Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. announced that the Scottish Medicines Consortium (SMC)has enabled access to volanesorsen on the National Health Service (NHS) in Scotland for the treatment of familial chylomicronaemia syndrome (FCS). This decision will allow patients in Scotland with this rare and life-threatening condition to access the treatment through NHS Scotland. SMC’s acceptance follows recent approval from the National Institute for Health and Care Excellence (NICE) for the use of volanesorsen on the NHS in England. Volanesorsen was evaluated by the SMC under the ultra-orphan medicines pathway, a new system for the assessment of medicines for extremely rare diseases, which was introduced at the start of this year. This means that eligible patients will be able to access volanesorsen through NHS Scotland while additional clinical effectiveness data are gathered. The SMC will review this data after three years and make a final decision on its routine use in NHS Scotland. FCS is an under-recognised condition characterised by extremely high triglyceride levels (10 to 100 times normal values) and abnormal accumulation of lipoprotein particles called chylomicrons in the blood. 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. 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. FCS is also associated with a breadth of cognitive impairments, significant emotional burden, and poor mental health. There is no cure, and the condition is currently managed by adopting a very strict low-fat diet of 10 to 20g of fat per day. This is incredibly challenging and does not remove the risk of pancreatitis and other symptoms of the condition. Volanesorsen will be the second treatment that Akcea Therapeutics has made available on the NHS in Scotland in the past 18 months. Volanesorsen, 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. Volanesorsen, which received conditional marketing authorisation in Europe in May 2019, is the only therapy indicated for people with familial chylomicronaemia syndrome (FCS). It was recommended for use in the treatment of FCS in routine care on the NHS by the National Institute for Health and Care Excellence (NICE) in England in September 2020. Volanesorsen is a self-administered, subcutaneous injection in a single-use, prefilled syringe. Volanesorsen, 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. The European Commission’s marketing authorisation of volanesorsen was based on results from the Phase 3 APPROACH study10 and the ongoing APPROACH Open Label Extension (OLE) study, and is supported by results from the Phase 3 COMPASS study. Results from the Phase 3 APPROACH trial, show that in comparison to placebo, following three months of treatment, volanesorsen reduced triglycerides by 94%. All patients in the trial maintained a low-fat diet. Volanesorsen is associated with risk of thrombocytopenia. Enhanced monitoring is required to support early detection and management of thrombocytopenia. The most frequently observed adverse reactions (more than 10%) during treatment with volanesorsen were events associated with injection site reactions and reduction in platelet levels/thrombocytopenia. FCS is an under-recognised condition caused by impaired function of the enzyme lipoprotein lipase, or LPL, and characterised by severe hypertriglyceridaemia (>880mg/dL or 10mmol/L) and a risk of potentially fatal acute pancreatitis. Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides. FCS, which is estimated to affect 3,000 to 5,000 patients worldwide, can lead to many health issues including chronic pain, fatigue, a milky appearance of retinal veins and arteries, neurological symptoms such as depression and memory loss, swelling of the liver and spleen, and fatty deposits in the skin.