Global Lysosomal Disease Treatments: Technologies and Markets, 2023 with Profiles of Major Players - Astellas Pharma, Astrazeneca, Eli Lilly and Co., Merck & Co. and Novo Nordisk A/S - ResearchAndMarkets.com

September 18, 2018

DUBLIN--(BUSINESS WIRE)--Sep 18, 2018--The “Lysosomal Disease Treatments: Technologies and Global Markets” report has been added to ResearchAndMarkets.com’s offering.

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 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.

The Report Includes:

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

Key Topics Covered:

Chapter 1 Introduction

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

Chapter 10 Company Profiles

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 For more information about this report visit https://www.researchandmarkets.com/research/r64vrn/global_lysosomal?w=4

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PUB: 09/18/2018 11:20 AM/DISC: 09/18/2018 11:20 AM


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