New guidance from NICE recommends volanesorsen for adults with genetically-confirmed familial chylomicronaemia syndrome (FCS) who are at high risk of pancreatitis, and when response to diet and triglyceride-lowering therapy has been inadequate.
The clinical evidence reviewed included:
- APPROACH (n=66) - a double-blind randomised placebo-controlled trial assessing the efficacy and safety of volanesorsen (n=33) compared with placebo (n=33). Volanesorsen was administered by subcutaneous injection (285 mg) once weekly. After the 52‑week trial period, patients could either have a 13‑week follow up or enter the APPROACH OLE open-label extension study.
- COMPASS (n=114) - a multicentre double-blind randomised placebo-controlled trial in patients with hypertriglyceridaemia from many different causes; only seven patients had FCS. Volanesorsen was administered by subcutaneous injection (285 mg) once weekly. After 26 weeks, patients could either have a 13‑week follow-up or enter the APPROACH OLE open-label extension study.
- APPROACH OLE - an ongoing single-arm open-label study assessing the safety and efficacy of dosing and extended dosing with volanesorsen administered by subcutaneous injection (285 mg) once weekly.
- The Early Access to Medicines Scheme (EAMS; n=20) that provides access to volanesorsen subcutaneously at a dose of 285 mg once every 2 weeks for people with FCS, including those who have previously had treatment in APPROACH and APPROACH OLE.
NICE concluded that the clinical trial evidence showed short-term benefits with volanesorsen, including reduced triglyceride levels, but said 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 uncertainties around volanesorsen's effect on acute pancreatitis and the utility values used in the economic model.
Despite these, NICE has concluded that 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, and has therefore recommended its use in the NHS.
