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Mucopolysaccharidosis (MPS) Treatment Market

Mucopolysaccharidosis (MPS) Treatment Market

Mucopolysaccharidosis (MPS) Treatment Market

Mucopolysaccharidosis Treatment Market by Treatment Type (Enzyme Replacement Therapies and Stem Cell Therapies), Type of MPS (MPS I, MPS II, MPS IV A, MPS VI and MPS VII) & Region - Forecast 2022-2029

Mucopolysaccharidosis Treatment Market Outlook – 2022-2029

[220 Pages Report] The global Mucopolysaccharidosis Treatment Market is estimated to be worth around US$ 2.38 Bn in 2022. With increasing prevalence of mucopolysaccharidosis (MSP) worldwide, the overall market is projected to grow at a robust CAGR of 5.9% between 2022 and 2029, reaching a valuation of US$ 3.37 Bn by 2029.



Mucopolysaccharidosis treatment Market Estimated Size in 2022

US$ 2.38 Bn

Mucopolysaccharidosis treatment Market Projected Size in 2029

US$ 3.37 Bn

Mucopolysaccharidosis treatment Market Historical CAGR (2014-2021)

5.2 %

Mucopolysaccharidosis treatment Market Value-Based CAGR (2022-2029)


MSP II segment will continue to dominate the mucopolysaccharidosis market, accounting for a share of 33.2% in 2022. Rising prevalence of MSP disorders along with growing adoption of mucopolysaccharidosis treatment for MSP II is boosting the market.

2014-2021 Mucopolysaccharidosis Treatment Market Outlook Compared to 2022-2029 Forecast

The global mucopolysaccharidosis treatment market is set to register a healthy CAGR of 5.9% between 2022 and 2029 in comparison to 5.2% CAGR registered from 2014 to 2021.

Rising health awareness, increasing prevalence of various forms of mucopolysaccharidosis worldwide, continuous advances in medical research and new drug launches and approvals are some of the factors driving the mucopolysaccharidosis treatment market.

Mucopolysaccharidosis is a group of inherited disorders in which the body is unable to breakdown glycosaminoglycan (long chains of sugars) due to absence or malfunctioning of certain enzymes. Presence of these disorders badly affects the physical and mental development of a person as well as damages the organs.

Although, there is no permanent cure for these disorders, mucopolysaccharidosis treatments such as enzyme replacement therapies and stem cell therapies can help to ease the symptoms.

Increasing prevalence of various type of mucopolysaccharidosis is a major force driving the global mucopolysaccharidosis treatment market. According to the National Organization for Rare Disorders (NORD), the total estimated prevalence of all forms of mucopolysaccharidosis is 1 in 25000 births.

Proper diagnosis of mucopolysaccharidosis is playing a vital role in expanding the growth of mucopolysaccharides treatment market. People with early diagnosis often opt for immediate treatment to relieve the symptoms and lead a normal life.

Key players operating in the market are continuously investing in research and development activities for new developing new therapeutics. Advances in stem cell therapies has provided a strong thrust to the mucopolysaccharidosis treatment market and the trend is likely to continue in the future.

Penetration of novel treatment approaches along with growing health awareness is anticipated to support the growth of mucopolysaccharidosis treatment market during the forecast period.

Spurred by aforementioned factors, the mucopolysaccharidosis treatment market is set to expand 1.4X through 2029.

How Will Focus on Clinical Trials Affect Mucopolysaccharidosis Treatment Market?

Clinical trials has remained the key focus of mucopolysaccharidosis treatment during the last few years. Amid rise in the cases of rare diseases, academic researchers, clinicians and leading market players are increasingly focusing on developing new treatment options that can significantly relieve the symptoms and help the patients to lead a normal life.

Increasing clinical research is in turn creating lucrative growth prospects within mucopolysaccharidosis treatment market and the trend is likely to continue in future.

Various regulatory bodies are approving the clinical trials of innovative treatments. For instance, in February 2020 – FDA granted fast track designation to Lysogene's LYS-SAF302 program for the treatment of MPS IIIA. Currently the new second generation gene therapy (LYS-SAF302) is being investigated in the international phase 2/3 clinical trial.

Similarly in Feb 2021, JCR Pharmaceuticals Co., Ltd. received clearance of its Investigational New Drug (IND) application from the US Food and Drug Administration (FDA) to initiate a phase 3 clinical trial of JR-141 for the treatment of MPS II (Hunter syndrome) in the US.

Consistent clinical trials conducted to encourage the discovery of novel therapeutics is anticipated to expand the growth of mucopolysaccharidosis treatment market during the forecast period.

What will be the Key Restraints to Mucopolysaccharidosis Treatment Market Growth?

