Sobi UK announced that the National Institute for Health and Care Excellence (NICE) has issued final draft guidance recommending Tryngolza (olezarsen) as an option for adults in England and Wales with genetically confirmed Familial Chylomicronaemia Syndrome (FCS), when response to diet and conventional triglyceride-lowering treatments, including statins and fibrates, has been inadequate.

FCS is an ultra-rare inherited lipid disorder characterised by extremely high triglyceride levels and an increased risk of acute pancreatitis. The condition can cause severe abdominal pain, recurrent hospitalisations and potentially life-threatening complications.

The recommendation is based on data from the Phase 3 BALANCE study, a global, randomised, double-blind, placebo-controlled trial evaluating the efficacy and safety of olezarsen in patients with FCS over 12 months.

In the study, patients receiving olezarsen 80 mg experienced a 32 percent reduction in fasting triglyceride levels from baseline at six months, compared with a 12 percent increase among patients receiving placebo. This represented a placebo-adjusted treatment difference of -43.5 percent. At 12 months, the placebo-adjusted treatment difference was -59 percent. 

Olezarsen also demonstrated a reduction in adjudicated acute pancreatitis events over the 12-month treatment period. Following completion of treatment and end-of-trial assessments, eligible patients could enter an open-label extension study and continue receiving olezarsen once every four weeks.

The most commonly reported adverse reactions in patients with FCS receiving olezarsen were injection-site erythema, headache, arthralgia and vomiting.

FCS is caused by impaired function of Lipoprotein Lipase (LPL), an enzyme involved in breaking down chylomicrons, which are lipoprotein particles containing approximately 90 percent triglycerides. The resulting accumulation of triglycerides can contribute to recurrent acute pancreatitis, severe abdominal pain and fatigue.

In the UK, around 114 people are diagnosed with FCS, although the condition is considered underdiagnosed. Its impact can extend to daily activities, education and employment.

Tryngolza is an RNA-targeted medicine designed to reduce the production of apolipoprotein C-III, a liver-produced protein that regulates triglyceride metabolism. Olezarsen was developed by Ionis Pharmaceuticals. Sobi and Ionis have an agreement under which Sobi holds exclusive commercialisation rights for Tryngolza in territories outside the US, excluding Canada and China.

NICE's final draft recommendation applies to eligible adults in England and Wales. The guidance is subject to the applicable NICE process before final guidance is issued.