Ultra Orphan Assessment

The Scottish Medicines Consortium (SMC) has completed its initial assessment of the evidence for the above product using the ultra orphan framework

volanesorsen sodium (Waylivra)

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.

Key points:

  • FCS is a rare disorder of lipid metabolism that has a major impact on quality of life. Patients are at high risk of pancreatitis, a potentially life-threatening condition.
  • Clinical study evidence shows that volanesorsen significantly reduces triglyceride levels compared with placebo from baseline to 3 months. Furthermore, a significantly greater proportion of volanesorsen-treated patients achieved the target triglyceride level of <8.5mmol/L compared with placebo. There is a currently a lack of information on the impact on direct health outcomes such as pancreatitis.
  • Treatment with volanesorsen is associated with reduced platelet counts and a strict dose algorithm and intense platelet monitoring must be adhered to in order to minimise this risk. Long-term efficacy and safety data are limited for this lifelong condition.
  • Volanesorsen may be associated with positive improvements in quality of life affecting personal, social and professional life.
  • The economic analysis suggested an incremental cost per quality-adjusted life-year (QALY) gained estimate for volanesoren of £76,571 versus standard of care, after taking account of a patient access scheme (PAS). The cost-effectiveness is uncertain primarily due to uncertainties in the impact volanesorsen has on reducing acute pancreatitis events and the incidence of chronic pancreatitis and diabetes over the lifetime horizon, and there are uncertainties with the health related quality of life and QALYs gained.
  • Despite a PAS that improves the cost-effectiveness of volanesorsen, the treatment’s cost in relation to its health benefits remains high.

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From 16 November 2020, volanesorsen (Waylivra) can be prescribed within the ultra-orphan pathway while further evidence on its effectiveness is generated.  After 3 years the company will provide an updated submission for reassessment to allow a decision on its routine use in NHSScotland.

Medicine details

Medicine name:
volanesorsen sodium (Waylivra)
SMC ID:
SMC2299
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
Ultra-orphan initial assessment
Date Published
09 November 2020
Additional notes

A new approach for assessing medicines for extremely rare conditions started in October 2018. Further information is available on our ultra-orphan webpage and in the Scottish Government’s guidance.