One of the major factors restraining the growth of mucopolysaccharidosis treatment market is the high cost associated with mucopolysaccharidosis treatment. Both ERT and stem cell therapies treatment are expensive. As a result, a large section population are not able to afford advanced treatments.

Moreover, lack of favorable reimbursement policies across and poor health awareness are limiting the growth of the market.

Country-wise Insights

What is driving the U.S. Mucopolysaccharidosis Treatment Market?

“Drug Development and Approvals to Spur the U.S. Market Growth”

As per FMI, the U.S. will continue to dominate the North America mucopolysaccharidosis treatment market during the forecast period. The U.S. mucopolysaccharides treatment market is driven by rising awareness, new drug approvals, presence of favorable reimbursement policies, well established healthcare infrastructure and presence of leading market players.

The U.S. is one of the most advanced nations in the world. It is home to some of the leading pharmaceutical companies. Advancements in medical research has enabled the market players to develop innovative products with effective features. New treatments are being continuously introduced to counter the burden of rare diseases. This is, in turn triggering the growth of mucopolysaccharides treatment market.

Presence of regulatory bodies such as the U.S Food and Drug Administration (FDA) is playing a key role in expanding the U.S. mucopolysaccharidosis treatment market. Various steps are being taken to foster the drug development for rare diseases. For instance, in February 2020, FDA issued a draft guidance regarding the rare disease, Sanofilippo syndrome. The draft is aimed to support the development of therapies for MPS III (Sanfilippo syndrome)

Moreover, the rise in the cases of mucopolysaccharidosis coupled with increasing diagnostic screening is positively impacting the growth of the U.S. mucopolysaccharidosis treatment market. According to an article published in the Orphanet Journal of Rare Diseases, prevalence of MPS in the U.S. is around 2.67 per 1 million.

Amid intensifying clinical trials, a healthy share has been projected for the U.S. mucopolysaccharides treatment market by 2029.

Why is Japan Emerging as One of the Global Leaders in Mucopolysaccharidosis Treatment Market?

“Focus on Clinical Trials Presenting Attractive Growth Prospects in Japan”

Japan boasts the presence of advanced healthcare system. The government in Japan spends increasingly on developing healthcare infrastructure to accommodate the nation’s healthcare needs in the most efficient manner possible.

Over the last few decades, there has been an increase in the cases of mucopolysaccharidosis disorders. This has triggered the growth of mucopolysaccharidosis treatment market in the country.

Various novel treatments such as hunterase are being introduced in Japan to help the patients suffering from genetic MPS disorders. Moreover, various clinical trials are being conducted to find new and effective treatments for various type of mucopolysaccharidosis disorders.

For instance, in 2021, JCR Pharmaceuticals initiated a phase 3 clinical trial of JR-141 for the treatment of hunter syndrome.

What are the Growth Projections for the U.K. Market?

“Rising Investments in Medical Research to Present Attractive Prospects for Growth in the U.K.”

As per FMI, the U.K. mucopolysaccharidosis treatment market is poised to exhibit strong growth during the forecast period. Increasing government support, rising prevalence of rare disorders and increasing investments in medical research are some of the factors driving the U.K. mucopolysaccharidosis treatment market.

The U.K. government spends millions of dollars on medical research annually. According to the National Institute for Health Research (NIHR), around € 4.5 billion was invested in the U.K. health research projects in 2018. This is positively impacting the market growth across the country.

Moreover, growing popularity of stem cell therapies for the effective treatment of various type of mucopolysaccharidosis will continue to accelerate the growth of the market during the forecast period from 2022 and 2029.

Category-wise Insights

Which is the Most Preferred Treatment for Mucopolysaccharidosis?

“Enzyme Replacement Therapy (ERT) Remains the Gold Standard Treatment for Mucopolysaccharidosis”

Based on treatment, mucopolysaccharidosis treatment market is segmented into enzyme replacement therapies (ERT) and stem cell therapies. Among these two, enzyme replacement therapies segment dominates the mucopolysaccharidosis treatment market.

ERT remains the primary treatment option for mucopolysaccharidosis across the globe. Easy availability and continuous approvals remain the key factors fueling the adoption of enzymes replacement therapies for relieving the symptoms of mucopolysaccharidosis.

Enzyme replacement therapies replace or generate the deficient enzyme and thus effectively helps to treat the condition. Drugs such as aldurazyme, naglazyme, vimizim, elaprase, mepsevii and hunterase are being consumed on large scales for the treatment of mucopolysaccharides.

Which Type of MSP Dominates the Mucopolysaccharidosis Treatment Market?

“MPS II Segment to Continue Accounting for Maximum Sales”

In terms of MPS type, the mucopolysaccharidosis treatment has been segmented into MPS I, MPS II, MPS IV A, MPS VI and MPS VII. Among these, MPS II (Hunter syndrome) segment dominates the global mucopolysaccharidosis treatment market, accounting for the largest share of 33.2% in 2022.

