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Mucopolysaccharidosis type I Market Insights, Epidemiology and Market Forecast-2028

  • ID: 4790889
  • Drug Pipelines
  • April 2019
  • Region: Global
  • 180 Pages
  • DelveInsight
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‘Mucopolysaccharidosis type I (MPS I) - Market Insights, Epidemiology and Market Forecast-2028’ report delivers an in-depth understanding of the disease, historical & forecasted epidemiology as well as the market trends of MPS I in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom), and Japan.

The Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Mucopolysaccharidosis type I (MPS I) from 2017 to 2028 segmented by seven major markets. The Report also covers current treatment practice/algorithm practice market drivers, market barriers and unmet medical needs to curate best of the opportunities and assess underlying potential of the market

Geography Covered

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

Study Period: 2017-2028

Mucopolysaccharidosis type I (MPS I) - Disease Understanding and Treatment Algorithm

The Mucopolysaccharidosis type I market report gives the thorough understanding of the Mucopolysaccharidosis type I by including details such as disease definition, classification, symptoms, etiology, pathophysiology, diagnostic trends. It also provides treatment algorithms and treatment guidelines for Mucopolysaccharidosis type I in the US, Europe, and Japan

Mucopolysaccharidosis type I Epidemiology

The Mucopolysaccharidosis type I epidemiology division provide the insights about historical and current patient pool and forecasted trend for every 7 major countries. It helps to recognize the causes of current and forecasted trends by exploring numerous studies and views of key opinion leaders. This part of The report also provides the diagnosed patient pool and their trends along with assumptions undertaken

The disease epidemiology covered in the report provides historical as well as forecasted epidemiology {total cases, Live Birth cases, overall prevalent cases, Severity Specific Cases [Hurler, Hurler-Scheie, Scheie] and diagnosed cases} scenario of Mucopolysaccharidosis type I in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom) and Japan from 2017-2028

According to this research, total prevalent population of Mucopolysaccharidosis type I in 7 major markets was found to be 3,529 in 2017

Mucopolysaccharidosis type I Drug Chapters

This segment of the Mucopolysaccharidosis type I report encloses the detailed analysis of marketed drugs and late stage (Phase-III and Phase-II) pipeline drugs. It also helps to understand the clinical trial details, expressive pharmacological action, agreements and collaborations, approval and patent details, advantages and disadvantages of each included drug and the latest news and press releases

Historically, treatment of MPS I was restricted to palliative care and symptom-based interventions such as surgery, physical, occupational, and speech therapies; respiratory support; hearing aids and other medications depends on the clinical manifestation in each individual patients which was the driving force for the market. However, the market scenario has been changed dramatically with the advent of hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy (ERT). HSCT is the mainstay of treatment, although ERT may be added as an adjunct therapy. Laronidase has been approved in the US in May 2003 for Hurler–Scheie and Scheie syndromes. Intrathecal enzyme replacement therapy is under investigation, but its safety and efficacy remain unclear. Detailed chapter for upcoming therapies like MGTA-456 (Magenta Therapeutics), Pentosan polysulfate sodium (Plexcera Therapeutics), Valanafusp alfa (ArmaGen) and SB-318 (Sangamo Therapeutics), have also been covered in the report

Mucopolysaccharidosis type I Market Outlook

The Mucopolysaccharidosis type I market outlook of the report helps to build the detailed comprehension of the historic, current and forecasted trend of the market by analyzing the impact of current therapies on the market, unmet needs, drivers and barriers and demand of better technology

This segment gives a through detail of market trend of each marketed drug and late-stage pipeline therapy by evaluating their impact based on annual cost of therapy, inclusion and exclusion criteria’s, mechanism of action, compliance rate, growing need of the market, increasing patient pool, covered patient segment, expected launch year, competition with other therapies, brand value, their impact on the market and view of the key opinion leaders. The calculated market data are presented with relevant tables and graphs to give a clear view of the market at first sight

According to this research, the market of Mucopolysaccharidosis type I in 7MM was found to be USD 333.31 million in 2017

