Australia Lysosomal Storage Disorders Drugs Market Analysis

Australia Lysosomal Storage Disorders Drugs Market Analysis


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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.

ID: IN10AURD097 CATEGORY: Rare Diseases GEOGRAPHY: Australia AUTHOR: Gaurav Bhongale

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Australia Lysosomal Storage Disorders Drugs Market Analysis Summary

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.

Market Dynamics

Market Drivers

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.

Market Restraints

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 players

BioMarin 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)

4.2.1 Company
4.2.1.1 Overview
4.2.1.2 Product Applications and Services
4.2.1.3 Recent Developments
4.2.1.4 Partnerships Ecosystem
4.2.1.5 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

Market Segmentations For Australia Lysosomal Storage Disease Drugs Market

By Treatment Therapy

  • Enzyme Replacement Therapy
  1. Velaglucerase Alfa
  2. Taliglucerase Alfa
  3. Agalsidase beta
  4. Laronidase
  5. Imiglucerase
  6. Other Enzyme Replacement Therapies
  • Substrate Reduction Therapy
  1. Eliglustat
  2. Miglustat
  3. Other Substrate Reduction Therapies
  • Stem cell transplantation

By Type

  • Gaucher disease
  • Cystinosis
  • Pompe Disease
  • Fabry Disease
  • Tay-Sachs disease

 

By Distribution channel

  • Hospital Pharmacies
  • Retail Pharmacies
  • Online Pharmacies

By Route of Administration

  • Oral
  • Parenteral
  • Others

By End-Users

  • Hospitals
  • Specialty Clinics
  • Homecare
  • Others

Insights10 will provide you with the reports within 10 key parameters which are:

  1. Market Overview
  2. Market Growth Drivers & Restraints
  3. Epidemiology of Disease Type
  4. Market Segmentation
  5. Market Share
  6. Competitive Landscape
  7. Key Company Profiles
  8. Healthcare Policies & Regulatory Framework
  9. Reimbursement Scenario
  10. Factors Driving Future Growth

Based on our many years of experience, we believe that these are the parameters that are critical to decision-making for business stakeholders. Our focused approach to developing reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

 

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Primary Interviews: We have developed a network of experts, freelancers, and researchers across countries through which we engage with local experts to gather key data points and assumptions about each market. We also engage regularly with some of the best market research agencies such as Atheneum, GuidePoint, GLG, etc. to conduct surveys and interviews, and build intelligence. We have language translators as a part of our team, who between them can cover 30+ languages allowing us to extract better local insights.

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Stage II: Market Data Analysis and Statistical Model

Market Trends: We generally look at macro parameters and micro indicators. The macro parameters include changes in government policies, demand and supply of the market, government intervention programs, and major market share. The micro indicators are GDP growth, market size, market volume, etc. We also understand nuances specific to each country like the US, Canada, India, Germany, etc., and have worked across 60+ countries and hence not only understand global trends but how these differ by country, how payment models, market structure, cultural parameters, etc. differ in each country.

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The study on the market covers the analysis of the leading geographies such as Asia-Pacific, Africa, Europe, Middle East, North America, and  Latin America for the period of 2022 to 2030. The qualitative analysis covers the industry landscape and trends, market opportunities, competitive landscape, and policy and regulatory scenario, and the quantitative analysis covers different market estimates and forecasts.

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We make sure to finalize the numbers in alignment with the market research. Firstly, our internal experts ensure thorough validation and checking to ensure accurate and precise analysis and then validation is also done using a multiple-data analysis model. Two-level validation is done and entails the finalization of the report scope and the way of representation pattern.

 

 

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analyzed and synthesized. The second series of interviews are done if necessary to check and validate. The future opportunities are analyzed by understanding product commercialization and many other factors. It also comprises the analysis of data discrepancies observed across various data sources. Information procured from secondary and primary results is then, interpreted by considering the following parameters: (a partial list)

  • Establishing market drivers and trends
  • Analyzing the regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue/volume
  • ​Analyzing current needs and determining penetration to estimate the market

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Data curated from the analysis and interpretation are drawn to portray all in one consolidated report.

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The foreign exchange rates are calculated on the respective dates and for the respective regions covered in the study.

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Last updated on: 04 July 2023
Updated by: Dhruv Joshi

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