NICE has published final draft guidance which now recommends volanesorsen (Waylivra) for treating familial chylomicronaemia syndrome (FCS).
In draft guidance issued in January, NICE said, because of the concerns around the clinical evidence and high cost, volanesorsen was not considered appropriate use of NHS resources.
However, in the latest draft, NICE says, despite some uncertainties in the evidence, volanesorsen is likely to provide important clinical and psychological benefits for people with FCS. Now, with the improved price discount, volanesorsen can be considered a cost-effective use of NHS resources for highly specialised technologies.
Volanesorsen is licensed for adults with FCS at high risk of pancreatitis who have not responded to diet and triglyceride-lowering therapy. Given as a once-weekly (decreasing to once-fortnightly after three months) 285 mg injection, Volanesorsen is used alongside a very low-fat diet to lower triglycerides in the blood. The list price of volanesorsen is £11,394 per single-use 285 mg syringe.
The appeal period for this appraisal will close at 5 pm, Friday 2 October 2020.
Final guidance is expected to be available next month (October 2020).
The prevalence of FCS is estimated to be one to two per million people, equating to about 55 to 110 people in England. At the time of the evidence submission, there were thought to be around 80 to 100 people with FCS eligible for treatment with volanesorsen in the UK.
Treatment with volanesorsen has been provided since March 2018 under an Early Access to Medicines Scheme at several specialist centres. At the time of evaluation, 20 people were receiving volanesorsen, with treatment duration ranging from one to 15 months. A further five people have been identified to start treatment.
This article originally appeared on Univadis, part of the Medscape Professional Network.