Mucopolysaccharidosis type I Drugs Uptake

This section focusses on the rate of uptake of the potential drugs recently launched in the market or will get launched in the market during the study period from 2017-2028. The analysis covers 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 allows 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

Mucopolysaccharidosis type I Report Insights

  • Patient Population
  • Therapeutic Approaches
  • Pipeline Analysis
  • Market Size and Trends
  • Market Opportunities
  • Impact of upcoming Therapies

Mucopolysaccharidosis type I Report Key Strengths

  • 10 Year Forecast
  • 7MM Coverage
  • Epidemiology Segmentation
  • Drugs Uptake
  • Highly Analyzed Market
  • Key Cross Competition

Mucopolysaccharidosis type I Report Assessment

  • Current Treatment Practices
  • Unmet Needs
  • Detailed Pipeline Product Profiles
  • Market Attractiveness
  • Market Drivers and Barriers

Key Benefits

  • This report will help to develop Business Strategies by understanding the trends shaping and driving Mucopolysaccharidosis type I market
  • Organize sales and marketing efforts by identifying the best opportunities for Mucopolysaccharidosis type I market
  • To understand the future market competition in the Mucopolysaccharidosis type I market.
Note: Product cover images may vary from those shown
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1. Key Insights

2. Mucopolysaccharidosis type I (MPS I): Market Overview at a Glance
2.1. Total Market Share (%) Distribution of Mucopolysaccharidosis type I (MPS I) in 2017
2.2. Total Market Share (%) Distribution of Mucopolysaccharidosis type I (MPS I) in 2028

3. Mucopolysaccharidosis type I (MPS I): Disease Background and Overview
3.1. Introduction
3.2. Symptoms
3.3. Clinical Manifestations of MPS I
3.4. Severity Spectrum
3.5. Genetic Causes
3.6. Inheritance pattern
3.7. Pathophysiological mechanisms of MPS I
3.8. Diagnosis
3.8.1. Differential diagnosis
3.9. Newborn screening for MPS I
3.10. Challenges in predicting disease severity in newborn screening
3.11. Easy-to-use algorithm for faster diagnoses of MPS I

4. Epidemiology and Patient Population
4.1. Key Findings

5. Total Cases of Mucopolysaccharidosis type I in 7MM

6. United States Epidemiology
6.1. Assumptions and Rationale
6.2. Total cases of Mucopolysaccharidosis type I in the US
6.3. Live Birth Cases of Mucopolysaccharidosis type I in the United States
6.4. Overall Prevalent Cases of Mucopolysaccharidosis type I in the United States
6.5. Diagnosed Cases of MPS Type I in the United States
6.6. Severity Specific Cases of MPS Type I in the United States

