Phosphoglucomutase (PGM 1) Deficiency - Market Insights, Epidemiology and Market Forecast-2028

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Date: 15-Jul-2019
No. of pages: 92
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DelveInsight's ‘Phosphoglucomutase (PGM 1) Deficiency - 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 IPF in the United States, EU5 (Germany, Spain, Italy, France and United Kingdom), and Japan.

This Report provides the current treatment practices, emerging drugs, market share of the individual therapies, current and forecasted market size of Phosphoglucomutase (PGM 1) Deficiency from 2017 to 2028 segmented by seven major markets. The Report also covers current treatment practice/algorithm, 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

Phosphoglucomutase (PGM 1) Deficiency - Disease Understanding and Treatment Algorithm

The DelveInsight PGM-1 deficiency market report gives the thorough understanding of the PGM1 by including details such as disease definition, cause, symptoms, pathophysiology, and diagnostic trends. It also provides treatment algorithms and treatment guidelines for etiology in the US, Europe, and Japan.

Phosphoglucomutase (PGM 1) Deficiency Epidemiology

The Phosphoglucomutase (PGM 1) Deficiency epidemiology division provide the insights about historical and current patient pool and forecasted trend for every eight 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 DelveInsight 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 prevalent cases, prevalence of PGM1 deficiency based on clinical manifestations and congenital malformations, and prevalence based on severity) scenario of PGM1 deficiency in the 7MM covering United States, EU5 countries (Germany, Spain, Italy, France and United Kingdom), and Japan from 2017-2028.

According to DelveInsight, total prevalent population of Idiopathic PGM1 deficiency in 7MM markets was found to be 2593 in 2017.

Phosphoglucomutase (PGM 1) Deficiency Drug Chapters

This segment of the PGM1 deficiency 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, and the latest news and press releases.

The current strategies for the treatment of PGM-1 deficiency primarily encompass galactose administration along with other off-label symptomatic medications to ensure the overall reduction of associated disease complications. In general, the current mainstays for the PGM-1 deficiency treatment includes dietary supplementation of sugar along with other cardiotonic, complex carbohydrates, hydrocortisone and symptomatic medications to make the affected individuals clinically comfortable. Furthermore, physical, occupational, and speech therapy are additional management modalities that impart clinical benefits to the PGM-1 deficient individuals. Currently two drugs are under development for the treatment of Phosphoglucomutase 1 (PGM1) deficiency. CERC-801 (Cerecor Inc.) an ultra-pure, oral, crystalline formulation of D-galactose and ORL-1G-D-galactose (Orpha Labs) are into eraly phase of clinical development.

Phosphoglucomutase (PGM 1) Deficiency Market Outlook

The PGM1 deficiency 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 marketed drug and 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 DelveInsight, the market of Idiopathic Pulmonary Fibrosis in 7MM was found to be USD 10.86 million in 2017.

Phosphoglucomutase (PGM 1) Deficiency Drugs Uptake

This section focusses on the rate of uptake of the potential drugs 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.

Phosphoglucomutase (PGM 1) Deficiency Report Insights

- Patient Population

- Therapeutic Approaches

- Pipeline Analysis

- Market Size and Trends

- Market Opportunities

- Impact of upcoming Therapies

Phosphoglucomutase (PGM 1) Deficiency Report Key Strengths

- 10 Year Forecast

- 7MM Coverage

- Epidemiology Segmentation

- Drugs Uptake

- Highly Analyzed Market

- Key Cross Competition

Phosphoglucomutase (PGM 1) Deficiency Report Assessment

- Current Treatment Practices

- Unmet Needs

- Detailed Pipeline Product Profiles

- Market Attractiveness

- Market Drivers and Barriers

Key Benefits

- This DelveInsight report will help to develop Business Strategies by understanding the trends shaping and driving Phosphoglucomutase (PGM 1) Deficiency market

- Organize sales and marketing efforts by identifying the best opportunities for Phosphoglucomutase (PGM 1) Deficiency market

- To understand the future market competition in the Phosphoglucomutase (PGM 1) Deficiency market.

