Cipaglucosidase alfa

Cipaglucosidase alfa (ATB200) is a novel recombinant human acid alpha-glucosidase (GAA) investigated for the treatment of patients with Pompe disease, a rare inherited metabolic disorder characterized by a deficiency in GAA. Other types of enzyme replacement therapy for the treatment of Pompe disease include alglucosidase alfa and avalglucosidase alfa. Cipaglucosidase alfa is conjugated with mannose-6-phosphate (M6P) N-glycans that bind to the cation-independent mannose-6-phosphate receptor (CI-MPR) in skeletal muscle, one of the main affected tissues in Pompe disease. Compared to alglucosidase alfa, cipaglucosidase alfa has a higher M6P content.

In December 2022, the EMA's Committee for Medicinal Products for Human Use (CHMP) recommended cipaglucosidase alfa be granted marketing authorization for the treatment of Pompe disease, and the EMA fully approved the drug on March 27, 2023. Cipaglucosidase alfa is coadministered with miglustat, a small-molecule pharmacological chaperone that stabilizes the conformation of the enzyme.

In Europe, cipaglucosidase alfa is a long-term enzyme replacement therapy used in combination with the enzyme stabilizer miglustat for the treatment of adults with late-onset Pompe disease, also known as acid α-glucosidase (GAA) deficiency.