− sNDA is Based on Full Findings from the
Positive HELIOS-B Phase 3 Study –
− Priority Review Voucher Utilized to
Accelerate Review Period –
− Alnylam to Host and Webcast TTR Investor Day
Event Today in New York City –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced the submission of its
supplemental New Drug Application (sNDA) to the U.S. Food and Drug
Administration (FDA) for vutrisiran, an investigational RNAi
therapeutic in development for the treatment of ATTR amyloidosis
with cardiomyopathy (ATTR-CM). Vutrisiran is the generic name for
AMVUTTRA®, which is currently approved by the U.S. FDA for the
treatment of the polyneuropathy of hereditary ATTR amyloidosis in
adults. As part of the submission, the Company utilized a Priority
Review Voucher, which obligates the FDA to an accelerated review
timeline.
“We are proud that with this first regulatory submission, we are
a significant step closer to bringing vutrisiran to patients with
ATTR amyloidosis with cardiomyopathy, which is a steadily
progressive, debilitating, and ultimately fatal disease. HELIOS-B
demonstrated rapid knockdown of TTR with vutrisiran and an
improvement in death and cardiovascular events, as well as delays
in disease progression, with vutrisiran as compared to placebo,
with consistent effects across all pre-specified subgroups,” said
Pushkal Garg, M.D., Chief Medical Officer, Alnylam. “We believe
that, pending regulatory approval, vutrisiran has the potential to
become a first-line therapy for ATTR amyloidosis with
cardiomyopathy. We look forward to working with the FDA over the
coming months on this application to bring this medicine to
patients as rapidly as possible. Additional filings in other
geographies are underway.”
The application to the FDA was based on positive results from
HELIOS-B, a Phase 3, randomized, double-blind, placebo-controlled
multicenter global study in patients with ATTR-CM with substantial
background use of other effective therapies. The study demonstrated
favorable effects of vutrisiran on outcomes of death and
cardiovascular events, functional capacity and quality of life in
patients with ATTR-CM. The safety profile of vutrisiran in HELIOS-B
was consistent with the established profile of the drug. In
HELIOS-B, rates of adverse events (AEs), serious AEs, severe AEs,
and AEs leading to study drug discontinuation were similar between
the vutrisiran and placebo arms.
Today, the Company will host its TTR Investor Day starting at
8:30 am ET in New York City. The event will feature presentations
from the Company’s management team, including senior leaders from
the commercial organization who will discuss launch preparation and
highlight the potential for market leadership in ATTR-CM. The event
will be webcast on the Investors section of the Company’s website,
www.alnylam.com. A replay will be available on the Alnylam website
within 48 hours after the event.
HELIOS-B Study Design
HELIOS-B (NCT: NCT04153149) was a Phase 3, randomized,
double-blind, placebo-controlled multicenter global study designed
and powered to evaluate the efficacy and safety of vutrisiran on
the reduction of all-cause mortality and recurrent cardiovascular
events as a primary composite endpoint in patients with ATTR
amyloidosis with cardiomyopathy. The study randomized 655 adult
patients with ATTR amyloidosis (hereditary or wild-type) with
cardiomyopathy. Patients were randomized 1:1 to receive vutrisiran
25mg or placebo subcutaneously once every three months during a
double-blind treatment period of up to 36 months. After the
double-blind period, all eligible patients remaining on the study
to were able receive vutrisiran in an open-label extension period
of HELIOS-B.
AMVUTTRA® (vutrisiran) U.S. Indication and Important Safety
Information
Indication
AMVUTTRA is indicated for the treatment of the polyneuropathy of
hereditary transthyretin-mediated amyloidosis in adults.
Important Safety Information
Reduced Serum Vitamin A Levels and Recommended
Supplementation
AMVUTTRA® (vutrisiran) treatment leads to a decrease in serum
vitamin A levels. Supplementation at the recommended daily
allowance (RDA) of vitamin A is advised for patients taking
AMVUTTRA. Higher doses than the RDA should not be given to try to
achieve normal serum vitamin A levels during treatment with
AMVUTTRA, as serum vitamin A levels do not reflect the total
vitamin A in the body.
Patients should be referred to an ophthalmologist if they
develop ocular symptoms suggestive of vitamin A deficiency (e.g.,
night blindness).
Adverse Reactions
The most common adverse reactions that occurred in patients
treated with AMVUTTRA were pain in extremity (15%), arthralgia
(11%), dyspnea (7%), and vitamin A decreased (7%).
For additional information about AMVUTTRA, please see the
full Prescribing Information.
