+353-1-416-8900REST OF WORLD
+44-20-3973-8888REST OF WORLD
1-917-300-0470EAST COAST U.S
1-800-526-8630U.S. (TOLL FREE)

PRINTER FRIENDLY

Lysosomal Disease Treatments: Technologies and Global Markets

  • ID: 4621149
  • Report
  • Region: Global
  • 118 Pages
  • BCC Research
UP TO OFF
until Nov 23rd 2019
1 of 5

FEATURED COMPANIES

  • Amicus Therapeutics
  • Astellas Pharma
  • Astrazeneca
  • Eli Lilly And Company
  • Merck & Co., Inc.
  • Novo Nordisk A/S
  • MORE

The lysosomal storage disease treatment market has been divided by type of indication into: Gaucher’s Diseases, Fabry Diseases, Pompe’s Syndrome, Mucopolysaccharidosis and more. Mucopolysaccharidosis is further segmented into Hunter syndrome, Hurlers Syndrome and Moriquo syndrome. The market is also segmented by routing of administration into IV and oral. The market has been divided into hospitals, clinics and other facilities into end users by type of treatment into enzyme replacement therapy and substrate reduction therapy. The market has been segmented into North America, Europe, Asia-Pacific and RoW.

The Report Includes:

  • 37 data tables and 25 additional tables
  • An overview of the global market for lysosomal disease treatments
  • Analyses of global market trends, with data from 2017, estimates for 2018 and projections of compound annual growth rates (CAGRs) through 2023
  • Segmentation of the global market by cancer therapy indication, treatment type, route of administration, end-use, application, and geographical region
  • Information on the current regulatory environment, and a trend analysis of recent government policies and regulations for lysosomal disease treatments
  • Assessment of lysosomal storage diseases by the accumulated substrate and the profitability of focusing on ultra-orphan diseases
  • Profiles of major players in the industry, including Astellas Pharma, Astrazeneca, Eli Lilly and Co., Merck & Co., Inc., and Novo Nordisk A/S

Membership Price:

Contact us for membership pricing.

Note: Product cover images may vary from those shown
2 of 5

FEATURED COMPANIES

  • Amicus Therapeutics
  • Astellas Pharma
  • Astrazeneca
  • Eli Lilly And Company
  • Merck & Co., Inc.
  • Novo Nordisk A/S
  • MORE

Chapter 1 Introduction

  • Study Goals and Objectives
  • Reasons for Doing This Study
  • Scope of Report
  • Information Sources
  • Methodology
  • Geographic Breakdown
  • Analyst's Credentials
  • Custom Research
  • Related Research Reports

Chapter 2 Summary and Highlights

Chapter 3 Market Overview and Background

  • History
  • Definition
  • Lysosomes
  • Lysosomal Storage Diseases
  • Most common LSDs
  • Symptoms of Lysosomal Storage Diseases
  • Diagnosis of Lysosomal Storage Diseases
  • Treatments for Lysosomal Storage Diseases
  • Market Dynamics
  • Drivers
  • Restraint
  • Importance of Patient Support Groups
  • Regulatory Options for Faster Drug Approval
  • Epidemiology Lysosomal Storage Disorders

Chapter 4 Treatment Options for Lysosomal Storage Diseases

  • Enzyme Replacement Therapy (ERT)
  • Oral Substrate Reduction Therapy (SRT)
  • Pharmacological Chaperone Therapy (PCT)
  • Gene Therapy

Chapter 5 Market Breakdown by Disease Type

  • Gauchers
  • Diagnosis
  • Treatment
  • Oral Substrate Reduction Therapy (SRT)
  • Fabry Disease
  • Type 1 Classic Phenotype
  • Pompe Disease
  • Causes
  • Treatment
  • Mucopolysaccharidosis (MPS)
  • Hurlers (MPS I)
  • Hunter Syndrome (MPS II)
  • Morquio Syndrome (MPS IV)
  • Others

Chapter 6 Market Breakdown by Route of Administration

  • Oral Route of Administration
  • Intravenous Route of Administration

Chapter 7 Market Breakdown by End Use

  • Hospitals
  • Clinics
  • Others

Chapter 8 Market Breakdown by Treatment Type

  • Enzyme Replacement Therapy Market
  • Substrate Reduction Therapy

Chapter 9 Market Breakdown by Region

  • North America
  • Europe
  • Asia-Pacific
  • China
  • India
  • Rest of the World

