Agalsidase alfa is a recombinant human alpha-galactosidase indicated to treat Fabry disease, a genetic deficiency in the enzyme leading to buildup of globotriaosylceramide. While patients generally do not experience a clinically significant difference in outcomes between the two drugs, some patients may experience greater benefit with agalsidase beta.
- Mechanism
- Curator reviewed · 3 references
α-galactosidase A is uptaken by cells via the mannose 6 phosphate receptor. Agalsidase alfa hydrolyzes globotriaosylceramide and other glycosphingolipids that would normally be hydrolyzed by endogenous α-galactosidase A. Preventing the accumulation of glycosphingolipids prevents or reduces the severity of manifestations of Fabry disease such as renal failure, cardiomyopathy, or cerebrovascular events.
- Primary indication
- Agalsidase alfa is indicated in the treatment of Fabry disease.Curator reviewed · 2 structured indications
- Formula / weight
- C2029H3080N544O587S27 · 45351.6 Da
- First approval
- Canada, 2021 · European Union, 2016
- Also known as
- Agalsidase alfa (genetical recombination) · Agalsidase alpha · alpha-D-galactopyranosidase · alpha-D-galactosidase · alpha-D-galactosidase enzyme
- Code names
- DRX-005B · EC 3.2.1.22
- Brand names
Resolves to
What you can answer from here — as of March 06, 2025
Which drugs share a target with Agalsidase alfa, and which of those have an active Phase 3 trial?
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