The global sales of mucopolysaccharidosis treatment is estimated to be worth USD 2,826.6 million in 2025 and anticipated to reach a value of USD 5,014.5 million by 2035. Sales are projected to rise at a CAGR of 5.9% over the forecast period between 2025 and 2035. The revenue generated by mucopolysaccharidosis treatment in 2024 was USD 2,669.1 million. The industry is anticipated to exhibit a Y-o-Y growth of 5.8% in 2024.
Mucopolysaccharidosis (MPS) is a rare genetic disorder where the cells fail to produce specific enzymes, which can break down mucopolysaccharides, therefore causing the buildup of those substances within cells. Due to the gradual damage inflicted on the organs and impairment in the developmental process, treatment is focused on methods such as ERT and HSCT.
Supportive care manages the symptoms in patients with MPS. Growing Advancements in biotechnology, improved diagnostic techniques which aid in early diagnosis of disease, along with increased awareness of rare diseases significantly anticipates the growing adoption of these treatments. Additionally, Availability of ERT and continuous research into gene therapies has increased the quality of treatment outcome and quality of life among patients with MPS which surges its market growth.
Global Mucopolysaccharidosis Treatment Market Outlook
| Attributes | Key Insights |
|---|---|
| Historical Size, 2024 | USD 2,669.1 million |
| Estimated Size, 2025 | USD 2,826.6 million |
| Projected Size, 2035 | USD 5,014.5 million |
| Value-based CAGR (2025 to 2035) | 5.9% |
Mucopolysaccharidosis is a rare genetic disorder arising from the deficiency of particular enzymes that cause an increase in mucopolysaccharides within the body. This increase eventually results in progressive damage to various organs and tissues and triggers symptoms such as skeletal anomalies, and cardiovascular problems.
Enzyme replacement therapy (ERT) has proven to replace the deficient enzyme in MPS patients. Physical therapy, surgeries, among other treatments, is employed to aid the disease from its progression.
Increase in number of cases suffering from the disease and an awareness for the same contributes to rise in demand for the effective treatment of options for these conditions. This increased number of diagnosed patients also encourages health care professionals to find treatments that could manage the condition. Many of the healthcare institutions are also working with pharmaceutical manufacturers to address the unmet needs of mucopolysaccharidosis patients also enhance the market.
Advances in biotechnology have resulted in improved ERT options, which show better efficacy and patient outcomes. Apart from that, huge investment by the manufacturers in the field of R&D are accelerating the innovations such as gene therapy, which can possibly lead to the development of curative treatment further anticipate the growth of the market.
These developments, while upgrading the quality of care, also provide health professionals with better tools for the management of the disease and lead to further growth in the treatment market.
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Comparative analysis of fluctuations in compound annual growth rate (CAGR) for the global mucopolysaccharidosis treatment market between 2024 and 2025 on six months basis is shown below.
By this examination, major variations in the performance of these markets are brought to light, and also trends of revenue generation are captured hence offering stakeholders useful ideas on how to carry on with the market's growth path in any other given year. January through June covers the first part of the year called half1 (H1), while half2 (H2) represents July to December
The table presents the expected CAGR for the global mucopolysaccharidosis treatment market over several semi-annual periods spanning from 2025 to 2035. In the first half (H1) of the decade from 2024 to 2035, the business is predicted to surge at a CAGR of 5.9%, followed by a slightly slower growth rate of 5.5% in the second half (H2) of the same decade.
| Particular | Value CAGR |
|---|---|
| H1 | 6.6% (2024 to 2034) |
| H2 | 6.3% (2024 to 2034) |
| H1 | 5.9% (2025 to 2035) |
| H2 | 5.4% (2025 to 2035) |
Moving into the subsequent period, from H1 2025 to H2 2035, the CAGR is projected to decrease slightly to 5.9% in the first half and decrease moderately at 5.4% in the second half. In the first half (H1) the market witnessed a decrease of 70 BPS while in the second half (H2), the market witnessed a decrease of 90 BPS.
Increase in Investment in Research and Development of Novel Drug Therapies Anticipate the Growth of the Market
Pharmaceutical companies are emphasizing on developing more effective and targeted treatments like advanced enzyme replacement therapies and potential gene therapies that can offer a cure for MPS patients for an extended period. These R&D work is majorly targeted towards improving the treatment outcomes, by reducing disease progression, in patients.
