0

Pompe Disease - A Pipeline Analysis Report

  • Published: May 2018
  • Pages: 88
  • SKU: IRTNTR22315
Technavio

Enjoy complimentary customisation on priority with our Enterprise License!

Get me the latest version! (includes COVID-19 impact)
Safe and Secure SSL Encrypted

info
close
  • Single:

    One user only.
    Quick & easy download option

  • Enterprise:

    Unlimited user access (Within your organization)
    Complimentary Customization Included

2500 USD

This pipeline analysis report provides detailed insights into the drug development landscape of the development of drugs for Pompe disease including molecules at pre-clinical, discovery, and other developmental stages. The report also offers comprehensive information about the therapeutic assessment of pipeline molecules based on the segmentations such as therapies employed, therapeutic modality, targets for drug development, mechanism of action, and route of administration.

Overview of the drug development pipeline for Pompe disease

Pompe disease is an autosomal recessive disorder and is also known as acid maltase deficiency disease or type II glycogen storage disease. Patients suffering from this disease experience glycogen accumulation primarily in skeletal and cardiac muscle, which is resulted from α-glucosidase (GAA) deficiency in lysosomes. The accumulation of glycogen impairs the ability of muscles to function properly. A nationwide newborn screening program for Pompe disease was started in Taiwan with high rate of successful results. Technavio’s market research analysts have predicted the introduction of such screening programs as one of the most promising approach to diagnose Pompe disease.

According to this pipeline analysis report, most of the drug development molecules in the pipeline are under the pre-clinical drug development stage. Our market research analysts have also identified that a small amount of drug molecules that are being developed comes under the phase III development stage. The pipeline landscape in the pipeline analysis report mentions the percentage of drug development molecules that are under the phase I/II and discovery stage.

Companies covered

This pipeline analysis report provides a detailed analysis of the companies that are actively involved in the development of drugs for the treatment of Pompe disease. In addition to providing information on the various stages of molecules developed by companies for different indications, this pipeline analysis report also provides information about the drug development molecules discontinued by companies.

Some of the companies covered in this pipeline analysis report are –

  • Pharming
  • OXYRANE
  • Etubics

Therapeutic assessment of the drug development pipeline for Pompe disease by route of administration

  • IV
  • Oral
  • IV + Oral

IV route of administration (ROA) involves the administration of drugs directly into the vein. According to this pipeline analysis report, the majority of drugs in the pipeline are being developed for IV administration.

Therapeutic assessment of the drug development pipeline for Pompe disease by therapeutic modality

  • Recombinant enzyme
  • Gene therapy
  • Small molecule

Recombinant enzyme is produced by the modification of gene sequences and it generates useful therapeutic products. According to this pipeline analysis report, the majority of molecules that are currently in the drug development pipeline for Pompe disease are being developed as recombinant enzymes.

Key questions answered in the report include

  • What are the drug development molecules in the various development stages for Pompe disease?
  • What are the companies that are currently involved in the development of drug molecules for Pompe disease?
  • Insight into discontinued/inactive molecules with appropriate reasoning?
  • What are the major regulatory authorities approving drugs in various regions?
  • Detailed profiling of each active molecule

Technavio also offers customization on reports based on specific client requirement.

PART 01: EXECUTIVE SUMMARY

PART 02: SCOPE OF THE REPORT

PART 03: RESEARCH METHODOLOGY

PART 04: INTRODUCTION

PART 05: MAJOR REGULATORY AUTHORITIES

  • US
  • Europe
  • China

PART 06: PIPELINE LANDSCAPE

PART 07: COMPARATIVE ANALYSIS

  • Discovery stage molecules
  • Pre-clinical stage molecules
  • Inactive and discontinued molecules

PART 08: INDICATION ANALYSIS

PART 09: THERAPEUTIC ASSESSMENT (THERAPY BASED)

PART 10: THERAPEUTIC ASSESSMENT (ROA BASED)

PART 11: THERAPEUTIC ASSESSMENT BY TARGET

PART 12: KEY COMPANIES

  • Active companies: Category and parameters

PART 13: APPENDIX

  • List of abbreviations

Research Framework

Technavio presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources. The analysts have presented the various facets of the market with a particular focus on identifying the key industry influencers. The data thus presented is comprehensive, reliable, and the result of extensive research, both primary and secondary.

TechnavioINFORMATION SOURCES

Primary sources

  • Manufacturers and suppliers
  • Channel partners
  • Industry experts
  • Strategic decision makers

Secondary sources

  • Industry journals and periodicals
  • Government data
  • Financial reports of key industry players
  • Historical data
  • Press releases
Technavio

TechnavioDATA ANALYSIS

Data Synthesis

  • Collation of data
  • Estimation of key figures
  • Analysis of derived insights

Data Validation

  • Triangulation with data models
  • Reference against proprietary databases
  • Corroboration with industry experts
Technavio

TechnavioREPORT WRITING

Qualitative

  • Market drivers
  • Market challenges
  • Market trends
  • Five forces analysis

Quantitative

  • Market size and forecast
  • Market segmentation
  • Geographical insights
  • Competitive landscape
Interested in this report?
Get your sample now!
  • What are the key global market and the regional market share?
  • What are the revenue-generating key market segments?
  • What are the key factors driving and challenging this market’s growth?
  • Who are the key market vendors and their growth strategies?
  • What are the latest trends influencing the growth of this market?
  • What are the variables influencing the market growth in the primary regions?
  • What are the factors influencing the growth of the parent market?
  • Off-the-shelf research reports
  • Reports can be tailored to meet the customer's needs
  • Trusted by more than 100 fortune 500 organizations
  • Information about the market's key drivers, trends, and challenges
  • Parent market analysis
  • Every week, 50,000 people visit our subscription platform
  • Detailed vendors report with competitive landscape
  • Covid-19 impact and recovery analysis
  • Data on revenue-generating market segments
  • Details on the market shares of various regions
  • Five-force market analysis

Certified ISO 9001 : 2015

We are ISO recognized

We are ISO 9001:2015 recognized that ensures quality services and product delivery to our clients.

We are GDPR and CCPA compliant

We are GDPR and CCPA compliant! Your transaction & personal information is protected from unauthorized use.

Safe and Secure SSL Encrypted
Technavio

info
close
  • Single:

    One user only.
    Quick & easy download option

  • Enterprise:

    Unlimited user access (Within your organization)
    Complimentary Customization Included

2500 USD

Technavio Get the report (PDF) sent to your email within minutes.

Subscribe & Save

Get lifetime access to our
Technavio Insights

Customized Report as per your Business Needs

  • Our analysts will work directly with you and understand your needs
  • Get data on specified regions or segments, competitor and Vendors
  • Data will be formatted and presented as per your requirements
  • We offer $1000 worth of FREE customization at the time of purchase

Let us help you make report more suited to your requirements.

  • Get a competitive breakdown as per your niche industry
  • Customize the data with various metrics that meet your business prerequisite
  • Understand Revenue Sources, Customers and many more
  • Details on Market Share
  • These customizations are done in a short amount of time by our analysts and industry experts
Technavio
Enquire Before Buying
17,000 reports available | Plans starting from just 5,000 USD | Get FREE trial>>