- FCS is a rare and debilitating genetic disease often
leading to significant risk of acute, potentially fatal
pancreatitis
- If approved, olezarsen would be the first approved treatment
for FCS in the U.S.
CARLSBAD, Calif., Jan. 31,
2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) today announced that the U.S. Food and Drug
Administration (FDA) has granted olezarsen Fast Track designation
for the treatment of familial chylomicronemia syndrome (FCS).
FCS is a debilitating genetic disease characterized by severely
high levels of plasma triglycerides and a risk of
unpredictable and potentially fatal acute pancreatitis. In addition
to acute pancreatitis, FCS patients are at risk of chronic
complications due to permanent organ damage, including chronic
pancreatitis and type 3c diabetes. There are currently no approved
therapies for the treatment of FCS in the U.S.
Fast Track designation is designed to expedite the FDA's review
of innovative, new drugs that demonstrate the potential to address
unmet medical need.
"The FDA Fast Track designation for olezarsen recognizes the
urgent need for an effective treatment for FCS, a debilitating rare
disease affecting people with very limited treatment options and an
elevated risk of painful and potentially fatal bouts of
pancreatitis," said Richard S.
Geary, Ph.D., executive vice president and chief development
officer at Ionis. "We look forward to working collaboratively with
the FDA to bring forward a safe and effective treatment for FCS
patients as quickly as possible."
Ionis fully enrolled its global Phase 3 BALANCE study of
olezarsen in adult patients with FCS last year. The company plans
to share data from the BALANCE study in the second half of 2023. In
addition to FCS, Ionis is evaluating olezarsen in severe
hypertriglyceridemia (SHTG). More information on the BALANCE study
(NCT04568434) is available at www.clinicaltrials.gov.
About familial chylomicronemia syndrome (FCS)
FCS is a rare, genetic disease estimated to affect 3,000 to
5,000 people worldwide and is characterized by extremely elevated
triglyceride levels. FCS can lead to many chronic health issues
including severe, recurrent abdominal pain, fatigue, high risk of
life-threatening pancreatitis and abnormal enlargement of the liver
or spleen. In addition, people with FCS are often unable to work,
adding to their disease burden. In severe cases, patients can have
bleeding into the pancreas, serious tissue damage, infection, and
cyst formation, as well as damage to other vital organs such as the
heart, lungs, and kidneys. Additional information on FCS is
available at www.fcsfocus.com and through the FCS Foundation
at www.livingwithfcs.org.
About Olezarsen
Olezarsen (formerly IONIS-APOCIII-LRx) is an
investigational LICA medicine designed to inhibit the production of
apoC-III for patients who are at risk of disease due to elevated
triglyceride levels. ApoC-III is a protein produced in the liver
that regulates triglyceride metabolism in the blood. People with
severely elevated triglycerides, such as people with FCS, are at
high risk for acute pancreatitis and an increased risk of
cardiovascular disease.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been the leader in
RNA-targeted therapy, pioneering new markets and changing standards
of care with its novel antisense technology. Ionis currently has
three marketed medicines and a premier late-stage pipeline
highlighted by industry-leading cardiovascular and neurological
franchises. Our scientific innovation began and continues with the
knowledge that sick people depend on us, which fuels our vision to
become the leader in genetic medicine, utilizing a multi-platform
approach to discover, develop and deliver life-transforming
therapies.
To learn more about Ionis visit www.ionispharma.com and follow
us on Twitter @ionispharma.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
Ionis' business and the therapeutic and commercial potential of
Ionis' technologies, olezarsen and other products in development.
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 Dec. 31, 2021, and the most
recent Form 10-Q quarterly filing, which are on file with the
Securities and Exchange Commission. Copies of these and other
documents are available from the Company.
In this press release, unless the context requires otherwise,
"Ionis," "Company," "we," "our," and "us" refers to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a trademark of Ionis
Pharmaceuticals, Inc.
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SOURCE Ionis Pharmaceuticals, Inc.