CARLSBAD, Calif., Feb. 15,
2024 /PRNewswire/ -- Ionis Pharmaceuticals,
Inc. (Nasdaq: IONS) announced today that the U.S. Food
and Drug Administration (FDA) has granted Orphan Drug
designation to the investigational medicine olezarsen for the
treatment of familial chylomicronemia syndrome (FCS), a rare,
genetic disease characterized by extremely elevated triglyceride
levels and recurrent acute pancreatitis (AP). The FDA grants Orphan
Drug designation to therapies intended for treatment, prevention or
diagnosis of a rare disease or condition that affects fewer than
200,000 people in the U.S. at the time of
designation.
"People living with FCS are in urgent need of a medicine that
may help stabilize their triglyceride levels and reduce the risk of
life-threatening AP attacks," said Sam
Tsimikas, M.D., senior vice president, global cardiovascular
development of Ionis. "If approved, olezarsen has the potential to
be the standard of care in the U.S. for the treatment of FCS. We
are deeply committed to advancing this important medicine that has
the potential to be transformative for people and families living
with FCS."
FCS prevents the body from breaking down fats, which can result
in fasting triglyceride levels that can range from 10 to 100 times
higher than normal levels.1,2 People
with FCS are at high risk of AP, or inflammation of the pancreas,
which often results in hospitalization and can be
life-threatening.3 In severe cases, vital organs such as
the heart, lungs, kidneys and pancreas can be
damaged.4
Under the FDA's Orphan Drug Act, orphan drug status
provides incentives, including tax credits, grants and waiver of
certain administrative fees for clinical trials, and seven years of
market exclusivity following drug approval.
About Olezarsen
Olezarsen is an RNA-targeted
investigational LIgand Conjugated Antisense
(LICA) medicine being evaluated for people at risk of disease due
to elevated triglyceride levels, including those with familial
chylomicronemia syndrome (FCS). Olezarsen is designed to inhibit
the body's production of apoC-III, a protein produced in the liver
that regulates triglyceride metabolism in the blood. The U.S. FDA
granted olezarsen Fast Track designation for the treatment
of FCS in January 2023. In
addition to FCS, Ionis is evaluating olezarsen for the
treatment of severe hypertriglyceridemia (sHTG) in Phase 3 clinical
trials.
About FCS
FCS is a rare, genetic disease characterized by extremely
elevated triglyceride levels. It is caused by impaired function of
the enzyme lipoprotein lipase (LPL). Because of limited LPL
production or function, people with FCS cannot
effectively break down chylomicrons, lipoprotein particles that are
90% triglycerides. FCS is estimated to impact one to two
people per million worldwide. People living with FCS are
at high risk of acute pancreatitis (AP) in addition to other
chronic health issues such as fatigue and severe, recurrent
abdominal pain. People living with FCS are sometimes unable to
work, adding to the burden of disease.
Currently, there are no U.S. FDA-approved therapies for the
treatment of FCS and standard triglyceride lowering therapies
are generally ineffective in patients with FCS. People living with
this condition currently rely solely on nutrition management
through extremely restrictive and difficult to adhere to diets to
navigate the health risks associated with FCS.
About Ionis Pharmaceuticals,
Inc.
For three decades, Ionis has invented medicines that bring
better futures to people with serious diseases. Ionis currently has
five marketed medicines and a leading pipeline in neurology,
cardiology, and other areas of high patient need. As the pioneer in
RNA-targeted medicines, Ionis continues to drive innovation in RNA
therapies in addition to advancing new approaches in gene editing.
A deep understanding of disease biology and industry-leading
technology propels our work, coupled with a passion and urgency to
deliver life-changing advances for patients. To learn more about
Ionis, visit Ionispharma.com and follow us on X (Twitter) and
LinkedIn.
Forward-looking
Statements
This press release includes forward-looking statements regarding
olezarsen, Ionis' business, and the therapeutic and commercial
potential of Ionis' commercial medicines, additional medicines in
development and technologies. 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 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, 2022, and most recent Form 10-Q, which are on
file with the SEC. Copies of these and other documents are
available at www.ionispharma.com.
Ionis Pharmaceuticals® is a registered trademark of Ionis
Pharmaceuticals, Inc.
1 Pallazola et al. Eur J Preventive Cardiol 2020
27:2276-2278.
2 Gaudet D, et al. N Engl J Med.
2014;371:2200-2206.
3 Chyzhyk V, Brown AS. Familial chylomicronemia
syndrome: A rare but devastating autosomal recessive disorder
characterized by refractory hypertriglyceridemia and recurrent
pancreatitis. Trends Cardiovasc Med. 2020;30(2):80-5.
4 "Pancreatitis." WebMD.
https://www.webmd.com/digestive-disorders/digestive-diseases-pancreatitis.
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SOURCE Ionis Pharmaceuticals, Inc.