– PDUFA Date Set for April 14, 2022 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the U.S. Food and Drug
Administration (FDA) has accepted the Company’s New Drug
Application (NDA) for vutrisiran, an investigational RNAi
therapeutic for the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults. The FDA has
set an action date of April 14, 2022 under the Prescription Drug
User Fee Act (PDUFA), and the Agency has indicated that they are
not currently planning an advisory committee meeting as part of the
NDA review.
“We are excited that the FDA has accepted our NDA for
vutrisiran, which was based on 9-month results from the HELIOS-A
study. Today’s announcement marks another important milestone as we
work to make vutrisiran available to hATTR amyloidosis patients
with polyneuropathy,” said Rena Denoncourt, Vice President, TTR
Franchise Lead. “If approved, once-quarterly, subcutaneously
administered vutrisiran may represent a new treatment option that
potentially reverses polyneuropathy manifestations of disease.”
Vutrisiran has been granted Orphan Drug Designation in the U.S.
and the European Union (EU) for the treatment of ATTR amyloidosis.
Vutrisiran has also been granted a Fast-Track designation in the
U.S. for the treatment of the polyneuropathy of hATTR amyloidosis
in adults. The Company plans to also submit regulatory filings in
the EU, Brazil, and Japan in 2021 based on the HELIOS-A
results.
About Vutrisiran
Vutrisiran is an investigational, subcutaneously administered
RNAi therapeutic in development for the treatment of ATTR
amyloidosis, which encompasses both hereditary (hATTR) and
wild-type (wtATTR) amyloidosis. It is designed to target and
silence specific messenger RNA, blocking the production of
wild-type and variant transthyretin (TTR) protein before it is
made. Quarterly administration of vutrisiran may help to reduce
deposition and facilitate the clearance of TTR amyloid deposits in
tissues and potentially restore function to these tissues.
Vutrisiran utilizes Alnylam’s Enhanced Stabilization Chemistry
(ESC)-GalNAc-conjugate delivery platform, designed for increased
potency and high metabolic stability that may allow for infrequent
subcutaneous injections. The safety and efficacy of vutrisiran have
not been evaluated by the U.S. Food and Drug Administration,
European Medicines Agency or any other health authority.
About the HELIOS-A Phase 3 Study
HELIOS-A (NCT03759379) is a Phase 3 global, randomized,
open-label study to evaluate the efficacy and safety of vutrisiran.
The study enrolled 164 patients with hATTR amyloidosis with
polyneuropathy at 57 sites in 22 countries. Patients were
randomized 3:1 to receive either 25mg of vutrisiran (N=122) via
subcutaneous injection once every three months or 0.3 mg/kg of
patisiran (N=42) via intravenous infusion once every three weeks
(as a reference comparator) for 18 months. The efficacy results of
vutrisiran in HELIOS-A are compared to the placebo group from the
landmark APOLLO Phase 3 study, which evaluated the efficacy and
safety of patisiran in a patient population similar to that studied
in HELIOS-A. The primary endpoint is the change from baseline in
mNIS+7 score at nine months. Secondary endpoints at 9 months are
the change from baseline in the Norfolk QoL-DN score and the timed
10-MWT. Changes from baseline in NT-proBNP were evaluated as an
exploratory endpoint at nine months. Additional secondary endpoints
at 18 months will be evaluated in the HELIOS-A study, including
change from baseline in mNIS+7, Norfolk QoL-DN, 10-MWT, modified
body mass index (mBMI), Rasch-built Overall Disability Scale
(R-ODS), and serum transthyretin (TTR) levels. Additional
exploratory cardiac endpoint data at the 18-month time point will
be evaluated, including NT-proBNP, echocardiographic measures and
cardiac amyloid assessments with technetium scintigraphy imaging.
Following the 18-month treatment period, all patients are eligible
to receive vutrisiran for an additional 18 months as part of the
randomized treatment extension where they will receive either 25mg
vutrisiran once quarterly or 50mg vutrisiran once every six
months.
About hATTR Amyloidosis
Hereditary transthyretin-mediated (hATTR) amyloidosis is an
inherited, progressively debilitating, and fatal disease caused by
variants (i.e., mutations) in the TTR gene. TTR protein is
primarily produced in the liver and is normally a carrier of
vitamin A. Variants in the TTR gene cause abnormal amyloid proteins
to accumulate and damage body organs and tissue, such as the
peripheral nerves and heart, resulting in intractable peripheral
sensory-motor neuropathy, autonomic neuropathy, and/or
cardiomyopathy, as well as other disease manifestations. hATTR
amyloidosis, represents a major unmet medical need with significant
morbidity and mortality affecting approximately 50,000 people
worldwide. The median survival is 4.7 years following diagnosis,
with a reduced survival (3.4 years) for patients presenting with
cardiomyopathy.
