Market Challenges And Opportunities

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.