POMPE DISEASE THERAPEUTIC MARKET ANALYSIS

Regional Analysis

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.