Updated Data Matrix Available

Market Value | Market Volume | Epidemiology | Clinical Trials | Patent Landscape | Regulatory Approvals

Lysosomal Storage Disorders (LSD) Market by Product [Gaucher’s Disease [Cerezyme (imiglucerase), Zavesca (miglustat), Vpriv (velaglucerase alfa), Elelyso (taliglucerase alfa), Cerdelga (eliglustat)], Fabry’s Disease [Replagal (agalsidase alfa), Fabrazyme (agalsidase beta), Galafold (migalastat)], Pompe’s Syndrome [Myozyme / Lumizyme (alglucosidase alfa), Nexviadyme (avalglucosidase alpha)], Mucopolysaccharidosis (MPS) – Hurler Syndrome / Hurler-Scheie Syndrome (MPS-I) [Aldurazyme (laronidase)], Hunter Syndrome (MPS-II) [Elaprase (idursulfase/ iduronate-2 sulfatase)], Morquio A Syndrome (MPS-IV A) [Vimizim (elosulfase alfa)], Maroteaux-Lamy Syndrome (MPS-IV) [Aryplase (N-acetylgalactosamine 4-sulfatase), Naglazyme (galsulfase)] and Forecast 2024-2031


Report Specifications: Market Size, Market Share, Growth Trends, Forecast, Geography

The lysosomal storage disorders (LSDs) are a group of over 70 rare inherited metabolic disorders that are caused for the most part by enzyme deficiencies within the lysosome resulting in accumulation of undegraded substrate. Examples of LSDs include the Gaucher disease, Fabry disease, Pompe disease, mucopolysaccharidoses, mucolipidoses, oligosaccharidoses, the Niemann-Pick disorders, and neuronal ceroid lipofuscinoses.

Several LSDs can be treated with approved, disease-specific therapies that are mostly based on enzyme replacement. However, small-molecule therapies, including substrate reduction and chaperone therapies, have also been developed and are approved for some LSDs, whereas gene therapy and genome editing are at advanced preclinical stages and, for a few disorders, have already progressed to the clinic. The main treatment methods include enzyme replacement therapy (ERT), substrate reduction therapy (SRT), use of molecular chaperones, bone marrow transplantation, and gene therapy.

The global lysosomal storage disorders (LSD) market research report provides market size ($million 2021 to 2031), market share analysis, growth trends and forecast (CAGR%, 2024 to 2031).

The global lysosomal storage disorders market segmented by product [Gaucher’s disease [Cerezyme (imiglucerase), Zavesca (miglustat), Vpriv (velaglucerase alfa), Elelyso (taliglucerase alfa), Cerdelga (eliglustat)], Fabry’s disease [Replagal (agalsidase alfa), Fabrazyme (agalsidase beta), Galafold (migalastat)], Pompe’s syndrome [Myozyme / Lumizyme (alglucosidase alfa), Nexviadyme (avalglucosidase alpha)], Mucopolysaccharidosis (MPS) – Hurler syndrome / Hurler-Scheie syndrome (MPS-I) [Aldurazyme (laronidase)], Hunter syndrome (MPS-II) [Elaprase (idursulfase/ iduronate-2 sulfatase)], Morquio A syndrome (MPS-IV A) [Vimizim (elosulfase alfa)], Maroteaux-Lamy syndrome (MPS-IV) [Aryplase (N-acetylgalactosamine 4-sulfatase), Naglazyme (galsulfase)], and geography.

The global lysosomal storage disorders market research report is further segmented by geography into North America (U.S., Canada), Europe (U.K., Germany, France, Italy, Spain, Rest of EU), Asia Pacific (Japan, China, India, Rest of APAC), Latin America (Brazil, Mexico, Rest of LA), and Rest of the World.

In addition, the global lysosomal storage disorders market report provides the detailed market landscape (market drivers, restraints, opportunities), market attractiveness analysis, and market profitability analysis by key products and regions or countries. The report also tracks the major competitors operating in the global market by company overview, financial snapshot, major products, technologies, services offered and recent developments.

Major players operating in the global lysosomal storage disorders market and profiled in this report include Amicus Therapeutics, Inc., BioMarin Pharmaceutical, Inc., Genzyme Corporation (Sanofi Genzyme), Johnson & Johnson (Actelion Pharmaceuticals, Inc.), Protalix Biotherapeutics, Inc., Pfizer, Inc., Sanofi S.A., Sumitomo Dainippon Pharma, and Takeda Pharmaceutical Company Ltd. (Shire plc).

DATA INCLUDED: Lysosomal Storage Disorders Market Size, Lysosomal Storage Disorders Market Share, Lysosomal Storage Disorders Market Growth Rates, Lysosomal Storage Disorders Market Trends, and Lysosomal Storage Disorders Market Forecast to 2031

Market Data:

  • Base year for estimation: 2023
  • Actual estimates/Historical data: 2021-2031
  • Forecast period: 2024-2031
  • Market representation: Revenue in $million and CAGR % from 2024 to 2031
  • Regional scope: North America, Europe, Asia Pacific, Latin America, Rest of the World (Middle East & Africa)
  • Country scope: U.S., Canada, U.K., Germany, France, Italy, Spain, Japan, China, India, South Korea, Brazil, Mexico, South Africa, South Korea, Saudi Arabia, UAE and others.
  • Report coverage: Revenue forecast, company share, competitive landscape, growth factors, and trends
  • 15% free customization scope: Any specific or custom requirements, which is not currently within the scope of the report, will be provided as a part of customization.

