– Filing Marks the First RNAi Therapeutic to be
Submitted for Potential Approval in Latin America –
– Submission Made Under the Accelerated
Regulatory Pathway for Rare Diseases –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that the marketing
authorization application of patisiran for the treatment of
patients with hereditary transthyretin-mediated (hATTR) amyloidosis
with polyneuropathy has been filed with the Brazilian Health
Regulatory Agency (ANVISA). Patisiran has been granted priority
review by ANVISA which is awarded to innovative medicines that
treat rare diseases under this accelerated regulatory pathway.
Alnylam expects a decision from ANVISA in the first half of
2020.
Patisiran – which will be commercialized following regulatory
approval under the brand name ONPATTRO® – is Alnylam’s first
investigational drug submitted for review in Brazil. If approved,
it will be the first product to be launched and marketed by Alnylam
in the country and the first ever RNAi therapeutic to be approved
in Latin America.
“The filing of our registration submission for patisiran in
Brazil is an important step forward in our continued commitment to
bring RNAi therapeutics to people around the world,” said Norton
Oliveira, Senior Vice President, Head of Latin America at Alnylam.
“hATTR amyloidosis is a rare progressive condition that is
considered endemic in Brazil, affecting more than 5,000 people.
Symptoms can manifest throughout the entire body and have a
devastating impact on patients. We look forward to working closely
with ANVISA to bring patisiran to these patients in need as quickly
as possible.”
The registration submission is based on positive data from the
APOLLO Phase 3 study, which evaluated the efficacy and safety of
patisiran in hATTR amyloidosis patients with polyneuropathy.
Results from the APOLLO study were published in the July 5, 2018
issue of The New England Journal of Medicine.
ONPATTRO is approved by the U.S. Food and Drug Administration
(FDA) for the treatment of the polyneuropathy of hATTR amyloidosis
in adults. ONPATTRO is also approved for this indication in Canada
and Japan, and is approved in the European Union and Switzerland
for the treatment of hATTR amyloidosis in adults with Stage 1 or
Stage 2 polyneuropathy.
About ONPATTRO® (patisiran) ONPATTRO is an RNAi
therapeutic that is approved by the U.S. Food and Drug
Administration (FDA) for the treatment of the polyneuropathy of
hATTR amyloidosis in adults. ONPATTRO is also approved in the
European Union and Switzerland for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, in
Canada for the treatment of hATTR amyloidosis with polyneuropathy
by Health Canada, and in Japan for the treatment of hATTR
amyloidosis with polyneuropathy by the Japanese Ministry of Health,
Labour and Welfare (MHLW). Patisiran is also being investigated in
patients with ATTR amyloidosis (hereditary [hATTR] or wild type
[wtATTR]) with cardiomyopathy in the APOLLO-B study. Based on Nobel
Prize-winning science, ONPATTRO is an intravenously administered
RNAi therapeutic targeting transthyretin (TTR) for the treatment of
hereditary ATTR amyloidosis. It is designed to target and silence
TTR messenger RNA, thereby blocking the production of TTR protein
before it is made. ONPATTRO blocks the production of TTR in the
liver, reducing its accumulation in the body’s tissues in order to
halt or slow down the progression of the disease.
Important Safety Information for ONPATTRO The most common
adverse reactions observed with ONPATTRO included infusion-related
reactions (IRRs) such as 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 prior to ONPATTRO infusion. If an IRR occurs,
consider slowing or interrupting the infusion and instituting
medical management as clinically indicated. In the case of a
serious or life-threatening IRR, the infusion should be
discontinued and not resumed. Because ONPATTRO reduces serum
vitamin A levels, patients should be advised to take the
recommended daily allowance (RDA) of vitamin A, and referred to an
ophthalmologist if they develop ocular symptoms suggestive of
vitamin A deficiency (e.g. night blindness).
About hATTR Amyloidosis Hereditary transthyretin
(TTR)-mediated amyloidosis (hATTR) is an inherited, progressively
debilitating, and often fatal disease caused by mutations in the
TTR gene. TTR protein is primarily produced in the liver and is
normally a carrier of vitamin A. Mutations in the TTR gene cause
abnormal amyloid proteins to accumulate and damage body organs and
tissue, such as the peripheral nerves and heart, resulting in
intractable peripheral sensory-motor neuropathy, autonomic
neuropathy, and/or cardiomyopathy, as well as other disease
manifestations. hATTR amyloidosis, represents a major unmet medical
need with significant morbidity and mortality affecting
approximately 50,000 people worldwide. The median survival is 4.7
years following diagnosis, with a reduced survival (3.4 years) for
patients presenting with cardiomyopathy.
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 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 Alnylam (Nasdaq: ALNY) is leading 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 genetic, cardio-metabolic, hepatic
infectious, and central nervous system (CNS)/ocular diseases. Based
on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of a wide
range of severe and debilitating diseases. Founded in 2002, Alnylam
is delivering on a bold vision to turn scientific possibility into
reality, with a robust discovery platform. Alnylam’s first
commercial RNAi therapeutic is ONPATTRO® (patisiran), approved in
the U.S., EU, Canada, and Japan. Alnylam has a deep pipeline of
investigational medicines, including five product candidates that
are in late-stage development. Looking forward, Alnylam will
continue to execute on its "Alnylam 2020" strategy of building a
multi-product, commercial-stage biopharmaceutical company with a
sustainable pipeline of RNAi-based medicines to address the needs
of patients who have limited or inadequate treatment options.
Alnylam employs over 1,200 people worldwide and 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 or on LinkedIn.
Alnylam Forward Looking Statements Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's expectations
regarding the review of its filing for marketing approval for
patisiran in Brazil by ANVISA and the expected timing of a decision
from ANVISA, the potential size of the patient population in Brazil
that could benefit from patisiran, if approved, and expectations
regarding its “Alnylam 2020” guidance for the advancement and
commercialization of RNAi therapeutics, 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, Alnylam's ability to discover and develop novel drug
candidates and delivery approaches, successfully demonstrate the
efficacy and safety of its product candidates, the pre-clinical and
clinical results for its product candidates, which may not be
replicated or continue to occur in other subjects or in additional
studies or otherwise support further development of product
candidates for a specified indication or at all, actions or advice
of regulatory agencies, which may affect the design, initiation,
timing, continuation and/or progress of clinical trials or result
in the need for additional pre-clinical and/or clinical testing,
delays, interruptions or failures in the manufacture and supply of
its product candidates, obtaining, maintaining and protecting
intellectual property, Alnylam's ability to enforce its
intellectual property rights against third parties and defend its
patent portfolio against challenges from third parties, obtaining
and maintaining regulatory approval, pricing and reimbursement for
products, progress in establishing a commercial and ex-United
States infrastructure, successfully launching, marketing and
selling its approved products globally, Alnylam’s ability to
successfully expand the indication for ONPATTRO in the future,
competition from others using technology similar to Alnylam's and
others developing or marketing products for similar uses, Alnylam's
ability to manage its growth and operating expenses, obtain
additional funding to support its business activities, and
establish and maintain strategic business alliances and new
business initiatives, Alnylam's dependence on third parties for
development, manufacture and distribution of products, the outcome
of litigation, the risk of 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 other filings that Alnylam makes
with the SEC. 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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version on businesswire.com: https://www.businesswire.com/news/home/20191010005075/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340
Josh Brodsky (Investors) 617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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