In draft guidance, the U.K.’s drug pricing watchdog the National Institute for Health and Care Excellence (NICE) said it does not recommend Akcea Pharmaceuticals’ Waylivra (volanesorsen) in the National Health Service in England for treating familial chylomecronamia syndrome because it was not cost effective.
NICE considered the drug for use in adults with genetically confirmed disease who are at high risk of pancreatitis and who do not respond to diet and triglyceride-lowering therapies.
Familial chylomecronamia syndrome (FCS) is an ultra-rare, inherited, and potentially life-threatening condition in which patients cannot metabolize triglyceride-rich lipid particles called chylomicrons due to a deficiency in the enzyme lipoprotein lipase. As a result, people with FCS have high levels of triglycerides in the blood, which leads to a range of symptoms including permanent organ damage and potentially fatal attacks of acute pancreatitis.
“NICE’s recommendation is a real blow for people living with FCS,” said Jill Prawer, founder and chair of the LPLD Alliance. “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.”
Although volanesorsen is the only authorized treatment for FCS, the recommendation from NICE states that it has concerns around the clinical evidence and high cost, and therefore did not consider it an appropriate use of NHS resources. NICE cited three reasons for its decision: although clinical trial evidence shows short term benefits of treatment, it’s uncertain the benefits are maintained in the long term; Akcea’s licensed dosage is not what was used in the clinical trials; and Waylivra’s cost-effectiveness falls short of what NICE considers acceptable for highly specialized drugs. The decision affects about 55 to 110 people in England.
NICE has asked Akcea to explain its data and clarify its pricing model. NICE did say that patients taking Waylivra through Akcea’s early access program can continue to receive treatment.
“We are disappointed with the draft guidance made by NICE today,” said Andy Caldwell, country manager, United Kingdom & Republic of Ireland, Akcea Therapeutics UK.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.”
In 2019, Akcea won European Commission approval for Waylivra for the treatment of FCS in adults. This came after the U.S. Food and Drug Administration rejected approving Waylivra for the treatment of FCS in August 2018, even after and advisory committee voted 12 to 8 to recommend its approval. Akcea is working with the FDA to resubmit the drug for marketing approval in the United States.
Photo: Jill Prawer, founder and chair of the LPLD Alliance