About AMVUTTRA® (vutrisiran)
AMVUTTRA® (vutrisiran) is an RNAi therapeutic that delivers
rapid knockdown of mutant and wild-type transthyretin (TTR),
addressing the underlying cause of transthyretin (ATTR)
amyloidosis. Administered quarterly via subcutaneous injection,
AMVUTTRA is approved and marketed in more than 15 countries for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated amyloidosis (hATTR-PN) in adults. Vutrisiran
is also in development for the treatment of ATTR amyloidosis with
cardiomyopathy (ATTR-CM), which encompasses both wild-type and
hereditary forms of the disease. For more information about
AMVUTTRA, including the full U.S. Prescribing Information,
visit AMVUTTRA.com.
About ATTR
Transthyretin amyloidosis (ATTR) is an underdiagnosed, rapidly
progressive, debilitating and fatal disease caused by misfolded
transthyretin (TTR) proteins, which accumulate as amyloid deposits
in various parts of the body, including the nerves, heart and
gastrointestinal tract. Patients may present with polyneuropathy,
cardiomyopathy or both manifestations of disease. There are two
different forms of ATTR – hereditary ATTR (hATTR), which is caused
by a TTR gene variant and affects approximately 50,000 people
worldwide, and wild-type ATTR (wtATTR), which occurs without a TTR
gene variant and impacts an estimated 200,000-300,000 people
worldwide.1-4
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.5 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.6 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 or
disease pathway proteins – thus preventing them from being made.5
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) has led 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 and prevalent diseases with unmet need. Based on Nobel
Prize-winning science, RNAi therapeutics represent a powerful,
clinically validated approach yielding transformative medicines.
Since its founding in 2002, Alnylam has led the RNAi Revolution and
continues to deliver on a bold vision to turn scientific
possibility into reality. Alnylam has a deep pipeline of
investigational medicines, including multiple 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 X (formerly Twitter) at @Alnylam, or
on LinkedIn, Facebook, or Instagram.
Alnylam Forward-Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. All statements
other than historical statements of fact regarding Alnylam’s
expectations, beliefs, goals, plans or prospects including, without
limitation, Alnylam’s expectations regarding the safety and
efficacy of vutrisiran for the treatment of ATTR amyloidosis with
cardiomyopathy; the potential for vutrisiran to obtain regulatory
approval for the treatment of ATTR amyloidosis with cardiomyopathy;
the safety and efficacy of vutrisiran for the treatment of ATTR
amyloidosis with cardiomyopathy, the potential of vutrisiran to
including its potential to become a first-line therapy and a
market-leading therapy for patients with ATTR amyloidosis with
cardiomyopathy,; and Alnylam’s view with respect to the timing of
regulatory review potential for a reduced review timeline by the
FDA and any resulting approval(s) of vutrisiran for the treatment
of ATTR amyloidosis in the U.S. and other countries Alnylam’s
global regulatory submissions for vutrisiran should be considered
forward-looking statements. 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, risks and
uncertainties relating to: Alnylam’s ability to successfully
execute on its “Alnylam P5x25” strategy; Alnylam’s ability to
successfully demonstrate the efficacy and safety of its product
candidates; the pre-clinical and clinical results for Alnylam’s
product candidates, including vutrisiran; actions or advice of
regulatory agencies and Alnylam’s ability to obtain regulatory
approval for its product candidates, including vutrisiran, as well
as favorable pricing and reimbursement; successfully launching,
marketing and selling Alnylam’s approved products globally; and any
delays, interruptions or failures in the manufacture and supply of
Alnylam’s product candidates or its marketed products; as well as
those risks more fully discussed in the “Risk Factors” filed with
Alnylam’s 2023 Annual Report on Form 10-K filed with the Securities
and Exchange Commission (SEC), as may be updated from time to time
in Alnylam’s subsequent Quarterly Reports on Form 10-Q 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.
1
Hawkins PN, Ando Y, Dispenzeri A, et al.
Ann Med. 2015;47(8):625-638.
2
Gertz MA. Am J Manag Care.
2017;23(7):S107-S112.
3
Conceicao I, Gonzalez-Duarte A, Obici L,
et al. J Peripher Nerv Syst. 2016;21:5-9.
4
Ando Y, Coelho T, Berk JL, et al. Orphanet
J Rare Dis. 2013;8:31.
5
Elbashir SM, Harborth J, Lendeckel W, et
al. Nature. 2001;411(6836):494-498.
6
Zamore P. Cell. 2006;127(5):1083-1086.
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version on businesswire.com: https://www.businesswire.com/news/home/20241009556041/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) +1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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