September 18, 2020: “Akcea Therapeutics UK Ltd., a subsidiary of Akcea Therapeutics Inc., announced 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 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.
“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.”
There are only 1-2 cases of FCS for every one million people, making the condition incredibly rare.
FCS is associated with a breadth of cognitive impairments, significant emotional burden, and poor mental health.
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.
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.
“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 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.
Volanesorsen will be the second treatment that Akcea Therapeutics has made available through the NHS in the last year. Akcea Therapeutics, Inc. is a majority-owned affiliate of Ionis Pharmaceuticals, Inc. Volanesorsen is an antisense oligonucleotide drug designed by Ionis Pharmaceuticals and co-developed by Akcea and Ionis to reduce the production of a protein that regulates plasma triglycerides.
WAYLIVRA▼® (volanesorsen) was granted a conditional marketing authorisation in May 2019 by the European Commission.
For important safety information for volanesorsen, including method of administration, special warnings, drug interactions and adverse drug reactions, please see the European Summary of Product Characteristics (SmPC), which is available on the EMA website at www.ema.europa.eu.
This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. Reporting forms and information can be found at www.yellowcard.mhra.gov.uk/.
Alternatively, search for MHRA Yellow Card in the Google Play or Apple App Store or contact the MHRA at [email protected] or 0800 731 6789 (10am to 2pm Monday to Friday only).
Volanesorsen, which received a marketing authorisation in Europe in May 2019, is the only therapy indicated for people with familial chylomicronaemia syndrome (FCS). Volanesorsen is a self-administered, subcutaneous injection in a single-use, prefilled syringe.
Volanesorsen, a product of Ionis’s 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 study 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, the largest study ever conducted in patients with FCS, 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 hypertriglyceridemia (>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 skinAdditional information on FCS is available through Action FCS at https://www.actionfcs.org/.”