1.1 Volanesorsen is recommended, within its marketing authorisation, as an option for treating familial chylomicronaemia syndrome in adults with genetically confirmed familial chylomicronaemia syndrome who are at high risk of pancreatitis, and when response to diet and triglyceride-lowering therapy has been inadequate. It is recommended only if the company provides volanesorsen according to the commercial arrangement.
Why the committee made these recommendations
Familial chylomicronaemia syndrome is a rare and potentially life-threatening condition that has a significant effect on the quality of life of people with the condition, and their families and carers. People with the condition have severe abdominal pain, unpredictable and recurrent acute pancreatitis and fatigue, and need to have a restricted low-fat diet. Current treatment options are limited.
Clinical trial evidence shows short-term benefits with volanesorsen, including reduced triglyceride (a type of fat found in the blood) levels. It is uncertain whether this is maintained in the longer term, particularly at the licensed dose, which was not used in clinical trials.
There are also other uncertainties, especially around volanesorsen's effect on acute pancreatitis and the utility values used in the economic model. Despite these, volanesorsen is likely to provide important clinical and psychological benefits for people with familial chylomicronaemia syndrome, and value for money within the context of a highly specialised service. It is therefore recommended for use in the NHS.