in the absence of a submission for reassessment through the ultra-orphan framework from the holder of the marketing authorisation
volanesorsen (Waylivra®) is not recommended for use within NHSScotland.
Indication under review: as an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.
The holder of the marketing authorisation has not made a submission to SMC regarding this product in this indication. As a result, we cannot recommend its use within NHSScotland.
Download detailed advice235KB (PDF)
Medicine details
- Medicine name:
- volanesorsen (Waylivra)
- SMC ID:
- SMC2716
- Indication:
As an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.
- Pharmaceutical company
- Akcea Therapeutics UK Ltd
- BNF chapter
- Nutrition and blood
- Submission type
- Non submission
- Status
- Not recommended
- Date advice published
- 09 September 2024
From 16 November 2020, volanesorsen (Waylivra) could be prescribed within the ultra-orphan pathway while further evidence on its effectiveness was generated. The company were expected to make a re-assessment submission to allow a decision on its routine use in NHSScotland. The company have not made a submission for re-assessment to SMC therefore not recommended advice has been issued.