7. EU5 Epidemiology
7.1. Germany Epidemiology
7.1.1. Total cases of Mucopolysaccharidosis type I in Germany
7.1.2. Live Birth Cases of Mucopolysaccharidosis type I in Germany
7.1.3. Overall Prevalent Cases of Mucopolysaccharidosis type I in Germany
7.1.4. Diagnosed Cases of MPS Type I in Germany
7.1.5. Severity Specific Cases of MPS Type I in Germany
7.2. France Epidemiology
7.2.1. Total cases of Mucopolysaccharidosis type I in France
7.2.2. Live Birth Cases of Mucopolysaccharidosis type I in France
7.2.3. Overall Prevalent Cases of Mucopolysaccharidosis type I in France
7.2.4. Diagnosed Cases of MPS Type I in France
7.2.5. Severity Specific Cases of MPS Type I in France
7.3. Italy Epidemiology
7.3.1. Total cases of Mucopolysaccharidosis type I in Italy
7.3.2. Live Birth Cases of Mucopolysaccharidosis type I in Italy
7.3.3. Overall Prevalent Cases of Mucopolysaccharidosis type I in Italy
7.3.4. Diagnosed Cases of MPS Type I in Italy
7.3.5. Severity Specific Cases of MPS Type I in Italy
7.4. Spain Epidemiology
7.4.1. Total cases of Mucopolysaccharidosis type I in Spain
7.4.2. Live Birth Cases of Mucopolysaccharidosis type I in Spain
7.4.3. Overall Prevalent Cases of Mucopolysaccharidosis type I in Spain
7.4.4. Diagnosed Cases of MPS Type I in Spain
7.4.5. Severity Specific Cases of MPS Type I in Spain
7.5. United Kingdom Epidemiology
7.5.1. Total cases of Mucopolysaccharidosis type I in the UK
7.5.2. Live Birth Cases of Mucopolysaccharidosis type I in the UK
7.5.3. Overall Prevalent Cases of Mucopolysaccharidosis type I in the UK
7.5.4. Diagnosed Cases of MPS Type I in the UK
7.5.5. Severity Specific Cases of MPS Type I in the UK
7.6. Japan Epidemiology
7.7. Assumptions and Rationale
7.7.1. Total cases of Mucopolysaccharidosis type I in Japan
7.7.2. Live Birth Cases of Mucopolysaccharidosis type I in Japan
7.7.3. Overall Prevalent Cases of Mucopolysaccharidosis type I in Japan
7.7.4. Diagnosed Cases of MPS Type I in Japan
7.7.5. Severity Specific Cases of MPS Type I in Japan
7.8. Management and Treatment of MPS I
7.8.1. Clinical management of MPS I
7.8.2. Treatment of MPS I
7.8.3. Recommendations for the diagnosis and treatment of MPS-I by Italian authorities
7.8.4. Treatment Algorithm

8. Unmet Needs

9. Marketed drugs
9.1. Aldurazyme: BioMarin Pharmaceutical/ Genzyme
9.1.1. Drug Description
9.1.2. Regulatory Milestones
9.1.3. Other Developmental Activities
9.1.4. Safety and Efficacy
9.1.5. Product Profile

10. Emerging Therapies
10.1. Key Cross Competition
10.2. MGTA-456: Magenta Therapeutics
10.2.1. Product Description
10.2.2. Other Developmental Activities
10.2.3. Clinical Development
10.2.4. Safety and Efficacy
10.2.5. Product Profile
10.3. Pentosan Polysulfate Sodium: Plexcera Therapeutics
10.3.1. Drug Description
10.3.2. Other Developmental Activities
10.3.3. Clinical Development
10.3.4. Safety and Efficacy
10.3.5. Product Profile
10.4. Valanafusp alfa: Armagen
10.4.1. Drug Description
10.4.2. Other Developmental Activities
10.4.3. Clinical Development
10.4.4. Safety and Efficacy
10.4.5. Product Profile
10.5. SB-318: Sangamo Therapeutics
10.5.1. Drug Description
10.5.2. Other Developmental Activities
10.5.3. Clinical Development
10.5.4. Safety and Efficacy
10.5.5. Product Profile

11. Mucopolysaccharidosis type I (MPS I): 7MM Market Analysis
11.1. Key Findings
11.2. Market Size of Mucopolysaccharidosis type I in 7MM

12. United States: Market Outlook
12.1. United States Market Size
12.1.1. Total Market size of Mucopolysaccharidosis type I
12.1.2. Market Size of Mucopolysaccharidosis type I by Therapies in the US

13. EU-5 countries: Market Outlook
13.1. Germany Market Size
13.1.1. Total Market size of Mucopolysaccharidosis type I
13.1.2. Market Size of Mucopolysaccharidosis type I by Therapies in Germany
13.2. France Market Size
13.2.1. Total Market size of Mucopolysaccharidosis type I
13.2.2. Market Size of Mucopolysaccharidosis type I by Therapies in France
13.3. Italy Market Size
13.3.1. Total Market size of Mucopolysaccharidosis type I
13.3.2. Market Size of Mucopolysaccharidosis type I by Therapies in Italy
13.4. Spain Market Size
13.4.1. Total Market size of Mucopolysaccharidosis type I
13.4.2. Market Size of Mucopolysaccharidosis type I by Therapies in Spain
13.5. United Kingdom Market Size
13.5.1. Total Market size of Mucopolysaccharidosis type I
13.5.2. Market Size of Mucopolysaccharidosis type I by Therapies in the UK

