2030] 4.3. Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030] 4.4. Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030] 5. Epidemiology of Hunter syndrome by Countries (2017–2030) 5.1. United States- Epidemiology (2017–2030) 5.1.1. Assumptions and Rationale 5.1.2. Total Prevalent Population of Hunter syndrome in the US [2017–2030] 5.1.3. Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030] 5.1.4. Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030] 5.2. EU-5 - Epidemiology (2017–2030) 5.2.1. Assumptions and Rationale 5.3. Germany-Epidemiology (2017–2030) 5.3.1. Total Prevalent Population of Hunter syndrome in Germany [2017–2030] 5.3.2. Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030] 5.3.3. Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030] 5.4. France-Epidemiology (2017–2030) 5.4.1. Total Prevalent Population of Hunter syndrome in France [2017–2030] 5.4.2. Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030] 5.4.3. Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030] 5.5. Italy-Epidemiology (2017–2030) 5.5.1. Total Prevalent Population of Hunter syndrome in Italy [2017–2030] 5.5.2. Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030] 5.5.3. Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030] 5.6. Spain-Epidemiology (2017–2030) 5.6.1. Total Prevalent Population of Hunter syndrome in Spain [2017–2030] 5.6.2. Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030]
  • 12. D e l v e I n s i g h t | P a g e | 11 DelveInsight Copyright Healthcare Market Research 5.6.3. Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030] 5.7. United Kingdom-Epidemiology (2017–2030) 5.7.1. Total Prevalent Population of Hunter syndrome in the UK [2017–2030] 5.7.2. Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030] 5.7.3. Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030] 5.8. Japan-Epidemiology (2017–2030) 5.8.1. Total Prevalent Population of Hunter syndrome in Japan [2017–2030] 5.8.2. Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030] 5.8.3. Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030] 6. Treatments & Medical Practices 6.1. Treatment Algorithm 6.2. Treatment Guidelines 7. Marketed Products 7.1. Elaprase: Shire 7.1.1. Drug description 7.1.2. Regulatory milestones 7.1.3. Generic Availability 7.1.4. Safety and efficacy 7.1.5. Product Profile 8. Emerging Therapies 8.1. Key Cross Competition 8.2. JR-141: JCR Pharmaceuticals 8.2.1. Product Description 8.2.2. Regulatory Milestones 8.2.3. Clinical Development 8.2.4. Clinical Pipeline Activity 8.2.5. Ongoing Trials Information 8.2.6. Safety and efficacy 8.2.7. Product Profile 8.3. GC1111B or Hunterase: Green Cross Corporation 8.3.1. Product Description 8.3.2. Regulatory Milestones 8.3.3. Clinical Development 8.3.4. Clinical Pipeline Activity 8.3.5. Ongoing Trials Information 8.3.6. Safety and efficacy 8.3.7. Product Profile 8.4. RGX-121: Regenxbio Inc. 8.4.1. Product Description 8.4.2. Regulatory Milestones
  • 13. D e l v e I n s i g h t | P a g e | 12 DelveInsight Copyright Healthcare Market Research 8.4.3. Clinical Development 8.4.4. Clinical Pipeline Activity 8.4.5. Ongoing Trials Information 8.4.6. Safety and efficacy 8.4.7. Product Profile List to be continued in report… 9. Hunter syndrome Market Size 9.1. Key Findings 9.2. Total 7MM Hunter syndrome Market Analysis 9.3. Overview of Total Hunter syndrome Market 9.4. Market size of Hunter syndrome Market by 7MM (2017–2030) 10. 7MM: Country-Wise Market Analysis 10.1. United States Market Size 10.1.1. Total Market Size of Hunter syndrome 10.1.2. Market Size of Hunter syndrome by Therapies 10.2. Germany Market Size 10.2.1. Total Market Size of Hunter syndrome 10.2.2. Market Size of Hunter syndrome by Therapies 10.3. France Market Size 10.3.1. Total Market Size of Hunter syndrome 10.3.2. Market Size of Hunter syndrome by Therapies 10.4. Italy Market Size 10.4.1. Total Market Size of Hunter syndrome 10.4.2. Market Size of Hunter syndrome by Therapies 10.5. Spain Market Size 10.5.1. Total Market Size of Hunter syndrome 10.5.2. Market Size of Hunter syndrome by Therapies 10.6. United Kingdom Market Size 10.6.1. Total Market Size of Hunter syndrome 10.6.2. Market Size of Hunter syndrome by Therapies 10.7. Japan Market Size 10.7.1. Total Market Size of Hunter syndrome 10.7.2. Market Size of Hunter syndrome by Therapies 11. Market Drivers 12. Market Barriers 13. Report Methodology
  • 14. D e l v e I n s i g h t | P a g e | 13 DelveInsight Copyright Healthcare Market Research 13.1. Sources Used 14. DelveInsight Capabilities 15. Disclaimer 16. About DelveInsight *Table of Contents is not exhaustive; will be provided in the final report
