-- Partnership with GENESIS Pharmamarks an
important step in the global commercial expansion of ONPATTRO, the
first-in-class ‘gene-silencing’ RNAi therapeutic --
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company and GENESIS Pharma, a leading regional
biopharma company operating in the broader region of South East
Europe, announced today an exclusive agreement to commercialize
ONPATTRO®, a first-in-class RNAi therapeutic, in 12 countries:
Greece, Cyprus, Bulgaria, Romania, Slovenia, Croatia, Serbia,
Bosnia and Herzegovina, Albania, Republic of North Macedonia,
Montenegro as well as Malta.
“Our partnership with GENESIS Pharma enables us to extend access
to ONPATTRO to patients suffering from hereditary ATTR (hATTR)
amyloidosis in areas of Europe where we currently don’t have a
presence,” said Theresa Heggie, SVP and Head of Europe, Middle East
and Africa, and Canada, Alnylam Pharmaceuticals. “We know that
patients in these countries have an urgent need for new treatment
options, and as the only company with an RNAi therapeutic approved
in the European Union, we are delighted to partner with GENESIS
Pharma to bring ONPATTRO and potential future therapies to patients
in South East Europe.”
Mr. Constantinos Evripides, Managing Director of GENESIS Pharma
stated: “We are very happy to announce our partnership with Alnylam
for the broader region of South East Europe. It is in challenging
therapeutic areas, such as rare, chronic and debilitating diseases,
that novel therapeutics have the most potential to bring meaningful
benefits for the patients and society at large. We are committed to
bringing such innovations closer to patients and the medical
community in the countries that we operate, through collaborations
with leading international companies such as Alnylam that transform
pioneering science into medicine.”
The agreement between Alnylam and GENESIS Pharma covers
ONPATTRO, approved in the EU in August 2018 for the treatment of
hATTR amyloidosis in adults with stage 1 or stage 2
polyneuropathy.
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 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 approved RNAi
therapeutic is ONPATTRO® (patisiran) available in the U.S., EU 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.
About GENESIS Pharma
GENESIS Pharma is a regional biopharma company focused on
innovative biopharmaceutical products targeting severe and rare
diseases. With offices in Athens, Nicosia, Sofia, Bucharest and
Zagreb, GENESIS Pharma was among the first pharmaceutical companies
in Europe to specialize in the marketing, sales and distribution of
biopharmaceutical products. Since its inception, GENESIS Pharma has
built a strong portfolio in therapeutic areas with high unmet
medical needs through long standing strategic alliances for the
broader region of South East Europe with some of the leading
multinational pharmaceutical companies committed to cutting edge
R&D. For more information please visit
www.genesispharma.com.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
Alnylam's partnership with Genesis Pharma to commercialize ONPATTRO
in South East Europe, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190729005574/en/
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Fiona McMillan (Media, Europe) +44 1628 244960
GENESIS Pharma
Natalia Karahaliou (Media, South Eastern Europe) +30 2108
771605
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