Phosphoglucomutase (PGM 1) Deficiency - Market Insights, Epidemiology and Market Forecast-2028

Table of Contents

1. Key Insights
2. Phosphoglucomutase 1 (PGM1) Deficiency Market Overview at a Glance
2.1. Market Share (%) Distribution of PGM1 Deficiency in 2017
2.2. Market Share (%) Distribution of PGM1 Deficiency in 2028
3. Disease Background and Overview
3.1. Introduction
3.2. Facts about Phosphoglucomutase-1
3.3. Pathophysiology
3.4. Causes
3.5. Symptoms
3.6. Multisystem phenotype in PGM1 deficiency
3.7. Diagnosis
4. Epidemiology and Patient Population
4.1. Key Findings
4.2. KOL's Views: Epidemiology
4.3. Assumptions and Rationale 7MM
5. 7MM Total Prevalent Patient Population of PGM1 Deficiency
6. Country Wise-Epidemiology of PGM1 Deficiency
6.1. The United States
6.1.1. Total Prevalent Cases of PGM1 Deficiency in the United States
6.1.2. Prevalence of PGM1 Deficiency based on Clinical Manifestations in the United States
6.1.3. Prevalence of PGM1 Deficiency based on Congenital malformations in the United States
6.1.4. Prevalence of PGM1 Deficiency based on Severity in the United States
6.2. EU5
6.2.1. Germany
6.2.2. Total Prevalent Cases of PGM1 Deficiency in Germany
6.2.3. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Germany
6.2.4. Prevalence of PGM1 Deficiency based on Congenital malformations in Germany
6.2.5. Prevalence of PGM1 Deficiency based on Severity in Germany
6.2.6. France
6.2.7. Total Prevalent Cases of PGM1 Deficiency in France
6.2.8. Prevalence of PGM1 Deficiency based on Clinical Manifestations in France
6.2.9. Prevalence of PGM1 Deficiency based on Congenital malformations in France
6.2.10. Prevalence of PGM1 Deficiency based on Severity in France
6.2.11. Italy
6.2.12. Total Prevalent Cases of PGM1 Deficiency in Italy
6.2.13. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Italy
6.2.14. Prevalence of PGM1 Deficiency based on Congenital malformations in Italy
6.2.15. Prevalence of PGM1 Deficiency based on Severity in Italy
6.2.16. Spain
6.2.17. Total Prevalent Cases of PGM1 Deficiency in Spain
6.2.18. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Spain
6.2.19. Prevalence of PGM1 Deficiency based on Congenital malformations in Spain
6.2.20. Prevalence of PGM1 Deficiency based on Severity in Spain
6.2.21. The United Kingdom
6.2.22. Total Prevalent Cases of PGM1 Deficiency in the UK
6.2.23. Prevalence of PGM1 Deficiency based on Clinical Manifestations in The UK
6.2.24. Prevalence of PGM1 Deficiency based on Congenital malformations in The UK
6.2.25. Prevalence of PGM1 Deficiency based on Severity in The UK
6.3. Japan
6.3.1. Total Prevalent Cases of PGM1 Deficiency in Japan
6.3.2. Prevalence of PGM1 Deficiency based on Clinical Manifestations in Japan
6.3.3. Prevalence of PGM1 Deficiency based on Congenital malformations in Japan
6.3.4. Prevalence of PGM1 Deficiency based on Severity in Japan
7. Treatment and Management of PGM1 Deficiency
8. Unmet Needs
9. Emerging Therapies
9.1. CERC-801: Cerecor Inc
9.1.1. Drug Description
9.1.2. Clinical Development
9.1.3. Other Developmental Activities
9.1.4. Product Profile
9.2. ORL-1G - D-galactose: Orpha Labs
9.2.1. Drug Description
9.2.2. Clinical Development
9.2.3. Clinical Trials Information
9.2.4. Product Profile
10. PGM1 Deficiency: 7 Major Market Analysis
10.1. Key Findings
10.2. KOL's Views: Treatment & Diagnosis
10.3. Market Outlook 7MM
10.4. Market Size of PGM1 Deficiency in 7MM
11. Market Outlook by Country
11.1. United States Market Size
11.1.1. Total Market Size of PGM1 Deficiency
11.1.2. Market Size of PGM1 Deficiency by Therapies
11.2. EU-5 Countries: Market Outlook
11.2.1. Germany
11.2.1.1. Total Market size of PGM1 Deficiency
11.2.1.2. Market Size of PGM1 Deficiency by Therapies
11.2.2. France
11.2.2.1. Total Market Size of PGM1 Deficiency
11.2.2.2. Market Size of PGM1 Deficiency by Therapies
11.2.3. Italy
11.2.3.1. Total Market Size of PGM1 Deficiency
11.2.3.2. Market Size of PGM1 Deficiency by Therapies
11.2.4. Spain
11.2.4.1. Total Market Size of PGM1 Deficiency
11.2.4.2. Market Size of PGM1 Deficiency by Therapies
11.2.5. United Kingdom
11.2.5.1. Total Market Size of PGM1 Deficiency
11.2.5.2. Market Size of PGM1 Deficiency by Therapies
11.3. Japan: Market Outlook
11.3.1. Total Market Size of PGM1 Deficiency
11.3.2. Market Size of PGM1 Deficiency by Therapies
12. Market Drivers
13. Market Barriers
14. Appendix
15. Report Methodology
16. DelveInsight Capabilities
17. Disclaimer
18. About DelveInsight