14. Japan Market Outlook
14.1. Japan Market Size
14.2. Total Market size of Mucopolysaccharidosis type I
14.3. Market Size of Mucopolysaccharidosis type I by Therapies in Japan

15. Market Drivers

16. Market Barriers

17. Appendix
17.1. Report Methodology

18. Publisher Capabilities

19. Disclaimer

20. About the Publisher

List of Tables
Table 1: MPS I Disease Spectrum and Progression of the Natural History
Table 1: MPS I Disease Spectrum and Progression of the Natural History
Table 1: MPS I Disease Spectrum and Progression of the Natural History
Table 4: Mucopolysaccharidosis type I newborn screening: positive clues
Table 5: Mucopolysaccharidosis newborn screening: major concern
Table 6: Challenges in predicting disease severity following positive NBS for MPS I
Table 7: Key signs and symptoms prompting early clinical suspicion of MPS I and timely referral of patients
Table 8: Total Cases of Mucopolysaccharidosis type I (MPS I) in 7MM (2017-2028)
Table 9: Total Cases of MPS Type I in the US (2017-2028)
Table 10: Live Birth Cases of Mucopolysaccharidosis type I in the US (2017-2028)
Table 11: Overall Prevalent Cases of Mucopolysaccharidosis type I in the US (2017-2028)
Table 12: Diagnosed Cases of MPS Type I in the US (2017-2028)
Table 13: Severity Specific Cases of MPS Type I in the US (2017-2028)
Table 14: Total Cases of MPS Type I in Germany (2017-2028)
Table 15: Live Birth Cases of Mucopolysaccharidosis type I in Germany (2017-2028)
Table 16: Overall Prevalent Cases of Mucopolysaccharidosis type I in Germany (2017-2028)
Table 17: Diagnosed Cases of MPS Type I in Germany (2017-2028)
Table 18: Severity Specific Cases of MPS Type I in Germany (2017-2028)
Table 19: Total Cases of MPS Type I in France (2017-2028)
Table 20: Live Birth Cases of Mucopolysaccharidosis type I in France (2017-2028)
Table 21: Overall Prevalent Cases of Mucopolysaccharidosis type I in France (2017-2028)
Table 22: Diagnosed Cases of MPS Type I in France (2017-2028)
Table 23: Severity Specific Cases of MPS Type I in France (2017-2028)
Table 24: Total Cases of MPS Type I in Italy (2017-2028)
Table 25: Live Birth Cases of Mucopolysaccharidosis type I in Italy (2017-2028)
Table 26: Overall Prevalent Cases of Mucopolysaccharidosis type I in Italy (2017-2028)
Table 27: Diagnosed Cases of MPS Type I in Italy (2017-2028)
Table 28: Severity Specific Cases of MPS Type I in Italy (2017-2028)
Table 29: Total Cases of MPS Type I in Spain (2017-2028)
Table 30: Live Birth Cases of Mucopolysaccharidosis type I in Spain (2017-2028)
