Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, and Medison Pharma, Israel’s leading
commercial partner for innovative pharmaceuticals, announced today
an exclusive agreement to commercialize ONPATTRO®, the first-ever
commercialized RNAi therapeutic, as well as other investigational
therapeutics under development in the Alnylam RNAi portfolio.
“Our partnership with Medison marks an important step in our
global commercial expansion and signals our intent to ensure that
patients suffering from serious rare diseases have access to our
medicines, regardless of location,” said Theresa Heggie, SVP and
Head of Europe, Middle East and Africa, and Canada, Alnylam
Pharmaceuticals. “Medison has a strong organization with a proven
track record of commercializing orphan products successfully,
together with an infrastructure uniquely suited to supporting
patients suffering from rare diseases in Israel and providing
access to our potentially transformational therapies. We look
forward to partnering with Medison to bring ONPATTRO and potential
future therapies to patients.”
“We are proud to partner and collaborate with Alnylam in
Israel,” said Meir Jakobsohn, Founder and CEO, Medison Pharma.
“Alnylam’s portfolio of ONPATTRO and potentially ground-breaking
medicines in late stage development will strengthen our rare
disease portfolio, fulfilling Medison’s vision to provide
innovative treatments to patients in Israel. Patients with hATTR
amyloidosis with polyneuropathy in Israel deserve to have the
earliest possible access to novel new treatments and we look
forward to making this a reality, beginning with ONPATTRO. Patients
and physicians in Israel are waiting for potentially disease
modifying treatments and we will do everything in our capabilities
to secure their access.”
The agreement between Alnylam and Medison includes ONPATTRO,
approved in the EU in August 2018 for the treatment of hATTR
amyloidosis in adults with stage 1 or stage 2 polyneuropathy;
givosiran, a late-stage investigational RNAi therapeutic for the
treatment of acute hepatic porphyria (AHP); and lumasiran, a
late-stage investigational RNAi therapeutic for the treatment of
Primary Hyperoxaluria Type 1 (PH1). These medicines are not
currently approved for use in Israel and givosiran and lumasiran
have not yet been approved by any regulatory authority.
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 specific messenger RNA, potentially
blocking the production of TTR protein before it is made. Patisiran
blocks the production of transthyretin 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.
Important Safety Information (ISI) for ONPATTRO
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients
treated with patisiran. In a controlled clinical study, 19% of
patisiran-treated patients experienced IRRs, compared to 9% of
placebo-treated patients. The most common symptoms of IRRs with
patisiran were flushing, back pain, nausea, abdominal pain,
dyspnoea, 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
patisiran 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
Patisiran treatment leads to a decrease in serum vitamin A
levels. Patients receiving patisiran should take oral
supplementation of approximately 2500 IU vitamin A per day to
reduce the potential risk of ocular toxicity due to vitamin A
deficiency. Doses higher than 2500 IU vitamin A per day should
not be given to try to achieve normal serum vitamin A levels during
treatment with patisiran, 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. including reduced night vision or night
blindness, persistent dry eyes, eye inflammation, corneal
inflammation or ulceration, corneal thickening or corneal
perforation).
Adverse Reactions
The most common adverse reactions that occurred in patients
treated with patisiran were peripheral oedema (30%) and
infusion-related reactions (19%).
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 new class of innovative medicines with
the potential to improve 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. ONPATTRO® (patisiran) lipid complex
injection, available in the U.S. for the treatment of the
polyneuropathy of hereditary transthyretin-mediated (hATTR)
amyloidosis in adults, is Alnylam’s first U.S. FDA-approved
RNAi therapeutic. In the EU, ONPATTRO is approved for the treatment
of hATTR amyloidosis in adults with stage 1 or stage 2
polyneuropathy. Alnylam has a deep pipeline of investigational
medicines, including three 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 1,000 people worldwide and is headquartered in Cambridge,
MA.
About Medison
Medison, Israel’s leading innovative pharmaceutical partner, is
an exclusive Israeli partner for global healthcare companies such
as Amgen, Biogen, Ipsen, Vertex and more. Backed by three
generations of experience in the healthcare industry since 1937,
Medison has built and maintained long-standing relations with HMOs,
local medical centers and physicians. Medison is uniquely qualified
to provide the complete spectrum of integrated services for
international companies looking to enter or expand their presence
in the Israeli market. Medison runs Medison Ventures, a corporate
arm with a dedicated research team boasting deep scientific and
commercial backgrounds. Medison Ventures operates a scouting
program to cater its partners and is an active investor in life
science projects around drug development and digital health.
Alnylam Forward Looking StatementsVarious statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's views with
respect to the potential for RNAi therapeutics, including without
limitation ONPATTRO, givosiran and lumasiran, plans to make
ONPATTRO available in additional countries and to continue the
filings for regulatory approval and launch of ONPATTRO for hATTR
patients with polyneuropathy around the world, 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 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.
With the exception of ONPATTRO (patisiran), none of Alnylam’s
investigational therapeutics have been approved by the U.S.
Food and Drug Administration, European Medicines Agency, or
any other regulatory authority and no conclusions can or should be
drawn regarding the safety or effectiveness of such investigational
therapies.
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version on businesswire.com: https://www.businesswire.com/news/home/20190121005029/en/
Alnylam Pharmaceuticals, Inc.Christine Regan
Lindenboom(Investors and Media)+1-617-682-4340
Fiona McMillan(Media, Europe)+44 1628 244960
Medison PharmaGil GurfinkelVice President, Corporate
Development+972 (0) 39250374
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