  • 15. D e l v e I n s i g h t | P a g e | 14 DelveInsight Copyright Healthcare Market Research List of Tables Table 1 Total Prevalent Population of Hunter syndrome in 7MM [2017–2030] Table 2 Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030] Table 3 Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030] Table 4 Total Prevalent Population of Hunter syndrome in the US [2017–2030] Table 5 Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030] Table 6 Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030] Table 7 Total Prevalent Population of Hunter syndrome in Germany [2017–2030] Table 8 Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030] Table 9 Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030] Table 10 Total Prevalent Population of Hunter syndrome in France [2017–2030] Table 11 Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030] Table 12 Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030] Table 13 Total Prevalent Population of Hunter syndrome in Italy [2017–2030] Table 14 Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030] Table 15 Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030] Table 16 Total Prevalent Population of Hunter syndrome in Spain [2017–2030] Table 17 Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030] Table 18 Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030] Table 19 Total Prevalent Population of Hunter syndrome in the UK [2017–2030] Table 20 Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030] Table 21 Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030] Table 22 Total Prevalent Population of Hunter syndrome in Japan [2017–2030] Table 23 Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030] Table 24 Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030] Table 25 JR-141, Clinical Trials by Recruitment status, 2020 Table 26 JR-141, Clinical Trials by Zone, 2020 Table 27 GC1111B, Clinical Trials by Recruitment status, 2020 Table 28 GC1111B, Clinical Trials by Zone, 2020 Table 29 RGX-121, Clinical Trials by Recruitment status, 2020 Table 30 RGX-121, Clinical Trials by Zone, 2020 Table 31 Market size of Hunter syndrome Market by 7MM (2017–2030) Table 32 Region-wise Market size of Hunter syndrome, USD Million (2017–2030) Table 33 7MM-Market Size by Therapy in USD, Million (2017–2030) Table 34 United States Market Size of Hunter syndrome in USD, Million (2017–2030) Table 35 United States Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 36 Germany Market Size of Hunter syndrome in USD, Million (2017–2030) Table 37 Germany Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 38 France Market Size of Hunter syndrome in USD, Million (2017–2030) Table 39 France Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 40 Italy Market Size of Hunter syndrome in USD, Million (2017–2030)
  • 16. D e l v e I n s i g h t | P a g e | 15 DelveInsight Copyright Healthcare Market Research Table 41 Italy Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 42 Spain Market Size of Hunter syndrome in USD, Million (2017–2030) Table 43 Spain Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 44 United Kingdom Market Size of Hunter syndrome in USD, Million (2017–2030) Table 45 United Kingdom Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Table 46 Japan Market Size of Hunter syndrome in USD, Million (2017–2030) Table 47 Japan Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) *List of Table is not exhaustive; will be provided in the final report
  • 17. D e l v e I n s i g h t | P a g e | 16 DelveInsight Copyright Healthcare Market Research List of Figures Figure 1 Total Prevalent Population of Hunter syndrome in 7MM [2017–2030] Figure 2 Severity-Specific Prevalent Population of Hunter syndrome in 7MM [2017–2030] Figure 3 Diagnosed and Treatable Population of Hunter syndrome in 7MM [2017–2030] Figure 4 Total Prevalent Population of Hunter syndrome in the US [2017–2030] Figure 5 Severity-Specific Prevalent Population of Hunter syndrome in the US [2017–2030] Figure 6 Diagnosed and Treatable Population of Hunter syndrome in the US [2017–2030] Figure 7 Total Prevalent Population of Hunter syndrome in Germany [2017–2030] Figure 8 Severity-Specific Prevalent Population of Hunter syndrome in Germany [2017–2030] Figure 9 Diagnosed and Treatable Population of Hunter syndrome in Germany [2017–2030] Figure 10 Total Prevalent Population of Hunter syndrome in France [2017–2030] Figure 11 Severity-Specific Prevalent Population of Hunter syndrome in France [2017–2030] Figure 12 Diagnosed and Treatable Population of Hunter syndrome in France [2017–2030] Figure 13 Total Prevalent Population of Hunter syndrome in Italy [2017–2030] Figure 14 Severity-Specific Prevalent Population of Hunter syndrome in Italy [2017–2030] Figure 15 Diagnosed and Treatable Population of Hunter syndrome in Italy [2017–2030] Figure 