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.

It was also granted Promising Innovative Medicine status by the MHRA in October 2017, followed by positive Scientific Opinion and eligibility as an Early Access to Medicines Scheme (EAMS) treatment in March 2018, by the MHRA.

“We are disappointed with the draft guidance made by NICE today,” said Andy Caldwell, country manager, United Kingdom & Republic of Ireland, Akcea Therapeutics UK. “We will continue to work with NICE so this much-needed treatment can be provided to the patients that need it the most.

“As a patient-focused company, we are dedicated to supporting patients with FCS and those patients who are currently receiving volanesorsen through the Early Access to Medicines Scheme will continue to have access to the medication.”

FCS is an ultra-rare disease caused by impaired function of the enzyme lipoprotein lipase and characterised by severe hypertriglyceridemia. It affects between one to two people per million.