Chapter 10 Company Profiles

  • ACTELION PHARMACEUTICALS LTD.
  • Overview
  • Financials
  • Products
  • Recent News and Developments
  • AMICUS THERAPEUTICS
  • Financial
  • ARENA PHARMACEUTICALS INC.
  • Overview
  • Financials
  • Recent News and Developments
  • ASTELLAS PHARMA
  • Overview
  • Financials
  • Recent News and Developments
  • ASTRAZENECA
  • Overview
  • Financials
  • BIOMARIN PHARMACEUTICAL INC.
  • Overview
  • Financials
  • Products
  • Recent News and Developments
  • ELI LILLY AND COMPANY
  • Overview
  • Financials
  • Recent News and Developments
  • LEXICON PHARMACEUTICALS, INC.
  • MERCK & CO., INC.
  • Overview
  • Financials
  • Recent News and Developments
  • NOVO NORDISK A/S
  • Overview
  • Product Areas:
  • Financials
  • Revenues by Business Segment
  • Recent News and Developments
  • SANGAMO THERAPEUTICS, INC.
  • Overview
  • Financials
  • Recent News and Developments
  • SANOFI GENZYME
  • Overview
  • Recent News and Developments
  • SHIRE
  • Overview
  • Financials
  • Recent News and Developments

Chapter 11 Appendix A: Global List of Lysosomal Storage Disease Societies

Chapter 12 Appendix B: Abbreviations

Chapter 13 Appendix C: Glossary of Terms

List of Tables
Summary Table: Global Market for Lysosomal Storage Diseases Treatment, by Type, Through 2023
Table 1: Major Events in Lysosomal Storage Diseases, 1882-2004
Table 2: Types of Lysosomal Storage Diseases
Table 3: Most Common LSDs
Table 4: Orphan Drug Regulations in Different Countries
Table 5: Comparison of Policies in Different Countries
Table 6: Orphan Drug Market Exclusivity in Various Countries
Table 7: Summary of Selective LSDs Intended for Newborn Screening, Clinical and Biochemistry Features and Comparison of Enzyme Methods
Table 8: Recommendations to Overcome the Restraints
Table 9: Therapeutic Options and Target Diseases
Table 10: Global Market for Gaucher’s Disease Treatment, by Region, Through 2023
Table 11: Fabry Disease Sign and Symptoms
Table 12: Global Market for Fabry Disease Treatment, by Region, Through 2023
Table 13: Global Market for Pompe’s Disease Treatment, by Region, Through 2023
Table 14: Global Market for Mucopolysaccharidosis Disease Treatment, by Region, Through 2023
Table 15: Global Market for Hurlers Disease Treatment, by Region, Through 2023
Table 16: Global Market for Hunter Disease Treatment, by Region, Through 2023
Table 17: Global Market for Morquio Disease Treatment, by Region, Through 2023
Table 18: Global Market for Other LSD Treatment, by Region, Through 2023
Table 19: Global Market for Lysosomal Storage Diseases Treatment, by Route of Administration, Through 2023
Table 20: Global Market for Lysosomal Storage Disease Via Oral Route of Administration, by Region, Through 2023
Table 21: Global Market for Lysosomal Storage Diseases Via IV Route of Administration, by Region, Through 2023
Table 22: Global Market for Lysosomal Storage Diseases Treatment, by End Use, Through 2023
Table 23: Global Market for Lysosomal Storage Diseases in Hospitals, by Region, Through 2023
Table 24: Global Market for Lysosomal Storage Diseases in Clinics, by Region, Through 2023
Table 25: Global Market for Lysosomal Storage Diseases in Others, by Region, Through 2023
Table 26: Global Market for Lysosomal Storage Diseases Treatment, by Types of Therapy, Through 2023
Table 27: Approved ERT for LSDs
Table 28: Global Market for Lysosomal Storage Diseases in Enzyme Replacement Therapy, by Region, Through 2023
Table 29: Global Market for Lysosomal Storage Diseases Treatment in Substrate Reduction Therapy, by Region, Through 2023
Table 30: Comparison of Different Countries and Rare Disease Policies
Table 31: North American Market for Lysosomal Storage Diseases Treatment, by Type, Through 2023
Table 32: European Market for Lysosomal Storage Diseases Treatment, by Type, Through 2023
Table 33: Asia-Pacific Market for Lysosomal Storage Diseases Treatment, by Type, Through 2023
Table 34: Comparison of South East Asia-Pacific Countries
Table 35: Rest of the World Market for Lysosomal Storage Diseases Treatment, by Type, Through 2023
Table 36: Actelion Pharmaceuticals: Revenues and R&D Expenditures, Through 2016
Table 37: Actelion Pharmaceuticals: Product Developments, 2017
Table 38: Amicus Therapeutics: Revenues and R&D Expenditures, Through 2016
Table 39: Arena Pharmaceuticals: Revenues and R&D Expenditures, Through 2016
Table 40: Arena Pharmaceuticals: Product Developments, 2017
Table 41: Astellas Pharma: Revenues and R&D Expenditures, Through 2017
Table 42: Astellas Pharma: Product Developments, 2017
Table 43: AstraZeneca: Revenues and R&D Expenditures, Through 2016
Table 44: AstraZeneca: Revenues, by Country/Region, 2014-2016
Table 45: BioMarin Pharmaceutical: Revenues and R&D Expenditures, Through 2016
Table 46: BioMarin Pharmaceutical: Product Developments, 2017
Table 47: Eli Lilly: Revenues and R&D Expenditures, Through 2016
Table 48: Eli Lilly’s Revenues: by Country/Region, 2014-2016
Table 49: Eli Lilly: Product Developments, 2017
Table 50: Merck & Co.: Revenues and R&D Expenditures, Through 2016
Table 51: Merck & Co.: Product Developments, 2017
Table 52: Novo Nordisk: Revenues and R&D Expenditures, Through 2016
Table 53: Novo Nordisk: Revenues, by Business Segment, 2014-2016
Table 54: Novo Nordisk: Product Developments, 2017
Table 55: Sangamo Therapeutics: Revenues and R&D Expenditures, Through 2016
Table 56: Sangamo Therapeutics’ Product Developments, 2015-2017
Table 57: Sanofi Genzyme: Product Developments, 2016-2017
Table 58: Shire: Revenues and R&D Expenditures, Through 2016
Table 59: Shire’s Product Developments, 2017
Table 60: Abbreviations Used in Lysosomal Disease Treatments
Table 61: Glossary of Terms Used in Lysosoaml Storage Diseases Treatment Market