Table 31: Overall Prevalent Cases of Mucopolysaccharidosis type I in Spain (2017-2028)
Table 32: Diagnosed Cases of MPS Type I in Spain (2017-2028)
Table 33: Severity Specific Cases of MPS Type I in Spain (2017-2028)
Table 34: Total Cases of MPS Type I in the UK (2017-2028)
Table 35: Live Birth Cases of Mucopolysaccharidosis type I in the UK (2017-2028)
Table 36: Overall Prevalent Cases of Mucopolysaccharidosis type I in the UK (2017-2028)
Table 37: Diagnosed Cases of MPS Type I in the UK (2017-2028)
Table 38: Severity Specific Cases of MPS Type I in the UK (2017-2028)
Table 39: Total Cases of MPS Type I in Japan (2017-2028)
Table 40: Live Birth Cases of Mucopolysaccharidosis type I in Japan (2017-2028)
Table 41: Overall Prevalent Cases of Mucopolysaccharidosis type I in Japan (2017-2028)
Table 42: Diagnosed Cases of MPS Type I in Japan (2017-2028)
Table 43: Severity Specific Cases of MPS Type I in Japan (2017-2028)
Table 44: Key Cross: Emerging Therapies (III & II)
Table 45: MGTA-456, Clinical Trial Description, 2019
Table 46: Pentosan polysulfate sodium, Clinical Trial Description 2019
Table 47: AGT-181, Clinical Trial Description 2019
Table 48: SB-318, Clinical Trial Description 2019
Table 49: Market Size of Mucopolysaccharidosis type I in 7MM in USD Million (2017-2028)
Table 50: The US Market Size of Mucopolysaccharidosis type I (MPS I) in USD Million (2017-2028)
Table 51: Market Size of MPS type I by therapies in the US in USD Million (2017-2028)
Table 52: Germany Market Size of Mucopolysaccharidosis type I in USD Million (2017-2028)
Table 53: Market Size of MPS type I by therapies in Germany in USD Million (2017-2028)
Table 54: France Market Size of Mucopolysaccharidosis type I in USD Million (2017-2028)
Table 55: Market Size of MPS type I by therapies in France in USD Million (2017-2028)
Table 56: Italy Market Size of Mucopolysaccharidosis type I in USD Million (2017-2028)
Table 57: Market Size of MPS type I by therapies in Italy in USD Million (2017-2028)
Table 58: Spain Market Size of Mucopolysaccharidosis type I in USD Million (2017-2028)
Table 59: Market Size of MPS type I by therapies in Spain in USD Million (2017-2028)
Table 60: UK Market Size of Mucopolysaccharidosis type I in USD Million (2017-2028)
Table 61: Market Size of MPS type I by therapies in the UK in USD Million (2017-2028)
Table 62: Japan Market Size of Mucopolysaccharidosis type I in USD Million (2017-2028)
Table 63:Market Size of MPS type I by therapies in Japan in USD Million (2017-2028)

