Hunter Syndrome Market

DelveInsight’s ‘Hunter Syndrome -Market Insights, Epidemiology and Market Forecast– 2030’ report delivers an in-depth understanding of the Hunter Syndrome, historical and forecasted epidemiology as well as the Hunter Syndrome market trends in the United States, EU5 (Germany, Spain, Italy, France, and United Kingdom) and Japan.

The Hunter Syndrome market report provides current treatment practices, emerging drugs, and market share of the individual therapies, current and forecasted 7MM Hunter Syndrome market size from 2017 to 2030. The report also covers current Hunter Syndrome treatment practice/algorithm, market drivers, market barriers and unmet medical needs to curate the best of the opportunities and assesses the underlying potential of the market.


Geography Covered

• The United States

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

• Japan


Study Period: 2017–2030


Hunter Syndrome Disease Understanding and Treatment Algorithm

Hunter Syndrome Overview

Hunter syndrome is also known as Mucopolysaccharidosis type II (MPS II). It is a condition that affects many different parts of the body and mainly affects males. It is a progressive disorder, but the rate of progression varies among affected individuals. It is a rare, X-linked disorder caused due to a deficiency of the lysosomal enzyme iduronate-2-sulfatase, which plays a major role in the catabolism of glycosaminoglycans (GAG). In patients with mucopolysaccharidosis II, glycosaminoglycans accumulate within tissues and organs, contributing to the signs and symptoms of the disease. Mucopolysaccharidosis II affects multiple organs and physiologic systems and has a variable age of onset and variable rate of progression. Common presenting features include excess urinary glycosaminoglycan excretion, facial dysmorphism, organomegaly, joint stiffness and contractures, pulmonary dysfunction, myocardial enlargement and valvular dysfunction, and neurologic involvement. In patients with neurologic involvement, intelligence is impaired, and death usually occurs in the second decade of life, whereas those patients with minimal or no neurologic involvement may survive into adulthood with normal intellectual development.


At birth, individuals with MPS II do not display any features of the condition. The vast majority of affected individuals are male; however, on rare occasions, heterozygous females’ manifest findings. In those with early progressive disease, CNS involvement (manifest primarily by progressive cognitive deterioration), progressive airway disease, and cardiac disease usually result in death in the first or second decade of life. In those with slowly progressive disease, the CNS is not (or is minimally) affected, although the effect of GAG accumulation on other organ systems may be early progressive to the same degree as in those who have progressive cognitive decline. Survival into the early adult years with normal intelligence is common in the slowly progressing form of the disease. Additional findings in both forms of MPS II include short stature; macrocephaly with or without communicating hydrocephalus, macroglossia, hoarse voice, conductive and sensorineural hearing loss, hepatosplenomegaly, dysostosis multiplex, spinal stenosis, and carpal tunnel syndrome. Generally, between ages 2–4, affected individuals develop full lips, large rounded cheeks, a broad nose, and an enlarged tongue (macroglossia). The vocal cords also enlarge, 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.


There are two types of MPS II, called 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 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.


Hunter Syndrome Diagnosis

The diagnosis of Hunter syndrome is established in a male by identifying the deficient iduronate 2-sulfatase (I2S) enzyme activity in white cells, fibroblasts, or plasma in the presence of normal activity of at least one other sulfatase. Detection of a hemizygous pathogenic variant in IDS confirms the diagnosis in a male with an unusual phenotype or a phenotype that does not match the results of GAG testing. The diagnosis of this indication is usually established in a female with suggestive clinical features by identification of a heterozygous IDS pathogenic variant on molecular genetic testing.

Although the disease is almost exclusively reported in males, rare cases in females also do occur. The diagnosis of MPS II is usually established in a female patient with suggestive clinical features, such as the identification of a heterozygous IDS pathogenic variant on molecular genetic testing.

Molecular genetic testing approaches can include a combination of gene-targeted testing (single-gene testing, multigene panel) and comprehensive genomic testing (exome sequencing, genome sequencing) depending on the phenotype. Gene-targeted testing requires that the clinician determine which gene(s) are likely involved, whereas genomic testing does not.


Hunter Syndrome Treatment

Even with the introduction of ERT, patients with MPS II still require supportive symptomatic treatment from a wide range of specialists. A comprehensive initial assessment of each patient at diagnosis should, therefore, be undertaken, and should be followed by regular reviews. Supportive management and the anticipation of possible complications can greatly improve the quality of life of affected individuals and their families. Family members should be offered genetic counselling, and contact with other affected families, patients, and support groups.


