The UK Sanfilippo Syndrome Therapeutics market size stood at around USD xx billion in 2020 and is projected to reach USD xx billion by 2028, exhibiting a CAGR of xx% during the forecast period.
Sanfilippo Syndrome (MPS III) is an ultra-rare autosomal recessive inherited disorder and a member of a group of hereditary metabolic diseases known as the mucopolysaccharidoses (MPS) which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). MPS III is a type of childhood dementia that causes fatal brain damage. Sanfilippo syndrome is caused by a lack of an enzyme that normally breaks down and recycles a large, complex sugar molecule called ‘heparan sulfate’. This heparan sulfate accumulates and causes damage to the cells of the central nervous system, including the brain.
Children with MPS III usually appear healthy at birth, but the developmental delay is usually evident by age 2-5 years. Mental and motor development peak by 3-6 years of age, after which intellectual decline usually occurs. Behavioral problems such as hyperactivity and irritability may become obvious earlier. The severe behavioral disturbance is a very common feature of Sanfilippo syndrome and one of the more difficult aspects of the disorder to manage.
Sanfilippo syndrome causes progressive intellectual disability and the loss of previously acquired skills (developmental regression). In later stages of the disorder, children with Sanfilippo Syndrome may develop seizures and movement disorders. The typical life expectancy for affected children is between 10-20 years old. MPS III affects males and females equally. It is estimated that nearly 6% of the UK population (around 3.5 million people) will be affected by a rare disease at some point in their lives. There are four subtypes of MPS III: types A, B, C, and D. Each type is caused by a mutation in a different gene. MPS IIIA is the most common subtype affecting around 1 in 100,000 births, closely followed by type B at 1 in 200,00.
The growing necessity for advanced therapeutics will impel the market players to launch novel therapies, which in turn, will boost the Sanfilippo syndrome therapeutics market growth in the forthcoming years. Moreover, the increasing acquisitions and deals between key players will also bolster the healthy growth of the market.
Additionally, the growing unmet clinical needs of patients along with better treatment outcomes, financial incentives for orphan drug development to recover R&D costs, success for the drugs in pipeline with faster market access, high burden of rare diseases, favorable regulations for orphan products development will spur demand for Sanfilippo syndrome therapeutics market during the forecast period.
While huge treatment costs and lack of awareness are more likely to hamper the Sanfilippo syndrome therapeutics market growth.