List of Tables

TABLE 1: PREVALENT PATIENT POPULATION OF PGM1 DEFICIENCY IN 7MM (2017-2028)
TABLE 2: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE US (2017-2028)
TABLE 3: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE US (2017-2028)
TABLE 4: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE US (2017-2028)
TABLE 5: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE US (2017-2028)
TABLE 6: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN GERMANY (2017-2028)
TABLE 7: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN GERMANY (2017-2028)
TABLE 8: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN GERMANY (2017-2028)
TABLE 9: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN GERMANY (2017-2028)
TABLE 10: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN FRANCE (2017-2028)
TABLE 11: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN FRANCE (2017-2028)
TABLE 12: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN FRANCE (2017-2028)
TABLE 13: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN FRANCE (2017-2028)
TABLE 14: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN ITALY (2017-2028)
TABLE 15: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN ITALY (2017-2028)
TABLE 16: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN ITALY (2017-2028)
TABLE 17: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN ITALY (2017-2028)
TABLE 18: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN SPAIN (2017-2028)
TABLE 19: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN SPAIN (2017-2028)
TABLE 20: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN SPAIN (2017-2028)
TABLE 21: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN SPAIN (2017-2028)
TABLE 22: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE UK (2017-2028)
TABLE 23: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE UK (2017-2028)
TABLE 24: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE UK (2017-2028)
TABLE 25: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE UK (2017-2028)
TABLE 26: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN JAPAN (2017-2028)
TABLE 27: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN JAPAN (2017-2028)
TABLE 28: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN JAPAN (2017-2028)
TABLE 29: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN JAPAN (2017-2028)
TABLE 30: ORL-1G - D-GALACTOSE CLINICAL TRIAL DESCRIPTION, 2019
TABLE 31: 7 MAJOR MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 32: THE UNITED STATES MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 33: THE US MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 34: MARKET SIZE OF PGM1 DEFICIENCY IN GERMANY, USD MILLION (2017-2028)
TABLE 35: GERMANY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 36: FRANCE MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 37: FRANCE MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 38: ITALY MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 39: ITALY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 40: SPAIN MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 41: SPAIN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 42: UNITED KINGDOM MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 43: THE UK MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
TABLE 44: JAPAN MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
TABLE 45: JAPAN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)

