About Lysosomal Storage Diseases
Lysosomal storage diseases (LSDs) refer to a group of diseases arising because of the deficiency of lysosomal enzymes, which leads to the accumulation of partially digested or undigested macromolecules inside the cell. LSDs includes progressive diseases with variable ages of onset and clinical symptoms. LSDs are autosomal recessive disorders that are passed on from generation to generation. However, Fabry disease and MPS II are X-linked recessive disorders, which are inherited through mutated alleles on X chromosome.
Technavio’s analysts forecast the global lysosomal storage diseases market to grow at a CAGR of 7.43% during the period 2017-2021.
Covered in this report
The report covers the present scenario and the growth prospects of the global lysosomal storage diseases market for 2017-2021. The report presents a detailed picture of the market by way of study, synthesis, and summation of data from multiple sources.
The market is divided into the following segments based on geography:
• Americas
• APAC
• EMEA
Technavio's report, Global Lysosomal Storage Diseases Market 2017-2021, has been prepared based on an in-depth market analysis with inputs from industry experts. The report covers the market landscape and its growth prospects over the coming years. The report also includes a discussion of the key vendors operating in this market.
Key vendors
• Actelion Pharmaceuticals
• BioMarin
• Genzyme
• Shire
Other prominent vendors
• Alexion Pharmaceuticals
• Amicus Therapeutics
• Chiesi Farmaceutici
• Greenovation Biotech and FGK Clinical Research
• Horizon Pharma
• Leadiant Biosciences
• Mylan
• Pfizer
• Protalix
• Raptor
• Recordati
• Synageva BioPharma
• Valerion Therapeutics
• Vtesse
Market driver
• Unmet medical needs
• For a full, detailed list, view our report
Market challenge
• Expensive treatment regimen for LSDs
• For a full, detailed list, view our report
Market trend
• Regulatory assistance in emerging nations
• For a full, detailed list, view our report
Key questions answered in this report
• What will the market size be in 2021 and what will the growth rate be?
• What are the key market trends?
• What is driving this market?
• What are the challenges to market growth?
• Who are the key vendors in this market space?
You can request one free hour of our analyst’s time when you purchase this market report. Details are provided within the report.
Table of Contents
PART 01: Executive summary
PART 02: Scope of the report
PART 03: Research methodology
PART 04: Introduction
• Market outline
PART 05: Understanding LSDs
• Introduction
PART 06: Pipeline
PART 07: Market landscape
• Market overview
• Five forces analysis
PART 08: Market segmentation by therapy
• Enzyme replacement therapy (ERT)
• Substrate reduction therapy (SRT)
• Cystine depleting agents
PART 09: Market segmentation by geography
• Geographical segmentation
• LSDs market in EMEA
• LSDs market in Americas
• LSDs market in APAC
PART 10: Decision framework
PART 11: Drivers and challenges
• Market drivers
• Market challenges
PART 12: Trends
• Advent of modern technologies
• Regulatory assistance in emerging nations
• Increased R&D activities
• Increased focus on gene therapy
• Emergence of immunotherapy
• Growing public awareness
PART 13: Vendor landscape
• Competitive scenario
PART 14: Key vendor analysis
• Actelion Pharmaceuticals
• BioMarin
• Genzyme
• Shire
• Other Prominent Vendors
PART 15: Appendix
• List of abbreviations
List of Exhibits?
Exhibit 01: Classification of LSDs
Exhibit 02: Pipeline snapshot
Exhibit 03: Pipeline landscape based on various LSDs
Exhibit 04: Key clinical trials
Exhibit 05: Global LSDs market 2016-2021 ($ millions)
Exhibit 06: Opportunity analysis of the global LSDs market
Exhibit 07: Five forces analysis
Exhibit 08: Global LSDs market segmentation by therapy
Exhibit 09: Global LSDs market share by therapy 2016
Exhibit 10: Global LSDs ERT market 2016-2021 ($ millions)
Exhibit 11: Potential benefits of using ERT for the treatment of various LSDs
Exhibit 12: Global LSDs SRT market 2016-2021 ($ millions)
Exhibit 13: Global LSDs cystine depleting agents market 2016-2021 ($ millions)
Exhibit 14: Global LSDs market share by geography 2016 and 2021
Exhibit 15: Global LSDs market revenue by geography 2016-2021
Exhibit 16: Global LSDs market share by geography 2016-2021
Exhibit 17: Market scenario in EMEA
Exhibit 18: LSDs market in EMEA 2016-2021 ($ millions)
Exhibit 19: Incentives provided for orphan drugs by EU regulation
Exhibit 20: Market scenario in Americas
Exhibit 21: LSDs market in Americas 2016-2021 ($ millions)
Exhibit 22: Orphan drug development and regulatory challenges
Exhibit 23: Incentives provided for orphan drugs by US regulation
Exhibit 24: LSDs market in APAC 2016-2021 ($ millions)
Exhibit 25: Incentives provided for orphan drugs by Japanese regulation
Exhibit 26: Incentives provided for orphan drugs by Australian regulation
Exhibit 27: ICD-10 coverage for some of the LSDs
Exhibit 28: US FDA filed to approved time: Orphan drugs versus non-orphan drugs and others (median time in months)
Exhibit 29: Usefulness of DNA-based testing
Exhibit 30: Competitive structure analysis of global LSDs market 2016
Exhibit 31: Actelion Pharmaceuticals: Key highlights
Exhibit 32: Actelion Pharmaceuticals: Strength assessment
Exhibit 33: Actelion Pharmaceuticals: Strategy assessment
Exhibit 34: Actelion Pharmaceuticals: Opportunity assessment
Exhibit 35: BioMarin: Key highlights
Exhibit 36: BioMarin: Strength assessment
Exhibit 37: BioMarin: Strategy assessment
Exhibit 38: BioMarin Pharmaceuticals: Opportunity assessment
Exhibit 39: Genzyme: Key highlights
Exhibit 40: Genzyme: Strength assessment
Exhibit 41: Genzyme: Strategy assessment
Exhibit 42: Genzyme: Opportunity assessment
Exhibit 43: Shire: Key highlights
Exhibit 44: Shire: Strength assessment
Exhibit 45: Shire: Strategy assessment
Exhibit 46: Shire: Opportunity assessment
