Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that the U.S. Food and Drug
Administration (FDA) has set a date of September 13, 2023 for the
meeting of the Cardiovascular and Renal Drugs Advisory Committee to
review the supplemental New Drug Application for patisiran, an
investigational treatment for the cardiomyopathy of
transthyretin-mediated (ATTR) amyloidosis. As previously announced,
the FDA has set an action date of October 8, 2023 under the
Prescription Drug User Fee Act. An advanced display of the public
notice is available in the Federal Register here.
Patisiran is the established name for ONPATTRO®, which is
currently approved by the U.S. 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 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 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 a rare, rapidly progressive, debilitating
disease caused by misfolded transthyretin (TTR) proteins which
accumulate as amyloid fibrils in multiple tissues including the
nerves, heart, and gastrointestinal (GI) tract. There are two
different types of ATTR amyloidosis – Hereditary ATTR (hATTR)
amyloidosis, caused by a TTR gene variant, and Wild-type ATTR
(wtATTR) amyloidosis, which occurs without a TTR gene variant.
hATTR amyloidosis affects approximately 50,000 people worldwide,
while wtATTR amyloidosis is estimated to impact 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 20 years ago, 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 Twitter at @Alnylam, on LinkedIn, or on
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, 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 and the effectiveness or timeliness of
Alnylam’s efforts to mitigate the impact of the pandemic; 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 Novartis, Sanofi,
Regeneron and Vir; the outcome of litigation; the potential impact
of a current government investigation and the risk 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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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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