It is now a decade since ERT with intravenous idursulfase (Elaprase), a recombinant form of human iduronate 2-sulfatase, has been approved in the United States and the European Union at a weekly dose of 0.5 mg/kg for the treatment of MPS II. The approval was mainly based on the results from a first trial on individuals with the slowly progressive form of the disease. In the following year several other studies were undertaken to investigate clinical safety and efficacy of ERT; these clearly showed that idursulfase has positive effects on functional capacity (distance walked in six minutes and forced vital capacity), liver and spleen volumes, and urine GAGs excretion. Recently, a 3.5-year independent study determined that long-term use of ERT is similarly effective in young (age 1.6–12 years at the start of ERT) and older individuals (age 12–27 years at the start of ERT). In addition, two recent studies have confirmed ERT efficacy in improving somatic signs and symptoms of the disease in all individuals, including infants younger than age 1 year and individuals with the early progressive MPS II phenotype.


Pretreatment with anti-inflammatory drugs or antihistamines, as is often done for ERT in other conditions, is not suggested on the label for Elaprase; however, if mild or moderate infusion reactions (e.g., dyspnea, urticaria, or systolic blood pressure changes of ≤ 20 mm Hg) cannot be ameliorated by slowing the infusion rate, the addition of treatment one hour before infusion with diphenhydramine and acetaminophen (or ibuprofen) to the regimen usually resolves the problem. Pretreatment can typically be discontinued after 6–10 weeks.

Hematopoietic stem cell transplantation (HSCT) using umbilical cord blood or bone marrow is a potential way of providing sufficient enzyme activity to slow or stop the progression of the disease, however, the use of HSCT is controversial because of the associated high risk of morbidity and mortality. The use of HSCT has been controversial because of limited information regarding the long-term outcomes and the associated high risk of morbidity and mortality.


Hunter Syndrome Epidemiology

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology segmented by Total Diagnosed Prevalent Population of Hunter Syndrome and Severity-based Diagnosed Prevalent Population of Hunter Syndrome in the 7MM market covering the United States, EU5 countries (Germany, France, Italy, Spain, and United Kingdom) and Japan from 2017 to 2030.


Key Findings

This section provides glimpse of the Hunter Syndrome epidemiology in the 7MM.

• The total diagnosed prevalent population of Hunter Syndrome in the 7 major markets was found to be 1,145 in 2017. In case of Hunter Syndrome patients in the United States, the diagnosed prevalent cases were found to be 503 in 2017.

• In the EU5 countries the diagnosed prevalence of Hunter Syndrome was found to be maximum in Germany with 83 cases, followed by the United Kingdom with 68 cases in 2017. While, the least number of cases were found in Spain, in 2017. The trend is expected to increase during the forecast period.

• In Japan, the diagnosed prevalence of Hunter Syndrome was found to be 309 in 2017, resulting into the second highest after the US across the 7MM. MPS II is very common in Asian countries, such as Japan, Korea, China, and Taiwan.


Country Wise- Hunter Syndrome Epidemiology

The epidemiology segment also provides the Hunter Syndrome epidemiology data and findings across the United States, EU5 (Germany, France, Italy, Spain, and the United Kingdom) and Japan.


Hunter Syndrome Drug Chapters

The drug chapter segment of the Hunter Syndrome report encloses the detailed analysis of Hunter Syndrome marketed drugs and mid and late stage pipeline drugs. It also helps to understand the Hunter Syndrome clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details of each included drug and the latest news and press releases.


Hunter Syndrome Marketed Drugs

Elaprase (idursulfase): Takeda

Elaprase with an active pharmaceutical ingredient idursulfase, is a hydrolytic lysosomal glycosaminoglycan (GAG)-specific enzyme indicated for patients with Hunter syndrome (MPS II), which was manufactured by the Shire, but after its acquisition in January 2019, the drug belonged to Takeda. It is known to improve walking capacity in patients 5 years and older with MPS II. Also, it is important to note that Elaprase is the first and only treatment approved for people suffering from Hunter syndrome. It has been designed to replace the natural enzyme, increasing the catabolism of certain accumulated glycosaminoglycans (GAG)—which abnormally accumulate in multiple tissue types in patients with Hunter syndrome. It is generally available as a sterile, aqueous clear to slightly-opalescent colorless solution for intravenous infusion.

