Velaglucerase alfa is an enzyme replacement therapy used for the long-term treatment of for pediatric and adult patients with type 1 Gaucher disease. A medication used to treat a rare genetic disorder called Gaucher disease, which is caused by the deficiency of a specific enzyme in the body.
- Mechanism
- Curator reviewed
Velaglucerase alfa catalyzes the hydrolysis of glucocerebroside, reducing the amount of accumulated glucocerebroside.
- Primary indication
- Velaglucerase alfa is a hydrolytic lysosomal glucocerebroside-specific enzyme indicated for long-term enzyme replacement therapy for pediatric and adult patients with type 1 Gaucher disease.Curator reviewed · 1 structured indication
- Formula / weight
- C2532H3850N672O711S16 · 63000.0 Da (approximate)
- First approval
- Canada, 2020 · United States, 2010 · European Union, 2016
- Also known as
- GA-GCB
- Brand names
Resolves to
Clinical / RWD
Targets
What you can answer from here — as of March 06, 2025
1Protein targetEach mapped to UniProt, with action and pharmacological actionListed above
Which drugs share a target with Velaglucerase alfa, and which of those have an active Phase 3 trial?
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