The mucopolysaccharidosis (MPS) is an inherited lysosomal storage disorders. It refers to the deficiency of the enzyme that is involved in the degradation of acid mucopolysaccharides. The enzyme is known as glycosaminoglycans (GAGs). Mucopolysaccharidosis affects many parts of the body and is caused due to the mutation of the IDUA gene. Due to the mutation of the IDUA gene, larger sugar molecules known as glycosaminoglycans, accumulate in the cells known as lysosomes. This results in the increase in the size of the lysosomes, causing enlargement of the different tissues and organs of the body. The mucopolysaccharidosis (MPS) is classified as Hurler syndrome (MPS IH), Hurler-Scheie syndrome (MPS I-H/S), Sanfilippo syndrome (MPS III), Scheie syndrome (MPS IS), Hunter syndrome (MPS II), Maroteaux-Lamy syndrome (MPS VI), Morquio syndrome (MP IV), and Sly syndrome (MPS VII). Though there is no cure to Mucopolysaccharidosis I (MPS I), the treatment option includes bone marrow transplant, enzyme replacement therapy, that helps to manage the symptoms to some extent.
The symptoms of the mucopolysaccharidosis I (MPS I) are not present at the time of birth, but apprers during childhood. The most common signs and symptoms includes, Enlarged vocal cords, resulting in a deep voice with enlarged head, lips, tongue and nose, sleep apnea, upper respiratory infection, hearing loss, Carpal tunnel syndrome, joint deformities and others. The symptoms vary from person to person. The management of mucopolysaccharidosis I (MPS I) includes primary care with pulmonology, gastroenterology, cardiology, dental, physical therapy specialist .The Aldurazyme is a drug used as an enzyme replacement therapy for MPS.
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Advancement in the treatment options includes intense research and development with government funding. The Orphan Disease Center is offering MPS I Pilot Grant Programs for improving the therapies for people suffering from this syndrome. The Lysosomal Disease Network includes doctors, nurses, researchers and others who are working to improve the lives of people suffering from this disorder. Increasing focus of major biopharmaceutical companies on research and development of drugs for treatment of rare diseases is expected to boost demand for mucopolysaccharidosis treatment drugs. Moreover, the number of treatment options currently in pipeline is expected to increase revenue growth during the forecast period.
Mucopolysaccharidosis treatment Market: Drivers and Restrains
Rise in number of people suffering from Lysosomal diseases, financial incentives for orphan drug development to recover R&D costs, including market exclusivity, tax waiver, fee reductions and, grants, success for the drugs in pipeline with faster market access, high burden of rare diseases, favorable regulations for orphan products development are some of the factors favoring market growth over the forecast period. The factors limiting the market growth is high treatment cost, heterogeneity of the disease, lack of treatment options, lack of awareness and others are some of the factors restraining the growth of the market . growth opportunities such as increase in R&D for orphan drugs driven by consolidation of Pharma-biotech firms in mucopolysaccharidosis treatment, initiatives to improve diagnosis, research and development in therapies targeting neuropathic lysosomal storage disorder by crossing blood brain barrier (BBB) is boosting the growth of the target market
Mucopolysaccharidosis treatment Market: Segmentation
The global Mucopolysaccharidosis treatment market has been classified on the basis of treatment type, end user and geography.
Based on Product Type, the global Mucopolysaccharidosis treatment market is divided into following:
- Enzyme Replacement Therapy
- Stem Cell Therapy
Based on the End User, the global Mucopolysaccharidosis treatment market is divided into following:
- Specialty Care Unit
Mucopolysaccharidosis treatment Market: Overview
The Mucopolysaccharidosis treatment market is segment based on treatment type, end-user and geography. Based on the product type, Mucopolysaccharidosis treatment market is segmented on the basis of enzyme replacement therapy, stem cell therapy and others. Reimbursement varies from state to state for Medicaid program and there are some restrictions for Medicaid coverage for rare disease drugs. For instance, In state of Georgis, clinically superior cystinosis agent is not reimbursed. In 2016, State of Oregon expanded the class of covered therapies to include drugs for Gaucher’s disease, MPS I, MPS II, MPSIV and MPS VI. On the basis on end user, the Mucopolysaccharidosis treatment market is segmented on the basis of hospital, clinics, and specialty care units. Increase in the number of patient population, focus of large pharmaceutical companies in research and development will increased the demand for Mucopolysaccharidosis treatment market.
Mucopolysaccharidosis treatment Market: Regional Overview
Region wise, the global Mucopolysaccharidosis treatments market is classified into regions namely, North America, Latin America, Western Europe, Eastern Europe, Asia-Pacific, Japan, Middle East and Africa. North America is estimated to account for major share due to favorable regulations for orphan products development, funding for research and development. Over the last few years, introduction of specific orphan drug regulations has enabled and accelerated research and development of therapies for treatment of several rare diseases. Health plan coverage and reimbursement policies for such costly drugs also influence the investment by pharmaceutical and biotechnology companies to invest in drug development of such costly drugs. Significant economic development has led to an increase in healthcare availability in Asia Pacific region providing an opportunity for biopharmaceutical companies to penetrate in Asia Pacific regions, rising awareness towards lysosomal storage diseases, collaborations to develop new class of medicine for lysosomal storage diseases are some of the factors fueling growth of the market.
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Mucopolysaccharidosis treatment Market: Key Players
Some of the key players in global mucopolysaccharidosis treatment market are BioMarin Pharmaceutical, Inc., Genzyme, a Sanofi Company, ArmaGen, Inc., Sangamo Therapeutics and others.