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  • To Be Published : Jul 2024
  • Code : CMI1316
  • Formats :
      Excel and PDF
  • Industry : Pharmaceutical

Pompe disease is an inherited Lysosomal Storage Disorder (LSD) caused by a deficiency of enzyme acid named alpha-glucosidase (GAA). This causes the glycogen to accumulate in different kinds of tissues, primarily into smooth muscles, skeletal muscles and cardiac muscle. It leads to muscle weakness, respiratory problems, and may also affects the liver and heart. Pompe disease is inherited as an autosomal recessive genetic trait. There are three major types of pompe disease, which differ in severity and appear at different age, these types are known as classic infantile-onset, which starts within a few months of birth, non-classic infantile-onset Pompe disease- usually appears by age 1, and late-onset type of Pompe disease that may shows the symptoms later in childhood, adolescence, or adulthood. Pompe disease therapeutic market can exhibit immense growth potential due to presence of limited number of manufacturers, which can significantly enhance and expand their business potential and scope in this market.

Pompe Disease Therapeutic Market Taxonomy:

By Therapy Type:

  • Gene Therapy
  • Enzyme Replacement Therapy (ERT)
  • Chaperone-Advanced Replacement Therapy (CHART)

By Administrational Route:

  • Oral
  • Parenteral

By Patient Type:

  • Infants
  • Adults

Increasing government initiatives is expected to augment growth of the pompe disease therapeutic market

Considering the public health problems, government is taking continuous initiatives in promoting and advancing the development of innovative products for the prevention and diagnosis or treatment of rare diseases or conditions such as Pompe disease. For instance, in 2015, Orphan Product Grants Program funded 18 new grant awards from 92 grant applications and provided funding and support to around 67 ongoing clinical study projects related to rare disease. Furthermore, initiatives from the governments of economies such as Sweden, the Netherlands, and South Korea that focus on approving guidelines for the reimbursement of individuals undergoing enzyme replacement therapy (ERT) are also expected to boost growth of the pompe disease therapeutic market in near future.

Increasing prevalence of pompe disease in many economies is expected to have a lucrative opportunities for pharmaceutical manufacturers to come with a tailored solution for diagnosis and treatment of disease. For instance, according to United Pompe Foundation (UPF), 2017, the estimated frequency occurrence of Pompe disease may vary among different ethnic groups and nationalities: in Holland: 1 in 40,000 (Adults: 1 in 57,000; Infantile: 1 in 138,000), Southern China and Taiwan: 1 in 50,000 births, African-Americans: 1 in 14,000 births and Caucasian: 1 in 100,000 suffer from pompe disease. Gene therapy and enzyme replacement therapy may be highly effective at reducing clinical manifestations of this rare disease. Currently, there are two commercialized products in enzyme replacement therapy, which are Myozyme for the treatment of infants and children with Pompe disease and Lumizyme for late-onset non-infantile Pompe disease. Furthermore, various pipeline products in phase 3 (NeoGAA GZ402666 (Sanofi), AT-982 (Audentes therapeutics)) and in phase 2 (BMN 701(Biomarin)) clinical trial are also expected to create a meteoric growth in this market during the forecast period. However, the high cost associated with the treatment and long duration of the treatment are the factors restraining growth of the pompe disease therapeutic market. For instance, according to a research paper published by Harvard School, in 2016, Lumizyme accounted for US$ 100,000 per year for children and US$ 300,000 per year for adult treatments.

Increasing investments in research and development by manufacturers is expected to propel the market growth

Manufacturers are focusing on finding new solutions by expanding their pipeline to cater the demand for pompe disease therapeutics market. For instance, in September, 2017, Avrobio Inc. expanded their products pipeline with gene therapy to treat pompe disease by utilizing a proprietary lysosomal targeting sequence to deliver high levels of enzyme to lysosomes. Furthermore, in October 2017, Amicus Therapeutics received orphan drug designation to ATB200/AT2221 from the U.S. FDA for the treatment of Pompe disease. This novel treatment paradigm consists of ATB200, a unique recombinant human acid alpha-glucosidase (rhGAA) enzyme with optimized carbohydrate structures, particularly mannose-6 phosphate (M6P) to enhance uptake, co-administered with AT2221, a pharmacological chaperone.

Key players operating in the pompe disease therapeutic market include Amicus Therapeutics, Inc., Valerion Therapeutics, LLC, Sanofi S.A., Oxyrane, and Sangamo Therapeutics, Inc.


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