NICE has issued final draft guidance recommending Tryngolza (olezarsen) as an option for adults in England and Wales with genetically confirmed familial chylomicronaemia syndrome (FCS), according to Sobi UK.

 

In people with FCS, response to diet and conventional triglyceride-lowering treatments, such as statins and fibrates, has been inadequate.

 

The recommendation could mark a significant step forward for people living with FCS, an ultra-rare inherited lipid disorder characterised by severe hypertriglyceridaemia and a markedly increased risk of acute pancreatitis — a potentially life-threatening medical emergency that can cause severe abdominal pain, hospitalisation, permanent organ dysfunction and death.

 

NICE’s recommendation is supported by positive results from the Phase 3 Balance study. Tryngolza 80 mg delivered a statistically significant reduction in fasting triglyceride levels at six months, with the reduction sustained through 12 months. Olezarsen also demonstrated a clinically meaningful reduction in acute pancreatitis events over 12 months.

 

'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," said Professor Handrean Soran, Consultant Physician and Endocrinologist at the Manchester University NHS Foundation Trust. 

 

"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."

 

For patients, the disease extends far beyond its clinical symptoms, with the need for an extremely fat-restricted diet placing significant restrictions on everyday life and a persistent fear of acute pancreatitis.

 

"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. 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," said Jill Prawer, Chair at Action FCS.

 

Sharon Hall, General Manager, Sobi UK, described the recommendation as an important milestone for the FCS community.

 

"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," said Sharon Hall, General Manager, Sobi UK. 

 

"At Sobi, we are committed to advancing understanding of lipid disorders and improving outcomes for people living with FCS. 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."