MPS Treatment – Focus Hinged at Clinical Trials and Drug Exclusivity
Clinical trials remain the focal point of MPS treatment drug manufacturers, in a bid to drive developments in new treatments, which range from gene therapy and enzyme replacement therapies, to pathophysiology. Drug exclusivity is one of the prime emphasized areas of research, encouraging the development of new biological products and drugs, for improving the provision of care.
Top 3 Factors shaping the mucopolysaccharidosis treatment Market
Drug Exclusivity Provides Continuous Value Addition for Companies
Regulatory boards are likely to grant the orphan drug designation to drugs proposed for the treatment of rare diseases or conditions. The orphan drug designation provides market exclusivity for a concerned drug type, which needs to be generated before submitting an application for marketing approval. Mucopolysaccharidosis treatment falls under the rare disease category due to which emerging market players are adopting various strategies to boost the sales of their products. The orphan drug designation for mucopolysaccharidosis treatment provides prospects such as financial backing for clinical trial costs, tax benefits, and FDA user fee benefits. Majority of the companies present in the mucopolysaccharidosis treatment market are directly involved in receiving drug exclusivity, which is expected to boost the revenue generation as well as result in a significant increase in competition.
Cost of mucopolysaccharidosis treatment is Hampering the Adoption of the Same
The cost of the treatment of rare diseases, such as mucopolysaccharidosis, is high irrespective of whether a patient opts for a standard treatment such as ERT or emerging and promising technologies such as stem cell therapies. The high cost of mucopolysaccharidosis treatment significantly indirectly affects the businesses of manufacturers due to factors such as potential patient count, sluggish awareness, and lack of epidemiological data for better calculations. The real cost of Mucopolysaccharidosis treatment differs by region due to factors such as socioeconomic conditions, birth rate, R&D investments, and healthcare expenditure, among others, which also influence the treatment cost in each region. The cost of mucopolysaccharidosis treatment in the North America region has been found to be significantly higher than the global average treatment cost.
Emerging Technologies are Contributing to the Growth of the mucopolysaccharidosis treatment Market in Terms of Revenue as well as New Treatment Options
A number of emerging treatments and therapies are currently in the development phase for MPS treatment. Nowadays, companies are involved in discovering the next major drug instead of waiting to benefit from the patent expiration of an exclusive drug type, which is proving to be an effective strategy. The existing drug types are facing competition from emerging candidates. The development of mucopolysaccharidosis treatment by companies such as Abeona Therapeutics, ArmaGen, and others is likely to provide a more progressive structure for the mucopolysaccharidosis treatment market in the coming years. For instance, on January 23, 2018, a Swedish Orphan Biovitrum AB received FDA acceptance based on its investigational new drug application for SOBI003, which has been issued for mucopolysaccharidosis treatment.
Mucopolysaccharidosis treatment Market Structure Analysis
- The current global Mucopolysaccharidosis treatment market is controlled by four major companies: Takeda Pharmaceutical Company Limited, Sanofi S.A., BioMarin, and Ultragenyx Pharmaceutical Inc.
- A number of companies have products in the clinical phases with an orphan drug exclusivity
- The Mucopolysaccharidosis treatment market is highly consolidated with very few market players, however, the emergence of new manufacturers is slow due the requirement of a long time period for the approval of drugs as well as lack of resources
Market Set to Grow 1.8X between 2019 and 2029
Clinical manifestations of MPS continue to derive a cascade of hypothetical research discoveries, which in turn have led realizations of pathways toward novel therapeutic targets. This will remain a key enabler of developments in adjunctive therapies for MPS in the foreseeable future.
Waves of Growth in Emerging Markets
Developed markets remain vital revenue pockets for stakeholders in the MPS treatment market, owing to a relatively aware population and economic vigour. However, emerging markets are considered to hold immense opportune potential, as governments in these economies increase expenditure on healthcare infrastructure & facilities. Comparatively denser proportion of demographic in emerging countries also add to greater occurrence possibility of rare genetic disorders, which in turn might create lucrative avenues for MPS treatment market players.
* This data is only a representation. Actual data may vary and will be available in report.
MPS treatment market stakeholders have directed their focus toward stem cell therapies which is leveraging analytics for effective correlations and calculative, personalized treatments. A palpable rise in incidences of MPS indications and notable growth in hospitalizations of associated chronic ailments, have provided an impetus to drug discovery & development activities. A prime focus area of research among players is breaking blood brain barrier via ERT treatment.
