Pompe disease treatment industry analysis

Market research analysts at Technavio predict that the global Pompe disease treatment market will grow steadily during the next four years and post a CAGR of almost 12% by 2020. This market research analysis identifies the availability of reimbursement policies for the treatment of Pompe disease as one of the primary factors that will have a positive impact on the growth of this market.

It has been observed that the treatment of Pompe disease costs around USD 100,000 to USD 300,000 for an individual every year, making it difficult for individuals to continue with the treatment for a long period. However, financial support from various organizations such as the Canadian Association of Pompe, Acid Maltase Deficiency Association, and International Pompe Association enables patients to continue their treatment, which will drive the growth of the market. Additionally, initiatives from the governments of countries such as Sweden, the Netherlands, and South Korea that focus on approving guidelines for the reimbursement of individuals undergoing enzyme replacement therapy (ERT) will also bolster the growth of the market in the next four years.

Pompe disease treatment market trends

One of the major trends that will gain traction in the Pompe treatment market is the emergence of immunotherapy for Pompe disease. Immunotherapy enhances the powers of the immune system and aids in fighting various respiratory, infectious, and autoimmune disorders. Advances in preventing Immunoglobin (IgG) production have shown a significant effect in both pre-clinical and clinical studies. This will result in the increased adoption of drugs such as mycophenolate mofetil, cyclophosphamide, and methotrexate for the prevention of responses to ERT as they have shown significant results in preventing the formation of anti-GAA antibody in both pre-clinical and clinical studies.

Pompe disease treatment market forecast: Competitive landscape and key vendors

Though the market exhibits immense growth potential, it is currently characterized by the presence of only a limited number of vendors. Amicus Therapeutics, Audentes Therapeutics, and Sanofi Genzyme are the major players in the market, where Sanofi Genzyme has already produced drugs that have been approved for the treatment for Pompe disease, while Amicus Therapeutics and Audentes Therapeutics have their respective drug candidates under clinical trials. As a result, any company that can manufacture and market medicinal products for the effective treatment of Pompe disease will gain a competitive edge over the other market players.

The leading vendors in the Pompe disease treatment market

• Amicus Therapeutics
• Audentes Therapeutics
• Sanofi Genzyme

Pompe disease treatment market growth by type 

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

The classic infantile-onset Pompe disease manifests in just a few months of the baby’s birth. The disease results in breathing complications and weight-loss and if untreated can lead to cardiac failure within the first year of life. The non-classic infantile-onset Pompe disease also occurs within the first year and results in the expansion of the heart, a condition known as cardiomegaly. The adult-onset Pompe disease does not manifest till adulthood and is milder than the infantile-onset Pompe disease. Symptoms mainly include progressive muscle weakness and often creates breathing problems that might result in respiratory failure.

Pompe disease treatment market based on geographical segmentation

• Americas
• APAC
• EMEA

This market study estimates that in terms of geographical regions, EMEA accounted for the major shares and dominated the Pompe disease treatment market during 2015. Though stringent regulations pose a major challenge to the market’s growth, the increasing awareness among the public, the assistance from regulatory bodies, and the exemption of Pompe treatment drugs from pricing constraints will boost market growth. Additionally, the availability of favorable reimbursement policies in several countries in Europe will also propel the market’s growth prospects in this region.

Key questions answered in the report include

• What will the Pompe disease treatment market size and the growth rate be in 2020?
• What are the key factors driving the global Pompe disease treatment market?
• What are the key market trends impacting the growth of the global Pompe disease treatment market?
• What are the challenges to Pompe disease treatment market growth?
• Who are the key vendors in the global Pompe disease treatment market?
• What are the market opportunities and threats faced by the vendors in the global Pompe disease treatment market?
• Trending factors influencing the market shares of the Americas, APAC, and EMEA.
• What are the key outcomes of the five forces analysis of the global Pompe disease treatment market?

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