NICE issues draft guidance, finding negative opinion for volanesorsen

The UK National Institute for Health and Care Excellence (NICE) has announced draft guidance that it will not be recommending Waylivra (volanesorsen) for the treatment of familial chylomicronaemia syndrome (FCS).

Volanesorsen, produced by Akcea Therapeutics, was found to be beneficial in the short-term, but showed little benefit for the long-term.

NICE reports that there was also uncertainty as the main clinical trials used a different dose to the dosage described in the marketing authorisation for the drug.

Another doubt from the committee came from aspects of the company’s economic model, which were found to be highly uncertain. The cost-effectiveness estimates were much higher than what NICE considers acceptable for highly specialised technologies, leading to the decision that volanesorsen could not be recommended as an appropriate use of NHS resource.

The company is now able to provide further clarification for public consultation, which could determine the final decision on the drug. NICE has also recommended that Akcea Therapeutics carries out a further scenario analysis to explore the impact of the condition on health-related quality of life carers.

Volanesorsen is licensed for adults with FCS at high risk for pancreatitis who have not responded to diet and triglyceride-lowering therapy. The drug is administered as a once-weekly (decreasing to once-fortnightly after three months) 285mg injection. The list price of volanesorsen is £11,394 per single-use 285mg syringe.