Enzyme replacement therapies are preferred treatment of choice for MPS-affected patients, accounting for bulk shares of the market.
Shares of stem cell therapies will continue to be marginal in the MPS treatment market
Hospitals continue to be the prime location for consultation and treatment among patients detected with MPS, accounting for over 90% shares.
Gains from specialty clinics will remain relatively significant compared to sluggish revenues from home infusion and medical research centres.
How Growth will Unfold
Gains from MPS II treatment are expected to account for leading market shares, as the prevalence of this MPS condition is relatively higher according to the International MPS Society. On the other hand, revenues from treatment of MPS I and MPS IVA are expected to grow at a relatively higher rate.
MPS II | MPS IVA | MPS I
* This data is only a representation. Actual data may vary and will be available in report.Click Here To Know How The Growth Will Unfold
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Mucopolysaccharidosis Treatment Market: Global Industry Analysis 2014–2018 and Opportunity Assessment 2019–2029
A recent market study published by FMI “Mucopolysaccharidosis Treatment Market: Global Industry Analysis 2014–2018 and Opportunity Assessment 2019–2029” comprises a comprehensive assessment of the key market dynamics. After conducting thorough research on the historic as well as current growth parameters of the mucopolysaccharidosis treatment market, the growth prospects of the market are obtained with maximum precision.
Mucopolysaccharidosis Treatment Market Taxonomy
The global mucopolysaccharidosis treatment market is segmented in detail to cover every aspect of the market and present a complete market intelligence approach in front of the reader.
By Treatment Type
- Enzyme Replacement Therapies
- Stem Cell Therapies
- Bone Marrow Transplantation
- Umbilical Cord Blood Transplantation
By Type of MPS
- MPS I
- MPS II
- MPS IV A
- MPS VI
- MPS VII
By End User
- Specialty Clinics
- Medical Research Centers
- North America
- Latin America
- South Asia
- East Asia
- Middle East & Africa
The mucopolysaccharidosis treatment market report commences with an executive summary of the key findings and key statistics of the mucopolysaccharidosis treatment market. It also includes the market value (US$ million) estimates of the leading segments of the mucopolysaccharidosis treatment market.
Readers can find detailed market taxonomy which highlights the inclusions and exclusions for the subject. The definition of mucopolysaccharidosis treatment market is included in this chapter, which helps in understanding the basic information about the concerned mucopolysaccharidosis treatment market, which helps the reader understand the scope of the mucopolysaccharidosis treatment market report.
This section explains about the key trends followed by the manufacturer and consumer in mucopolysaccharidosis treatment market. This section helps reader to understand the both supply-side and demand-side trend impacting the growth of mucopolysaccharidosis treatment market.
This chapter highlights the key success factors of the mucopolysaccharidosis treatment market, which include regulatory scenario, pipeline analysis, snapshot of developments for mucopolysaccharidosis Type 3, rare disease framework and designed designation for each treatment present.
This section explain the global market analysis and forecast for the mucopolysaccharidosis treatment market. It also highlights the incremental opportunity for the mucopolysaccharidosis treatment market along with the absolute dollar opportunity for every year between the forecast period of 2019-2029.
This chapter explains the key macro-economic factors that are expected to influence the growth of the mucopolysaccharidosis treatment market over the forecast period. Along with macroeconomic factors, this section also highlights the opportunity analysis for the mucopolysaccharidosis treatment market. This chapter also highlights the key dynamics of the mucopolysaccharidosis treatment market, which include the drivers and restraints.
Based on the Treatment Type, the mucopolysaccharidosis treatment market is segmented into Enzyme Replacement Therapies and Stem Cell Therapies. Stem Cell Therapies is further segmented into Bone Marrow Transplantation and Umbilical Cord Blood Transplantation. In this chapter, readers can find a detailed analysis of the mucopolysaccharidosis treatment market by different Treatment Type and their expected growth over the forecast period.
Based on the Type of MPS, the mucopolysaccharidosis treatment market is segmented into MPS I, MPS II, MPS IV A, MPS VI and MPS VII. This section helps readers understand the prevalence of different Type of MPS in the mucopolysaccharidosis treatment market over the forecast period.
