The UK Mucopolysaccharidosis VI 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.
Mucopolysaccharidosis VI (MPS VI), also known as Maroteaux-Lamy syndrome is a member of a group of hereditary metabolic diseases known as the mucopolysaccharidoses which, in turn, are part of a larger group of diseases known as lysosomal storage disorders (LSDs). MPS VI is a rare genetic disorder characterized by a complete or partial lack of activity of the enzyme arylsulfatase B (also called N-acetylgalactosamine-4-sulfatase), encoded by the ARSB gene. MPS VI occurs due to mutations in the ARSB gene and is inherited as an autosomal recessive disorder. The deficiency or absence of this enzyme activity leads to the accumulation of complex carbohydrates called glycosaminoglycans (previously known as mucopolysaccharides) in the body. Abnormal accumulation of mucopolysaccharides leads to progressive involvement of multiple organ systems.
The symptoms and severity of Maroteaux-Lamy syndrome can vary dramatically from one person to another; some individuals only develop mild symptoms, while others develop severe, even life-threatening complications. Common symptoms can include coarse facial features, corneal clouding, joint abnormalities, various skeletal malformations, an abnormally enlarged liver and/or spleen (hepatosplenomegaly), and hearing loss. 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, and the MPS VI had the highest birth prevalence of 7.85 per 100,000 live births, comprising 46% of all MPS cases diagnosed.
Initial diagnosis is usually made between 6 and 24 months of age, and life expectancy depends on the severity of symptoms and is around 20 to 30 years. People with MPS VI often begin to show signs and symptoms of MPS VI during early childhood, these include changes to the physical appearance as well as developing other clinical features in the cardiovascular and respiratory systems. Importantly, MPS VI does not affect intelligence.
Market Growth Drivers
The growth in the number of products in the preliminary and advanced stages of R&D along with the focus on the treatment of Maroteaux-Lamy syndrome is anticipated to drive the growth in the MPS VI treatment market at a considerably fast rate. With the approval of BioMarin’s Naglazyme (galsulfase), the market is expected to be boosted several products currently in the pipeline of various companies can be anticipated to undergo regulatory approvals for the marketing, if they have favorable clinical trials results.
The increasing incidence and prevalence also boost the MPS VI treatment market growth during the forecast period. Market players are more focused on the adoption of inorganic growth strategies such as collaborations and partnerships to strengthen their position in the MPS VI treatment market. The adoption of inorganic growth strategies by key players is expected to drive the market growth over the forecast period.
However, rising treatment costs and a lack of awareness among patients and physicians about MPS VI are among the major factors acting as restraints and will continue to pose a challenge to the MPS VI treatment market in the forecast period.