Overview

Mucopolysaccharidosis Market is estimated to value over USD 4 billion by 2027 end with a CAGR of over 10.2% during the forecast period 2020 to 2027.

Mucopolysaccharidosis, or MPS, are a group of rare metabolic disorders that are caused by the absence of lysosomal or malfunctioning enzymes known as glycosaminoglycan. This disorder creates an inability of breaking down sugars in the body, resulting in the over accumulation of compounds like heparan sulphate, keratan sulphate and dermatan sulphate that interferes the normal functioning of body cells. Severe neurological symptoms like Sanfilippo syndrome and hunter syndrome are some of the primary symptoms of mucopolysaccharidosis and key players are involved in the research and development of advanced therapeutics for managing these symptoms.
Rising number of candidates from such key players in rare disease therapeutics sector shall boost the mucopolysaccharidosis market growth. This is mainly because it is easy to develop major drugs for rare diseases in comparison with other pharma portfolios and it is also mandatory for pharmaceutical companies to conduct studies with large scale results for traditional therapies and medications as compared to the therapies required for rare diseases. Furthermore, the rare diseases like MPS have a limited number of therapies like the enzyme replacement therapy for treating MPS II or hunter syndrome and this monopoly shall propel the proliferation of the market. However, some of the therapies like Vimizim, which is used for treating MPS IV A, costs over USD 380,000 which is expensive for patients from developing nations like China. Additionally, some of these therapies are unable to identify the neurological symptoms and other complications present in MPS patients. For instance, therapeutics like Aldurazyme and Elaprase are unable to cross the brain-blood barrier which is essential for managing the disorder effectively. Such factors shall encourage companies for conducting extensive researches in developing advanced therapeutic measures that can identify these neurological symptoms in earlier stages and manage the disorder.
There has been a substantial rise in the number of patients diagnosed with MPS. As per the data provided by the National Organization for Rare Disorders, the occurrence of all types of MPS is predicted to be one in over 24,000 births. It was reported in April 2019 that over 140 individuals in Japan were diagnosed with MPS type II (Hunter Syndrome). Such a rise shall bolster the augmentation of the market.
However, the high prevalence of MPS in countries where patients are unable to afford the therapeutic treatments shall restrain the growth of the market. A substantial proportion of MPS patients could not undergo treatment owing to their financial conditions and to add to that, people in developing countries are unaware about treatment alternatives for rare disease like MPS and the healthcare insurance policies do not cover such rare diseases in their reimbursements which further limits the number of patients opting for these therapies. As per a study conducted by the Orphanet Journal of Rare Diseases, in case of MPS type I and MPS type III, there has been a massive failure in reducing the diagnostic gap in these diseases and reasons like these shall substantially impede the market growth.

Mucopolysaccharidosis Market

Market Segmentation:

By Treatment
•   Enzyme Replacement Therapy
•   Others
By Disease Type
•   Mucopolysaccharidosis Type I
•   Mucopolysaccharidosis Type II
•   Mucopolysaccharidosis Type IV A
•   Mucopolysaccharidosis Type VI
•   Others
By Route of Administration
•   Intravenous
•   Intracerebroventricular
By End User
•   Hospitals
•   Specialty Clinics
•   Others
By Region
•   North America
•   Europe
•   Asia-Pacific
•   Latin America
•   Middle East and Africa

The market is classified into treatment, disease type, route of administration, end user and region. 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. ERTs are the only approves therapeutics for MPS like for instance, MPS type II treatment has two therapeutics options, Hunterase and Elaprase, both of which are enzyme replacement therapies. Such a monopoly shall let this segment dominate the market during the forecast period. Other treatments such as hematopoietic stem cell transplants are under clinical trials which once approved, shall increase its demand amongst patients, thereby boosting its growth during the forecast period.
The disease type segment is categorised into mucopolysaccharidosis type I, mucopolysaccharidosis type II, mucopolysaccharidosis IV A, mucopolysaccharidosis VI and others. The mucopolysaccharidosis type II or Hunter syndrome, is predicted to lead the market growth during the forecast period owing to the rising number of MPS type II patients across the world along with the availability of Elaprase and Hunterase. The route of administration segment is fragmented into intravenous and intracerebroventricular. The intravenous segment is predicted to hold the mucopolysaccharidosis market share as MPS therapies like Elaprase are administered with the help of intravenous injections. The end user segment is bifurcated into specialty clinics, hospitals and others. The hospitals segment is poised to dominate the market, and this can be credited to the availability of trained healthcare professionals who can administer these therapies in hospital settings.

Regional Analysis:

The North America region is expected to dominate the market owing to the increased rate of treatment of rare diseases and the higher rate of rare disease diagnosis in the region. Furthermore, there has been significant improvements in the reimbursement policies for these therapies couple with a rising awareness amongst patients regarding the treatment of MPS. Additionally, a strong presence of key market players in the region who are constantly looking for alternate cost-effective therapies which shall bolster the market domination.

Competitive Landscape:

Some of the key players are Shire (Takeda Pharmaceutical Co. Ltd.), Denali Therapeutics, ArmaGen, REGENXBIO Inc., Sangamo Therapeutics, BioMarin, Lysogene, Abeona Therapeutics Inc., Ultragenyx Pharmaceutical, Genzyme Corp.

FutureWise Key Takeaways:

• Growth prospects
• SWOT analysis
• Key market trends
• Key data-points affecting market growth

Objectives of the Study:

• To provide with an exhaustive analysis on the global mucopolysaccharidosis market by treatment, by disease type, by route of administration, by end user and by region
• To cater comprehensive information on factors impacting market growth (drivers, restraints, opportunities, and industry-specific restraints)
• To evaluate and forecast micro-markets and the overall market
• To predict the market size, in key regions (along with countries)— North America, Europe, Asia Pacific, Latin America and Middle East and Africa
• To record, evaluate and analyze competitive landscape mapping- product launches, technological advancements, mergers and expansions

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