The Global Sanfilippo syndrome drugs market size stood at USD 0.7 billion in 2019 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during
the forecast period.
Sanfilippo Syndrome, also known as Mucopolysaccharidosis type III (MPSIII), is a rare genetic condition that causes fatal brain damage. It is referred to as a childhood disease because most patients fail to reach adulthood. Sanfilippo is a metabolic disorder. One in 70,000 children is born with this inherited condition, which is caused by an enzyme deficiency.
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.