Akcea Therapeutics' Volanesorsen Receives Promising Innovative Medicine (PIM) Status for the Treatment of Familial Chylomicronaemia Syndrome (FCS)
(Thomson Reuters ONE) -
LONDON, Oct. 10, 2017 (GLOBE NEWSWIRE) -- Akcea Therapeutics United Kingdom (UK)
today announced that volanesorsen has been granted a Promising Innovative
Medicine (PIM) Designation by the Medicines and Healthcare products Regulatory
Agency (MHRA) for the treatment of familial chylomicronaemia syndrome (FCS), a
rare genetic lipid disorder. Akcea Therapeutics UK, the UK subsidiary of Akcea
Therapeutics Inc. (NASDAQ:AKCA), an affiliate of Ionis Pharmaceuticals, Inc., is
focused on developing and commercialising drugs to treat patients with serious
cardiometabolic lipid disorders.
A PIM Designation is an early indication that a medicinal product is a promising
candidate for the Early Access to Medicines Scheme (EAMS) in the UK; intended
for the treatment, diagnosis or prevention of a life-threatening or seriously
debilitating condition, with the potential to address an unmet medical need.
"We are delighted about the MHRA's decision to designate volanesorsen as a PIM.
This designation demonstrates the potential to address clear unmet medical needs
in the treatment of this debilitating condition," comments Luke Robinson,
general manager, Akcea Therapeutics, UK, Ireland & Nordics.
FCS is a severe, rare disorder characterised by extremely elevated levels of
triglycerides,(1) symptoms such as abdominal pain that affect daily
living,(1) and the risk of recurrent, potentially fatal, acute
pancreatitis.(1) People with FCS are unable to effectively metabolise large,
triglyceride-rich, lipid particles called chylomicrons due to a deficiency in
lipoprotein lipase,(1) an enzyme that helps to break down triglycerides.
"FCS is a significant burden on people affected by the condition - from their
day to day health, to what they can eat, their employment, their sense of self-
worth and their relationships with their friends and family," says Dr. Louis
O'Dea, chief medical officer, Akcea Therapeutics. "FCS patients have a high
unmet need with potentially life-threatening consequences, and we seek to bring
this new treatment to these patients as expeditiously as possible."
Patients with FCS have triglyceride levels that can reach 20 to 30 times that of
healthy individuals.(2) This can often lead to repeated episodes of acute
pancreatitis, which can be fatal.(1) There is currently no effective therapy
available.(3)
Investor and Media Contact
Kathleen Gallagher
Head of Communications, Akcea Therapeutics, UK, Ireland & Nordics
E: kgallagher(at)akceatx.com
T: +1 617 207 8509
Notes to Editors
About Volanesorsen in FCS
Volanesorsen, a product of Ionis' proprietary antisense technology, is in
development for rare metabolic disorders including familial chylomicronaemia
syndrome (FCS). Volanesorsen is designed to reduce the production of ApoC-III, a
protein produced in the liver that plays a central role in the regulation of
plasma triglycerides and may also affect other metabolic parameters.(2)
FCS is a severe, rare disorder characterised by extremely elevated levels of
triglycerides, symptoms such as abdominal pain that affect daily living, and the
risk of recurrent, potentially fatal, acute pancreatitis.(1)People with FCS are
unable to effectively metabolise large, triglyceride-rich lipid particles called
chylomicrons due to a deficiency in lipoprotein lipase, an enzyme that helps to
break down triglycerides.(1)Patients with FCS have triglyceride levels that can
reach 20 to 30 times that of healthy individuals,(2) which can often lead to
repeated episodes of acute pancreatitis, which can be fatal.(1) There is
currently no effective therapy available.(3)
About Akcea Therapeutics UK
Akcea Therapeutics UK is the UK subsidiary of Akcea Therapeutics Inc., an
affiliate of Ionis Pharmaceuticals, Inc. Akcea is a biopharmaceutical company
focused on transforming the lives of patients with serious cardiometabolic lipid
disorders. Akcea has a robust portfolio of development-stage drugs covering
multiple targets and diseases using advanced RNA-targeted antisense
therapeutics.
Akcea is advancing a mature pipeline of four novel drugs, including
volanesorsen, AKCEA-APO(a)-L(Rx), AKCEA-ANGPTL3-L(Rx) and AKCEA-APOCIII-L(Rx),
all with the potential to treat multiple diseases. All four drugs were
discovered by and are being co-developed with Ionis, a leader in antisense
therapeutics, and are based on Ionis' proprietary antisense technology. The most
advanced drug in its pipeline, volanesorsen, is under regulatory review in the
U.S., EU and Canada for the treatment of familial chylomicronaemia syndrome, or
FCS. Akcea is building the infrastructure to commercialise its drugs globally
with a focus on lipid specialists as the primary call point. Akcea's global
headquarters is located in Cambridge, Massachusetts. Additional information
about Akcea is available at www.akceatx.com.
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 and other products in development. Any statement describing Akcea's
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, particularly those
inherent in the process of discovering, developing and commercializing 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 materialize 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. As a result, you are cautioned not
to rely on these forward-looking statements. These and other risks concerning
Akcea's programs are described in additional detail in its final prospectus for
its initial public offering and its most recent quarterly report on Form 10-Q,
which is on file with the SEC.
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(TM) is a trademark of Ionis Pharmaceuticals, Inc. Akcea
Therapeutics(TM) is a trademark of Ionis Pharmaceuticals, Inc.
References
1. Brunzell JD. Familial lipoprotein lipase deficiency. GeneReviews 2011.
2. Gaudet D, et al. Targeting APOC3 in the familial chylomicronemia syndrome.
New England Journal of Medicine 2014; 371:2200-2206
3. NORD. The physician's guide to lipoprotein lipase deficiency (LPLD).
Available at: https://rarediseases.org/physician-guide/lipoprotein-lipase-
deficiency-lpld Accessed October 2017.
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: Akcea Therapeutics, Inc. via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 10.10.2017 - 08:00 Uhr
Sprache: Deutsch
News-ID 562997
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contact information:
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Kategorie:
Business News
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