The UK Morquio 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.
Morquio Syndrome (MPS IV) 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 IV is caused by the deficiency of the lysosomal hydrolase, N-acetylglucosamine-6-sulfate sulfatase (GALNS) enzyme. The deficiency of this enzyme accumulates glycosaminoglycans (GAG) such as keratan sulfate (KS) and chondroitin-6-sulfate (C6S) in multiple tissues, mainly bone, cartilage, heart valves, and cornea, leading to devastating skeletal dysplasia with incomplete ossification and successive imbalance of growth.
Patients with MPS IVA typically appear healthy at birth, but gradually develop multi-systemic manifestations that progress over time. MPS IV is an ultra-rare autosomal recessive inherited disorder with a point prevalence ranging between 1/599,000 live births in the UK. The frequency of imaging depends on both the patient’s age and clinical condition. Physical growth slows and often stops around age 8. Children with the more severe form of MPS IV may not live beyond their twenties or thirties. On average, female patients lived 4 years longer than male patients.
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 Morquio syndrome is anticipated to drive the growth in the MPS IV treatment market during the forecast period. With the recent approval of the Vimizim (elosulfase alpha), 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.
Increasing incidence of MPS IV syndrome, rising health care industries, and increase in government investment to develop health care infrastructure also drive the market growth. Market players are more focused on the adoption of inorganic growth strategies such as collaborations and partnerships to strengthen their position and expected by key players to drive the market growth over the forecast period.
The huge price tag associated with Morquio syndrome therapeutics such as Vimizim (elosulfase alfa) approved by NICE, has an exceptionally high price – around £395,000 per patient per year – making it one of the most expensive drugs in the world, is one of the major factors expected to limit the growth of MPS IV treatment market. Other factors which may restrict the growth of the MPS IV treatment market are the lack of awareness and the regulatory and clinical hurdles.