− Prescription Drug User Fee Act (PDUFA) Date
Set for March 23, 2025 −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that the U.S. Food and Drug
Administration (FDA) has accepted for review the Company’s
supplemental New Drug Application (sNDA) for vutrisiran, an
investigational RNAi therapeutic in development for the treatment
of ATTR amyloidosis with cardiomyopathy (ATTR-CM). Based on the
Company’s use of a Priority Review Voucher, the FDA has set an
action date goal of March 23, 2025, under the Prescription Drug
User Fee Act (PDUFA). The FDA has informed the Company that it is
not planning to hold an advisory committee meeting at this time to
review the application.
Vutrisiran is the generic name for AMVUTTRA®, which is currently
approved by the FDA for the treatment of the polyneuropathy of
hereditary transthyretin-mediated amyloidosis (hATTR-PN) in adults.
By rapidly knocking down both mutant and wild‑type transthyretin
(TTR), vutrisiran addresses the underlying cause of transthyretin
amyloidosis (ATTR). If approved, vutrisiran would become the first
therapeutic approved in the U.S. to treat both the polyneuropathy
manifestations of hATTR and cardiomyopathy manifestations of ATTR
amyloidosis.
“We are pleased that the FDA has accepted our sNDA for
vutrisiran for the treatment of ATTR with cardiomyopathy – a
steadily progressing, debilitating and ultimately fatal disease,”
said Pushkal Garg, M.D., Chief Medical Officer of Alnylam. “In
HELIOS-B, treatment with vutrisiran improved cardiovascular
outcomes, survival, disease progression and quality of life, as
compared to placebo, in a population reflective of today’s patients
on substantial background treatment. We look forward to working
with the FDA to support their review of the application and bring
vutrisiran to patients with ATTR-CM in the U.S. early next
year.”
The supplemental application to the FDA was based on positive
results from HELIOS-B, a randomized, double-blind,
placebo-controlled multicenter global Phase 3 study in patients
with ATTR-CM. 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.
These treatment effects were seen on top of substantial background
standard of care treatments. Vutrisiran demonstrated encouraging
safety and tolerability consistent with the established profile of
the drug. Detailed results from the study were presented at the
European Society of Cardiology Congress and simultaneously
published in The New England Journal of Medicine on August 30,
2024.
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 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 U.S. 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.
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 or disease pathway 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) 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’s commercial
RNAi therapeutic products are ONPATTRO® (patisiran), AMVUTTRA®
(vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and
Leqvio® (inclisiran), which is being developed and commercialized
by Alnylam’s partner, Novartis. 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, including
the safety, tolerability and favorable effects of vutrisiran on
outcomes of death and cardiovascular events, functional capacity
and quality of life in patients with ATTR-CM; whether the FDA will
convene an advisory committee in connection with the FDA’s review
of Alnylam’s supplemental application for approval of vutrisiran
for the treatment of patients with ATTR-CM; the potential for
vutrisiran to obtain FDA approval for the treatment of patients
with ATTR-CM, and the anticipated timing of such FDA approval; and
the potential for Alnylam to achieve its Alnylam P5x25 vision of
becoming a leading biopharma company 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.
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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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