The National Institute for Health and Care Excellence refuses recommendation for Waylivra to treat Familial Chylomicronaemia Syndrome.- Akcea Therapeutics

The National Institute for Health and Care Excellence (NICE) has issued an Evaluation Consultation Document (ECD), refusing Waylivra (volanesorsen) as a treatment for Familial Chylomicronaemia Syndrome (FCS) from Akcea Therapeutics. NICE states that the drug is not recommended for use on the NHS, despite being “innovative and the only authorised treatment for FCS”. The draft negative guidance states that NICE has “concerns around the clinical evidence and high cost, and therefore did not consider it an appropriate use of NHS resources.” The main clinical trials used to approve the drug used a different dose to that described in its EU licence has added to NICE’s negative opinion. NICE has recommended that Akcea provides further clarification and analyses to capture the health related quality of life of those affected. It also wants an analysis exploring the impact of FCS on the health-related quality of life of carers.