Akcea’s rare inherited disorder drug Waylivra has been handed a resounding no by the UK’s cost-effectiveness watchdog NICE, on the basis that it is far too costly for the NHS.
Waylivra (volanesorsen) is an antisense drugs, used for the treatment of a rare inherited disorder called familial chylomicronemia syndrome (FCS).
FCS is a rare genetic disease that prevents the body from breaking down fats, and results in extremely high levels of triglycerides in the blood.
This causes a range of symptoms in sufferers, including severe abdominal pain, fatal attacks of acute pancreatitis, hepatosplenomegaly, diabetes and a lack of concentration.
Akcea’s drug was handed conditional approval by the European Medicines Agency (EMA) last May, making Europe its first market.
It is licensed for use in adults with FCS at high-risk of pancreatitis who have not responded to diet and triglyceride lowering therapy.
The US FDA turned down its marketing application for Waylivra in August 2018, despite a 12-8 vote in favour of approval from its own advisory committee.
The FDA had concerns about the drug’s safety, particularly fears surrounding some reductions in blood platelet counts among patients treated with the antisense agent.
Although NICE did not raise any concerns about Waylivra’s safety, it did take issue with it long-term clinical benefit, as well as its steep price.
Despite acknowledging that clinical trials of the drug demonstrated some benefit in the short-term, NICE said there is “a lack of evidence to show whether this benefit is maintained in the longer term”.
The cost-effectiveness of the drug were also deemed as “much higher” than what NICE would consider appropriate for such a highly specialised treatment, which only has a small patient population in England.
The list price of Waylivra is £11,394 per single-use 285mg syringe, and would be able to help around 55 to 110 people in England who are effected by FCS disorders.
The only other treatment option for these patients is a severely restricted low fat diet of ten to 20 grams of fat a day which often fails to remove the threat of pancreatitis.
“We will continue to work with NICE so this much-needed treatment can be provided to the patients that need it the most,” said Andy Caldwell, country manager, United Kingdom & Republic of Ireland.
“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,” he added.
Waylivra was Akcea’s second drug to reach the market last year after transthyretin-related amyloidosis (ATTR) therapy Tegsedi (inotersen).