Amicus Therapeutics is a biotherapeutics company operating globally offering treatment for patients suffering from genetic devastating disorders. The firm has introduced Galafold, which is an advanced precision medicine that treats persons suffering from Fabry disease across Europe. Fabry is an unusual X-linked genetic lysosomal disease which comes as a result of sphingolipids being improperly metabolized. Galafold is a medicine which is administered orally. It is the first drug to be approved by European Medicines Agency for treating patients suffering from Fabry disease. Moreover, the medicine has not yet been approved by the Food and Drug Administration. Amicus Therapeutics has several goals which are to be achieved in 2017. These include launching a clinical plan for treating Pompe disease, presenting a J-NDA for migalastat, and finishing the Phase 3 clinical trial for treating and managing epidermolysis bullosa.
In late 2016, Amicus Therapeutics announced its regulatory strategy with the Food and Drug Administration for treating and managing Galafold in the United States of America. The plan was for advancing the Fabry drug program including two phase 3 clinical trials. Galafold conferred its significance statistically when 50 patients suffering from mutant galactosidase alleles were treated. Amicus Therapeutics worth in business investment is estimated to be approximately $1.2 billion by the end of 2024 (Twitter). The company reported a positive early phase 1/2 data for managing and treating Pompe disease. Pompe disease is an unusual genetic infection that leads to the growth of glycogen in the body. The disease weakens the muscles making them impaired in function. Additionally, Amicus Therapeutics conducted a study which revealed a safety profile that is positive and has no severe events. The study also manifested musculatoprotection as exhibited by biomarkers of muscle destruction. The study is divided into groups; ERT-switch, non-ambulatory ERT-switch, and ERT-naive. Aspartate aminotransferase, alanine aminotransferase, and creatine kinase levels displayed a trend towards improvement in half the people admitted and were stable at all levels.
AT2221/ ATB200 uses ATB200, a treatment mechanism that is unique. ATB200 is a recombinant functional alpha-glucosidase enzyme that is capable of carrying mannose-6 phosphate moieties which are meant for increasing uptake. AT2221 is a co-treated pharmacological chaperone used to stabilize the compound.