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

September 18, 2020 3:00 AM EDT

National Institute for Health and Care Excellence (NICE) issues a positive recommendation for volanesorsen to treat adults with Familial Chylomicronaemia Syndrome (FCS)


Akcea 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.

“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.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

“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.6

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

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 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).

About volanesorsen

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.6,7 The European Commission’s marketing authorisation of volanesorsen was based on results from the Phase 3 APPROACH study8 and the ongoing APPROACH Open Label Extension (OLE) study7, and is supported by results from the Phase 3 COMPASS study.9 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.7

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.6,7

About FCS

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.4 Because of limited LPL function, people with FCS cannot breakdown chylomicrons, lipoprotein particles that are 90% triglycerides.4 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.4 Additional information on FCS is available through Action FCS at


Akcea Therapeutics, Inc., a majority-owned affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), is a biopharmaceutical company focused on developing and commercialising medicines to treat patients with serious and rare conditions. Akcea is commercialising Tegsedi® (inotersen) and Waylivra® (volanesorsen), as well as advancing a mature pipeline of novel medicines, including AKCEA-APO(a)-LRx, vupanorsen (AKCEA-ANGPTL3-LRx), AKCEA-APOCIII-LRx, and AKCEA-TTR-LRx, with the potential to treat multiple conditions. All six medicines were discovered by Ionis, a leader in antisense therapeutics, and are based on Ionis' proprietary antisense technology. Tegsedi is approved in the U.S., E.U., Canada and Brazil, and volanesorsen is approved in the E.U. Akcea is headquartered in Boston, Massachusetts, and is building the infrastructure to commercialise its medicines globally. Additional information about Akcea Therapeutics, Inc. is available at and you can follow the Company on Twitter at @akceatx.

Additional Information about Akcea Therapeutics UK Ltd can be found at


This press release includes forward-looking statements regarding the business of Akcea Therapeutics, Inc. and the therapeutic and commercial potential of volanesorsen. Any statement describing Akcea’s goals, expectations, financial or other projections, intentions or beliefs, including commercial potential of volanesorsen, or other of Akcea’s drugs in development is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, particularly those inherent in the process of discovering, developing and commercialising drugs that are safe and effective for use as human therapeutics, and in the endeavour of building a business around such drugs. Akcea’s forward-looking statements also involve assumptions that, if they never materialise or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Akcea’s forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Akcea. In particular, we caution you that our forward-looking statements are subject to the ongoing and developing circumstances related to the COVID-19 pandemic, which may have a material adverse effect on our business, operations and future financial results. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Akcea's programmes are described in additional detail in Akcea's annual report on Form 10-K, and its most recent quarterly report on Form 10-Q, which are on file with the SEC in the U.S. Copies of these and other documents are available from the Company.

In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies,” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.

Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics®, Tegsedi® and Waylivra® are trademarks of Akcea Therapeutics, Inc.




1 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.

2 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.

3 Heart UK. FCS. Available at: Last accessed June 2020.

4 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.

5 Heart UK. Eating for FCS. Available at: Last accessed June 2020.

6 European Medicines Agency. Waylivra Summary of Product Characteristics. Available at: Last accessed February 2020.

7 The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients with Familial Chylomicronemia Syndrome. Available at: Last accessed June 2020.

8 Gaudet D, et al. (2017) The APPROACH Study: A Randomized, Double-Blind, Placebo-Controlled, Phase 3 Study of Volanesorsen Administered Subcutaneously to Patients with Familial Chylomicronemia Syndrome (FCS). Journal of Clinical Lipidology; 11(3):814-5.

9 The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients with Hypertriglyceridemia. Available at: Last accessed June 2020.

UK Media:
Sarah Herbert Evoke KYNE
T: +44 0203 928 6834
[email protected]

US Media:
Bill Berry
Berry & Co.
T: +1 212-253-8881
[email protected]

Matt Roache
Director, Investor Relations
T: +1 617-841-9535
[email protected]

Source: Akcea Therapeutics UK Ltd.

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