Amicus Therapeutics has received approval from the European Commission (EC) for Opfolda (miglustat), its treatment for Pompe disease, a rare disorder that causes progressive muscle weakness. The approval specifically covers the drug in combination with Pombiliti (cipaglucosidase alfa). Results from a recent phase 3 study showing positive changes in mobility and breathing, both affected by the disorder, supported the EC’s decision.
According to Emily Kimber, “Pompe disease is a rare, progressive and debilitating muscle disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA), which leads to a build-up of glycogen in cells. The disease ranges from a rapidly deteriorating infantile form with significant impact on heart function to a more slowly progressive, late-onset form primarily affecting skeletal muscle and progressive respiratory involvement.”
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(Source: PM Live, June 29th, 2023)