UK – NICE refuses Akcea’s Waylivra

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).

The document states that the drug isn’t recommended for use on the NHS, despite being “innovative and the only authorised treatment for FCS”.

The draft negative guidance goes on to 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 drug in question is an antisense oligonucleotide drug – designed by Ionis and co-developed by Akcea and Ionis to reduce the production a protein that regulates plasma triglycerides.

Waylivra was initially granted a marketing authorisation in May 2019 by the European Commission, based on results from the Phase III APPROACH study and the ongoing APPROACH Open Label Extension (OLE) study…