The therapy is given as a monthly subcutaneous injection and gained approval through the International Recognition Procedure, expediting UK access by leveraging trusted international regulatory decisions.

MHRA approved olezarsen (Tryngolza) for adults with familial chylomicronaemia syndrome (FCS), a rare fat-metabolism disorder that can trigger life-threatening pancreatitis.

In a phase 3 NEJM trial, olezarsen reduced triglycerides by an average of 32% at six months versus a 12% rise with placebo, with benefits sustained or enhanced at one year and fewer acute pancreatitis events.

Olezarsen is an antisense oligonucleotide that lowers hepatic apoC-III production, enabling triglyceride breakdown by lipoprotein lipase.

MHRA official Julian Beach described the approval as providing a new treatment option for adults with FCS.

FCS is an exceedingly rare autosomal recessive disorder, affecting roughly 1–2 individuals per million, often caused by LPL mutations, with symptoms including hypertriglyceridaemia, recurrent pancreatitis, joint pain, eruptive xanthomata, steatorrhoea, and lipaemia retinalis.