Cipaglucosidase Alfa: First Approval.

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Blair HA

Cipaglucosidase Alfa: First Approval.

Drugs. 2023 Jun;83(8):739-745. doi: 10.1007/s40265-023-01886-5.

PubMed ID

37184753 [ View in PubMed

]

Abstract

Cipaglucosidase alfa (Pombiliti()) is a recombinant human acid alpha-glucosidase (GAA) product being developed by Amicus Therapeutics along with the enzyme stabilizer miglustat as a two-component therapy for Pompe disease. Pompe disease is a rare, inherited lysosomal disease caused by a deficiency of the enzyme GAA, which leads to accumulation of glycogen in various tissues. On 27 March 2023, cipaglucosidase alfa was approved in the EU as a long-term enzyme replacement therapy (ERT) used in combination with miglustat for the treatment of adults with late-onset Pompe disease. This article summarizes the milestones in the development of cipaglucosidase alfa leading to this first approval.

DrugBank Data that Cites this Article

Drugs

Cipaglucosidase alfa

Drug Targets

Drug	Target	Kind	Organism	Pharmacological Action	Actions
Cipaglucosidase alfa	Cation-independent mannose-6-phosphate receptor	Protein	Humans	Yes	Ligand	Details