In October 2007, Shire received approval from Japan's Ministry of Health, Labour and Welfare for Elaprase, a human enzyme replacement therapy for the treatment of Hunter syndrome, sale and marketing in Japan. As part of an agreement with Genzyme Corporation, Genzyme was accounted to manage sales and distribution of Elaprase in Japan as well as certain other countries in the Asia Pacific region.

In January 2007, the European Commission granted a marketing authorization valid throughout the European Union for Elaprase to treat patients with Hunter syndrome.

In July 2006, the US FDA granted marketing approval for Elaprase, a human enzyme replacement therapy for the treatment of Hunter syndrome, also known as MPS II.

Product detail in the report…


Hunter Syndrome Emerging Drugs

JR-141: JCR Pharmaceuticals

JR-141 is an anti-human transferrin receptor (J-Brain Cargo) antibody fused IDS (iduronate-2-sulfatase), which is expected to cross the blood–brain barrier (BBB). It consists of intact human IDS and anti-human transferrin receptor (hTfR) antibody, which is being developed by JCR Pharmaceuticals. Transferrin is a protein that crosses BBB through a mechanism of receptor-mediated transcytosis, which is utilized in the delivery of JR-141 to the brain. JR-141 is distributed in the brain parenchyma of hTfR knock-in cells after intravenous administration. Additionally, intravenous administration of human IDS fused with anti-mouse TfR antibody has reduced GAG accumulations in both the peripheral tissues and the brains of MPS II patients, leading to significant improvements of neurocognitive deficits. In addition, after administration of JR-141, the heparin sulfate concentrations in the cerebrospinal fluid (CSF) decreases in parallel with those in the brain, indicating that GAGs in CSF can be used as an important biomarker of the pathophysiological activities of neuropathic MPS II.

Currently, the drug is in phase III stage of clinical development for patients with Hunter Syndrome in Japan and is in phase II preparation in Brazil. JCR Pharmaceuticals has received Orphan Drug Designation from both the FDA and the EMA and is expecting to file for marketing approval of JR-141 in 2020 in Japan. However, the company intends to initiate trial of JR-141 in the US and Europe as well in near future.

Product detail in the report…


TAK-609/SHP609/HGT-2310: Takeda

SHP-609 is an investigational formulation of idursulfase administered intrathecally for the treatment of pediatric patients with Hunter syndrome and cognitive impairment. In clinical trials, SHP609 is administered to patients using an intrathecal drug delivery device. SHP609 is administered directly into the cerebrospinal fluid as a means of delivering the drug to the central nervous system.

According to the clinicaltrials.gov, the drug is in phase II/III stage of clinical development for patients with Hunter Syndrome and cognitive impairment, and the Shire is conducting the trial. However, in January 2019, Shire was acquired by Takeda, which has included the drug by the name TAK-609 in its pipeline in phase II clinical developmental trial.

Product detail in the report…


GC1111 (Hunterase): Green Cross Corporation/GC Pharma

Green Cross’s Hunterase drug eases the symptoms of Hunter Syndrome, which is caused by deficiency or absence of the enzyme iduronate-2-sulfatase (IDS), by injecting cell-culture-produced enzyme. This drug reduces the levels of mucopolysaccharidosis excreted through the urine by about 30–40% following treatment. It also leads to an average increase in the 6 min walking distance. Hunterase has shown potentially better safety levels, including proportionately fewer occurrences of adverse drug reactions compared to the reference drug.

In January 2012, Hunterase was approved by the Korean Food & Drug Administration (KFDA) for the treatment of Hunter Syndrome in Korea. It is currently in phase II stage of clinical development for patients with Hunter Syndrome in the United States. However, GC Pharma has entered into a licensing agreement with Clinigen K.K. to commercialize Hunterase in Japan. Also, as per clinicaltrials.gov the drug is in phase III stage of clinical development.

GC Pharma aspires toward increasing Hunterase’s global market share to over 50%, thereby shifting the paradigm on Hunter syndrome treatment. The company also intends to continue to invest in treatments for various other rare diseases, including Fabry disease, to ensure effective treatment and better quality of life for struggling people in Korea and around the world. The company has also submitted a New Drug Application to gain marketing approval for Hunterase in China.

Products detail in the report…


DNL310: Denali Therapeutics

DNL310 is a recombinant form of the iduronate 2-sulfatase (IDS) enzyme engineered to cross the BBB using Denali’s proprietary Enzyme Transport Vehicle technology. DNL310 is intravenously administered and intended to improve overall clinical manifestations of Hunter syndrome, including both peripheral and neurological symptoms, which are not adequately addressed by currently approved therapies.

