− Type II Variation Submission Based on the
Positive HELIOS-B Phase 3 Study in which Vutrisiran Significantly
Reduced the Risk of Death and Cardiovascular Events Relative to
Placebo –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA
interference (RNAi) therapeutics company, today announced the
submission of a Type II Variation to the European Medicines Agency
(EMA) 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 in the European Union (EU)
for the treatment of hereditary transthyretin-mediated (hATTR)
amyloidosis in adult patients with stage 1 or stage 2
polyneuropathy.
“Today marks another important milestone in our journey to bring
RNAi therapeutics to patients with high unmet need around the
world,” said Pushkal Garg, M.D., Chief Medical Officer of Alnylam.
“ATTR-CM is a rapidly progressive, debilitating, and
life-threatening disease that is an increasingly recognized cause
of heart failure. Vutrisiran rapidly knocks down TTR, and in the
HELIOS-B study treatment with vutrisiran substantially reduced
all-cause mortality and cardiovascular events, underscoring the
potential of this therapy for those living with the disease. We
look forward to working closely with the EMA with the aim to bring
this new treatment option to patients as soon as possible.”
The regulatory application is based on positive results from the
pivotal HELIOS-B Phase 3, randomized, double-blind,
placebo-controlled multicenter global study which met all 10 of its
primary and secondary endpoints across both the overall and
monotherapy populations, each with statistical significance. The
findings demonstrated the effects of vutrisiran on outcomes of
mortality and cardiovascular events, as well as functional capacity
(6-minute walk test), quality of life (Kansas City Cardiomyopathy
Questionnaire), and heart failure symptoms and severity (NYHA
class) in patients with ATTR-CM. The safety profile of vutrisiran
in HELIOS-B was consistent with the established profile of the drug
for hATTR amyloidosis in adult patients with polyneuropathy. 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. Detailed results from the HELIOS-B
study were published in The New England Journal of Medicine.
A supplemental New Drug Application (sNDA) for vutrisiran has
been submitted to the U.S. Food and Drug Administration (FDA) for
the treatment of ATTR-CM. Additional regulatory submissions are
planned globally.
AMVUTTRA® (vutrisiran) INDICATION AND IMPORTANT SAFETY
INFORMATION
Indication In Europe and the UK, vutrisiran is indicated
for the treatment of hATTR amyloidosis in adult patients with stage
1 or stage 2 polyneuropathy.
Important Safety Information
Reduced Serum Vitamin A Levels and Recommended
Supplementation
Vutrisiran treatment leads to a decrease in serum vitamin A
levels. Supplementation of approximately, but not exceeding, 2500
IU to 3000 IU vitamin A per day is advised for patients taking
vutrisiran. 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 frequently occurring adverse reactions in patients
treated with vutrisiran were pain in extremity and arthralgia.
Other commonly reported adverse reactions with vutrisiran were
dyspnoea, injection site reaction and increase in blood alkaline
phosphatase.
For additional information about vutrisiran, please see the
full Summary of Product Characteristics.
About AMVUTTRA® (vutrisiran) AMVUTTRA® (vutrisiran) is an
RNAi therapeutic that delivers rapid knockdown of variant and
wild‑type transthyretin (TTR), addressing the underlying cause of
transthyretin (ATTR) amyloidosis. Administered quarterly via
subcutaneous injection, vutrisiran 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.
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.
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 and the
potential of vutrisiran to improve outcomes for patients with ATTR
amyloidosis with cardiomyopathy; the potential for vutrisiran to
obtain regulatory approval for the treatment of ATTR amyloidosis
with cardiomyopathy, in the EU or elsewhere, and the timing of any
such regulatory approval(s) 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/20241016045541/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) +1-617-682-4340 Josh Brodsky (Investors)
+1-617-551-8276 Emily Bunting (Media, Europe) +41 79 866 97 03
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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