NICE recommendsfor Redemplo (plozasiran) for treating familial chylomicronaemia syndrome – Arrowhead

Plozasiran 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.

Plozasiran can only be used if the company provides it according to the commercial arrangement.

Why the committee made these recommendations: 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 to diet and conventional triglyceride-lowering therapies has been inadequate, usual treatment is volanesorsen, an apolipoprotein C-III (APOC-3) triglyceride-lowering treatment. Plozasiran works in a similar way to volanesorsen, and would be offered to the same population.

Clinical trial evidence shows that plozasiran is more effective than placebo. The clinical trial did not directly compare plozasiran 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 plozasiran works the same as, or 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 plozasiran would be used as an alternative to volanesorsen at the same point in the care pathway. Evidence also indicates that plozasiran is likely to be better tolerated than volanesorsen. It also needs fewer injections and avoids the requirement for routine platelet monitoring. The costs for plozasiran are similar to or lower than volanesorsen.

So, it can be used.