Denali is planning to initiate a phase I/II stage clinical development of DNL310 for patients with Hunter Syndrome in June 2020.

Products detail in the report…


RGX-121: Regenxbio

RGX-121 is being developed as a novel, one-time, treatment for MPS II, which is directly administered intra-cisternally into the CNS. It includes the NAV AAV9 vector encoding for human IDS (iduronate-2-sulfatase). Delivery of the enzyme that is deficient within cells in the CNS could provide a permanent source of secreted IDS beyond the blood–brain barrier, allowing long-term cross-correction of cells throughout the CNS. This strategy could also provide rapid IDS delivery to the brain, potentially preventing the progression of cognitive deficits that otherwise occurs in MPS II patients.

Product detail in the report…


SB-913: Sangamo Therapeutics

SB-913, a zinc finger nuclease (ZFN) in vivo genome editing product candidate for the treatment of patients with MPS II in development by Sangamo Therapeutics. It is an investigational product candidate, which is designed to insert a normal copy of the IDS gene into a precise location in the DNA of liver cells. The goal of SB-913 treatment is to enable a patient's liver to produce a continuous supply of functional IDS enzyme.

Product detail in the report…


Hunter Syndrome Market Outlook

According to the National Organization for Rare Disorders (NORD), Hunter Syndrome (MPS II) is a rare lysosomal inborn error of metabolism that affects every organ of the body. Though the onset age, disease severity and the rate of progression of the disease vary significantly, initial symptoms and findings associated with the indication usually become apparent in children from 2–4 years of age. Hunter Syndrome typically affects only males as it is an X-linked recessive genetic condition. The disorder occurs in approximately 1 in 100,000 to 1 in 170,000 male births. A few affected females have been observed as well, due to the selective inactivation of the X chromosome inherited by the father.


Treatments include the enzyme replacement therapy along with the new upcoming therapeutic strategies that work on the principle of iduronate 2-sulfatase (I2S) enzyme replacement and gene therapies. The medical management for Hunter Syndrome comprises several aspects such as symptomatic treatment as well as prevention of secondary risks associated with Hunter Syndrome. The current 7MM possess only one approved product namely Elaprase (idursulfase) to treat patients with Hunter Syndrome.


Since, intravenous ERT is unable to cross the BBB, it does not improve or even halt the neurological symptoms and neurodegeneration that occur in patients with neuronopathic forms of Hunter syndrome. However, hematopoietic stem cell transplantation has become more secure and accessible after the development of new protocols and techniques and the creation of bone marrow donor registries and umbilical cord banks. Additionally, the advent of gene therapies, which can target and subsequently edit specific stretches of a genetic code can also prove to be a big competition in near future for ERT based therapies as they are cheaper and once in a life-time therapy.


Key Findings

This section includes a glimpse of the Hunter Syndrome 7MM market.

• The market size of Hunter Syndrome in the seven major markets is USD 376.62 Million in 2017.

• The United States accounts for the largest market size of Hunter Syndrome, in comparison to EU5 (the United Kingdom, Germany, Italy, France, and Spain) and Japan.

• Among the EU5 countries, Germany had the highest market size with USD 20.09 Million in 2017, while Spain had the lowest market size of Hunter Syndrome with USD 12.49 Million in 2017, during the forecast period 2017–2030.

• Japan accounts for the second highest market size in the 7MM during the forecast period 2017–2030, which was USD 74.75 Million in 2017 .


The United States Market Outlook

This section provides the total Hunter Syndrome market size and market size by therapies in the United States.


EU-5 Market Outlook

The total Hunter Syndrome market size and market size by therapies in Germany, France, Italy, Spain, and the United Kingdom are provided in this section.


Japan Market Outlook

The total Hunter Syndrome market size and market size by therapies in Japan are provided.


Hunter Syndrome Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the Hunter Syndrome market or expected to get launched in the market during the study period 2017–2030. The analysis covers Hunter Syndrome market uptake by drugs; patient uptake by therapies; and sales of each drug.  

This helps in understanding the drugs with the most rapid uptake, reasons behind the maximal use of new drugs and allow the comparison of the drugs on the basis of market share and size which again will be useful in investigating factors important in market uptake and in making financial and regulatory decisions.


Hunter Syndrome Development Activities

The report provides insights into different therapeutic candidates in phase III, phase II and phase I/II stage. It also analyzes key players involved in developing targeted therapeutics.


Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing and patent details for Hunter Syndrome emerging therapies.


