Australia Sanfillipo Syndrome (MPS-III) Market Analysis

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  • Type: Rare Disease Area
  • Published : April 2021
  • Region: APAC
  • Country: Australia
  • Report ID : 4086

  • Format: PPT, PDF

Australian Sanfillipo Syndrome (MPS III) Therapeutics Market: Segmented by Therapeutics (Enzyme Replacement Therapy, Gene Therapy, Stem cell Therapy, Use of Pharmacological Chaperones and Other therapies), by Route of Administration (Oral, Parenteral & Others), and by Distribution Channel (Hospital Pharmacies, Retail Pharmacies & Online Pharmacies) – Growth, Trends, and Forecasts (2020 – 2028F)

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Report Overview

The Australian Sanfillipo Syndrome Therapeutics Market size stood at around USD XX billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Mucopolysaccaridosis  III (MPS III) is a rare genetic condition that causes fatal brain damage. It is also known as Sanfilippo syndrome and is a type of childhood dementia. MPS III is caused by a lack of an enzyme that normally breaks down and recycles a large, complex sugar molecule called ‘heparan sulphate’. This heparan sulphate accumulates and causes damage to the cells of the central nervous system, including the brain. Sanfilippo belongs to a group of disorders known as the “Mucopolysaccharidoses” (MPS), which are part of a larger group of disorders known as “lysosomal storage disorders”. Children with MPS III usually appear healthy at birth, but developmental delay is usually evident by age 2-5 years. Mental and motor development peak by 3-6 years of age, after which intellectual decline usually occurs. Behavioral problems such as hyperactivity and irritability may become obvious earlier. Severe behavioral disturbance is a very common feature of Sanfilippo syndrome, and one of the more difficult aspects of the disorder to manage.

Sanfilippo is considered a rare disease: 1 in 70,000 children are born with the inherited condition. There are between 75 and 100 children living with Sanfilippo in Australia.

Market Drivers

The growing necessity for advanced therapeutics will impel companies to launch novel therapies, which in turn, will boost the Sanfillipo Syndrome (MPS III) Therapeutics market growth in the forthcoming years. Moreover, the growing unmet clinical needs of patients along with better treatment outcomes, financial incentives for orphan drug development to recover R&D costs, success for the drugs in pipeline with faster market access, high burden of rare diseases, favorable regulations for orphan products development will spur demand for Mucopolysaccharidosis Therapeutics market during the forecast period. Moreover,  increasing acquisitions and deals between key players will bolster healthy growth of the market. High Treatment Costs, Lack of treatment options & lack of awareness will continue to hamper market.

1. Australian Sanfillipo Syndrome Therapeutics Market Overview………………..
A. Market Size
2. Market Growth Drivers…………………………………………………………………………………….
A. Advancement in Therapies
B. Increase in Awareness Levels of Rare Diseases
3. Major Sanfillipo Syndrome Types………………………………………………..………………..
A. Type A
B. Type B
C. Type C
D. Type D
4. Australian Sanfillipo Syndrome Therapeutics Market Segmentation……….
A. By Therapeutics
I. Enzyme Replacement Therapy
II. Gene Therapy
III. Stem cell Therapy
IV. Use of Pharmacological Chaperones
V. Others therapies
B. By Distribution Channel
I. Hospital Pharmacies
II. Retail Pharmacies
III. Online Pharmacies
C. By Routes of Administration
I. Oral
II. Parenteral
III. Others
5. Australian Sanfillipo Syndrome Major Products Market Share……………
A. Market Analysis, Insights and Forecast – By Revenue Type
6. Competitive Landscape…………………………………………………………………
A. Major Players
B. Products in Pipeline
7. Key Company Profiles…………………………………………………………………..
A. Biomarin Company overview, Product & Services, Strategies & Financials
B. Genzyme Company overview, Product & Services, Strategies & Financials
C. Abeona Therapeutics Company overview, Product & Services, Strategies & Financials
8. Healthcare Policies and Regulatory Landscape……………………………………
A. Policy changes and Reimbursement scenario
9. Factors Driving Future Growth…………………………………………………………
A. New Trends and Development of Sanfillipo Syndrome Therapeutics market
B. Future Opportunities
10. Conclusion

Market Segmentation

By Distribution Channel:

Based on distribution channel, the Sanfillipo Syndrome Therapeutics market consists of hospital pharmacies, retail pharmacies & online pharmacies.

By Routes of administration:

Based on routes of administration, the Sanfillipo Syndrome Therapeutics market is segmented into oral, parenteral and others.

By Therapeutics:

Based on Therapeutics, the Sanfillipo Syndrome Therapeutics market involves enzyme replacement therapy, gene therapy, stem cell therapy, use of pharmacological chaperones along with other therapies.

Other hematopoietic stem cell transplantation involves the transplants was highly under doubts of capability for providing neurological safety and benefits thus is not considered an effective option. At present, the clinical trials for enzyme replacement therapy (ERT) are underway for Sanfilippo syndrome treatment. Gene Therapy although highly complex, is the most viable option for rectifying the genes. Another challenge the therapy poses is the insertion of the normal gene into hundreds of millions of cells that contain the defective gene; which requires a carrier to transport the gene inside the cells.

Top Major Players

The market payers from Sanfillipo Syndrome Therapeutics market are anticipated to lucrative growth opportunities in the future with the rising demand for Sanfillipo Syndrome Therapeutics in the market. Major players operating in the market include BioMarin Pharmaceuticals Australia Pty Ltd., Genzyme, a Sanofi Company, ArmaGen, Inc., Abeona Therapeutics among others.

Research Methodology

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

  1. Market overview
  2. Market growth drivers & restraints
  3. Epidemiology or 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 develop reports focused on 10 key parameters, enabled us to arrive at the name “Insights10”.

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The top-down approach mainly used for new product forecasting and bottom-up approach is used for demand estimation of any product for different countries summed up to form total market. We are able to round off insights and build stronger forecasts because we always do both these methods and triangulate the final numbers.

The study on the market covers the analysis of the leading geographies such as North America, Europe, Asia-Pacific, and Africa for the period of 2020 to 2028. 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 to the market research. Firstly, our internal experts ensure thorough validation and checking to ensure the accurate and precise analysis and then validation is also done using multiple data analysis model.  Two level validation is done and entails finalization of report scope and to way of representation pattern.

Stage III: Interpretation and Presentation

Analysis & Interpretation: The information gathered is then analysed and synthesized. A second series of interviews are done if necessary to check and validate. The future opportunities are analysed 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 are then, interpreted by considering following parameters: (a partial list)

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  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
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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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