Pombiliti Atga

1 INDICATIONS AND USAGE POMBILITI is indicated, in combination with Opfolda, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency) weighing ≥40 kg and who are not improving on their current enzyme replacement therapy (ERT). POMBILITI is a hydrolytic lysosomal glycogen-specific enzyme indicated, in combination with Opfolda, an enzyme stabilizer, for the treatment of adult patients with late-onset Pompe disease (lysosomal acid alpha-g...

Pombiliti Atga (CIPAGLUCOSIDASE ALFA-ATGA) belongs to the General Medicine class of medications. It was first approved by the FDA in Yes. This medication requires a prescription from a licensed healthcare provider.

12.1 Mechanism of Action Pompe disease (also known as glycogen storage disease type II, acid maltase deficiency, and glycogenosis type II) is an inherited disorder of glycogen metabolism caused by a deficiency of lysosomal acid alpha‑glucosidase (GAA) that degrades glycogen to glucose in the lysosome. GAA deficiency results in intra-lysosomal accumulation of glycogen in various tissues. Cipaglucosidase alfa-atga provides an exogenous source of GAA. The bis-M6P on cipaglucosidase alfa-atga mediates binding to M6P receptors on the cell surface with high affinity. After binding, it is internalized and transported into lysosomes where it undergoes proteolytic cleavage and N‑glycans trimming which are both required to yield the most mature and active form of GAA. Cipaglucosidase alfa-atga then