As new therapies come into the market, there will be a wider range of alternatives available to healthcare providers to improve patient access to proper treatments and expand market reach. Moreover, ongoing R&D can lead to the creation of personalized treatments for individual MPS types, thus leading to more accurate management of the disease.
For instance, In February 2024, REGENXBIO Inc., announced phase 3 trials Phase I/II/III CAMPSIITE® trial of RGX-121 for the treatment of patients up to 5 years diagnosed with Mucopolysaccharidosis Type II (MPS II).
This trials demonstrated statistical significant results. This factors certainly fuel the demand for MPS treatments, eventually driving the expansion and growth of the market for MPS treatment.
Emphasis on Making Strategic Acquisitions for Expansion of Drug Candidate Product Pipeline
Strategic acquisitions to develop drug candidate products in pipelines represent an essential driver in the growth of the Mucopolysaccharidosis (MPS) treatment market. With the help of strategic acquisition of small biotech companies or in-license innovative technology, large pharmaceutical firms get an opportunity for the promising new drug candidates with huge unmet medical needs areas like MPS.
These acquisitions enable rapid development of new treatments, including next-generation ERT and gene therapies, which offer improved efficacy and long-term solutions for patients.
With these advanced treatments entering the market, healthcare professionals are able to provide more diverse options for personalized care for patients with MPS. Moreover, strategic acquisitions often accelerate clinical trials and regulatory approvals, which get new therapies to market more quickly.
An enlarged pipeline of available treatments increases competition, can reduce costs and improve accessibility, and finally contribute to immense market growth of treatments for MPS.
For instance, in January 2024, Kyowa Kirin Co., Ltd., a Japanese pharmaceutical company acquired Orchard Therapeutics plc. This acquisition expanded company’s drug candidate’s pipeline.
Focus on Developing Combination Therapies can bring Growth opportunities to the company
New business opportunities that will be opened up to manufacturing companies by focusing on developing combination therapies for MPS treatment include the fact that combination therapies can offer more comprehensive treatment options that will target multiple facets of the disease. For instance, the integration of ERT with gene therapy can help manage diseases more effectively and potentially offer curative solutions that are in great demand in the market.
Combination therapies can also help manufacturers be front-runners in the treatment option advancements. It makes their products unique from the competitors. Such products also often lead to strategic alliances with research institutes or other pharma companies for accelerating faster developmental and market entry.
This provides companies with an opportunity in the increasing demand for custom treatment approaches by enabling them to tailor their healthcare providers to the specific needs of every patient. All this goes a long way to stimulate longer-term revenue growth, fortify market dominance, and increase the number of patients with better MPS treatment outcomes.
High Treatment Costs of Mucopolysaccharidosis hinders its treatment adoption rate and market growth
High treatment costs for Mucopolysaccharidosis (MPS) greatly affect the adoption rates and growth of the market. Most ERT, gene therapies, and stem cell transplants are expensive, hence out of reach for most patients, especially in low- and middle-income countries.
Such therapies have to be administered for life, increasing the burden on the patient and the healthcare system. Such high costs also limit access of patients, and reduce the willingness of providers of healthcare services and insurers to adopt them.
Thus, many patients will not receive early diagnosis and treatment; hence, they suffer from negative health outcomes. This limits the extent of access, limiting the investment of pharmaceutical companies in new treatment therapies. This would therefore keep progressing the costs and continue this financial obstacle, attributing to lower the rates of higher application of MPS treatment drugs across the world.
Principal Consultant
The global sales of mucopolysaccharidosis treatment drugs recorded a CAGR of 5.2% during the historical period between 2020 and 2024. The drug sales for mucopolysaccharidosis treatment was positive as it reached a value of USD 2,669.1 million in 2024 from USD 2,107.4 million in 2020.
Mucopolysaccharidosis is a group of rare, inherited metabolic disorders caused by deficiencies in enzymes. Development of enzyme replacement therapy (ERT) and hematopoietic stem cell transplantation (HSCT), significantly contributes to the growth of market owing to the improvement demonstrated by these therapies in patients.
