Global Pompe Disease Market was valued at 1241.24 million and is expected to register a CAGR of 3% during the forecast period of 2020 to 2027 and reach USD 1450 million in 2027.
Pompe disease is a genetic metabolic disorder that occurs in infants and is caused due to the mutations in the GAA gene responsible for producing the acid, alpha-glycosidase enzyme that converts glycogen into a simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and results to have a heart problem, muscle weakness, liver damage that can lead to premature death in the new-born.
The demand for Pompe disease treatment is increasing due to the increasing government initiatives and rising R&D activities by manufacturers. However, the high cost of treatment is hampering the market growth.
The rising prevalence of Pompe disease is creating opportunities for manufacturers to find new solutions for the diagnosis and treatment. According to the National Organization for Rare Disorders, the estimated frequency occurrence of Pompe disease is approximately 1 in 40,000 people in the US. Currently, manufacturers are focusing on expanding their pipeline to cater to the increasing demand for Pompe disease treatment. For instance, in September 2017, AVROBIO’s initiated a pre-clinical project which includes third gene therapy to treat lysosomal storage disorders by utilizing lysosomal targeting sequence to deliver high levels of enzymes to lysosomes. This project helped the company to expand its research & development pipeline and develop effective products for the treatment of Pompe disease.
Governments in various countries are taking continuous initiatives for developing and manufacturing innovative products for the diagnosis & treatment of rare diseases such as Pompe disease. For instance, in the US the Orphan Product Grants funded 18 new grants from a total of 92 grant applications in 2015 to support around 67 ongoing clinical studies that are related to rare diseases.
Pompe disease is a genetic metabolic disorder that occurs in infants and is caused due to the mutations in the GAA gene responsible for producing the acid, alpha-glycosidase enzyme that converts glycogen into a simple form. The absence or mutation in the GAA gene leads to the accumulation of glycogen and results to have a heart problem, muscle weakness, liver damage that can lead to premature death in the new-born.
The demand for Pompe disease treatment is increasing due to the increasing government initiatives and rising R&D activities by manufacturers. However, the high cost of treatment is hampering the market growth.
The rising prevalence of Pompe disease is creating opportunities for manufacturers to find new solutions for the diagnosis and treatment. According to the National Organization for Rare Disorders, the estimated frequency occurrence of Pompe disease is approximately 1 in 40,000 people in the US. Currently, manufacturers are focusing on expanding their pipeline to cater to the increasing demand for Pompe disease treatment. For instance, in September 2017, AVROBIO’s initiated a pre-clinical project which includes third gene therapy to treat lysosomal storage disorders by utilizing lysosomal targeting sequence to deliver high levels of enzymes to lysosomes. This project helped the company to expand its research & development pipeline and develop effective products for the treatment of Pompe disease.
Governments in various countries are taking continuous initiatives for developing and manufacturing innovative products for the diagnosis & treatment of rare diseases such as Pompe disease. For instance, in the US the Orphan Product Grants funded 18 new grants from a total of 92 grant applications in 2015 to support around 67 ongoing clinical studies that are related to rare diseases.
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