Agalsidase beta · Fabrazyme

What is Agalsidase beta?

Agalsidase beta is a recombinant human alpha-galactosidase A enzyme that provides an exogenous source of the deficient enzyme in Fabry disease. It is internalized into lysosomes where it reduces accumulated globotriaosylceramide (GL-3), the pathogenic substrate that accumulates due to enzyme deficiency.

Summary written with AI from the sources listed at the end of this page.

At a glance

Also known as

Fabrazyme

Drug type

Recombinant protein

Mechanism

Protein replacement

Pathways

lysosomal glycosphingolipid catabolism

Therapy area

Cardiovascular, Neurology

Developer

Genzyme

In trials of

IQV

FDA status

Approved as Fabrazyme (April 24, 2003), BLA103979 · FDA label

UNII

RZD65TSM9U

✅ FDA-approved uses

• Fabry disease

From the indications section of the FDA label (version of Aug 22, 2025). Read the full label for the exact wording, limitations and safety information.

🧪 Clinical trials

Trials of the companies BioRadar tracks, from ClinicalTrials.gov, most advanced phase first.

Conditions studied

Fabry Disease

Sources

• FDA Purple Book
• FDA label (DailyMed)
• FDA label Fabrazyme (2025-08-22), indications
• ClinicalTrials.gov NCT05843916

Updated Oct 3, 2026.