Growing advancements in biotechnology and genetic research have played a significant role in making these therapies more effective, it significantly aid in slowing down progression of disease.
The latest directions in MPS treatments include novel ERT next-generation formulation, which appears to be more efficient with fewer adverse effects. Gene therapy also seems to gain speed for the treatment as it directly repairs genetic mutations-a permanent correction.
The future industry outlook for MPS treatment market is quite positive, since awareness is being increased and diagnostic tools are being developed continuously, further developed by ongoing therapy development. More targeted and effective solutions would be the resultant of increasing research and development spending, particularly on gene therapies and combination treatments.
Early diagnosis through expanded newborn screening programs have paved ways for timely interventions and continuously contribute to expand the scope of the market.
Tier 1 companies comprise market leaders with a market revenue of above USD 100 million capturing significant market share of 68.6% in global market. These market leaders are characterized by high production capacity and a wide product portfolio.
These market leaders are distinguished by their extensive expertise in providing their services, and a broad geographical reach, underpinned by a robust consumer base. Prominent companies within tier 1 include BioMarin Pharmaceutical, Sanofi S.A., Takeda Pharmaceutical Company Limited, and Sumitomo Pharma Co., Ltd.
Tier 2 companies include mid-size players with revenue of USD 50 to 100 million having presence in specific regions and highly influencing the local market and holds around 26.6% market share. These are characterized by a strong presence overseas and strong market knowledge.
These market players have good technology, ensure regulatory compliance but may not have global reach. Companies having prominent role in tier 2 segment are Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Sangamo Therapeutics, Inc., and JCR Pharmaceuticals.
Finally, Tier 3 companies, act as suppliers to the established market players. They are essential for the market as they specialize in specific products and cater to niche markets, adding diversity to the industry.
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The section below covers the industry analysis for the mucopolysaccharidosis treatment market for different countries. Market demand analysis on key countries in several regions of the globe, including North America, Asia Pacific, Europe, and others, is provided. The United States is anticipated to remain at the forefront in North America, with a maximum value share through 2035.
| Countries | Value CAGR (2025 to 2035) |
|---|---|
| United States | 7.4% |
| Germany | 7.1% |
| Japan | 6.3% |
| UK | 5.4% |
| China | 4.1% |
| India | 3.7% |
| Australia | 5.2% |
The increasing focus of the USA FDA on giving orphan drug designation to candidates for Mucopolysaccharidosis (MPS) contributes much to the growth of the market as it offers them variety of incentives, such as market exclusivity, tax credits, and accelerated approval processes.
This designation also makes candidate more attractive to pharmaceutical companies it provides regulatory support and reduces financial burden on companies.
Therefore, the FDA's focus on speed make the introduction of innovative therapies faster to the market which helps in fulfilling the unmet medical needs of MPS patients. Such factors help expand treatment options, increase market competition, and hasten the availability of life-saving therapies for such rare disease.
The sales of mucopolysaccharidosis treatment therapies in Japan is anticipated to grow at a CAGR of 4.1% throughout the forecast period.
Strong emphasis in the development of innovative therapy, including superior enzyme replacement therapies, advanced gene therapy, and latest generation, increases the chance of providing better treatment facilities to Japanese patients suffering from MPS. Investment in research and development by manufacturers, for the development of rare disease therapies makes Japan a significant market for MPS treatments.
For instance, in September 2022, Ultragenyx Pharmaceutical Inc., commercially launched a new drug for the treatment of patients suffering with Mucopolysaccharidosis Type VII (MPS VII or Sly Syndrome).
Introduction of new drug options for the treatment not only improves the outcome of patients but also increases the competition among market players, encouraging innovation from future prospects. Along with these incentives provided by Japanese government for rare disease therapies, makes Japan one of the prominent country that can ensure better availability of treatment for MPS.
Strategic alliances between research institutions and Hospitals in the UK significantly attributes to the growth of the Mucopolysaccharidosis (MPS) treatment market in the country as these Partnerships accelerate the development of new treatments.
Collaborative efforts overcome financial and technical barriers toward ensuring faster market access of new treatments. Moreover, collaborations with research centers also help in understanding the disease better, which leads to a discovery of more targeted and effective therapies.
