Global Market of Mucopolysaccharidosis I Drug

$3,000.00$7,500.00

  • Type: Rare Disease Area
  • Published : March 2021
  • Region: Worldwide
  • Country: Other
  • Report ID : 385

  • Format: PPT, PDF

GLOBAL MUCOPOLYSACCHARIDOSIS I DRUG MARKET – SIZE, SHARE, IMPACT, GROWTH, TRENDS, AND FORECASTS, GLOBAL, NORTH AMERICA, ASIA-PACIFIC, U.S. (2019 – 2028)

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Report Overview

The Global Mucopolysaccharidosis I drugs market size stood at USD 1.9 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.

Mucopolysaccharidosis refers to a group of inherited conditions in which the body is unable to properly breakdown mucopolysaccharides, a long chain of sugar molecules that are found in the body. As a result, these sugars build up in cells, blood, and connective tissues, which can lead to several health problems. There are seven forms of mucopolysaccharidosis. Generally, most of the affected people appear healthy at birth and experience a period of normal development, followed by a decline in physical and/or mental function. As the condition progresses, it may affect appearance, physical abilities, organ and system functioning, and in most cases, cognitive development.

Market Drivers

The growing necessity for advanced therapeutics will impel companies to launch novel therapies, which in turn, will boost the mucopolysaccharidosis treatment market growth in the forthcoming years. Moreover, the growing unmet clinical needs of patients along with better treatment outcomes will spur demand for mucopolysaccharidosis treatment market during the forecast period.

Moreover, the increasing acquisitions and deals between key players will bolster healthy growth of the market. For instance, in April 2019, GC Pharma announced the signing of a licensing deal with Clinigen KK in Japan for their product offering of Hunterase ICV, which is used for the treatment of Mucopolysaccharidosis Type II (Hunter syndrome).

In addition, the increasing R&D by pharmaceutical companies for the mass production of conventional medications or therapies to treat diseases such as heart disease and diabetes along with rare disorders such as the types of mucopolysaccharidosis. This factor will aid the mucopolysaccharidosis treatment market revenue in the foreseeable future.

  1. Global Mucopolysaccharidosis I Drugs Market Overview…….………..……………
    1. Market Size
  2. Market Growth Drivers……………………………………………………………………….
    1. Rise in Global Prevalence of Mucopolysaccharidosis I
    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. Mucopolysaccharidosis I 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. Mucopolysaccharidosis I 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. Shire Company Product & Services, Strategies & Financials
    2. ArmaGen 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 Mucopolysaccharidosis I 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.

By Distribution Channel:

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

By Region:

The market in North America generated a revenue of USD 1,019.4 million in 2018. The growth in the region is attributed to the higher diagnosis and treatment rates for a number of rare disorders. The favorable reimbursement policies for such therapies will also have a weighty impact on the market in North America.

The rising awareness among patient population towards advanced treatment options will also spur demand for mucopolysaccharidosis treatment market. The presence of significant players and biopharmaceutical companies along with ongoing clinical trials will further augment growth in North America. Europe is predicted to witness high growth during the forecast period owing to the high sales of Elaprase and ALDURAZYME in the region.

Asia Pacific is predicted to grow significantly due to launch of novel therapeutics such as Hunterase in Japan. The growing patient population in various countries will enable growth of the market in the region.

Major Players
Key players operating in the market are focusing on obtaining approvals for their advanced therapeutics and this is expected to drive the market growth over the forecast period. For instance, in June 2019, Biomarine Pharmaceuticals Inc. received approval for its Vimizim (elosulfase alfa) from National Medical Products Administration (NMPA) for the treatment of patients with mucopolysaccharidosis type IVA (MPS IVA), also known as Morquio A syndrome. Vimizim is the first treatment in China approved for this condition.

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)

  • Establishing market drivers and trends
  • Analyzing regulatory landscape to understand future growth
  • Market Segment based analysis to obtain revenue / volume
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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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