Products
Elosulfase alfa was approved in the EU and US in 2014 and in many countries in 2016 as a concentrate for the preparation of an infusion solution (Vimizim).
Structure and properties
Elosulfase alfa is the recombinant form of the human enzyme -acetylgalactosamine-6-sulfatase. It is produced by biotechnological methods.
Effects
Elosulfase alfa (ATC A16AB12) replaces the absent or reduced active enzyme -acetylgalactosamine-6-sulfatase. This lysosomal enzyme is involved in the metabolic degradation of the glycosaminoglycans keratan sulfate and chondroitin-6-sulfate.
Indications
For the treatment of IVA-type mucopolysaccharidosis (Morquio A syndrome, MPS IVA).
Dosage
According to the SmPC. The drug is usually administered as an intravenous infusion once a week.
Contraindications
- Hypersensitivity
For complete precautions, see the drug label.
Adverse effects
The most common possible adverse effects include headache, nausea, vomiting, indigestion, fever, chills, muscle pain, dyspnea, and abdominal pain. These are predominantly infusion-related reactions that occur after administration.
