NICE rules against the use of Akcea’s Waylivra in the NHS

pharmafile | January 3, 2020 | News story | Medical Communications |  Akcea, European Commission, FCS, NHS, NICE 

NICE has not recommended Akcea’s drug Waylivra (volanesorsen) to be used for the treatment of Familial Chylomicronaemia Syndrome (FCS) in the National Health Service (NHS) in England.

The drug got its European license last May, and NICE did acknowledge that the trial evidence did demonstrate the drug showed some benefit in the short term.

However NICE’s Evaluation Consultation Document’s raised concerns around the clinical evidence and the high cost of the drug. The fact that the main clinical trials used to approve the drug had a different dose to that described in its EU licence has added to NICE’s uncertainties.

Andy Caldwell, Country Manager at Akcea Therapeutics UK, said: “We are disappointed with the draft guidance made by NICE today. We will continue to work with NICE so this much-needed treatment can be provided to the patients that need it the most”.

Jill Prawer, of the Lipoprotein Lipase Deficiency (LPLD) Alliance, said: “NICE’s recommendation is a real blow for people living with FCS. There are currently no treatments available for FCS. Volanesorsen is the only therapeutic option proven to change the lives of people affected by the condition.”

Waylivra is an antisense oligonucleotide drug designed by Ionis, and co-developed by Akcea, to reduce the production a protein that regulates plasma triglycerides.

FCS is an ultra-rare life-altering genetic disease that affects between 1 to 2 people per million. It is caused by impaired function of the enzyme lipoprotein lipase, which causes elevations in certain types of fat in the blood. It can lead to significant health risks, including severe abdominal pain and potentially fatal attacks of acute pancreatitis, as well as chronic complications such as permanent organ damage and diabetes.

Conor Kavanagh