− ONPATTRO is the First RNAi Therapeutic
Approved in Japan –
− Approval Marks the Arrival of an Entirely New
Treatment Approach for People Living with hATTR Amyloidosis with
Polyneuropathy in Japan –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, today announced that the Japanese
Ministry of Health, Labour and Welfare (MHLW) has approved
ONPATTRO® (patisiran) for the treatment of hereditary
transthyretin-mediated (hATTR) amyloidosis with polyneuropathy.
ONPATTRO is the first approved RNAi therapeutic in Japan and
Alnylam will launch and directly market it in the country.
“hATTR amyloidosis is a genetic disease that can cause a variety
of debilitating symptoms and can often be fatal. Historically,
treatment options for people living with this condition in Japan,
where the V30M mutation of the condition is prevalent, were
limited,” said Professor Yukio Ando, President of ISA
(International Society of Amyloidosis) and JSA (Japan Society of
Amyloidosis), Chairman and Professor/Department of Amyloidosis
Research of Nagasaki International University and Professor
Emeritus and Visiting Professor of Kumamoto University, Japan. “The
approval of ONPATTRO, a treatment that has the potential to
mitigate and potentially reverse symptoms of polyneuropathy and to
improve quality of life, marks a paradigm shift in the way we
approach and treat this serious disease.”
“We are very excited about the approval of ONPATTRO and are
proud to be bringing this important new treatment to patients with
hATTR amyloidosis with polyneuropathy in Japan,” said Masako
Nakamura, Senior Vice President, Head of Asia, Alnylam. “With this
approval, we look forward to working with the hATTR amyloidosis
community to continue to raise disease awareness, increase
diagnosis rates and ultimately provide treatment to patients
suffering from this devastating disease. This is a significant
milestone in our efforts to bring RNAi therapeutics to people
around the world and we look forward to continuing to build our
presence in Asia.”
Alnylam submitted a New Drug Application (NDA) to Japan’s
Pharmaceuticals and Medical Devices Agency (PMDA) on September 27,
2018 based on the results 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. Patisiran had previously received orphan drug designation
from the MHLW, which made it eligible for priority review as well
as 10 years of market exclusivity in Japan.
About hATTR AmyloidosisHereditary 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 RNAiRNAi (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 ONPATTRO® (Patisiran)Patisiran, based
on Nobel Prize-winning science, 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. Patisiran 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. In August 2018,
Patisiran received U.S. Food and Drug Administration (FDA) approval
for the treatment of the polyneuropathy of hATTR amyloidosis in
adults, as well as European Medicines Agency marketing
authorization for the treatment of hATTR amyloidosis in adults with
Stage 1 or Stage 2 polyneuropathy. Patisiran was approved in Japan
in June 2019 for the treatment of hereditary transthyretin-mediated
(hATTR) amyloidosis with polyneuropathy.
IMPORTANT SAFETY INFORMATIONONPATTRO is a medicine that
treats the polyneuropathy caused by an illness called hereditary
transthyretin-mediated amyloidosis (hATTR amyloidosis). ONPATTRO is
used in adults only.
Infusion-Related ReactionsInfusion-related reactions (IRRs) have
been observed in patients treated with ONPATTRO. In a controlled
clinical study, 19 percent of ONPATTRO-treated patients experienced
IRRs, compared to 9 percent 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, paracetamol, 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
SupplementationONPATTRO 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 ReactionsThe most common adverse reactions that occurred
in patients treated with ONPATTRO were respiratory-tract infection
(29 percent) and infusion-related reactions (19 percent).
About Alnylam PharmaceuticalsAlnylam (Nasdaq: ALNY) is
leading the translation of RNA interference (RNAi) into a 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/ocular diseases. Based on
Nobel Prizewinning science, RNAi therapeutics represent a powerful,
clinically validated approach for the treatment of diseases with
high unmet need. ONPATTRO® (patisiran) is the first-ever RNAi
therapeutic approved by the U.S. FDA for the treatment of the
polyneuropathy of hereditary transthyretin-mediated (hATTR)
amyloidosis in adults and by the EMA for the treatment of hATTR
amyloidosis in adults with stage 1 or stage 2 polyneuropathy.
Alnylam has a deep pipeline of investigational medicines, including
five product candidates in Phase 3 studies and one in registration.
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. Headquartered in Cambridge, MA,
Alnylam employs over 1,200 people worldwide. 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 StatementsVarious statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's plans to launch
ONPATTRO (patisiran) in Japan, plans to increase awareness of hATTR
amyloidosis, increase diagnosis rates and increase Alnylam’s
presence in Japan, 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/20190618005119/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)617-682-4340Josh
Brodsky(Investors)617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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