- U.S. FDA approval based on Phase 3 NEURO-TTRansform results
showing WAINUA demonstrated consistent and sustained benefit
halting neuropathy disease progression and improving neuropathy
impairment and quality of life
- Additional regulatory reviews for WAINUA underway in rest of
world
- WAINUA will be available in the U.S. in January 2024
CARLSBAD, Calif., Dec. 21,
2023 /PRNewswire/ -- Ionis Pharmaceuticals, Inc.
(Nasdaq: IONS) announced today that the U.S. Food and Drug
Administration (FDA) has approved Ionis and AstraZeneca's WAINUA™
(eplontersen) for the treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis in adults, commonly referred to
as hATTR-PN or ATTRv-PN. WAINUA is the only approved medicine for
the treatment of ATTRv-PN that can be self-administered via an
auto-injector.
The approval was based on the positive 35-week interim analysis
from the Phase 3 NEURO-TTRansform study which showed patients
treated with WAINUA demonstrated consistent and sustained benefit
on the co-primary endpoints of serum transthyretin (TTR)
concentration and neuropathy impairment measured by modified
Neuropathy Impairment Score +7 (mNIS+7), and key secondary endpoint
of quality of life (QoL) on the Norfolk Quality of Life
Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN). Positive
results from the Phase 3 NEURO-TTRansform study were published in
The Journal of the American Medical Association
(JAMA) further demonstrating the benefit of WAINUA across
the spectrum of ATTRv-PN at 35, 66 and 85-weeks.
"Many people living with hereditary transthyretin-mediated
amyloid polyneuropathy are unable to fully enjoy their lives
because of the relentless, progressive and debilitating effects of
the disease," said Michael J.
Polydefkis, M.D., professor of neurology at Johns Hopkins University School of Medicine and an
investigator in the NEURO-TTRansform study. "Approval of WAINUA
represents a meaningful advancement in treatment, one that gives
those who are living with transthyretin-mediated amyloid
polyneuropathy help managing the disease."
ATTRv-PN is a debilitating disease that leads to peripheral
nerve damage with motor disability within five years of diagnosis
and, without treatment, is generally fatal within a decade. WAINUA
is a ligand-conjugated antisense oligonucleotide (LICA) medicine
designed to reduce the production of TTR protein at its source.
"The FDA approval of WAINUA marks an important milestone for
people living with hereditary transthyretin-mediated amyloid
polyneuropathy, who will now have an effective, well-tolerated
treatment that can be self-administered via auto-injector to combat
this devastating disease," said Brett P.
Monia, Ph.D., chief executive officer at Ionis. "It is also
a pivotal moment for Ionis as WAINUA will be the first in a steady
cadence of potential commercial launches for the company. We are
proud to have discovered and, together with AstraZeneca, developed
WAINUA, and are grateful to the patients, caregivers and
investigators who participated in our clinical studies, as well as
for the dedication of our scientists and researchers."
"People with hereditary transthyretin-mediated amyloid
polyneuropathy, and other forms of amyloidosis, are often
misdiagnosed since symptoms can mirror other conditions," said
Isabelle Lousada, President and CEO,
Amyloidosis Research Consortium "The path to getting an
accurate diagnosis can often be a long, arduous journey and it is
critical that a timely and accurate diagnosis is made not only for
the individual experiencing symptoms but for their families and
loved ones. It is exciting to see new innovations coming through
and increased efforts to raise awareness in an area that has often
been overlooked or neglected."
WAINUA will be available in the U.S. in January 2024.
"There is an urgent medical need for new therapies for people
living with hereditary transthyretin-mediated amyloid
polyneuropathy," said Ruud Dobber, executive vice president,
BioPharmaceuticals Business Unit, AstraZeneca. "The U.S. approval
of WAINUA offers a new treatment option that provides consistent
and sustained reduction in serum TTR concentration compared to
baseline while halting disease progression and improving quality of
life for people living with this debilitating condition."
As part of a global development and
commercialization agreement, AstraZeneca and Ionis will
commercialize WAINUA for the treatment of ATTRv-PN in the U.S. and
are seeking regulatory approval in Europe and other parts of the world. This
agreement was recently expanded to include exclusive rights for
AstraZeneca to commercialize WAINUA in Latin America in addition to all other
countries outside the U.S. WAINUA was granted Orphan Drug
Designation in the U.S. and in the EU for the treatment of
transthyretin-mediated amyloidosis (ATTR).
Eplontersen is currently being evaluated in the Phase 3
CARDIO-TTRansform study for transthyretin-mediated amyloid
cardiomyopathy (ATTR-CM), a systemic, 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.
About WAINUA™ (eplontersen)
WAINUA™ (eplontersen) is a
ligand-conjugated antisense (LICA) medicine designed to inhibit the
production of transthyretin, or TTR protein. WAINUA has been
approved in the U.S. for the treatment of the polyneuropathy of
hereditary transthyretin-mediated amyloidosis in adults (also
referred to as ATTRv-PN). Please see full Prescribing
Information
INDICATION for WAINUATM
(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 WAINUATM
(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 Hereditary Transthyretin-mediated Amyloid
Polyneuropathy (ATTRv-PN)
ATTRv-PN is caused by the accumulation of misfolded mutated TTR
protein in the peripheral nerves. Patients with ATTRv-PN experience
ongoing debilitating nerve damage throughout their body resulting
in the progressive loss of motor functions, such as walking. These
patients also accumulate TTR in other major organs, which
progressively compromises their function. The damage from misfolded
TTR protein accumulation leads to disability within five years of
diagnosis and is generally fatal within a decade.
About the NEURO-TTRansform Study
NEURO-TTRansform is a global, open-label, randomized trial
evaluating the efficacy and safety of eplontersen in patients with
ATTRv-PN at week 35, week 66 and week 85. The final analysis
comparing eplontersen to an external placebo group was completed at
week 66. All patients were then followed on treatment until week 85
and evaluated four weeks after the last dose in an end-of-trial
assessment. Following treatment and the end-of-trial assessments,
patients were eligible to enter an open-label extension study to
continue receiving eplontersen once every four weeks or enter a
20-week post-treatment evaluation period.
About Ionis Pharmaceuticals, Inc.
For more than 30 years, Ionis has been a leader in RNA-targeted
therapy, pioneering new markets and changing standards of care.
Ionis currently has four marketed medicines and a promising
late-stage pipeline highlighted by 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
WAINUA, Ionis' technologies 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 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 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" all refer to Ionis
Pharmaceuticals and its subsidiaries.
Ionis Pharmaceuticals® is a registered trademark of
Ionis Pharmaceuticals, Inc. WAINUA™ is a trademark of AstraZeneca
plc.
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SOURCE Ionis Pharmaceuticals, Inc.