On Sept. 28, 2023, Amicus Therapeutics announced that the U.S. Food and Drug Administration (FDA) had approved Pombilitiﾙ (cipaglucosidase alfa-atga) + Opfoldaﾙ (miglustat) 65mg capsules. This two-component therapy is indicated for adults living with late-onset Pompe disease (LOPD) weighing =40 kg and who are not improving on their current enzyme replacement therapy (ERT).

Late-onset Pompe disease is a rare, debilitating, and life-threatening lysosomal disorder caused by a deficiency of the enzyme acid alpha-glucosidase (GAA).