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cipaglucosidase alfa
- Development stage
- Approved
- Lead developer
- Amicus Therapeutics
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Cipaglucosidase alfa is a recombinant human acid α-glucosidase enzyme replacement therapy used in combination with miglustat for the long-term treatment of adults with late-onset Pompe disease (lysosomal acid alpha-glucosidase [GAA] deficiency). It provides an exogenous source of GAA to break down accumulated glycogen in lysosomes, thereby reducing tissue damage and improving muscle function. Cipaglucosidase alfa is conjugated with mannose-6-phosphate N-glycans to enhance uptake via the cation-independent mannose-6-phosphate receptor (CI-MPR), facilitating delivery to lysosomes where it becomes active and hydrolyzes glycogen. Miglustat acts as an enzyme stabilizer, maintaining the activity of cipaglucosidase alfa in circulation[1][4][9].
- Brand names
- Pombiliti
- Other names
- recombinant human acid alpha-glucosidaserhGAA
02
Targets
GAA (Lysosomal acid alpha-glucosidase)IGF2R (Cation-independent mannose-6-phosphate receptor)
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