cipaglucosidase alfa + miglustat

**Cipaglucosidase alfa + miglustat** is a combination therapy approved for treating symptomatic adults with late-onset Pompe disease (LOPD). Cipaglucosidase alfa is a recombinant human acid alpha-glucosidase (GAA) enzyme replacement designed with enhanced bis-phosphorylated mannose-6-phosphate (bis-M6P) N-glycans, facilitating efficient cellular uptake via CI-MPR receptor-mediated endocytosis[1]. Miglustat is a small molecule administered orally, which serves as a stabilizer for cipaglucosidase alfa in plasma before cell uptake, increasing its bioavailability and exposure[1][2]. Miglustat transiently inhibits the enzyme in circulation, preventing inactivation, but releases it in the low pH of lysosomes, allowing GAA activity to hydrolyze accumulated glycogen in muscle cells[1]. The combination improves muscle function and reduces biomarkers of muscle damage in clinical trials, with demonstrated durable benefits over standard ERT[1][2].

Brand names

Pombiliti + Opfolda

Other names

ATB200 + AT2221