Global Lysosomal Storage Diseases Market 2017-2021
- Published: Sep 2017
- Pages: 104
- SKU: IRTNTR14696
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This industry research report identifies Actelion Pharmaceuticals, BioMarin, Genzyme, and Shire as the key vendors in the global lysosomal storage diseases market. This industry research report also presents a comprehensive analysis of the market by therapy (Enzyme replacement therapy (ERT), Substrate reduction therapy (SRT), and Cystine depleting agents) and by geography (the Americas, APAC, and EMEA).
Overview of the global lysosomal storage diseases market
There is a rising need for efficacious therapeutics that can either slow or reverse the progression of the lysosomal storage disease (LSD). The available medicines in the market are ineffective to improve neurological symptoms associated with LSDs due to the limited penetration of blood brain barrier. Technavio’s market study identifies this unmet need for effective treatment as one of the primary growth factors for the lysosomal storage diseases market. In addition to this, the high cost of treatment will also contribute to the growth of the market. Our market research analysts estimate that this market will grow steadily at a CAGR of more than 7% by 2021.
Lysosomal storage diseases market trends
Several legislative changes are aimed to promote research and development activities for rare diseases in the developing economies. Increasing awareness of the importance of orphan drug designations in these developing regions is encouraging the development of regulatory protocols. Pharmaceutical manufacturers in the developing countries are also focusing extensively on the research and development of novel therapeutics. This industry research report identifies the increase in regulatory assistance in emerging nations as one of the key trends that will contribute to the growth of the lysosomal storage diseases market.
Lysosomal storage diseases market forecast: Competitive landscape and key vendors
The lysosomal storage diseases market is highly consolidated due to the presence of a few key vendors who account for the major market share. The key vendors compete intensely based on factors such as product pipeline, emerging indications/applications, and collaborations. The competition among the vendors in the lysosomal diseases market will intensify in the coming years due to product portfolio expansions and the development of novel therapeutics using emerging technologies.
The leading vendors in the lysosomal storage diseases market
- Actelion Pharmaceuticals
- BioMarin
- Genzyme
- Shire
Lysosomal storage diseases market segmentation by therapy
- Enzyme replacement therapy (ERT)
- Substrate reduction therapy (SRT)
- Cystine depleting agents
End users primarily prefer enzyme replacement therapy (ERT) therapy as ERT contains targeted therapies that are efficient and effective for treating LSDs. Additionally, ERT also provides high efficacy and is administered through intravenous infusion where they metabolize the accumulated substrate effectively.
Lysosomal storage diseases market as per regions
- Americas
- APAC
- EMEA
In EMEA, the rising awareness about the treatment of rare diseases, government initiatives to encourage orphan drug development, and favorable regulatory policies contribute to the growth of the lysosomal storage diseases market. Additionally, the availability of favorable reimbursement policies in Europe will also contribute to the growth of the lysosomal diseases market in the region.
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Key questions answered in the report include
- What will the lysosomal storage diseases marke size and the growth rate be in 2021?
- What are the key factors driving the global lysosomal storage diseases market?
- What are the key lysosomal storage diseases marke trends impacting the growth of the market?
- What are the challenges to market growth?
- Who are the key vendors in the global lysosomal storage diseases market?
- What are the market opportunities and threats faced by the vendors in the global lysosomal storage diseases market?
- Trending factors influencing the market shares of the Americas, APAC, and EMEA.
- What are the key outcomes of the five forces analysis of the global lysosomal storage diseases market?
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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
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.
INFORMATION 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

DATA 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

REPORT WRITING
Qualitative
- Market drivers
- Market challenges
- Market trends
- Five forces analysis
Quantitative
- Market size and forecast
- Market segmentation
- Geographical insights
- Competitive landscape
Key Questions Answered
- 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?
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