About RNAi
RNAi (RNA interference) is a natural cellular process of gene
silencing that represents one of the most promising and rapidly
advancing frontiers in biology and drug development today. Its
discovery has been heralded as “a major scientific breakthrough
that happens once every decade or so,” and was recognized with the
award of the 2006 Nobel Prize for Physiology or Medicine. By
harnessing the natural biological process of RNAi occurring in our
cells, a new class of medicines, known as RNAi therapeutics, is now
a reality. Small interfering RNA (siRNA), the molecules that
mediate RNAi and comprise Alnylam’s RNAi therapeutic platform,
function upstream of today’s medicines by potently silencing
messenger RNA (mRNA) – the genetic precursors – that encode for
disease-causing proteins, thus preventing them from being made.
This is a revolutionary approach with the potential to transform
the care of patients with genetic and other diseases.
About Alnylam Pharmaceuticals
Alnylam (Nasdaq: ALNY) is leading the translation of RNA
interference (RNAi) into a whole new class of innovative medicines
with the potential to transform the lives of people afflicted with
rare genetic, cardio-metabolic, hepatic infectious, and central
nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning
science, RNAi therapeutics represent a powerful, clinically
validated approach for the treatment of a wide range of severe and
debilitating diseases. Founded in 2002, Alnylam is delivering on a
bold vision to turn scientific possibility into reality, with a
robust RNAi therapeutics platform. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), GIVLAARI®
(givosiran), OXLUMO® (lumasiran), and Leqvio® (inclisiran) being
developed and commercialized by Alnylam’s partner, Novartis.
Alnylam has a deep pipeline of investigational medicines, including
six product candidates that are in late-stage development. Alnylam
is executing on its “Alnylam P5x25” strategy to deliver
transformative medicines in both rare and common diseases
benefiting patients around the world through sustainable innovation
and exceptional financial performance, resulting in a leading
biotech profile. Alnylam is headquartered in Cambridge, MA. For
more information about our people, science and pipeline, please
visit www.alnylam.com and engage with us on Twitter at @Alnylam, on
LinkedIn, or on Instagram.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam’s
expectations, plans, aspirations, and goals, including those
related to the investigational therapeutic vutrisiran and its
potential as a low-dose, once quarterly, subcutaneously
administered treatment option with encouraging safety profile for
patients with hATTR amyloidosis with polyneuropathy that has the
potential to reverse polyneuropathy manifestations of disease, the
expected timing for FDA review of the NDA for vutrisiran, our
progress toward building in the future a franchise of medicines for
the treatment of ATTR amyloidosis, our aspiration to become a
leading biotech company, and the planned achievement of our
“Alnylam P5x25” strategy, constitute forward-looking statements for
the purposes of the safe harbor provisions under The Private
Securities Litigation Reform Act of 1995. Actual results and future
plans may differ materially from those indicated by these
forward-looking statements as a result of various important risks,
uncertainties and other factors, including, without limitation: the
direct or indirect impact of the COVID-19 global pandemic or any
future pandemic on Alnylam’s business, results of operations and
financial condition and the effectiveness or timeliness of
Alnylam’s efforts to mitigate the impact of the pandemic; Alnylam's
ability to discover and develop novel drug candidates and delivery
approaches and successfully demonstrate the efficacy and safety of
its product candidates; the pre-clinical and clinical results for
its product candidates; actions or advice of regulatory agencies
and Alnylam’s ability to obtain and maintain regulatory approval
for its product candidates, as well as favorable pricing and
reimbursement; successfully launching, marketing and selling its
approved products globally; delays, interruptions or failures in
the manufacture and supply of its product candidates or its
marketed products; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to successfully expand the
indication for ONPATTRO (or vutrisiran, if approved) in the future;
Alnylam's ability to manage its growth and operating expenses
through disciplined investment in operations and its ability to
achieve a self-sustainable financial profile in the future without
the need for future equity financing; Alnylam’s ability to maintain
strategic business collaborations; Alnylam's dependence on third
parties for the development and commercialization of certain
products, including Novartis, Regeneron and Vir; the outcome of
litigation; the risk of government investigations; and unexpected
expenditures; as well as those risks more fully discussed in the
“Risk Factors” filed with Alnylam's most recent Quarterly Report on
Form 10-Q filed with the Securities and Exchange Commission (SEC)
and in its other SEC filings. In addition, any forward-looking
statements represent Alnylam's views only as of today and should
not be relied upon as representing its views as of any subsequent
date. Alnylam explicitly disclaims any obligation, except to the
extent required by law, to update any forward-looking
statements.
This release is not intended to convey conclusions about
efficacy or safety as to any investigational uses or dosing
regimens of any investigational RNAi therapeutics. There is no
guarantee that any investigational therapeutics or dosing regimens
for such therapeutics will successfully complete clinical
development or gain health authority approval.
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version on businesswire.com: https://www.businesswire.com/news/home/20210624005230/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Josh Brodsky
(Investors) 617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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