Lysosomal alpha glucosidase is a recombinant enzyme used for the treatment of lysosomal storage disorder known as Pompe disease. Pompe disease, also known as Acid Maltase Deficiency or Glycogen Storage Disease type II. Pompe disease is considered to be an inherited fatal muscular myopathy disorder caused due to glycogen build-up in the body cells such as in cardiac, smooth, and skeletal muscle cells. Pompe disease is characterized by absence or deficiency of the lysosomal enzyme alpha-glucosidase (GAA). Large number of pipeline product is the major factor driving lysosomal alpha glucosidase market.
Lysosomal Alpha Glucosidase Market – Driver
Increasing research activities along with the number of clinical trials and pipeline products is expected to boost the lysosomal alpha glucosidase market in near future. For instance, in 2017, Valerion Therapeutic, a clinical-stage biotechnology company specialized in the development of therapies for orphan genetic diseases, initiated phase 1/2 clinical trials for its candidate drug, VAL-1221 in patients with late-onset of Pompe disease. VAL-1221 combines company’s proprietary antibody-mediated delivery technology with recombinant human acid alpha-glucosidase, offering improved glycogen clearance and better patient outcomes.
Moreover, JCR Pharmaceuticals Co. Ltd., evaluated its candidate genetically engineered enzyme, JR-162, a therapeutic enzyme product for Pompe disease. As on August 2017, JR-162 was in preclinical stage. JR-162 is developed by applying J-Brain Cargo, an innovative technology by the company that enables the delivery of intravenously administered drug compounds of large molecular weight such as protein to cross blood brain barrier.
Further, in Feb 2018, Amicus Therapeutics, patient-centric biotechnology company focused on discovering, developing and delivering novel high-quality medicines for rare metabolic disease, is investigating ATB200/AT2221 in patients with Pompe disease and is in Phase1/2 clinical study. ATB200 is recombinant human acid alpha-glucosidase enzyme co-administered with AT2221, a pharmacological chaperone. In 2016, Genzyme, a unit of Sanofi, initiated phase III clinical trial for NeoGAA, a second generation alglucosidase alfa enzyme based enzyme replacement therapy for Pompe disease. In Feb 2018, Audentes Therapeutics, Inc., a biotechnology company focused on developing and commercializing gene therapy products, conducts phase 1/2 clinical trial for AT982, to treat Pompe disease.
Moreover, in 2012, Oxyrane collaborated with VIB researchers from Ghent University and Vrije Universiteit Brussels, have developed a new technology that enables a more efficient approach to ERT production for LSD treatments. Further, Oxyrane, company involved in enzyme replacement therapies (ERTs) for the treatment of lysosomal storage diseases (LSDs), also has a pipeline product named OXY 2810 for Pompe disease.
Lysosomal Alpha Glucosidase Market - Regional Analysis
Based on geography, the lysosomal alpha glucosidase market has been segmented into North America, Latin America, Europe, Middle East, Asia Pacific and Africa. Europe is expected to be dominant market during the forecast period attributed to higher rate of incidence in these region. For instance, according to Feb 2018 published article of BMJ Paediatrics Open journal, the incidence of infantile Pompe disease in the Maroon community of France is roughly 50 times higher than elsewhere in the world.
Lysosomal Alpha Glucosidase Market – Competitor
The global market of lysosomal alpha glucosidase market is led by Genzyme
Corp., a unit of Sanofi. The player with pipeline product includes Pharming Group N.V., Valerion Therapeutic, Audentes Therapeutics, Inc. and Oxyrane.
Lysosomal Alpha Glucosidase Market - Taxonomy
By Distributional Channel