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Lysosomal Disease Treatments: Technologies and Global Markets

Published By :

BCC Research

Published Date : Sep 2018

Category :

Diseases & Conditions

No. of Pages : 118 Pages

"Summary

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 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 deficiency of 1 or more of themany 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.

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 efficient diagnostic systems to detect greater numbers of patients. The market for Lysosomal storage diseases is estimated to reach a value of nearly REDACTED in 2018 and is expected to grow at a compound annual growth rate (CAGR) of REDACTED to nearly REDACTED by 2023. Themarket for Lysosomalstorage diseases is a concentrated market with many large players present in the market. Major players in themarket are Sanofi, Pfizer, Shi re and others."

"Report Scope:

The lysosomal storage disease treatment market has been divided by type of indication into: Gauchers Diseases, Fabry Diseases, Pompes 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.

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 indiscation, treatment type, route of administration, end-use, application and geographical region
- Information on 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"
Table of Contents
Chapter 1 Introduction
Study Goals and Objectives
Reasons for Doing This Study
Scope of Report
Information Sources
Methodology
Geographic Breakdown
Analyst's Credentials
BCC Custom Research
Related BCC 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 Table

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 Gauchers 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 Pompes 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 Lillys 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 : Shires 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 Chart

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

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