Based on end user, the mucopolysaccharidosis treatment market is segmented into Hospital, Specialty Clinics, Medical Research Centers and Home-infusion. In this chapter, readers can also understand the market attractive analysis based on the end user.
This chapter explains how the mucopolysaccharidosis treatment market will grow across various geographic regions such as North America, Latin America, Europe, East Asia, South Asia, Oceania and the Middle East & Africa (MEA).
This chapter includes a detailed analysis of the growth of the North America mucopolysaccharidosis treatment market along with a country-wise assessment, which includes the U.S. and Canada. Readers can also find the key takeaways of this region, and market growth based on treatment type, type of MPS, end user and country of mucopolysaccharidosis treatment in the North America region.
This chapter contains a snapshot of the Latin America mucopolysaccharidosis treatment market. It includes the growth prospects of the mucopolysaccharidosis treatment market in the leading LATAM countries such as Brazil, Mexico, Argentina and the rest of the Latin America region.
The important growth prospects of the mucopolysaccharidosis treatment market based on treatment type, type of MPS and end user in several European countries, such as the U.K., Germany, France, Italy, Spain, Russia and the rest of Western Europe, is included in this chapter.
This chapter highlights the growth of the mucopolysaccharidosis treatment market in Eastern Europe by focusing on China, Japan and South Korea. This section also helps readers understand the key factors that are responsible for the growth of the mucopolysaccharidosis treatment market in East Asia.
India, Indonesia, Thailand , Malaysia are the leading countries in the South Asia region that are the prime subjects of assessment to obtain the growth prospects of the South Asia mucopolysaccharidosis treatment market in this chapter. Readers can find detailed information about the growth parameters of the South Asia mucopolysaccharidosis treatment market during the period 2019-2029.
Readers can find important factors that can significantly impact the growth of the mucopolysaccharidosis treatment market in Australia and New Zealand during the forecast period based on the market segmentation.
This chapter provides information about the growth of the mucopolysaccharidosis treatment market in the major countries of the MEA region, such as GCC Countries and South Africa, during the period 2019-2029.
This section explains the tier structure for global mucopolysaccharidosis treatment market which helps reader to understand the percent share of market cover by tier 1, tier 2, and tier 3 players in the mucopolysaccharidosis treatment market. This section also explains the company share analysis for mucopolysaccharidosis treatment market which helps readers to understand the market share taken by key players available in the market.
In this chapter, readers can find a comprehensive list of all the leading manufacturers in the mucopolysaccharidosis treatment market, along with detailed information about each company, which includes the company overview, revenue shares, strategic overview, and recent company developments. Some of the players featured in the mucopolysaccharidosis treatment market report are Sanofi S.A., Shire, BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc., ArmaGen, Eloxx Pharmaceuticals, Esteve, Immusoft Corporation, Inventiva.
This chapter includes a list of acronyms and assumptions that provide a base to the information and statistics included in the report.
This chapter helps readers understand the research methodology followed to obtain various conclusions and important qualitative information & quantitative information about the mucopolysaccharidosis treatment market
How the research was conducted?
- C - Level Executives
- Marketing Directors
- Sales Heads
- Portfolio Managers
- Purchasing Managers
- Medical Officers
- Device Development Heads
- Strategy Advisors
- R&D Managers
- Product Manufacturers
- Component Suppliers / OEMs
- Hospital Purchasing Departments
- Physicians / Nursing Managers
- Industry Experts
- Current Market Dynamics and Challenges
- Market Characteristics
- Market Performance and Growth Quadrants
- Competition Structure and Market Structure
- Strategic Growth Initiatives
- Product Adoption, Installed Base, Replacement Cycles, Refurbishment
- Regulatory Landscape
- Products in Pipeline and Clinical Development
- Near-term and Long-term Market Growth Prospects
- Market Segment Splits and Authenticity
- Opinions on Market Projections and Validity of Assumptions
- Industry Magazines
- Company Press Releases
- Annual Reports and Investor Presentations
- Peer-reviewed Journals
- Government Websites and Publications
- Trade Websites
- Clinical Trial Registries
- Country-specific Regulatory Authorities
- World Bank, WHO, IMF, OECD
Primary Interview Splits
Mucopolysaccharidosis Treatment Market Reports - Table of Contents
20. Assumptions and Acronyms Used
21. Research Methodology