List of Figures

FIGURE 1: SCHEMATIC REPRESENTATION OF PHOSPHOGLUCOMUTASE 1 (PGM1) KEY ROLE IN METABOLISM AND PROTEIN INTERACTION
FIGURE 2: ROLE OF PHOSPHOGLUCOMUTASE 1 IN SUGAR METABOLISM.
FIGURE 3: (A) N-LINKED GLYCOSYLATION REQUIRES THE NUCLEOTIDE SUGARS CMP SIALIC ACID,
FIGURE 4: N-LINKED GLYCOSYLATION REQUIRES THE NUCLEOTIDE SUGARS CMP SIALIC ACID
FIGURE 5: ASSOCIATED SIGNS AND SYMPTOMS OF PGM-1 DEFICIENCY
FIGURE 6: PREVALENT PATIENT POPULATION OF PGM1 DEFICIENCY IN 7MM (2017-2028)
FIGURE 7: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE US (2017-2028)
FIGURE 8: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE US (2017-2028)
FIGURE 9: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE US (2017-2028)
FIGURE 10: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE US (2017-2028)
FIGURE 11: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN GERMANY (2017-2028)
FIGURE 12: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN GERMANY (2017-2028)
FIGURE 13: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN GERMANY (2017-2028)
FIGURE 14: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN GERMANY (2017-2028)
FIGURE 15: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN FRANCE (2017-2028)
FIGURE 16: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN FRANCE (2017-2028)
FIGURE 17: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN FRANCE (2017-2028)
FIGURE 18: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN FRANCE (2017-2028)
FIGURE 19: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN ITALY (2017-2028)
FIGURE 20: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN ITALY (2017-2028)
FIGURE 21: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN ITALY (2017-2028)
FIGURE 22: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN ITALY (2017-2028)
FIGURE 23: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN SPAIN (2017-2028)
FIGURE 24: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN SPAIN (2017-2028)
FIGURE 25: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN SPAIN (2017-2028)
FIGURE 26: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN SPAIN (2017-2028)
FIGURE 27: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN THE UK (2017-2028)
FIGURE 28: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN THE UK (2017-2028)
FIGURE 29: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN THE UK (2017-2028)
FIGURE 30: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN THE UK (2017-2028)
FIGURE 31: TOTAL PREVALENT CASES OF PGM1 DEFICIENCY IN JAPAN (2017-2028)
FIGURE 32: PREVALENCE OF PGM1 DEFICIENCY BASED ON CLINICAL MANIFESTATIONS IN JAPAN (2017-2028)
FIGURE 33: PREVALENCE OF PGM1 DEFICIENCY BASED ON CONGENITAL MALFORMATIONS IN JAPAN (2017-2028)
FIGURE 34: PREVALENCE OF PGM1 DEFICIENCY BASED ON SEVERITY IN JAPAN (2017-2028)
FIGURE 35: UNMET NEEDS OF PGM1 DEFICIENCY
FIGURE 36: 7 MAJOR MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
FIGURE 37: THE UNITED STATES MARKET SIZE OF PGM1 DEFICIENCY IN USD MILLION (2017-2028)
FIGURE 38: THE US MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 39: MARKET SIZE OF PGM1 DEFICIENCY IN GERMANY, USD MILLION (2017-2028)
FIGURE 40: GERMANY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 41: MARKET SIZE OF PGM1 DEFICIENCY IN FRANCE USD MILLION (2017-2028)
FIGURE 42: FRANCE MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 43: MARKET SIZE OF PGM1 DEFICIENCY IN ITALY, USD MILLION (2017-2028)
FIGURE 44: ITALY MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 45: MARKET SIZE OF PGM1 DEFICIENCY IN SPAIN, USD MILLION (2017-2028)
FIGURE 46: SPAIN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 47: MARKET SIZE OF PGM1 DEFICIENCY IN THE UNITED KINGDOM, USD MILLION (2017-2028)
FIGURE 48: THE UK MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 49: MARKET SIZE OF PGM1 DEFICIENCY IN JAPAN, USD MILLION (2017-2028)
FIGURE 50: JAPAN MARKET SIZE OF PGM1 DEFICIENCY BY THERAPIES IN USD MILLION (2017-2028)
FIGURE 51: MARKET DRIVERS OF PGM1 DEFICIENCY
FIGURE 52: MARKET BARRIERS OF PGM1 DEFICIENCY
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