List of Figures
Summary Figure: Global Market for Lysosomal Storage Diseases Treatment, by Type, 2017-2023
Figure 1: Timeline of Lysosomal Storage Diseases
Figure 2: Market Dynamics

Note: Product cover images may vary from those shown
3 of 5

Loading
LOADING...

4 of 5

FEATURED COMPANIES

  • Amicus Therapeutics
  • Astellas Pharma
  • Astrazeneca
  • Eli Lilly And Company
  • Merck & Co., Inc.
  • Novo Nordisk A/S
  • MORE

Lysosomal storage diseases are a rare group of genetic disorders that resulted from the malfunctioning or lack of lysosomal enzymes. Tay-Sachs disease was the first lysosomal storage disorder (LSD). It was described in 1881 and was followed by Gaucher disease in 1882. The lysosomal storage diseases (LSD) are a group of 50 diseases that are characterized by the accumulation of waste product in the lysosomes. They are rare, inborn defects and have a combined incidence rate of 1 in 1,500 to 7,000 live births. In lysosomal storage diseases, there is a deficiency of 1 or more of the many catabolic enzymes that are located within the lysosome. Many of the diseases known today were described even before the discovery of lysosomes. The last decade has witnessed major advances in our understanding of the clinical, biochemical and genetical aspects of lysosomal storage diseases.

The major drivers influencing the market are orphan drug status; this is a major driver of the market. Increasing awareness, rising focus by pharmaceutical companies and early diagnosis as well as newborn screening are the other drivers. The market also faces a few restraints that include expensive drugs, change in payer policies, lack of proper training and knowledge, nature of disease and concerns in diagnosis. Restraints are overcome by the drugs that aid in improving the lifespan and quality of life for the patients.

The U.S. is the major market for lysosomal storage disease treatment. It was the first country to implement orphan drug status and to provide many incentives for orphan drugs for treating rare diseases. Europe is the second-largest market; it also stands by the Orphan Drug Act. Asia-Pacific is showing the highest growth rate during the forecasted period. In China and India, the market is limited due to the high prices of the drugs.

The lysosomal storage diseases market is a subset of the larger orphan drugs market and is impacted by any change in regulation and policy on orphan drugs. There is an increasing focus on LSD as there are effective diagnostic systems to detect greater numbers of patients. The market for Lysosomal storage diseases is a concentrated market with many large players present in the market. Major players in the market are Sanofi, Pfizer, Shire, and others.

Note: Product cover images may vary from those shown
5 of 5
  • Actelion Pharmaceuticals Ltd.
  • Amicus Therapeutics
  • Arena Pharmaceuticals Inc.
  • Astellas Pharma
  • Astrazeneca
  • Biomarin Pharmaceutical Inc.
  • Eli Lilly And Company
  • Lexicon Pharmaceuticals, Inc.
  • Merck & Co., Inc.
  • Novo Nordisk A/S
  • Sangamo Therapeutics, Inc.
  • Sanofi Genzyme
  • Shire
Note: Product cover images may vary from those shown
Adroll
adroll