BOSTON, Aug. 13, 2020 /PRNewswire/ -- Akcea Therapeutics,
Inc. (NASDAQ: AKCA), a majority-owned affiliate of Ionis
Pharmaceuticals, Inc., today announced that the German Association
of Statutory Sick Funds has agreed upon new pricing and
reimbursement of WAYLIVRA® (volanesorsen) as an adjunct
to diet in adult patients with genetically confirmed familial
chylomicronemia syndrome (FCS) and at high risk for pancreatitis,
in whom response to diet and triglyceride lowering therapy has been
inadequate. WAYLIVRA has been commercially available in
Germany since August 15, 2019. As part of reimbursement and
market access protocol in Germany,
Akcea was required to renegotiate the WAYLIVRA launch price 12
months after first commercialization, which will go into effect on
August 15, 2020.
"We are pleased that renegotiations in Germany were swiftly completed,
demonstrating that payers in the region continue to recognize
the value that WAYLIVRA offers to FCS patients. While WAYLIVRA has
been commercially available to FCS patients in the region for some
time, achieving this new, 2-year mutually agreed upon pricing
and reimbursement is a significant milestone for the global FCS
community of patients and caregivers, who are currently
underserved," said Michael Pollock,
SVP, head of Europe at Akcea. "We
continue to build on our momentum in Europe and are on track to launch WAYLIVRA in
additional European countries with the goal of bringing this much
needed therapy to all eligible patients living with FCS."
"FCS patients in Germany as
well as their families have been living with the significant burden
of this disease for years, including physical, social, emotional
and financial effects, and I am pleased to be able to offer
appropriate patients a treatment option supported by extensive
clinical data including results from the Phase 3 APPROACH study,"
said Professor Ioanna Gouni-Berthold
from University of Cologne.
FCS is a debilitating genetic disease caused by impaired
function of the enzyme lipoprotein lipase (LPL), which results in
elevated triglyceride levels and significant risk and disease
burden, including unpredictable and potentially fatal acute
pancreatitis as well as chronic complications due to permanent
organ damage. It is estimated that there are between 3,000 to 5,000
people living with FCS worldwide. WAYLIVRA is designed to reduce
the production of apolipoprotein C-III (apoC-III), a protein
produced in the liver that plays a central role in the regulation
of plasma triglycerides.
About WAYLIVRA® (volanesorsen)
With
conditional marketing authorization from the European
Commission (EC) as an adjunct to diet in adult patients with
genetically confirmed FCS and at high risk for pancreatitis, in
whom response to diet and triglyceride lowering therapy has been
inadequate, WAYLIVRA is now the only therapy indicated for people
with familial chylomicronemia syndrome (FCS). Akcea is working to
confirm a path forward for WAYLIVRA in the U.S. and Canada.
WAYLIVRA, 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.
The European Commission's marketing authorization of WAYLIVRA is
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, treatment with WAYLIVRA
reduced triglycerides 77% (-94% when compared to placebo). All
patients in the trial maintained a low-fat diet.
WAYLIVRA 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 WAYLIVRA were events
associated with injection site reactions, immunogenicity and
reduction in platelet levels.
The WAYLIVRA Early Access Program (EAP) has been initiated in
Europe, the U.S. and Canada and is currently enrolling eligible
patients. Click here for more information on the WAYLIVRA
EAP. For more information on WAYLIVRA, please
visit www.WAYLIVRA.eu.
About FCS
FCS is an ultra-rare disease caused by impaired function of
the enzyme lipoprotein lipase (LPL) and characterized by severe
hypertriglyceridemia (>880mg/dL or 10mmol/L) and a risk of
unpredictable and potentially fatal acute pancreatitis. Because of
limited LPL production or function, people
with FCS cannot breakdown chylomicrons, lipoprotein
particles that are 90% triglycerides. In addition to
pancreatitis, FCS patients are at risk of chronic
complications due to permanent organ damage, including chronic
pancreatitis and pancreatogenic (type 3c) diabetes. They can
experience daily symptoms including abdominal pain, generalized
fatigue and impaired cognition that affect their ability to work.
People with FCS also report major emotional and
psychosocial effects including anxiety, social withdrawal,
depression and brain fog. Additional information
on FCS is available at www.fcsfocus.com, through
the LPLD Alliance at www.lpldalliance.org and
through The FCS Foundation
at http://www.livingwithfcs.org. For a full list of
organizations supporting the FCS community worldwide,
please click here.
ABOUT AKCEA THERAPEUTICS, INC.
Akcea Therapeutics, Inc., a majority-owned affiliate
of Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), is a
biopharmaceutical company focused on developing and commercializing
medicines to treat patients with serious and rare diseases. Akcea
is commercializing 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
diseases. 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 WAYLIVRA
is approved in the E.U. Akcea is headquartered in Boston,
Massachusetts, and is building the
infrastructure to commercialize its medicines globally. Additional
information about Akcea is available
at www.akceatx.com and you can follow us on Twitter at
@akceatx.
FORWARD-LOOKING STATEMENT
This press release includes forward-looking statements regarding
the business of Akcea Therapeutics, Inc. Any statement
describing Akcea's goals, expectations, financial or other
projections, intentions or beliefs, including the commercial
potential of TEGSEDI®, WAYLIVRA® and Akcea's
other medicines 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
commercializing medicines that are safe and effective for use as
human therapeutics, and in the endeavor of building a business
around such medicines. 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. 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 programs are described in additional detail in
Akcea's quarterly reports on Form 10-Q and annual reports on Form
10-K, which are on file with the SEC. 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.
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SOURCE Akcea Therapeutics, Inc.