Competitive Intelligence Analysis

We perform competitive and market Intelligence analysis of the Hunter Syndrome market by using various competitive intelligence tools that include–SWOT analysis, PESTLE analysis, Porter’s five forces, BCG Matrix, Market entry strategies, etc. The inclusion of the analysis entirely depends upon the data availability.


Scope of the Report

• The report covers the descriptive overview of Hunter Syndrome, explaining its causes, signs and symptoms, pathogenesis and currently available therapies.

• Comprehensive insight has been provided into the Hunter Syndrome epidemiology and treatment.

• Additionally, an all-inclusive account of both the current and emerging therapies for Hunter Syndrome are provided, along with the assessment of new therapies, which will have an impact on the current treatment landscape.

• A detailed review of Hunter Syndrome market; historical and forecasted is included in the report, covering the 7MM drug outreach.

• The report provides an edge while developing business strategies, by understanding trends shaping and driving the 7MM Hunter Syndrome market.


Report Highlights

• In the coming years, Hunter Syndrome market is set to change due to the rising awareness of the disease, and incremental healthcare spending across the world; which would expand the size of the market to enable the drug manufacturers to penetrate more into the market.

• The companies and academics are working to assess challenges and seek opportunities that could influence Hunter Syndrome R&D. The therapies under development are focused on novel approaches to treat/improve the disease condition.

• Report covers Severity-based Diagnosed Prevalence of Hunter Syndrome, including both severe (early onset) and mild (late) type of Hunter Syndrome subgroup.

• Expected Launch of potential therapies may increase the market size in the coming years, assisted by an increase in diagnosed prevalent population of Hunter Syndrome. Owing to the positive outcomes of the several products during the developmental stage by key players such as JCR Pharmaceuticals, GC Pharma, Takeda, Denali Therapeutics, Regenxbio and Sanagamo Therapeutics, the market is expected to witness a significant positive shift in the Hunter Syndrome Market Size.

• The current US market of Hunter Syndrome is mainly dominated by the use of enzyme replacement therapy (Elaprase) along with the new upcoming therapeutic strategies that work on the principle of iduronate 2-sulfatase (I2S) enzyme replacement and gene therapies.

• DelveInsight estimates that there are several upcoming therapies that can cross the blood brain barrier (BBB), which Elaprase (only approved therapy) failed to do. The companies who claim that their drug candidate can cross this BBB include JCR Pharmaceuticals, Takeda, GC Pharma and Denali Therapeutics.

• One of the major key players of Hunter Syndome market include JCR Pharmaceuticals, which is developing JR-141 and Takeda’s TAK-609.

• The companies involved in developing gene therapies for Hunter Syndrome patients are Regenxbio and Sangamo Therapeutics. However, these products are still in their early phase of clinical trials.


Hunter Syndrome Report Insights

• Patient Population

• Therapeutic Approaches

• Hunter Syndrome Pipeline Analysis

• Hunter Syndrome Market Size and Trends

• Market Opportunities

• Impact of upcoming Therapies


Hunter Syndrome Report Key Strengths

• Eleven Years Forecast

• 7MM Coverage

• Hunter Syndrome Epidemiology Segmentation

• Key Cross Competition

• Highly Analyzed Market

• Drugs Uptake


Hunter Syndrome Report Assessment

• Current Treatment Practices

• Unmet Needs

• Pipeline Product Profiles

• Market Attractiveness

• Market Drivers and Barriers


Key Questions

Market Insights:

• What was the Hunter Syndrome market share (%) distribution in 2017 and how it would look like in 2030?

• What would be the Hunter Syndrome total market size as well as market size by therapies across the 7MM during the forecast period (2020–2030)?

• What are the key findings pertaining to the market across the 7MM and which country will have the largest Hunter Syndrome market size during the forecast period (2020–2030)?

• At what CAGR, the Hunter Syndrome market is expected to grow at the 7MM level during the forecast period (2020–2030)?

• What would be the Hunter Syndrome market outlook across the 7MM during the forecast period (2020–2030)?

• What would be the Hunter Syndrome market growth till 2030 and what will be the resultant market size in the year 2030?

• How would the market drivers, barriers and future opportunities affect the market dynamics and subsequent analysis of the associated trends?


Epidemiology Insights:

• What is the disease risk, burden and unmet needs of Hunter Syndrome?

• What is the historical Hunter Syndrome patient pool in the United States, EU5 (Germany, France, Italy, Spain, and the UK) and Japan?

• What would be the forecasted patient pool of Hunter Syndrome at the 7MM level?

• What will be the growth opportunities across the 7MM with respect to the patient population pertaining to Hunter Syndrome?

