EP3871687A1 — Enzyme replacement therapy for treating pompe disease

USPTO Abstract

The present invention relates to a method of treating a patient with Pompe disease comprising providing a composition of a recombinant acid alpha-glucosidase, which comprises a glycosylation structure of formula 1, wherein a "G" represents N-acetylglucosamine, a "M" represents mannose, and a "G/M" represents a terminal N-acetylglucosamine or terminal mannose, wherein one but not both of the "G/M" subunits may be absent in which case the present "G/M" subunit may represent two terminal mannose subunits, wherein one or more of the "G" or "M" subunits are not α1,3-fucosylated, not α1,6-fucosylated and not β1,2-xylosylated, and wherein any one of "G", "M" or "G/M" may be methylated; and said glycosylation structure of formula 1 is present in at least 20% of all glycosylation structures of the recombinant acid alpha-glucosidases of the composition; and administering the composition to the patient.