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How it works

It is an antisense oligonucleotide that binds apolipoprotein C-III messenger RNA, reducing its translation; lowering apoC-III decreases plasma triglycerides through clearance pathways that are partly independent of lipoprotein lipase.

Prescribing in practice

• It commonly causes thrombocytopenia, which can be severe, so platelet counts must be monitored regularly before and throughout treatment, with treatment adjusted or stopped according to the count.
• Injection-site reactions are very common, and bleeding events can occur in association with low platelets.
• Renal function and the development of antibodies should be considered, and it is used under specialist lipid supervision in a confirmed diagnosis.

Monitoring

Monitor platelet counts regularly, together with serum triglycerides, liver and renal function, and observe for injection-site reactions and any signs of bleeding.

Counselling the patient

• Regular blood tests are essential to check your platelet count during treatment.
• Report any unusual bruising, bleeding, or a rash of small red or purple spots straight away.
• Continue your prescribed low-fat diet, as this remains central to managing the condition.

Evidence & guidelines

Triglyceride-lowering efficacy in familial chylomicronaemia syndrome was shown in a randomised placebo-controlled trial supporting its approval for this rare condition.