NICE issues final draft guidance recommending ▼ Tryngolza® (olezarsen) as an option to treat genetically confirmed familial chylomicronaemia syndrome (FCS) in adults in England and Wales

CAMBRIDGE, England--(BUSINESS WIRE)--FOR UK HEALTHCARE/TRADE MEDIA ONLY

Sobi^® UK today announced that the National Institute for Health and Care Excellence (NICE) has issued final draft guidance which recommends Tryngolza^® (olezarsen) as an option to treat genetically confirmed familial chylomicronaemia syndrome (FCS) in adults in England and Wales, only if response to diet and conventional triglyceride-lowering treatments (such as statins and fibrates) has been inadequate.

FCS is an ultra-rare inherited lipid disorder characterised by severe hypertriglyceridaemia and a markedly increased risk of acute pancreatitisⁱ - a potentially life-threatening medical emergency that can cause severe abdominal pain, hospitalisation, permanent organ dysfunction and death.^ii,iii

The recommendation is based on positive data from the Phase 3 Balance study, in which Tryngolza^® 80 mg demonstrated a statistically significant reduction in fasting triglyceride levels at 6 months that was sustained through 12 months. Additionally, olezarsen demonstrated a clinically meaningful reduction in acute pancreatitis events over 12 months.^iv

‘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.’

‘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.

‘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.’

Olezarsen is developed by Ionis Pharmaceuticals. Sobi and Ionis entered into a licence agreement under which Sobi has exclusive rights to commercialise Tryngolza in ex-U.S. geographies except Canada and China.

About the Balance study
BALANCE was a global, multicentre, randomised, double-blind, placebo-controlled Phase 3 study evaluating the efficacy and safety of olezarsen in patients with FCS over 12 months. The primary endpoint was the percent change from baseline in fasting triglyceride levels at 6 months compared with placebo. In patients receiving olezarsen 80 mg (n=22), fasting triglyceride levels decreased by 32% from baseline at 6 months, compared with a 12% increase with placebo (n=23), corresponding to a placebo-adjusted treatment difference of −43.5% (95% CI: −69.1 to −17.9; p<0.05). At 12 months, the placebo-adjusted treatment difference was −59.4% (95% CI: −90.7 to −28.1; nominal p<0.05). Secondary endpoints included percent changes in triglyceride levels at 12 months, percent changes in other lipid parameters at 6 and 12 months, and adjudicated acute pancreatitis event rates over the treatment period. In patients with FCS, the most commonly reported adverse reactions during treatment with olezarsen were injection site erythema (17%), headache (16%), arthralgia (15%), and vomiting (10%). Further details can be obtained from currently approved SMPC at: https://www.medicines.org.uk/emc/product/102176/smpc#gref

Following treatment and end-of-trial assessments, patients were eligible to enter an open-label extension study to continue receiving olezarsen once every 4 weeks.

About familial chylomicronaemia syndrome (FCS)
FCS is a rare, genetic disease characterised by extremely elevated triglyceride levels. In the UK, around 114 people are diagnosed with FCS; however, it is thought that the condition is under-diagnosed and that numbers may be higher.^v It is caused by impaired function of the enzyme lipoprotein lipase (LPL). Because of limited LPL production or function, people with FCS cannot effectively break down chylomicrons, lipoprotein particles that are 90% triglycerides.^vi People living with FCS are at high risk of acute pancreatitis and can experience recurrent episodes, alongside other health issues, including fatigue and severe, recurrent abdominal pain.^ii,iii People living with FCS are sometimes unable to work, adding to the burden of disease.^vii

About Tryngolza^® (olezarsen)
Tryngolza^® (olezarsen) is an RNA-targeted medicine designed to lower the body’s production of apolipoprotein C-III, a protein produced in the liver that is a key regulator of triglyceride metabolism.

About Sobi^® UK and Ireland
At Sobi, we’re committed to investing in medicines in areas of high unmet need, which make a real difference to the lives of people living with rare diseases across the UK and Ireland. With our early roots in Sweden, we started as pioneers in haemophilia. We have since grown into an innovative leader across immunology and speciality care, with a portfolio spanning chronic and acute conditions, genetic diseases, cardiometabolic and genetic metabolic disorders.

Sobi headquarters are located in Stockholm, Sweden. You can find more information about Sobi in the UK and the Republic of Ireland at www.sobi.com/uk/en.

ⁱ Moulin P, Dufour R, Averna M, et al. Identification and diagnosis of patients with familial chylomicronaemia syndrome (FCS): Expert panel recommendations and proposal of an “FCS score”. Atherosclerosis. 2018;275:265–272.
ⁱⁱ Saadatagah S, Larouche M, Naderian M, et al. Recognition and management of persistent chylomicronemia: A Joint Expert Clinical Consensus by the National Lipid Association and the American Society for Preventive Cardiology. J Clin Lipidol. 2025;19(4):723–736.
ⁱⁱⁱ Endocrine Society. What is Familial Chylomicronemia Syndrome (FCS). Endocrine Library.
^iv Stroes ESG, Alexander VJ, Karwatowska-Prokopczuk E, et al. Olezarsen, Acute Pancreatitis, and Familial Chylomicronemia Syndrome. N Engl J Med. 2024;390:1781–1792.
^v Bashir B, Forrester N, Downie, P, et al. Rare variant genetic landscape of familial chylomicronemia syndrome (FCS) in the United Kingdom. Genet Med Open. 2025;3:103445.
^vi Javed F, Hegele RA, Garg A, et al. Familial chylomicronemia syndrome: An expert clinical review from the National Lipid Association. J Clin Lipidol. 2025;19(3):382–403.
^vii Davidson M, Stevenson M, Hsieh A, et al. The burden of familial chylomicronemia syndrome: Results from the global IN-FOCUS study. J Clin Lipidol. 2018;12(4):898–907.e2.

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