Mucopolysaccharidosis (MPS) Treatment Market

Mucopolysaccharidosis (MPS) Treatment Market: Stem Cell Therapy Research to Prominently Uphold Growth Outlook: Global Industry Analysis 2014-2018 and Opportunity Assessment 2019-2029


  • 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.


  • 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

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.

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.

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.


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

By End User

  • Hospital
  • Specialty Clinics
  • Medical Research Centers
  • Home-infusion


  • North America
  • Latin America
  • Europe
  • South Asia
  • East Asia
  • Oceania
  • Middle East & Africa

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Mucopolysaccharidosis (MPS) Treatment Market