NICE has issued final draft guidance recommending Tryngolza (olezarsen) for adults in England and Wales with genetically confirmed familial chylomicronaemia syndrome, but only when response to diet and conventional triglyceride‑lowering treatments has been inadequate.
FCS is an ultra‑rare inherited lipid disorder marked by severe hypertriglyceridaemia and a high risk of acute pancreatitis. The condition can lead to severe abdominal pain, hospitalisation, permanent organ dysfunction and death.
The recommendation is supported by data from the phase 3 Balance study, in which Tryngolza 80 mg delivered a statistically significant reduction in fasting triglyceride levels at 6 months, sustained through 12 months. The therapy also reduced acute pancreatitis events over 12 months. Sobi said the decision represents meaningful progress for people living with FCS.
Professor Handrean Soran, Consultant Physician and Endocrinologist at the Manchester University NHS Foundation Trust, said: “People living with FCS have extremely high triglyceride levels since childhood, which causes severe recurrent pain and unpredictable and potentially life-threatening episodes of acute pancreatitis. Historically we have had very limited treatments to manage this.”
He added: “Access to olezarsen through the NHS in England and Wales will represent an important step forward. It will provide adults living with FCS a once-monthly option that provides sustained reductions in triglyceride levels and risk of occurrence of pancreatitis and subsequent reduction in hospitalisations due to acute pancreatitis.”
Jill Prawer, Chair at Action FCS, explained: “We warmly welcome today’s decision from NICE. Due to the need for an extremely fat-restricted diet, living with FCS affects every area of life, and every meal eaten.”
She continued: “Alongside this comes the constant worry of a sudden and painful attack of pancreatitis that can lead to time in hospital – time away from family, education and work.”
Sharon Hall, General Manager at Sobi UK, said: “Today’s recommendation from NICE marks a significant moment for people living with FCS in England and Wales. It represents meaningful progress in addressing the significant burden of this ultra-rare condition and is an important milestone for the FCS community.”
She added: “Our focus now is on working with the NHS, clinical experts and the wider FCS community to support the effective implementation of this recommendation and ensure eligible patients can benefit from this new treatment option.”