Qualitative Analysis:

  • Industry overview
  • Industry trends
  • Market drivers and restraints
  • Key market opportunities
  • Market size
  • Growth trends
  • Porter’s five forces analysis
  • Market attractiveness analysis
  • Competitive landscape
    • Company overview
    • Financial performance
    • Product portfolio
    • Strategic insights
    • Recent developments

Quantitative Analysis:

  • Market size estimates and forecast for all segments
  • Regional market size estimated and forecast for major countries
  • Company financial performance
  • Competitive landscape: Company market share analysis

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  1. Introduction
  2. Executive Summary
    • Market Size Estimation ($million, 2021-2031)
    • Forecast Estimation ($million and CAGR%, 2024-2031)
  3. Research Methodology
  4. Market Landscape
    • Market Dynamics
      • Drivers
      • Barriers
      • Opportunities
    • Market Share Analysis
      • Companies
      • Drugs
    • Market Trends Analysis
      • Key success factors
      • Market Growth Rate
    • Market Attractiveness Analysis
    • Market Profitability Analysis
      • Buyer power
      • Supplier power
      • Barriers to entry
      • Threat of substitute products
      • Rivalry among firms in the industry
    • Distribution Channels
  5. Market Segmentation [refer Market Segments and Companies Tab]
    • Drug Class or Drug (Brand) Type
    • Drug or Disorder Test
    • Indication Type
    • Drug Treatment
    • Mechanism of Action
    • Therapeutic Area or Nature of Application
    • End User Groups
  6. Geography (Region, Country)
    • North America (U.S., Canada)
    • Europe (U.K., Germany, France, Italy, Spain, Rest of EU)
    • Asia Pacific (Japan, China, India, Rest of APAC)
    • Latin America (Brazil, Mexico, Rest of LA)
    • Rest of the World (Middle East & Africa)
  7. Regulatory Overview 
    • IND, NDA filed, FDA, EMEA Approvals
  8. Pipeline Analysis(Phase 1, 2 and 3 Drugs)
    • Phase 3 Drugs Forecast Estimation (Approval to 2031)
    • Phase 1 and 2 Drugs – Qualitative Analysis
  9. Company Profiles [refer Market Segments and Companies Tab]
    • Company Overview
    • Financial Snapshot
    • Product Portfolio
    • Business Strategies
    • Recent Developments
  10. Recommendations
  11. References

Key Questions Answered:
1. What are the factors driving and limiting this market, key success factors and challenges?
2. Which are the leading competitors in the market, their market share, profitability and key trends by product, technology, services portfolio?
3. How do markets perform between countries and regions?
4. What market segments are contributing to maximum share and greatest growth?
5. What are the product or procedure volumes by country and regions?

Actionable Insights Included:
1. Product portfolio matrix (Share vs. Growth)
2. Market attractiveness (Region vs. Growth)
3. Spot matrix (Product vs. Region)
4. Geographic distribution matrix (Share vs. Growth)

Lysosomal Storage Disorders (LSD) Market

1. Product
1.1. Gaucher’s Disease
1.1.1. Cerezyme (imiglucerase)
1.1.2. Zavesca (miglustat)
1.1.3. Vpriv (velaglucerase alfa)
1.1.4. Elelyso (taliglucerase alfa)
1.1.5. Cerdelga (eliglustat)
1.2. Fabry’s Disease
1.2.1. Replagal (agalsidase alfa)
1.2.2. Fabrazyme (agalsidase beta)
1.2.3. Galafold (migalastat)
1.3. Pompe’s Syndrome
1.3.1. Myozyme / Lumizyme (alglucosidase alfa)
1.3.2. Nexviadyme (avalglucosidase alpha)
1.4. Mucopolysaccharidosis (MPS)
1.4.1. Hurler Syndrome / Hurler-Scheie Syndrome (MPS-I) Aldurazyme (laronidase)
1.4.2. Hunter Syndrome (MPS-II) Elaprase (idursulfase/ iduronate-2 sulfatase)
1.4.3. Morquio A Syndrome (MPS-IV A) Vimizim (elosulfase alfa)
1.4.4. Maroteaux-Lamy Syndrome (MPS-IV) Aryplase (N-acetylgalactosamine 4-sulfatase) Naglazyme (galsulfase)
1.5. Others

2. Indication
2.1. Gaucher’s Disease
2.2. Fabry’s Disease
2.3. Pompe’s Syndrome
2.4. Mucopolysaccharidosis (MPS)
2.5. Others

3. Geography
3.1. North America (U.S., Canada)
3.2. Europe (U.K., Germany, France, Italy, Spain, Rest of EU)
3.3. Asia Pacific (Japan, China, India, Rest of APAC)
3.4. Latin America (Brazil, Mexico, Rest of LA)
3.5. Rest of the World

4. Company Profiles
4.1. Amicus Therapeutics, Inc.
4.2. BioMarin Pharmaceutical, Inc.
4.3. Genzyme Corporation (Sanofi Genzyme)
4.4. Johnson & Johnson (Actelion Pharmaceuticals, Inc.)
4.5. Protalix Biotherapeutics, Inc.
4.6. Pfizer, Inc.
4.7. Sanofi S.A.
4.8. Sumitomo Dainippon Pharma
4.9. Takeda Pharmaceutical Company Ltd. (Shire plc)

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