Akcea Announces FDA Advisory Committee Meeting to Review Volanesorsen as a Treatment for Familial Chylomicronemia Syndrome
February 15 2018 - 4:05PM
Akcea Therapeutics, Inc. (NASDAQ:AKCA), an affiliate of Ionis
Pharmaceuticals, Inc., focused on developing and commercializing
drugs to treat patients with serious cardiometabolic diseases
caused by lipid disorders, today announced that the Division of
Metabolism and Endocrinology Products of the U.S. Food and
Drug Administration (FDA) will hold an advisory committee meeting
to review data supporting the new drug application (NDA) for
volanesorsen. The meeting is tentatively scheduled for May 10,
2018. Volanesorsen is an investigational therapy for the treatment
of a rare lipid disorder called familial chylomicronemia syndrome
(FCS), which is characterized by the buildup of fats in the body
due to a deficiency in the enzyme lipoprotein lipase.
“Today, there is no effective therapy approved to treat people
with FCS. Standard triglyceride-lowering agents are generally
ineffective in lowering the extremely high triglyceride levels that
cause the severe acute and chronic symptoms in people with FCS,”
said Paula Soteropoulos, chief executive officer of Akcea
Therapeutics. “If approved, volanesorsen would be the first therapy
indicated for these patients. We look forward to reviewing the
supporting data with members of the advisory committee.”
The FDA set a PDUFA (Prescription Drug User Fee Act) goal date
of August 30, 2018 for the completion of its review.
ABOUT VOLANESORSEN AND FCS Volanesorsen, a
product of Ionis’ proprietary antisense technology, 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. Volanesorsen is
under regulatory review in the U.S., EU and Canada as a treatment
for familial chylomicronemia syndrome (FCS). The U.S. and EU
regulatory agencies have granted Orphan Drug Designation to
volanesorsen for the treatment of people with FCS.
FCS is a severe, rare disorder characterized by extremely high
levels of triglycerides, daily symptoms such as abdominal pain, and
the risk of recurrent, potentially fatal, acute pancreatitis.
People with FCS are unable to effectively metabolize large,
triglyceride-rich lipid particles called chylomicrons due to a
deficiency in lipoprotein lipase, an enzyme that helps to break
down triglycerides. There is no effective therapy available.
Additional information on FCS is available
at www.fcsfocus.com. For a full list of organizations
supporting the FCS community worldwide, please click here.
Volanesorsen is also currently in Phase 3 clinical development
for the treatment of patients with familial partial lipodystrophy,
or FPL. Akcea anticipates reporting top-line data from this
study in 2019.
ABOUT AKCEA THERAPEUTICSAkcea Therapeutics, an
affiliate of Ionis Pharmaceuticals, Inc. (NASDAQ:IONS), is a
biopharmaceutical company focused on developing and commercializing
drugs to treat patients with serious cardiometabolic diseases
caused by lipid disorders. Akcea is advancing a mature pipeline of
four novel drugs, including volanesorsen, AKCEA-APO(a)-LRx,
AKCEA-ANGPTL3-LRx and AKCEA-APOCIII-LRx, 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
chylomicronemia syndrome, or FCS, and is currently in Phase 3
clinical development for the treatment of patients with familial
partial lipodystrophy, or FPL. Akcea is building the infrastructure
to commercialize its drugs globally with a focus on lipid
specialists as the primary call point. Akcea is located in
Cambridge, Massachusetts. Additional information about Akcea is
available at www.akceatx.com.
FORWARD-LOOKING STATEMENTThis press release
includes forward-looking statements regarding the business of Akcea
Therapeutics, Inc. and the therapeutic and commercial potential of
volanesorsen or 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 endeavor 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™ is a trademark of Ionis Pharmaceuticals,
Inc. Akcea Therapeutics™ is a trademark of Ionis Pharmaceuticals,
Inc.
Media and Investor Contact: Kathleen
Gallagher
Head of Communications and Investor Relations, Akcea
Therapeutics (617)-207-8509 kgallagher@akceatx.com
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