Products
Agalsidase is commercially available as an infusion preparation and has been approved in many countries since 2001 and 2003, respectively:
- Replagal: agalsidase alfa
- Fabrazyme: agalsidase beta
Structure and properties
Agalsidase is a recombinant human α-galactosidase A produced by biotechnological methods. The amino acid sequence is identical to the natural lysosomal enzyme. It is a homodimer with a molecular weight of about 100 kDa. One subunit consists of 398 amino acids.
Effects
Agalsidase (ATC A16AB03, ATC A16AB04) replaces the missing enzyme α-galactosidase A in the organism and catalyzes the hydrolysis of globotriaosylceramide (GL-3) and other glycosphingolipids.
Indications
For enzyme replacement therapy in patients with Fabry disease (α-galactosidase A deficiency).
Dosage
According to the SmPC. The drugs are administered as an intravenous infusion.
Contraindications
The drugs are contraindicated in case of hypersensitivity. For complete precautions, see the drug label.
Interactions
Combination with chloroquine, amiodarone, benoquine, or gentamicin is not indicated because these agents may inhibit α-galactosidase activity.
Adverse effects
The most common potential adverse effects include:
- Reactions associated with infusion
- Redness
- Nausea
- Muscle rigidity
- Fever
- Pain
- Fatigue
- Headache
