Amicus Therapeutics, a biotechnology company focused on developing medicines for rare diseases, announced the approval of Opfolda (miglustat) 65mg capsules by the European Commission. This is an enzyme stabilizer of cipaglucosidase alfa, a long-term therapy used to treat adult patients with late-onset Pompe disease, an inherited lysosomal disorder caused by deficiency of the enzyme acid α-glucosidase (GAA). Amicus is launching Pombiliti + Opfolda in Germany and is commencing reimbursement processes with healthcare authorities in other European countries. The EC approval was given based on clinical data from the PROPEL trial which studied the population of both ERT-naïve and ERT-experienced participants in a controlled setting.


Tagged: chaperone, ERT