16 Total Prevalent Population of Hunter syndrome in Spain [2017–2030] Figure 17 Severity-Specific Prevalent Population of Hunter syndrome in Spain [2017–2030] Figure 18 Diagnosed and Treatable Population of Hunter syndrome in Spain [2017–2030] Figure 19 Total Prevalent Population of Hunter syndrome in the UK [2017–2030] Figure 20 Severity-Specific Prevalent Population of Hunter syndrome in the UK [2017–2030] Figure 21 Diagnosed and Treatable Population of Hunter syndrome in the UK [2017–2030] Figure 22 Total Prevalent Population of Hunter syndrome in Japan [2017–2030] Figure 23 Severity-Specific Prevalent Population of Hunter syndrome in Japan [2017–2030] Figure 24 Diagnosed and Treatable Population of Hunter syndrome in Japan [2017–2030] Figure 25 Unmet Needs Figure 26 Market size of Hunter syndrome Market by 7MM (2017–2030) Figure 27 Region-wise Market size of Hunter syndrome, USD Million (2017–2030) Figure 28 7MM-Market Size by Therapy in USD, Million (2017–2030) Figure 29 United States Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 30 United States Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 31 Germany Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 32 Germany Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 33 France Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 34 France Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 35 Italy Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 36 Italy Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 37 Spain Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 38 Spain Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 39 United Kingdom Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 40 United Kingdom Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030)
  • 18. D e l v e I n s i g h t | P a g e | 17 DelveInsight Copyright Healthcare Market Research Figure 41 Japan Market Size of Hunter syndrome in USD, Million (2017–2030) Figure 42 Japan Market Size of Hunter syndrome, by Therapy in USD, Million (2017–2030) Figure 43 Market Drivers Figure 44 Market Barriers *List of Figures is not exhaustive; will be provide in the final report
  • 19. D e l v e I n s i g h t | P a g e | 18 DelveInsight Copyright Healthcare Market Research Appendix Report Methodology DelveInsight is a premier source for pharmaceutical and biotechnology sector analysis, delivering exclusive, trusted commercial insight into industry performance through its proprietary platform. DelveInsight’s reports are continuously updated and revised. The following research methodology is followed for all databases and reports. Coverage The objective of updating DelveInsight coverage is to ensure that it represents the most up-to- date vision of the industry possible. The DelveInsight is a fully integrated solution for comprehensive intelligence on various pharmaceutical products, both in the market and in the pipeline, across the globe. Research Design Secondary Research The secondary research on internal and external sources is being carried out to source qualitative and quantitative information relating to each market. The secondary research sources that are typically referred to include, but are not limited to: • Company websites, annual reports, financial reports, broker reports, investor presentations, and SEC Filings Industry trade journals and other literature. • Internal and external proprietary databases. • National government documents, statistical databases, and market reports. • News articles, press releases, and web-casts specific to the companies operating in the market. Primary Research DelveInsight conducts primary research by interacting with the healthcare professionals, doctors, industry’s experts, and industry participants and commentators in order to validate its data and analysis. The discussion provides first-hand information on the market size, market trends, growth trends, competitive landscape, and future outlook, for example. • It helps in validating and strengthening the secondary research findings. • It further develops the Analysis Team’s expertise and market understanding. • An interactive one-stop, fully integrated business intelligence (BI) platform, catering the need of the global pharmaceuticals market, across all the value chains. • The comprehensive integration allows for the seamless comparison of the market dynamics, helps strategic planning, and decision making to stay competitive in the global pharmaceuticals industry. • We help our clients to find answers relevant to business decision-making, including potential market opportunities, competitor assessments, and business environmental dynamics enhances companies to plan development, trials or process-based research on their competitor focus.
