The UK Pompe Disease 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.
Pompe Disease also known as Type II glycogen storage disease (GSDII), is a rare autosomal recessively inherited metabolic disorder caused by the accumulation of glycogen takes place in the lysosome that affects people of all ages. The GAA gene contains the genetic information for the production and function of a protein called ‘acid alpha-glucosidase (GAA), which converts glycogen into a simple sugar called glucose. Therefore, glycogen starts to accumulate in all kinds of tissues, but primarily in skeletal muscle, smooth muscle, and cardiac muscle, where it causes damage to tissue structure and function and can lead to premature death in the newborn.
Pompe disease is an ultra-orphan disease that is currently diagnosed in less than 200 people in the UK. Pompe disease is the only glycogen storage disease that is also a lysosomal storage disease (LSD). The incidence rate of Pompe disease is approximately 1 in 40,000 live births. Progressive muscle weakness, poor muscle tone, an enlarged liver are the major symptoms. Enzyme replacement therapy (ERT), one of the effective treatments, aims to replenish the shortage of GAA by intravenous administration of industrially made ‘rhGAA’ (recombinant human GAA).
Market Growth Drivers
The rising prevalence of Pompe disease is creating opportunities for manufacturers to find new solutions for the diagnosis and treatment. Currently, manufacturers are focusing on expanding their pipeline and develop effective products to cater to the increasing demand for Pompe disease treatment.
Increasing developments in enzyme replacement therapies (ERT) and gene therapies are one of the key factors driving the growth of the Pompe disease market. Moreover, research activities performed to find an effective treatment for the disease and the growth in the special regulatory drug designations for the treatment can further create growth opportunities for the Pompe disease market.
Regulatory bodies are taking continuous initiatives for developing and manufacturing innovative products for the diagnosis & treatment of Pompe disease. However, inflated costs associated with Pompe disease therapy could hinder the market growth opportunities.