Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that NASDAQ has halted
trading of the Company’s common stock.
The U.S. Food and Drug Administration’s (FDA) Cardiovascular and
Renal Drugs Advisory Committee is meeting today to review the
supplemental New Drug Application for patisiran, an investigational
RNAi therapeutic in development for the treatment of the
cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
The Advisory Committee meeting is scheduled to begin at 9:00
a.m. ET today. The briefing materials can be found on the FDA
website here. The Company is not responsible for the content of,
nor the statements made in, the briefing materials prepared by the
FDA.
As previously announced, the FDA has set an action date of
October 8, 2023 under the Prescription Drug User Fee Act.
Patisiran is the established name for ONPATTRO®, which is
currently approved by the FDA for the treatment of the
polyneuropathy of hereditary ATTR amyloidosis in adults.
ONPATTRO Indication and Important Safety Information
Indication ONPATTRO is indicated for the treatment of the
polyneuropathy of hereditary transthyretin-mediated amyloidosis in
adults.
Important Safety Information Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients
treated with ONPATTRO. In a controlled clinical study, 19% of
ONPATTRO-treated patients experienced IRRs, compared to 9% of
placebo-treated patients. The most common symptoms of IRRs with
ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea,
and headache.
To reduce the risk of IRRs, patients should receive
premedication with a corticosteroid, acetaminophen, and
antihistamines (H1 and H2 blockers) at least 60 minutes prior to
ONPATTRO infusion. Monitor patients during the infusion for signs
and symptoms of IRRs. If an IRR occurs, consider slowing or
interrupting the infusion and instituting medical management as
clinically indicated. If the infusion is interrupted, consider
resuming at a slower infusion rate only if symptoms have resolved.
In the case of a serious or life-threatening IRR, the infusion
should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended Supplementation
ONPATTRO 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 ONPATTRO. Higher doses than the
RDA should not be given to try to achieve normal serum vitamin A
levels during treatment with ONPATTRO, as serum 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 ONPATTRO were upper respiratory
tract infections (29%) and infusion-related reactions (19%).
For additional information about ONPATTRO, please see the full
U.S. Prescribing Information.
About ONPATTRO® (patisiran) ONPATTRO (patisiran) is an
RNAi therapeutic that is approved in the United States and Canada
for the treatment of the polyneuropathy of hereditary ATTR (hATTR)
amyloidosis in adults. ONPATTRO is also approved in the European
Union, Switzerland and Brazil for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with
polyneuropathy. ONPATTRO is an intravenously administered RNAi
therapeutic targeting transthyretin (TTR). It is designed to target
and silence TTR messenger RNA, thereby reducing the production of
TTR protein before it is made. Reducing the pathogenic protein
leads to a reduction in amyloid deposits in tissues. Patisiran is
also being evaluated for the treatment of the cardiomyopathy of
transthyretin-mediated (ATTR) amyloidosis; the safety and efficacy
of patisiran in this indication have not been established or
evaluated by the FDA, EMA or any other health authority.
About ATTR Amyloidosis Transthyretin-mediated (ATTR)
amyloidosis 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
amyloidosis – hereditary ATTR (hATTR) amyloidosis, which is caused
by a TTR gene variant and affects approximately 50,000 people
worldwide, and wild-type ATTR (wtATTR) amyloidosis, which occurs
without a TTR gene variant and impacts an estimated 200,000 –
300,000 people worldwide.
About LNP Technology Alnylam has licenses to Arbutus
Biopharma LNP intellectual property for use in RNAi therapeutic
products using LNP technology.
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 Pharmaceuticals
(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,
expectations regarding Alnylam’s aspiration to become a leading
biotech company and the planned achievement of its “Alnylam P5x25”
strategy, the potential for Alnylam to identify new potential drug
development candidates and advance its research and development
programs, Alnylam’s ability to obtain approval for new commercial
products or additional indications for its existing products, the
action date by the FDA for patisiran and Alnylam’s projected
commercial and financial performance, 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: 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; Alnylam’s ability to successfully execute on its
“Alnylam P5x25” strategy; 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 Alnylam’s product candidates,
including patisiran and vutrisiran; actions or advice of regulatory
agencies and Alnylam’s ability to obtain and maintain regulatory
approval for its product candidates, including patisiran and
vutrisiran, as well as favorable pricing and reimbursement;
successfully launching, marketing and selling Alnylam’s approved
products globally; delays, interruptions or failures in the
manufacture and supply of Alnylam’s product candidates or its
marketed products; delays or interruptions in the supply of
resources needed to advance Alnylam’s research and development
programs, including as may arise from recent disruptions in the
supply of non-human primates; obtaining, maintaining and protecting
intellectual property; Alnylam’s ability to successfully expand the
indication for ONPATTRO or AMVUTTRA 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 Roche, Novartis, Sanofi, Regeneron and Vir; the
outcome of litigation; the risks of future government
investigations; and unexpected expenditures; as well as those risks
more fully discussed in the “Risk Factors” filed with Alnylam's
2022 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.
This release discusses investigational RNAi therapeutics and
uses of previously approved RNAi therapeutics in development and is
not intended to convey conclusions about efficacy or safety as to
those investigational therapeutics or uses. Patisiran has not been
approved by any regulatory agency for the treatment of ATTR
amyloidosis with cardiomyopathy. No conclusions can or should be
drawn regarding its safety or effectiveness in treating
cardiomyopathy in this population. There is no guarantee that any
investigational therapeutics or expanded uses of commercial
products will successfully complete clinical development or gain
health authority approval.
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version on businesswire.com: https://www.businesswire.com/news/home/20230913894129/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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