– sNDA Submission is Based on Findings from the
APOLLO-B Phase 3 Study That Showed Patisiran Demonstrated
Significant Improvement on Functional Capacity, Health Status and
Quality of Life Compared to Placebo at Month 12 –
– Study Also Demonstrated Encouraging Safety
Profile in Patients with Cardiomyopathy of ATTR Amyloidosis –
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 patisiran, an investigational RNAi
therapeutic in development for the treatment of
transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy.
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.
“We are excited by today’s announcement as the submission of our
sNDA brings us one step closer to making patisiran available to
patients with ATTR amyloidosis with cardiomyopathy. Patisiran has
the potential to improve the functional capacity and quality of
life of patients with ATTR amyloidosis with cardiomyopathy, an
increasingly recognized cause of heart failure for which there are
limited treatment options,” said Rena N. Denoncourt, Vice
President, TTR Franchise Lead. “The positive results of the
APOLLO-B Phase 3 study of patisiran validate the therapeutic
hypothesis that TTR silencing by an RNAi therapeutic may be an
effective approach to treating cardiomyopathy of both wild-type and
hereditary ATTR amyloidosis. This is an important milestone as we
work to build an industry-leading franchise for the treatment of
ATTR amyloidosis.”
The application to the FDA was based on positive results from
APOLLO-B, a Phase 3, randomized, double-blind, placebo-controlled
multicenter global study that demonstrated the effects of patisiran
on functional capacity and quality of life in patients with ATTR
amyloidosis with cardiomyopathy. The safety profile in APOLLO-B was
consistent with what was observed in APOLLO and in postmarketing
use of ONPATTRO. In APOLLO-B, the majority of adverse events (AEs)
were mild or moderate in severity. The 12-month results from the
study were presented at the 18th International Symposium on
Amyloidosis (ISA) on September 8, 2022.
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.
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 (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),
GIVLAARI® (givosiran), OXLUMO® (lumasiran), AMVUTTRA® (vutrisiran)
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 Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam’s views with
respect to the safety and efficacy of patisiran for the treatment
of ATTR amyloidosis with cardiomyopathy, the potential of treatment
with patisiran to address multiple aspects of ATTR amyloidosis,
including cardiac manifestations of disease, Alnylam’s commitment
to develop impactful RNAi therapeutic options for patients living
with all forms of ATTR amyloidosis, and Alnylam’s aspiration to
become a leading biotech company and the planned achievement of its
“Alnylam P5x25” strategy, constitute forward-looking statements for
the purposes of the safe harbor provisions under The Private
Securities Litigation Reform Act of 1995. 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; the
potential impact of the January 2022 leadership transition on
Alnylam’s ability to attract and retain talent and 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 its 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 its approved products
globally; delays, interruptions or failures in the manufacture and
supply of its product candidates or its marketed products;
obtaining, maintaining and protecting intellectual property;
Alnylam’s ability to successfully expand the indication for
ONPATTRO, AMVUTTRA or OXLUMO 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 most recent
Quarterly Report on Form 10-Q filed with the Securities and
Exchange Commission (SEC) 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/20221208005974/en/
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
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