Market Trends of Lysosomal Storage Disease Treatment Industry
Pompe Disease Segment is Expected to Hold a Major Market Share in the Lysosomal Storage Disease Treatment Market
Pompe disease is a rare disease classified as early infantile or late-onset, majorly affecting skeletal muscle, heart defects, and breathing problems.
As to the National Institute of Health, in April 2022, Pompe disease affects 1 in 40,000 individuals in the United States. Moreover, according to the study titled "Carrier frequency and predicted genetic prevalence of Pompe disease based on a general population database" Molecular Genetics and Metabolism report in June 2021, Pathogenic or Likely Pathogenic Variants (PLPVs) were found in higher proportions in East Asian and African patients with classic infantile-onset Pompe disease. In the general population, total carrier frequency (CF) and predicted genetic prevalence (pGP) was 1.3 % (1 in 77) and 1:23,232, respectively. Thus, the rising prevalence of pompe disease is anticipated to boost the growth of the market segment over the forecast period.
Moreover, product approvals and launches by the key market players with respect to the segment are anticipated to drive the market over the forecast period. For Instance, In August 2021, The US FDA approved a new enzyme replacement therapy, avalglucosidase alfa, for patients aged 1 year and older with late-onset Pompe disease.
Thus, all aforementioned factors are anticipated to drive the segment growth over the forecast period.
North America is Expected to Hold a Significant Share in the Market and Expected to do Same in the Forecast Period
North America is expected to hold a major market share in the global lysosomal storage disease treatment market due to the increasing incidence of lysosomal storage disorders in this region.
According to NORD, data updated in March 2021, approximately 6,000 individuals are affected with Gaucher disease in the United States. Moreover, Type 1 Gaucher disease is the most common form of the disease in western countries, making up roughly 95 percent of patients. Furthermore, increasing research and development for diagnosis and drug development for the treatment of lysosomal diseases and the presence of well-established healthcare infrastructure are also fueling the growth of the overall regional market to a large extent.
Moreover, with increasing product launches and the US FDA approval, and the activities by the Key players the market is expected to see a surge. For Instance, In August 2021, the US FDA approved Nexviazyme for intravenous infusion to treat patients 1 year of age and older with late-onset Pompe disease. Moreover, The US FDA granted Orphan Drug Designation (ODD) to AceLink Therapeutics ' AL01211 to treat Fabry disease. Such, instances are expected to boost the growth of the market over the forecast period.
Furthermore, increasing awareness about rare disorders and developed healthcare infrastructure are also expected to fuel the growth of the market in this region.