  • 20. D e l v e I n s i g h t | P a g e | 19 DelveInsight Copyright Healthcare Market Research • Forecast Methodology • DelveInsight Reports are prepared considering different epidemiology and market factors, keeping past and current trends in focus. Our forecasting models are prepared based on the Hybrid approach, where we use both top to bottom and bottom to top approaches and analyze different factors and parameters to forecast the numbers. • • We develop a deep understanding by doing systemic literature research for Epidemiological data around different geographies. We prepare the forecast model based on different data sources, considering past and current trends from different factors such as improvements in survival and disease therapy, unmet need, the risk of disease by gender and age group, population size, diagnose trends, treatment factors, and others. • • Our Market Forecast model consider factors such as unmet medical need, drug competitor factor, patient compliance, product price, marketed drugs calibration based on actual sales and their impact in different geographies, emerging drugs and their impact, novel agents analysis based on their results, therapeutic potential, regulatory success, launch dates, etc., market challenges, drivers and barriers, and other factors. • • Our major sources are company annual reports, scientific literature, epidemiology studies conducted by different agencies, Pub-Med Articles, the country-specific government released population sources, and others. To keep up with current market trends, we try to take doctors, KOLs, and industry experts’ opinion through secondary as well as primary research to come up with the actual market size. • • Expert Panel Validation • DelveInsight uses a panel of experts to cross verify research and forecast methodologies and drive its analytical content. The panel comprises of marketing managers, product specialists, international sales managers from pharmaceutical companies; academics from research universities and consultants from professional services companies.
  • 21. D e l v e I n s i g h t | P a g e | 20 DelveInsight Copyright Healthcare Market Research DelveInsight Capabilities Our Consulting Services collaborate with Biotech & Pharmaceutical companies, Venture Capitalist, R&D units, and API manufacturers to help them in critical business decisions, building commercial excellence and growing businesses in an increasingly competitive and challenging environment. DelveInsight delivers strategic CI Inputs to clients for monitoring competition and staying ahead via our expertise in gathering industry intelligence, leading to better decision-making. Our in-depth insights help market research and business development teams of global bio-Pharma companies in understanding the market, identifying and targeting the untapped opportunities. DelveInsight’s forecasting through epidemiology- and unit-based methodologies help create flexible and robust models to accommodate key inputs for effective market forecasting. DelveInsight’s CI team has supported many global clients by delivering CI projects with high-quality insights, helping improve their business processes, combining primary and secondary research methods and validating information to provide un-biased insights. 1 2 3 4 5 6 Our subject matter experts help deliver high-quality insights, supporting clients in key decisions like prioritizing indications, analyzing clinical trials, and analyzing the patents and publications. Our in-house conference/congress intelligence experts gather information from various websites of congresses and analyze the data to map the KOLs attending the congresses, for effective planning and providing an un-biased analysis. Competitive Intelligence Market/BD Assessment Studies Forecasting Primary CI R&D Analytics Conference/Congress Intelligence
  • 22. D e l v e I n s i g h t | P a g e | 21 DelveInsight Copyright Healthcare Market Research Disclaimer All Rights Reserved. No part of this publication may be reproduced, stored in a retrieval system, or transmitted in any form by any means, electronic, mechanical, photocopying, recording or otherwise, without the prior permission of the publisher, DelveInsight. The facts of this report were believed to be correct at the time of publication but cannot be guaranteed. Please note that the findings, conclusions, and recommendations that DelveInsight delivers will be based on information gathered in good faith from both primary and secondary sources, whose accuracy we were not always in a position to guarantee. As such DelveInsight can accept no liability whatsoever for the actions taken based on any information that may subsequently prove to be incorrect. About DelveInsight DelveInsight is a Business Consultant company and serves as a Knowledge partner across the value chain of the Pharmaceutical Industry. With the use of proprietary databases and analytical models, DelveInsight provides cutting-edge market and pipeline analysis and API intelligence across all therapy areas to the Pharma and biotech sector, helping clients to quantify market events and evaluate their impact on the valuation of products, portfolios, and companies. Additional information is available at www.delveinsight.com. For further information or queries, please contact the Sales Team, DelveInsight at salessupport@delveinsight.com or info@delveinsight.com
  • 23. D e l v e I n s i g h t | P a g e | 22 DelveInsight Copyright Healthcare Market Research REPORT PRICE Hunter Syndrome Market Insights, Epidemiology, and Market Forecast-2030 Site License 2-3 Users License Single User License Corporate License USD 20850 USD 13900 USD 10450 USD 6950