Australia Lysosomal Storage Disease Drugs Market is projected to grow from $xx Mn in 2023 to $xx Mn by 2030, registering a CAGR of xx% during the forecast period of 2023 ? 2030. The rise in lysosomal diseases, greater public awareness of unusual lysosomal storage disorders, increased research and development for diagnostics, and increased drug development for treating lysosomal diseases are the main drivers impacting the market for lysosomal storage disease treatments. Major global players in Lysosomal Storage Disease Drugs Market are Pfizer Inc, Takeda Pharmaceutical Company Limited (Shire Plc), Sanofi (Genzyme Corporation), BioMarin, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Drugs, Inc, Alexion Pharmaceuticals, Inc, Sigilon Drugs, Inc, and Orphazyme A/S are some of the companies that participate in this industry.
Australia Lysosomal Storage Disorders Drugs Market is valued at around $0.12 Bn in 2022 and is projected to reach $0.29 Bn by 2030, exhibiting a CAGR of 11.5% during the forecast period 2023-2030.
Lysosomal enzyme shortage or dysfunction results in a set of uncommon inherited metabolic disorders known as lysosomal storage diseases. Fabry disease, Gaucher disease, glycogen storage disease II, Tay-Sachs disease, aspartyl glucosaminuria, Batten disease, cystinosis etc are examples of lysosomal storage diseases. Currently, 11 lysosomal storage diseases can be treated using 23 orphan drugs. There are roughly 50 of these ailments in all, and they can affect the skeleton, brain, skin, heart and central nervous system among other bodily components. Treatments available are enzyme replacement therapy in which missing or deficient enzyme is given to individuals example Imiglucerase (Cerezyme) and velaglucerase alfa (VPRIV): Used for the treatment of Gaucher disease. Agalsidase alfa (Replagal) and agalsidase beta (Fabrazyme): Used for the treatment of Fabry disease. Alglucosidase alfa (Myozyme) and alglucosidase alfa (Lumizyme): Used for the treatment of Pompe disease. Substrate reduction therapy main aim is to reduce the production or accumulation of substrates that cannot be metabolized due to enzyme deficiencies example miglustat (Zavesca), used for the treatment of Gaucher disease. Chaperone Therapy in which the use of small molecules that can stabilize and improve the function of misfolded enzymes is done example Migalastat (Galafold). Major global players in Lysosomal Storage Disease Drugs Market are Pfizer Inc, Takeda Pharmaceutical Company Limited (Shire Plc), Sanofi (Genzyme Corporation), BioMarin, Johnson & Johnson (Actelion Pharmaceuticals Ltd), Amicus Drugs, Inc, Alexion Pharmaceuticals, Inc, Sigilon Drugs, Inc, and Orphazyme A/S are some of the companies that participate in this industry.
It is anticipated that throughout the course of the projection period, the global market for therapies for lysosomal storage diseases will expand due to the significant prevalence of LSDs. Additionally, it is hoped that increased public awareness of uncommon diseases, Orphan Drug Designation and Incentives for research and development will encourage market expansion.
The high cost associated with the treatment and lack of skilled professionals treating this disease acts as market growth restraint.
Developments of Australia Lysosomal Storage Disease Drugs Market
In January 2020, Quest Diagnostics bought Blueprint Genetics to increase the range of its product offerings for uncommon and genetic disorders.
Key playersBioMarin Pharmaceutical Inc. Pfizer Sanofi Shire plc Catalyst Biosciences uniQure Ionis Pharmaceuticals Alnylam Pharmaceuticals Intercept Pharmaceuticals Novartis
1. Executive Summary
1.1 Disease Overview
1.2 Global Scenario
1.3 Country Overview
1.4 Healthcare Scenario in Country
1.5 Patient Journey
1.6 Health Insurance Coverage in Country
1.7 Active Pharmaceutical Ingredient (API)
1.8 Recent Developments in the Country
2. Market Size and Forecasting
2.1 Epidemiology of Disease
2.2 Market Size (With Excel & Methodology)
2.3 Market Segmentation (Check all Segments in Segmentation Section)
3. Market Dynamics
3.1 Market Drivers
3.2 Market Restraints
4. Competitive Landscape
4.1 Major Market Share
4.2 Key Company Profile (Check all Companies in the Summary Section)
22.214.171.124 Product Applications and Services
126.96.36.199 Recent Developments
188.8.131.52 Partnerships Ecosystem
184.108.40.206 Financials (Based on Availability)
5. Reimbursement Scenario
5.1 Reimbursement Regulation
5.2 Reimbursement Process for Diagnosis
5.3 Reimbursement Process for Treatment
6. Methodology and Scope
By Treatment Therapy
By Distribution channel
By Route of Administration
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