• Out of the above-mentioned countries, which country would have the highest prevalent population of Hunter Syndrome during the forecast period (2020–2030)?

• At what CAGR the population is expected to grow across the 7MM during the forecast period (2020–2030)?

Current Treatment Scenario, Marketed Drugs and Emerging Therapies:

• What are the current options for the treatment of Hunter Syndrome along with the approved therapy?

• What are the current treatment guidelines for the treatment of Hunter Syndrome in the US and Europe?

• What are the Hunter Syndrome marketed drugs and their MOA, regulatory milestones, product development activities, advantages, disadvantages, safety and efficacy, etc.?

• How many companies are developing therapies for the treatment of Hunter Syndrome?

• How many therapies are developed by each company for the treatment of Hunter Syndrome?

• How many emerging therapies are in the mid-stage and late stage of development for the treatment of Hunter Syndrome?

• What are the key collaborations (Industry–Industry, Industry–Academia), Mergers and acquisitions, licensing activities related to the Hunter Syndrome therapies?

• What are the recent novel therapies, targets, mechanisms of action and technologies developed to overcome the limitation of existing therapies?

• What are the clinical studies going on for Hunter Syndrome and their status?

• What are the key designations that have been granted for the emerging therapies for Hunter Syndrome?

• What are the 7MM historical and forecasted market of Hunter Syndrome?


Reasons to buy

• The report will help in developing business strategies by understanding trends shaping and driving the Hunter Syndrome.

• To understand the future market competition in the asprgillosis market and Insightful review of the key market drivers and barriers.

• Organize sales and marketing efforts by identifying the best opportunities for Hunter Syndrome in the US, Europe (Germany, Spain, Italy, France, and the United Kingdom) and Japan.

• Identification of strong upcoming players in the market will help in devising strategies that will help in getting ahead of competitors.

• Organize sales and marketing efforts by identifying the best opportunities for Hunter Syndrome market.

• To understand the future market competition in the Hunter Syndrome market.


What is Hunter Syndrome?

Hunter Syndrome is a rare, inherited disorder in which the body does not properly digest sugar molecules in the body. When these molecules accumulate in organs and tissues over time, they can cause damage that affects physical and mental development and abilities.


At what CAGR Hunter Syndrome market size is growing in the 7 major markets (MM)? 

Hunter Syndrome Market Size is expected to increase at a CAGR of 7.5% for the study period (2017-2030) in the 7MM.


What is the diagnosed prevalence of Hunter Syndrome in the 7MM in 2020?   

The diagnosed prevalence of Hunter Syndrome in 7MM is 1,166 in 2020.


What is the diagnosed prevalence of Hunter Syndrome in the United States in 2020?

The diagnosed prevalence of Hunter Syndrome in the United States is 515 in 2020.


What are the Hunter Syndrome market drivers?

Rich pipeline, one approved therapy, better awareness, and no cure are Hunter Syndrome market drivers.


Which are the emerging therapies in the Hunter Syndrome market?

JR-141, GC1111B or Hunterase, RGX-121, DNL310, SB-913 are the emerging therapies in the Hunter Syndrome market.


Which are the leading companies in the Hunter Syndrome market? 

Takeda, JCR Pharmaceuticals, GC Pharma, Denali Therapeutics, Regenxbio, Sangamo Therapeutics, ArmaGen are the leading companies in the Hunter Syndrome market.


How is epidemiology segmented for Hunter Syndrome?

Hunter Syndrome epidemiology is segmented as Total Prevalent Cases of Hunter Syndrome, Severity Specific Prevalence of Hunter Syndrome [Mild and Severe Form], and Diagnosed and Treatable Population of Hunter Syndrome in the 7MM from 2017 to 2030.

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

11.10. 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

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)


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

Takeda 

JCR Pharmaceuticals

Takeda

Green Cross Corporation/GC Pharma

Denali Therapeutics

Regenxbio

Sangamo Therapeutics

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  • Hunter Syndrome market
  • Hunter Syndrome market research
  • Hunter Syndrome market insight
  • Hunter Syndrome market trends
  • Hunter Syndrome market forecast
  • Hunter Syndrome market share
  • Hunter Syndrome pipeline drugs
  • Hunter Syndrome treatment algorithm
  • Hunter Syndrome drugs
  • Hunter Syndrome sales forecasting
  • Hunter Syndrome market size
  • Hunter Syndrome disease
  • Hunter Syndrome epidemiology
  • Hunter Syndrome

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