According to FutureWise analysis, the market for Mucopolysaccharidosis is to reach USD 4 billion by 2027, and is projected to expand at a CAGR of 10.2% from 2023-2031.
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.