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
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Table of Contents
Executive 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 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.
Companies Mentioned
- 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
