WAYLIVRA▼® (volanesorsen), the First and Only Therapy for FCS, an Ultra-Rare and Life-Threatening Condition, to Be Available on the NHS in England

About this update from Ionis Pharmaceuticals, Inc.

[{"type":"text","content":"\nNational Institute for Health and Care Excellence (NICE) issues a positive recommendation for volanesorsen to treat adults with Familial Chylomicronaemia Syndrome (FCS)\n\n LONDON--(BUSINESS WIRE)--\n\nAkcea Therapeutics UK Ltd., a subsidiary of Akcea Therapeutics Inc. (NASDAQ: AKCA), announced today that the National Institute for Health and Care Excellence (NICE) has issued a positive Final Evaluation Document (FED) for volanesorsen for the treatment of Familial Chylomicronaemia Syndrome (FCS) for routine care on the National Health Service (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.1,2 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.3 Volanesorsen is the first and only treatment for FCS currently available to patients in the UK.\n\n“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.”\n\nThere are only 1-2 cases of FCS for every one million people, making the condition incredibly rare.3 FCS is associated with a breadth of cognitive impairments, significant emotional burden, and poor mental health.2,4 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.5 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.4\n\n“This is a landmark day for people with FCS as they have ...

More updates from Ionis Pharmaceuticals, Inc.