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avalglucosidase alfa
- Development stage
- Approved
- Lead developer
- Sanofi
- Modality
- Replacement Enzymes → Therapeutic Enzymes → Recombinant Proteins and Enzymes
- Administration
- Intravenous
01
Overview
Avalglucosidase alfa is a next-generation recombinant human lysosomal acid alpha-glucosidase (GAA) enzyme replacement therapy designed to treat late-onset Pompe disease, a rare genetic disorder caused by GAA deficiency leading to glycogen accumulation in muscle and other tissues. The drug is engineered with enhanced mannose-6-phosphate (M6P) glycan content, which increases its binding affinity for the cation-independent mannose-6-phosphate receptor (CI-MPR), improving cellular uptake and muscle targeting compared to earlier therapies. Once internalized via CI-MPR-mediated endocytosis, avalglucosidase alfa is trafficked to lysosomes where it restores GAA activity and catalyzes the hydrolysis of accumulated glycogen into glucose, thereby reducing pathological storage and improving clinical outcomes in patients with late-onset Pompe disease[2][5][9].
- Brand names
- Nexviazyme
- Other names
- avalglucosidase alfa-ngpt
02
Targets
GAA (Lysosomal acid alpha-glucosidase)IGF2R (Cation-independent mannose-6-phosphate receptor)
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