List of Figures
Figure 1: Spectrum of Disease Severity MPS I
Figure 2: Inheritance pattern of MPS I
Figure 3: Secondary gangliosides accumulation following primary GAGs storage in MPSs
Figure 4: Inflammation as a pathological mechanism in MPS I and other LSDs
Figure 5: Potential pathogenetic effects of lysosomal membrane permeabilization in MPS I cells
Figure 6: Alteration of cell signaling pathways due to modified HS storage in MPS I
Figure 7: Algorithm for Mucopolysaccharidosis type I (MPS I)-positive newborn screening
Figure 8: Algorithm to prompt early clinical suspicion of MPS I and timely referral of patients
Figure 9: Total Cases of Mucopolysaccharidosis type I in 7MM (2017-2028)
Figure 10: Total Cases of MPS Type I in the US (2017-2028)
Figure 11: Live Birth Cases of Mucopolysaccharidosis type I in the US (2017-2028)
Figure 12: Overall Prevalent Cases of Mucopolysaccharidosis type I in the US (2017-2028)
Figure 13: Diagnosed Cases of MPS Type I in the US (2017-2028)
Figure 14: Severity Specific Cases of MPS Type I in the US (2017-2028)
Figure 15: Total Cases of MPS Type I in Germany (2017-2028)
Figure 16: Live Birth Cases of Mucopolysaccharidosis type I in Germany (2017-2028)
Figure 17: Overall Prevalent Cases of Mucopolysaccharidosis type I in Germany (2017-2028)
Figure 18: Diagnosed Cases of MPS Type I in Germany (2017-2028)
Figure 19: Severity Specific Cases of MPS Type I in Germany (2017-2028)
Figure 20: Total Cases of MPS Type I in France (2017-2028)
Figure 21: Live Birth Cases of Mucopolysaccharidosis type I in France (2017-2028)
Figure 22: Overall Prevalent Cases of Mucopolysaccharidosis type I in France (2017-2028)
Figure 23: Diagnosed Cases of MPS Type I in France (2017-2028)
Figure 24: Severity Specific Cases of MPS Type I in France (2017-2028)
Figure 25: Total Cases of MPS Type I in Italy (2017-2028)
Figure 26: Live Birth Cases of Mucopolysaccharidosis type I in Italy (2017-2028)
Figure 27: Overall Prevalent Cases of Mucopolysaccharidosis type I in Italy (2017-2028)
Figure 28: Diagnosed Cases of MPS Type I in Italy (2017-2028)
Figure 29: Severity Specific Cases of MPS Type I in Italy (2017-2028)
Figure 30: Total Cases of MPS Type I in Spain (2017-2028)
Figure 31: Live Birth Cases of Mucopolysaccharidosis type I in Spain (2017-2028)
Figure 32: Overall Prevalent Cases of Mucopolysaccharidosis type I in Spain (2017-2028)
Figure 33: Diagnosed Cases of MPS Type I in Spain (2017-2028)
Figure 34: Severity Specific Cases of MPS Type I in Spain (2017-2028)
Figure 35: Total Cases of MPS Type I in the UK (2017-2028)
Figure 36: Live Birth Cases of Mucopolysaccharidosis type I in the UK (2017-2028)
Figure 37: Overall Prevalent Cases of Mucopolysaccharidosis type I in the UK (2017-2028)
Figure 38: Diagnosed Cases of MPS Type I in the UK (2017-2028)
Figure 39: Severity Specific Cases of MPS Type I in the UK (2017-2028)
Figure 40: Total Cases of MPS Type I in Japan (2017-2028)
Figure 41: Live Birth Cases of Mucopolysaccharidosis type I in Japan (2017-2028)
Figure 42: Overall Prevalent Cases of Mucopolysaccharidosis type I in Japan (2017-2028)
Figure 43: Diagnosed Cases of MPS Type I in Japan (2017-2028)
Figure 44: Severity Specific Cases of MPS Type I in Japan (2017-2028)
Figure 45: Treatment algorithm for patients with a diagnosis of MPS I
Figure 46: Treatment algorithm for MPS I
Figure 47: Unmet Needs of Mucopolysaccharidosis type I (MPS I)
Figure 48: Market Size of Mucopolysaccharidosis type I in 7MM in USD Million (2017-2028)
Figure 49: Market Size of Mucopolysaccharidosis type I in the US, USD Millions (2017-2028)
Figure 50: Market Size of MPS type I by therapies in the US in USD Million (2017-2028)
Figure 51: Market Size of Mucopolysaccharidosis type I in Germany, USD Millions (2017-2028)
Figure 52: Market Size of MPS type I by therapies in Germany in USD Million (2017-2028)
Figure 53: Market Size of Mucopolysaccharidosis type I in France, USD Millions (2017-2028)
Figure 54: Market Size of MPS type I by therapies in France in USD Million (2017-2028)
Figure 55: Market Size of Mucopolysaccharidosis type I in Italy, USD Millions (2017-2028)
Figure 56: Market Size of MPS type I by therapies in Italy in USD Million (2017-2028)
Figure 57: Market Size of Mucopolysaccharidosis type I in Spain, USD Millions (2017-2028)
Figure 58: Market Size of MPS type I by therapies in Spain in USD Million (2017-2028)
Figure 59: Market Size of Mucopolysaccharidosis type I in the UK, USD Millions (2017-2028)
Figure 60: Market Size of MPS type I by therapies in the UK in USD Million (2017-2028)
Figure 61: Market Size of Mucopolysaccharidosis type I in Japan, USD Millions (2017-2028)
Figure 62: Market Size of MPS type I by therapies in Japan in USD Million (2017-2028)
Figure 63: Market Drivers
Figure 64: Market Barriers
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