Akcea Therapeutics UK’s Waylivra (volanesorsen), the first and only therapy for Familial Chylomicronaemia Syndrome (FCS), will be funded on the NHS after all, following a u-turn by the National Institute for Health and Care Excellence (NICE).
FCS is the name for a group of rare genetic disorders that affect between 55 and 110 people in England. The condition results in very high levels of triglycerides in the blood, causing symptoms such as repetitive episodes of severe abdominal pain, unpredictable and recurrent episodes of acute pancreatitis, enlargement of the liver and spleen and fatigue.
Waylivra, a product of Ionis’ proprietary antisense technology, is designed to reduce the production of ApoC-III, a protein that regulates plasma triglycerides and may also affect other metabolic parameters.
NICE initially rejected routine NHS funding earlier this year. The new positive recommendation for Waylivra follows an improved discount to its price offered by the company and using the committee’s preferred assumptions in the economic model, it said.
“Despite some uncertainties in the evidence the committee considered that volanesorsen is likely to provide important clinical and psychological benefits for people with FCS. With the improved discount to its price volanesorsen can be considered a cost-effective use of NHS resources for highly specialised technologies,” the Institute noted.
“We are delighted that NICE has recognised the value of volanesorsen and has given a positive recommendation for its use on the NHS,” said Andy Caldwell, country manager, UK & Ireland, Akcea Therapeutics UK.
“This is Akcea’s second treatment to receive a positive recommendation from NICE and builds on our vision to bring treatment to patients with rare conditions as soon as possible. This positive decision will transform the lives of patients, who, until now, have had no other treatment options available to them. This news is a real step change for patients with FCS.”
“This is a landmark day for people with FCS as they have finally had their voices heard,” said Jill Prawer of Action FCS (formerly LPLD Alliance). “Patients often present in A&E with symptoms such as sudden intense abdominal pain, pancreatitis and fat-filled spots, which can be wrongly attributed to poor diet or even alcohol misuse. We are delighted that NICE has acknowledged the unique unmet needs of FCS patients. This important milestone will allow patients with this severely debilitating condition to access treatment on the NHS, drastically improving their quality of life.”
Akcea Therapeutics initially made Waylivra available to patients with FCS through the Early Access to Medicines Scheme (EAMS). Since initiation of the EAMS, 22 patients with FCS have been treated with the drug, at no cost to the NHS.
The firm has also implemented their complimentary patient support programme, Akcea Connect, which allows patients initiated on Waylivra to continue to access treatment, without having to travel to a hospital, to ensure that people living with this condition are supported.