MPS II or hunter syndrome is a rare inherited genetic disorder caused by absence or malfunctioning of enzyme 2-sulfatas. Absence of this enzyme causes permanent progressive damage affecting mental development, appearance and organ functions. As a result, medical products such as hunterase and elaprase are being consumed to relieve the symptoms.

Increasing prevalence of MPS II coupled with presence of multiple treatment options is triggering the growth of MPS II segment and the trend is likely to continue in the future.

Who is the Leading End User of Mucopolysaccharidosis Treatment?

“Favorable Scenario Enables Dominance of Hospitals Segment”

In terms of end user, the global donor egg IVF market is segmented into hospital, specialty clinics, medical research centers and home-infusion.

Among these, the hospitals segment accounts for major revenue share in the global mucopolysaccharides treatment market in 2022. Presence of trained medical professionals and low-cost treatment are some of the factors prompting patients to opt for mucopolysaccharidosis treatment in hospitals.

Hospitals are expected to remain the only means of accessing the treatment for maximum number of people living in developing and underdeveloped regions. However, specialty clinics and home infusion segments are anticipated to grow at a robust CAGR over the forecast period.

Competitive Landscape

Leading players operating in mucopolysaccharidosis treatment market are vigorously investing in research and development activities. They are adopted various organic and inorganic growth strategies such as mergers, new product launches and approvals, partnerships and acquisitions to gain a competitive edge in the market.

  • In March 2021, JCR Pharmaceuticals Co., Ltd. announced that the approval of IZCARGO (pabinafusp alfa 10 mL, intravenous drip infusion) by Ministry of Health, Labour and Welfare (MHLW) for the treatment of mucopolysaccharidosis type II (MPS II, or Hunter syndrome).
  • In September 2021, Takeda collaborated with JCR Pharmaceutical to commercialize the next generation hunter syndrome therapy (JR-141) outside the U.S.
  • In July 2020, Lysogene collaborated with the Weizmann Institute of Science with the aim of developing a novel AAV gene therapy approach for various genetic disorders.
  • In September 2020, REGENXBIO Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, expanded its RGX-121 program for the treatment of Mucopolysaccharidosis Type II.
  • In November 2017, the U.S. Food and Drug Administration (FDA) approved Mepsevii for the treatment of Sly syndrome (MPS VII) in pediatric and adult persons.

Scope of the Report



Historical Data Available for


Forecast Period


Market Analysis

Units for Volume and US$ Mn for Value

Key Regions Covered

North America, Latin America, Europe, South Asia, East Asia, Oceania, and the Middle East & Africa

Key Countries Covered

US, Canada, Mexico, Brazil, Germany, U.K., France, Italy, Spain, Russia, Poland, China, Japan, South Korea, India, ASEAN, Turkey and South Africa

Key Segments Covered

Cycle Type, End Use and Region

Key Companies Profiled

  • BioMarin
  • Takeda Pharmaceutical Company Limited
  • Sanofi S.A.
  • Ultragenyx Pharmaceutical Inc.
  • Sangamo Therapeutics, Inc.
  • Sarepta Therapeutics
  • Abeona Therapeutics, Inc.

Report Coverage

Market Forecast, Company Share Analysis, Competition Intelligence, DROT Analysis, Market Dynamics and Challenges, and Strategic Growth Initiatives

Customization & Pricing

Available upon Request

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Mucopolysaccharidosis Treatment Market by Category


  • Enzyme Replacement Therapies
  • Stem Cell Therapies
    • Bone Marrow Transplantation
    • Umbilical Cord Blood Transplantation

Type of MPS

  • MPS I
  • MPS II
  • MPS IV A
  • MPS VI

End User

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

Key Questions Answered in the Report

What is the current mucopolysaccharidosis treatment market valuation?

The mucopolysaccharidosis treatment market is estimated to be worth around US$ 2.34 Bn in 2022.
As per FMI, the mucopolysaccharidosis treatment market is forecast to register a steady CAGR of 5.9% between 2022 and 2029
The mucopolysaccharidosis treatment market grew at a CAGR of 5.2% between 2014 and 2021.
Continuous drug approvals, increasing government initiatives to tackle the burden of rare diseases and rising penetration of novel treatments across developing economies are some of the key trends shaping the growth of mucopolysaccharidosis treatment market.
BioMarin, Takeda Pharmaceutical Company Limited, Sanofi S.A., and Ultragenyx Pharmaceutical Inc. BioMarin, Sangamo Therapeutics, Inc., REGENXBIO Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc. are some of the leading players operating in the mucopolysaccharidosis treatment market.

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