Pompe Disease Market in the US 2015-2019

Pompe disease industry analysis in the US

Pompe disease is a type of lysosomal storage disease (LSD) that is caused by mutations in the GAA gene. These mutations inhibit the action of GAA, which is an enzyme responsible for catabolizing glycogen. The market for Pompe disease in the US is estimated to show healthy growth during the forecast period and is positively influenced by factors like the reimbursement of treatment expenses by governments and vendors in some countries. These factors propel market growth during the forecast period and result in its healthy CAGR of more than 10% by 2019.

Based on the Pompe disease industry insights, eminent factors, such as the high-unmet medical needs of patients, to drive market growth during the forecast period. Drugs like Myozyme and Lumizyme fail to improve the neurological symptoms associated with the disease. This unavailability of a drug that is safe, effective, and less expensive is expected to bolster market growth during the forecast period.

Types of Pompe disease

• Infantile-onset Pompe disease
• Adult-onset Pompe disease

The infantile-onset Pompe disease generally manifests itself within a few months after the baby is born and is characterized by symptoms like myopathy, hypotonia, hepatomegaly, and a defective heart.  The adult-onset Pompe disease generally manifests itself during adolescence or adulthood. This disease is generally milder than its infantile-onset form and is characterized by symptoms like progressive muscle weakness in the legs and the trunk.

Competitive landscape and key vendors

This market is highly lucrative and is characterized by the presence of a limited number of vendors. Out of all the vendors in this market, only Genzyme has produced an approved drug for the treatment of the disease. Vendors that have the capability to manufacture and market a drug that can effectively treat Pompe disease and also exhibit a high efficacy profile are expected to gain maximum traction in the market.

Key vendors in this market are 

• Amicus Therapeutics
• BioMarin Pharmaceutical
• Genzyme

Other prominent vendors analyzed in this market study are Audentes Therapeutics, EpiVax, Oxyrane, Sangamo BioSciences, and Valerion Therapeutics.

Growth drivers, challenges, and upcoming trends: Pompe disease

Technavio’s market research analyst has estimated upcoming trends, such as the recent rise in public awareness, to spur market growth during the forecast period. The organization of various events and fundraising programs by the government and non-government organizations is expected to raise awareness and educate people about the disease, its diagnosis, and its treatment options. Organizations like the United Pompe Foundation, International Pompe Association, Canadian Association of Pompe, Acid Maltase Deficiency Association, and NORD help to spread awareness by promoting various online and offline events.

This report provides a number of factors contributing to the adoption, limitations, and opportunities of the pompe disease market in the US. It also offers an analysis of each factor and an estimation of the extent to which the factors are likely to impact the overall market growth.

Key questions answered in the report include

• What will the market size and the growth rate be in 2019?
• What are the key factors driving the pompe disease market in the US?
• What are the key market trends impacting the growth of the pompe disease market in the US?
• What are the challenges to market growth?
• Who are the key vendors in the pompe disease market in the US?
• What are the market opportunities and threats faced by the vendors in the pompe disease market in the US?
• What are the key outcomes of the five forces analysis of the pompe disease market in the US?

Technavio also offers customization on reports based on specific client requirement.

Related reports

• Global Pompe Disease Market 2015-2019
• Autoimmune Diseases Market in the US 2015-2019
• Neurodegenerative Diseases Market - Global Trends & Forecast 2015-2019