This approach therefore fosters innovation, enriches treatment options for the patients, and strengthens the UK MPS treatment market overall against the growing demand for an effective therapy.
For instance, in November 2023, The University of Manchester (UoM) initiated the recruitment activities for a phase 1/2 clinical trial (NCT05665166) of AVR-RD-05 at Royal Manchester Children’s Hospital. This AVR-RD-05, candidate is an investigational autologous hematopoietic stem cell (HSC) gene therapy intended for the treatment of neuronopathic mucopolysaccharidosis type II.
The section contains information about the leading segments in the industry. By treatment, the enzyme replacement therapy segment dominated the market by holding largest share of 56.4% in 2025.
| Treatment | Enzyme Replacement Therapy |
|---|---|
| Value Share (2025) | 56.4% |
The enzyme replacement therapy (ERT) takes the dominant share of the Mucopolysaccharidosis (MPS) treatment market as it has shown to be effective in controlling symptoms and slowing down the progression of the disease.
ERT corrects the deficiency of the underlying enzymes in patients by administering synthetic versions of the missing enzymes. With approved therapies such as Naglazyme, Vimizim, and Aldurazyme, the prognosis has improved significantly, life expectancy is increased, and quality of life is enhanced.
ERT is the current treatment for many types of MPS, with a well-established and clinically valid solution. Furthermore, due to its extensive availability, regulatory clearance, and continued development for next-generation ERT formulations, it remains the preferred agent in MPS management. Gene therapies are just evolving, and ERT is yet to be dethroned because of its experience in the clinical arena and availability.
| End User | Hospital |
|---|---|
| Value Share (2035) | 53.0% |
Hospital segment to contribute for a significant revenue share in the mucopolysaccharidosis treatment by 2035 end.
The hospital segment leads in the Mucopolysaccharidosis (MPS) treatment market since MPS management is complex and demands specific care. It encompasses complete diagnostic services, such as advanced treatment options with enzyme replacement therapy and stem cell transplants, as well as the expertise of a multidisciplinary medical team.
MPS patients need prolonged, intensive care and constant monitoring, and thus, are often treated in hospitals. Hospitals are also well-endowed with infrastructure to handle complex therapies and complications arising from the disease. Centers of excellence in rare genetic disorders also exist, thus increasing the preference for hospital-based treatment for MPS, thereby making them the largest end-user segment in the market.
Substantial investments are seen in the mucopolysaccharidosis treatment by the pharmaceutical industry. Their major focus towards development of potential drug candidates for launch into the market anticipates the growth of the market. In addition, their growing focus towards launch and commercialization of their new products further surge their market growth.
Recent Industry Developments in Mucopolysaccharidosis Treatment Industry
In terms of treatment, the industry is divided into enzyme replacement therapies and stem cell therapies (Bone marrow transplantation (BMT) and Umbilical cord blood transplantation (UCBT))
The industry is classified by type of MPS as MPS I, MPS II, MPS IV A, MPS VI and MPS VII
The industry is classified by Hospital, Specialty Clinics, Medical Research Centers, Home-infusion
Key countries of North America, Latin America, Western Europe, Eastern Europe, East Asia, South Asia and Pacific, and Middle East and Africa (MEA) have been covered in the report.
The global sales of mucopolysaccharidosis treatment is projected to witness CAGR of 5.9% between 2025 and 2035.
The global sales of mucopolysaccharidosis treatment stood at USD 2,669.1 million in 2024.
The global sales of mucopolysaccharidosis treatment is anticipated to reach USD 5,014.5 million by 2035 end.
United States is set to record the highest CAGR of 7.4% in the assessment period.
The key players operating in this sector include Sanofi S.A., Shire, BioMarin Pharmaceutical Inc., Ultragenyx Pharmaceutical Inc., Sarepta Therapeutics, Abeona Therapeutics, Inc., ArmaGen, Eloxx Pharmaceuticals, Esteve, Immusoft Corporation, Inventiva, Orchard Therapeutics Limited, REGENXBIO Inc. and Sangamo Therapeutics, Inc. are the key players in this market.
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Mucopolysaccharidosis Treatment Market
