Sebelipase alfa

How it works

It replaces deficient lysosomal acid lipase, hydrolysing accumulated cholesteryl esters and triglycerides within lysosomes.

Prescribing in practice

• Hypersensitivity and infusion-associated reactions, including anaphylaxis, can occur; administer under supervision with resuscitation facilities available.
• It is given by intravenous infusion at regular intervals; refer to the SPC for administration details and the approach in rapidly progressive infantile disease.
• Anti-drug antibodies may develop and can influence response and tolerability.

Monitoring

Patients are monitored during and after infusions for hypersensitivity, with review of lipid profile, hepatic parameters and antibody status as indicated.

Counselling the patient

• This treatment is given as a regular drip and is a long-term therapy.
• Report any rash, breathing difficulty, flushing or feeling unwell during or after an infusion.
• Attend scheduled appointments for ongoing monitoring.

Evidence & guidelines

Approval was supported by clinical trials in infants, children and adults with lysosomal acid lipase deficiency.