Products
Sebelipase alfa was approved in the EU and US in 2015 and in many countries in 2016 as a concentrate for the preparation of an infusion solution (Kanuma).
Structure and properties
Sebelipase alfa is a recombinant human lysosomal acid lipase (rhLAL) with the same amino acid sequence as the endogenous enzyme. The protein is produced by biotechnological methods.
Effects
Sebelipase alfa (ATC A16AB14) replaces the missing or insufficiently active enzyme lysosomal acid lipase in the body. It is an enzyme replacement therapy. Sebelipase alfa is taken up into lysosomes and reduces lipid accumulation by hydrolyzing cholesterol esters and triglycerides to fatty acids, cholesterol and glycerol. The therapy lowers liver lipid content and has a lipid-lowering effect.
Indications
For long-term enzyme replacement therapy in patients with lysosomal acid lipase (LAL) deficiency.
Dosage
According to the SmPC. The drug is administered as an intravenous infusion.
Contraindications
- Life-threatening hypersensitivity reactions.
For complete precautions, see the drug label.
Interactions
No information is available.
Adverse effects
The most serious possible adverse effect is anaphylaxis.