NICE recommends Tryngolza for ultra-rare lipid disorder FCS

The UK's National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Tryngolza (olezarsen), from Swedish Orphan Biovitrum (STO: SOBI), the Swedish rare disease specialist, to treat genetically confirmed familial chylomicronaemia syndrome (FCS) in adults in England and Wales whose response to diet and conventional triglyceride-lowering treatments, such as statins and fibrates, has been inadequate.

FCS is an ultra-rare inherited lipid disorder causing severe hypertriglyceridaemia and a markedly increased risk of acute pancreatitis, a potentially life-threatening complication. Around 114 people are diagnosed with FCS in the UK, though the condition is thought to be under-diagnosed.