Pompe Disease Market
size is $1,277Bn in 2019, growing at a CAGR of 3.7% during the forecast period 2020-2025. Pompe disease is also known as GAA deficiency or type II glycogen storage disease (GSD). It is a genetic disorder in which complex sugar called glycogen builds up metabolism in the body’s cells. The disease results from the deficiency of an enzyme called Acid Alfa Glucosidase (GAA), which breaks downs complex sugars in the body. This buildup occurs in organs and tissues, especially in muscles, causing them to break down. The additional glycogen builds up in the patient and is stored in skeletal muscle, heart and other tissues, which causes the progressive indications of pompe disease. It causes muscle weakness and trouble breathing affecting the liver, heart (Cardiomegaly), and muscles that can lead to premature death in the new-born.
By Therapy Type – Segment Analysis
In 2019, Enzyme Replacement Therapy (ERT) segment dominated the Pompe Disease Market in terms of revenue is estimated to grow at a CAGR of 3.0%. Enzyme Replacement Therapy is used to increase the levels of GAA in the body and reduce the accumulation of glycogen inside cells. ERT is administered into the bloodstream and has been shown to reduce the abnormal thickening of the heart walls that is characteristic of Pompe disease. It also helps maintain muscle function and improve patients’ quality of life. Lumizyme (alglucosidase alfa) is an FDA approved drug for Enzyme Replacement Therapy abetting towards the market’s growth.
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Geography – Segment Analysis
In 2019, the North America region dominated Pompe Disease Market in terms of revenue with a market share of 39% owing towards owing to the presence of established payers and an increase in the number of Pompe disease patients in the region. This growth can be attributed towards the increasing research & development initiatives and government support for the improvement of the healthcare sector. Government has taken initiatives to promote the healthcare expenditure, growing awareness among patients regarding rare disorders in this region are key factors in the growth of the Pompe Disease market. Europe is estimated to record the fastest growth rate during the forecast period 2020-2025.
Drivers – Pompe Disease Market
- Increase In The Prevalence of Pompe Disease
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. The increase in the prevalence of pompe disease is driving the Pompe Disease Market owing towards the increase of manufacturers focusing on expanding their pipeline to cater to the demand for Pompe disease treatment. Governments have taken initiatives for developing and manufacturing innovative products for the diagnosis & treatment of Pompe disease is the other major factor driving growth of the Pompe Disease market.
Challenges – Pompe Disease Market
- Lack Of Cost-Effective Treatment
The lack of cost effective treatments is restraining the growth of the market. The treatment is very expensive owing to the lack of FDA approved drugs which leads to the less affordability rate. The scarcity of low cost treatment hampers the growth of the market owing towards the patients being uncured.
Pompe Disease Industry Outlook
Product Launches was the key strategy of the players in the Pompe Disease Industry. Pompe Disease top 10 companies include Amicus Therapeutics, Inc., Sanofi S.A., Audentes Therapeutics, Oxyrane , Valerion Therapeutics , AVROBIO, Inc., CENTOGENE AG, Sangamo BioSciences, BioMarin Pharmaceutical and Sanofi Genzyme.
- In January 2020, Sanofi S.A. acquired Synthorx, Inc. This acquisition helped the company in increasing the production for genetic disorders, oncology and immunological therapy research.
- In April 2019, Audentes Therapeutics collaborated AAV Technology Platform and Pipeline with New Development Programs for Duchenne Muscular Dystrophy and Myotonic Dystrophy.
- Increased research in the field of Pompe Disease is set to propel the growth of the market.
- Increasing developments in the gene therapy and enzyme replacement therapies are driving factors for the growth of Pompe disease market.
- Europe region is estimated to record the fastest growth rate during the forecast period 2020-2025.
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