The global Pompe disease market is expected to witness significant growth through the forecast period. Pompe disease (glycogenosis type II) is a rare disorder affecting approximately 1 in 40,000 people in the U.S. Factors such as continued uptake of long-term therapies, launch of novel drugs, and high treatment cost are propelling market growth.
Pompe disease is an autosomal recessive glycogen storage disorder occurring as a result of deficiency of lysosomal enzyme acid α-glucosidase (GAA). Approximately 20% of patients with Pompe disease have the infantile or early-onset form of disease, which proves fatal within a year of birth. The more common form of the disease is known as juvenile or adult-onset form and is reported in 80% of patients. The disorder primarily affects cardiac and skeletal muscle and manifests as progressive respiratory distress, cardiomyopathy, and skeletal muscles atrophy.
Based on treatment type, the Pompe disease market can be segmented into enzyme replacement therapy (ERT), substrate reduction therapy (SRT), chaperone-advanced replacement therapy (CART), and supportive care. In 2006 and 2010, Genzyme’s ERT therapy Myozyme and Lumizyme garnered approval for the treatment of pediatric and adult-onset Pompe disease, respectively and currently remain the only long-term therapies available for the treatment of the disease.
By region, North America is one of the leading markets and this trend is expected to continue in future. Asia Pacific (APAC) is projected to show strong growth through the forecast period, driven by increasing awareness among patients regarding rare disorders and improved healthcare facilities in the APAC countries.
Some of the key players in the global Pompe disease market include Genzyme, Amicus Therapeutics, Valerion Therapeutics, Audentes Therapeutics, among others. At present, manufacturers are focusing on the development of novel therapies for the treatment of this life threatening disease. Increasing advancement in the gene therapy and modified enzyme replacement therapies are driving factors for the growth of Pompe disease market.
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