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

November 9, 2020 3:00 AM EST

The Scottish Medicines Consortium (SMC) enables access to volanesorsen, a treatment for adults with familial chylomicronaemia syndrome (FCS)

LONDON--(BUSINESS WIRE)-- Akcea Therapeutics, a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), announced today 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).1 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.2

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.

“Today’s announcement is an incredibly important milestone for those living with FCS in Scotland,” said Andy Caldwell, Country Manager, UK & Ireland at Akcea Therapeutics UK. “We welcome this innovative new pathway in Scotland that allows treatments for extremely rare conditions that meet specific criteria, to be evaluated under the ultra-orphan medicines pathway. This allows patients with extremely rare conditions to benefit from access to much-needed treatments. As no other treatments are currently available for FCS in Scotland, this is a landmark moment for this patient population and it moves us forward on our journey to ensure patients with the rarest conditions, such as FCS, are not left behind.”

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.3,4 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.

There are only one to two cases of FCS for every one million people, making the condition incredibly rare.5 FCS is also associated with a breadth of cognitive impairments, significant emotional burden, and poor mental health.4,6 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.4 This is incredibly challenging and does not remove the risk of pancreatitis and other symptoms of the condition.7

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.6,9

WAYLIVRA▼® (volanesorsen) was granted conditional marketing authorisation in May 2019 by the European Commission.8

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 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 2020.2 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.8,9 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) study9, and is supported by results from the Phase 3 COMPASS study.11 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.9

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.8,9

About FCS

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.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. is a wholly owned subsidiary of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), the leader in RNA therapeutics. Akcea commercializes TEGSEDI® (inotersen) and WAYLIVRA® (volanesorsen), and with Ionis, is advancing a mature pipeline of novel medicines discovered by Ionis and based on Ionis' proprietary antisense technology. TEGSEDI is approved in the U.S., E.U., Canada and Brazil, and WAYLIVRA is approved in the E.U. For more information about Akcea please visit Additional information about Ionis is available at


This press release includes forward-looking statements regarding Ionis’ business, financial guidance and the therapeutic and commercial potential of WAYLIVRA (volanesorsen) and Ionis' technologies and products in development, including the business of Akcea Therapeutics, Inc., Ionis' wholly owned subsidiary. Any statement describing Ionis’ goals, expectations, financial or other projections, intentions or beliefs is a forward-looking statement and should be considered an at-risk statement. Such statements are subject to certain risks and uncertainties, including those related to the impact COVID-19 could have on our business, and including but not limited to those related to our commercial products and the medicines in our pipeline, and particularly those inherent in the process of discovering, developing and commercializing medicines that are safe and effective for use as human therapeutics, and in the endeavor of building a business around such medicines. Ionis’ forward-looking statements also involve assumptions that, if they never materialize or prove correct, could cause its results to differ materially from those expressed or implied by such forward-looking statements. Although Ionis’ forward-looking statements reflect the good faith judgment of its management, these statements are based only on facts and factors currently known by Ionis. As a result, you are cautioned not to rely on these forward-looking statements. These and other risks concerning Ionis' programs are described in additional detail in Ionis' annual report on Form 10-K for the year ended December 31, 2019, and the most recent Form 10-Q quarterly filing, which are on file with the SEC. Copies of these and other documents are available from the Company.

Ionis Pharmaceuticals® is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics® TEGSEDI® and WAYLIVRA® are trademarks of Akcea Therapeutics, Inc.



1 Scottish Medicines Consortium (SMC). volanesorsen sodium (Waylivra). Available at: Last accessed October 2020.
2 National Institute for Health and Care Excellence (NICE). Final evaluation document. Volanesorsen for treating familial chylomicronaemia syndrome. Available at: Last accessed October 2020.
3 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.
4 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.
5 Heart UK. FCS. Available at: Last accessed June 2020.
6 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.
7 Heart UK. Eating for FCS. Available at: Last accessed June 2020.
8 European Medicines Agency. Waylivra Summary of Product Characteristics. Available at: Last accessed February 2020.
9 The Approach Open Label Study: A Study of Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients with Familial Chylomicronemia Syndrome. Available at: Last accessed June 2020.
10 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.
11 The COMPASS Study: A Study of Volanesorsen (Formally ISIS-APOCIIIRx) in Patients with Hypertriglyceridemia. Available at: Last accessed June 2020.

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

US Media Contact:
Roslyn Patterson
T: +1 760 603 2681 
[email protected]

Akcea/Ionis Investor Contact:
Wade Walke, Ph.D.
Vice President, Investor Relations
T: +1 760 603 2331
[email protected]

Source: Akcea Therapeutics, Inc.

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