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
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
yellowcard@mhra.gov.uk 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
https://www.actionfcs.org/.
ABOUT AKCEA THERAPEUTICS
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 www.akceatx.com and you can follow the Company on Twitter at
@akceatx.
Additional Information about Akcea Therapeutics UK Ltd can be
found at www.akceatx.co.uk
AKCEA’S FORWARD-LOOKING STATEMENT
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.
References
________________________________
1 Stroes E, Moulin P, Parhofer KG, Rebours V, L�hr 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:
https://www.heartuk.org.uk/genetic-conditions/familial-chylomicronaemia-symdrome-fcs
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:
https://www.heartuk.org.uk/eating-for-fcs-/eating-for-fcs- Last
accessed June 2020.
6 European Medicines Agency. Waylivra Summary of Product
Characteristics. Available at: www.ema.europa.eu/en. Last accessed
February 2020.
7 ClinicalTrials.gov. The Approach Open Label Study: A Study of
Volanesorsen (Formerly IONIS-APOCIIIRx) in Patients with Familial
Chylomicronemia Syndrome. Available at: https://clinicaltrials.gov/ct2/show/NCT02658175.
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 ClinicalTrials.gov. The COMPASS Study: A Study of Volanesorsen
(Formally ISIS-APOCIIIRx) in Patients with Hypertriglyceridemia.
Available at: https://clinicaltrials.gov/ct2/show/NCT02300233. Last
accessed June 2020.
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version on businesswire.com: https://www.businesswire.com/news/home/20200918005029/en/
UK Media: Sarah Herbert Evoke KYNE T: +44 0203 928 6834
sarah.herbert@evokegroup.com
US Media: Bill Berry Berry & Co. T: +1 212-253-8881
bberry@berrypr.com
Investor: Matt Roache Director, Investor Relations T: +1
617-841-9535 mroache@akceatx.com
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