Global MPS-III (Sanfilippo Syndrome) Drug Market Analysis


  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 387

  • Format: PPT, PDF



Report Overview

The Global Sanfilippo syndrome drugs market size stood at USD 0.7 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during
the forecast period.

Sanfilippo Syndrome, also known as Mucopolysaccharidosis type III (MPSIII), is a rare genetic condition that causes fatal brain damage. It is referred to as a childhood disease because most patients fail to reach adulthood. Sanfilippo is a metabolic disorder. One in 70,000 children is born with this inherited condition, which is caused by an enzyme deficiency.

Market Drivers

Rise in number of people suffering from Lysosomal diseases, financial incentives for orphan drug development to recover R&D costs, including market exclusivity, tax waiver, fee reductions and grants, success for the drugs in pipeline with faster market access, high burden of rare diseases, favorable regulations for orphan products development are some of the factors favoring market growth over the forecast period. The factors limiting the market growth is high treatment cost, heterogeneity of the disease, lack of treatment options, lack of awareness and others are some of the factors restraining the growth of the market. Growth opportunities such as increase in R&D for orphan drugs driven by consolidation of Pharma-biotech firms in mucopolysaccharidosis treatment, initiatives to improve diagnosis, research and development in therapies targeting neuropathic lysosomal storage disorder by crossing blood brain barrier (BBB) is boosting the growth of the target market.

  1. Global Sanfilippo syndrome Drugs Market Overview…….……………..……………
    1. Market Size
  2. Market Growth Drivers……………………………………………………………………….
    1. Rise in Global Prevalence of Sanfilippo Syndrome
    2. Growing R&D Investments and Reimbursement Policies
  3. Major Types of Treatments…….………..……………..……………..…….……………….
    1. Enzyme Replacement Therapy
    2. Gene Therapy
    3. Stem Cell Therapy
    4. Others
  4. Sanfilippo syndrome Drugs Market Segmentation….………………………….………
    1. Drug Type
      1. Oral
      2. Parental
      3. Others
    2. Geography
      1. North America Market Analysis
      2. Europe Market Analysis
      3. Asia-Pacific Market Analysis
      4. Middle East and Africa Market Analysis
    3. Distribution Channel
      1. Hospital Pharmacies
      2. Retail Pharmacies
      3. Online Pharmacies
      4. Others
  5. Sanfilippo syndrome Drugs Market Share…………..……………..……………………
    1. Key Findings
    2. Market Analysis, Insights and Forecast – By Drug Type
  6. Competitive Landscape…………………………….……………………………………….
    1. Major Players
    2. Products in Pipeline
  7. Key Company Profiles…………………………………………………………………..
    1. Seelos Therapeutics Product & Services, Strategies & Financials/li>
    2. Lysogene Pharmaceutical Product & Services, Strategies & Financials
    3. Biomarin Company, Product & Services, Strategies & Financials
  8. Healthcare Policies and Regulatory Landscape…………………….……..……….…
    1. Policy changes and Reimbursement scenario
  9. Factors Driving Future Growth……………………………………………………………
    1. New Trends and Development of Sanfilippo Syndrome Drugs Market
    2. Future Opportunities
  10. Conclusion

Market Segmentation

By Drug Class:

The treatment segment is divided into enzyme replacement therapy and others. The enzyme replacement therapy is the primary treatment for MPS disorders, and they aim at replacing or generating the enzymes that are missing in the body, which is the primary reason for causing MPS III.

By Distribution Channel:

Based on the distribution channel, the market has been segregated into hospitals, retail, online and other pharmacies.

By Region:

Region wise, the global Mucopolysaccharidosis III treatments market is classified into regions namely, North America, Latin America, Western Europe, Eastern Europe, Asia-Pacific, Japan, Middle East, and Africa. North America is estimated to account for major share due to favourable regulations for orphan products development, funding for research and development. Over the last few years, introduction of specific orphan drug regulations has enabled and accelerated research and development of therapies for treatment of several rare diseases. Health plan coverage and reimbursement policies for such costly drugs also influence the investment by pharmaceutical and biotechnology companies to invest in drug development of such costly drugs. Significant economic development has led to an increase in healthcare availability in Asia Pacific region providing an opportunity for biopharmaceutical companies to penetrate in Asia Pacific regions, rising awareness towards lysosomal storage diseases, collaborations to develop new class of medicine for lysosomal storage diseases are some of the factors fueling growth of the market.

Major Players

Some of the key players in global Mucopolysaccharidosis III treatment market are BioMarin Pharmaceutical, Inc., Genzyme, a Sanofi Company, ArmaGen, Inc., Sangamo Therapeutics and 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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Market Sizing and Analysis: Our expert data analytics team has created various market forecast models by employing the top-down approach i.e. start with large overall market and segment different areas and bottom-up approach i.e. start with population and epidemiology and roll up based on spend, etc., estimating the size of the market, and distributing among the geographic and/or product segments.

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.

Data Triangulation & Validation:

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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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  • 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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