WAYLIVRA▼® (volanesorsen) for FCS to be available on the NHS in England

Image of a contract and pen to show WAYLIVRA ▼® (volanesorsen) for FCS to be available on the NHS in England

The National Institute for Health and Care Excellence (NICE) has issued a positive recommendation for WAYLIVRA▼® (volanesorsen), the first and only therapy for adults with FCS, an ultra-rare and life-threatening condition.

Akcea Therapeutics UK Ltd announced that NICE has issued a positive Final Evaluation Document for volanesorsen for the treatment of Familial Chylomicronaemia Syndrome (FCS) for routine care on the NHS in England. FCS is an under-recognised condition characterised by extremely high triglycerides levels (10 to 100 times normal values) and abnormal accumulation of lipoprotein particles called chylomicrons in the blood.[i],[ii] FCS can put the patient at an increased risk of recurrent episodes of potentially fatal acute pancreatitis as well as chronic abdominal pain and long-term morbidity.[iii] Volanesorsen is the first and only treatment for FCS currently available to patients in the UK.

There are only one to two cases of FCS for every one million people, making the condition incredibly rare.[iii] FCS is associated with a breadth of cognitive impairments, significant emotional burden, and poor mental health.[ii],[iv] There is no cure, and the condition is currently managed through adopting a very strict, extremely low-fat diet of 10-20g of fat per day.2 This is incredibly challenging and doesn’t remove the risk of pancreatitis and the other symptoms of the condition.[v] The severe physical implications of FCS and its long-term complications have a significant burden on the day-to-day life of those affected and their families from an emotional, social and professional standpoint.[iv]

Akcea Therapeutics initially made volanesorsen 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 volanesorsen, at no cost to the NHS. Akcea Therapeutics has also implemented their complimentary patient support programme, Akcea Connect, which allows patients initiated on volanesorsen to continue to access treatment, without having to travel to a hospital, to ensure that people living with this condition are supported.

WAYLIVRA▼® (volanesorsen) was granted a conditional marketing authorisation in May 2019 by the European Commission.[vi]

“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.”


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[i] Stroes E, Moulin P, Parhofer KG, Rebours V, Löhr J-M, Averna M. Diagnostic algorithm for familial chylomicronemia syndrome. Atheroscler Suppl. 2017;23:1-7. doi:10.1016/j.atherosclerosissup.2016.10.002.

[ii] Williams L, Rhodes KS, Karmally W, Welstead LA, Alexander L, Sutton L; for patients and families living with FCS. Familial chylomicronemia syndrome: bringing to life dietary recommendations throughout the life span. J Clin Lipidol. 2018;12(4):908-919. doi:10.1016/j.jacl.2018.04.010.

[iii] Heart UK. FCS. Available at: https://www.heartuk.org.uk/genetic-conditions/familial-chylomicronaemia-symdrome-fcs Last accessed June 2020.

[iv] Davidson M, et al. (2017) The burden of familial chylomicronemia syndrome: interim results from the IN-FOCUS study. Expert Review of Cardiovascular Therapy; 15(5):415-423.

[v] Heart UK. Eating for FCS. Available at: https://www.heartuk.org.uk/eating-for-fcs-/eating-for-fcs- Last accessed June 2020.

[vi] European Medicines Agency. Waylivra Summary of Product Characteristics. Available at: www.ema.europa.eu/en. Last accessed February 2020.