Pombiliti® (cipaglucosidase alfa-atga)
for Pompe Disease
Pombiliti (cipaglucosidase alfa-agta) is an intravenous infusion given over 4 hours in combination with oral Opfolda (miglustat) capsules for treating late-onset Pompe disease in adults weighing at least 40kg (88 lb) who are not improving on current enzyme replacement therapy (ERT). Pombiliti provides exogenous GAA, which binds to muscle cell receptors, is internalized, and activated in lysosomes. It then breaks down glycogen, reducing its concentration in the cells.
