Report Overview

The Australian Pompe Disease (GSDII) 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 disorders caused by the accumulation of glycogen take 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), that converts glycogen into 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 new-born.

Three infants are born with Pompe every year in Australia. Pompe is much less common in Australia, with an incidence ofone in 145,000people. Progressive muscle weakness, poor muscle tone, an enlarged liver are the major symptoms. Enzyme replacement therapy (ERT), one of the effective treatments, aims to replenishing the shortage of GAA by intravenous administration of industrially made rhGAA (recombinant human GAA).



Market 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 the enzyme replacement therapies (ERT) and gene therapies are one of the key factors driving the growth of 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 the growth opportunities for Pompe Disease market.

However, inflated costs associated with the Pompe Disease therapy could hinder the market growth opportunities.