CARLSBAD, Calif., Feb. 8, 2024
/PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS)
announced today that the U.S. Food and Drug Administration (FDA)
has granted Fast Track designation to Ionis and AstraZeneca's
eplontersen, an investigational therapy for the treatment of
transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) in adults.
The FDA grants development programs Fast Track designation to
facilitate the development and expedite the review of drugs that
demonstrate the potential to treat serious conditions and fill an
unmet medical need.
"Receiving Fast Track designation from the FDA reinforces our
belief that eplontersen has the potential to be a transformational
treatment for patients with ATTR-CM, which remains a progressive
and fatal condition for hundreds of thousands of people worldwide
despite available treatment options," said Eugene Schneider, M.D., executive vice president
and chief clinical development officer, Ionis. "CARDIO-TTRansform
is the largest, most comprehensive study ever conducted in ATTR-CM
patients, with results expected as early as next year."
Sarah Walters, vice president,
U.S. cardiovascular, renal and metabolic diseases, AstraZeneca
said: "We are committed to accelerating innovation and addressing
the spectrum of unmet patient needs in amyloidosis. Together with
our partner Ionis, AstraZeneca is building on our heritage in
cardiovascular disease and RNA-targeted therapeutics to bring a
potential best-in-class treatment for people living with
ATTR-CM."
In December 2023, eplontersen was
approved in the U.S. for the treatment of the polyneuropathy of
hereditary transthyretin-mediated amyloidosis in adults, commonly
referred to as hATTR-PN or ATTRv-PN, under the brand name WAINUA™
(eplontersen). As part of a global development and
commercialization agreement, AstraZeneca and Ionis are
commercializing WAINUA for the treatment of ATTRv-PN in
the U.S. and are seeking regulatory approval
in Europe and other parts of the world. WAINUA
was granted Orphan Drug Designation in
the U.S. and in the EU for the treatment of
transthyretin-mediated amyloidosis (ATTR).
The global CARDIO-TTRansform Phase 3 study of eplontersen in
adults with ATTR-CM is fully enrolled with more than 1,400 patients
– making it the largest study in this patient population to date.
The company plans to share data from the CARDIO-TTRansform study as
early as 2025. More information on the CARDIO-TTRansform study
(NCT04136171) is available at www.clinicaltrials.gov.
About Transthyretin-Mediated Amyloid Cardiomyopathy
(ATTR-CM)
Transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) is an
underdiagnosed and potentially fatal disease. It is caused by the
accumulation of misfolded TTR protein in the cardiac muscle.
Patients experience ongoing debilitating heart damage resulting in
progressive heart failure, which results in death within three to
five years from disease onset. ATTR-CM includes both the genetic
and wild-type form of the disease. Worldwide, there are an
estimated 300,000 – 500,000 patients with
ATTR-CM.1,2,3
About Eplontersen
Eplontersen is a LIgand-Conjugated
Antisense (LICA) medicine designed to inhibit the production
of transthyretin, or TTR protein. The investigational medicine is
currently being evaluated in the Phase 3 CARDIO-TTRansform study
for hereditary or wild-type transthyretin-mediated amyloid
cardiomyopathy (ATTR-CM), a progressive and fatal condition that
typically leads to progressive heart failure and often death within
three-to-five years from disease onset. The CARDIO-TTRansform Phase
3 study is fully enrolled with more than 1,400 patients – making it
the largest study in this patient population to date.
Eplontersen was recently approved in the U.S. for the treatment
of the polyneuropathy of hereditary transthyretin-mediated
amyloidosis in adults, commonly referred to as hATTR-PN or ATTRv-PN
under the brand name WAINUA™ (eplontersen). Please see full
Prescribing Information.
INDICATION for WAINUA™ (eplontersen)
WAINUA injection for subcutaneous use 45 mg is indicated for
treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults.
IMPORTANT SAFETY INFORMATION for WAINUA™
(eplontersen)
WARNINGS AND PRECAUTIONS
Reduced Serum Vitamin A Levels and Recommended
Supplementation WAINUA leads to a decrease in serum vitamin A
levels. Supplement with recommended daily allowance of vitamin A.
Refer patient to an ophthalmologist if ocular symptoms suggestive
of vitamin A deficiency occur.
ADVERSE REACTIONS
Most common adverse reactions (≥9% in WAINUA-treated patients)
were vitamin A decreased (15%) and vomiting (9%).
Please see link to U.S. Full Prescribing Information for
WAINUA.
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 Ionis.com and follow us on X (Twitter) and
LinkedIn.
Ionis' Forward-looking Statements
This press release includes forward-looking statements regarding
eplontersen, 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. WAINUA™ is a trademark of AstraZeneca
plc.
1 Mohamed-Salem L, et al. Prevalence of wild type
ATTR assessed as myocardial uptake in bone scan in the elderly
population. Int J Cardiol. 2018 Nov
1;270:192-196. doi: 10.1016/j.ijcard.2018.06.006.
2 Cuscaden C, et al. Estimation of prevalence of
transthyretin (ATTR) cardiac amyloidosis in an Australian
subpopulation using bone scans with echocardiography and clinical
correlation. J Nucl Cardiol. 2020 May
8. doi: 10.1007/s12350-020-02152-x.
3 Columbia University
Irving Medical Center [Internet]. Drug Reduces Death from
Underdiagnosed Form of Heart Failure [last accessed 5 February 2024]. Available
from: https://www.cuimc.columbia.edu/news/drug-reduces-deaths-underdiagnosed-form-heart-failure.
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SOURCE Ionis Pharmaceuticals, Inc.