NICE recommends Tryngolza (olezarsen) for familial chylomicronaemia syndrome – Sobi

Olezarsen can be used as an option to treat genetically confirmed familial chylomicronaemia syndrome in adults, only if response to diet and conventional triglyceride-lowering treatments (such as statins and fibrates) has been inadequate. Olezarsen can only be used if the company provides it according to the commercial arrangement.

Why these recommendations were made: Usual treatment for familial chylomicronaemia syndrome is a restricted low-fat diet, and may also include conventional treatments for lowering triglyceride levels such as fibrates and statins. When response has been inadequate, usual treatment is volanesorsen, an apolipoprotein C-III (APOC-3) triglyceride-lowering treatment. Olezarsen works in a similar way to volanesorsen, and would be offered to the same population. Clinical trial evidence shows that olezarsen is more effective than placebo. The clinical trial did not directly compare olezarsen with volanesorsen. Evidence from indirect comparisons suggests that the reduction in fasting triglyceride levels is similar for the 2 treatments. But these results are uncertain. So, it is unclear from the indirect comparisons alone whether olezarsen works the same as, better, or less well than volanesorsen. But because the 2 medicines work in a similar way, it is likely that their clinical effectiveness is similar. Clinical expert feedback supports this and indicates that olezarsen would be used as an alternative to volanesorsen at the same point in the care pathway. Evidence also indicates that olezarsen is likely to be better tolerated than volanesorsen. It also needs fewer injections and avoids the requirement for routine platelet monitoring. The costs for olezarsen are similar to or lower than volanesorsen.

So, it can be used.