Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading
RNAi therapeutics company, today announced that the European
Medicines Agency (EMA) Committee for Orphan Medicinal
Products (COMP) has issued a positive opinion recommending
ALN-TTRsc02, an investigational, subcutaneously administered RNAi
therapeutic, for designation as an orphan medicinal product for the
treatment of transthyretin (TTR)-mediated (ATTR) amyloidosis.
"We are very pleased to have received a positive opinion from
the EMA COMP on our application for Orphan Drug Designation for
ALN-TTRsc02," said Rena Denoncourt, Program Leader,
ALN-TTRsc02 Program at Alnylam. "With potent and durable TTR
knockdown, we believe ALN-TTRsc02 holds great promise as an
investigational, once-quarterly, low volume, subcutaneously
administered RNAi therapeutic for the treatment of a broad spectrum
of patients with ATTR amyloidosis. We look forward to advancing
ALN-TTRsc02 into Phase 3 later this year."
Orphan Drug Designation by the European
Commission provides regulatory and financial incentives for
companies to develop and market therapies that treat a
life-threatening or chronically debilitating condition affecting no
more than five in 10,000 persons in the European
Union (EU), and where no satisfactory treatment is available.
In addition to a 10-year period of marketing exclusivity in the EU
after product approval, Orphan Drug Designation provides incentives
for companies seeking protocol assistance from the EMA during the
product development phase and direct access to centralized
marketing authorization.
About ALN-TTRsc02
ALN-TTRsc02 is an investigational, subcutaneously administered
RNAi therapeutic targeting transthyretin (TTR) in development for
the treatment of ATTR amyloidosis. It is designed to target and
silence specific messenger RNA, potentially blocking the production
of TTR protein before it is made. This may help to reduce the
deposition and facilitate the clearance of TTR amyloid deposits in
peripheral tissues and potentially restore function to these
tissues. The safety and efficacy of ALN-TTRsc02 have not been
evaluated by the U.S. Food and Drug Administration, European
Medicines Agency or any other health authority.
About ATTR amyloidosis
Transthyretin (TTR)-mediated (ATTR) amyloidosis is a rare,
progressively debilitating, and often fatal disease caused by
misfolded TTR proteins that accumulate as amyloid deposits in
multiple tissues including the nerves, heart, and GI tract. TTR
protein is primarily produced in the liver and is normally a
carrier of vitamin A. Hereditary ATTR (hATTR) amyloidosis is an
inherited, progressive disease that occurs when mutations in the
TTR gene cause abnormal amyloid proteins to accumulate and damage
body organs and tissues, such as the peripheral nerves and heart,
resulting in intractable peripheral sensory 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. Wild-type ATTR (wtATTR)
amyloidosis is a nonhereditary, progressive disease of undefined
etiology that occurs when misfolded TTR proteins accumulate as
amyloid deposits in multiple organs, including the heart, resulting
predominantly in cardiomyopathy, leading to heart failure and
mortality within 2 to 6 years. Prevalence of wtATTR amyloidosis is
uncertain, however estimates suggest fewer than 200,000 patients
across the U.S. and Europe.
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 major new class of medicines, known as RNAi therapeutics,
is on the horizon. 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 Pharmaceuticals
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, and hepatic infectious 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 and deep pipeline of
investigational medicines, including four 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 800 people in the U.S. and Europe 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,
its expectations as to the prospects of ALN-TTRsc02, and 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, 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
Annual Report on Form 10-K 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.
ALN-TTRsc02 has not 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 this investigational therapeutic.
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version on businesswire.com: https://www.businesswire.com/news/home/20180423005383/en/
Alnylam Pharmaceuticals, Inc.Christine Regan Lindenboom,
617-682-4340(Investors and Media)orJosh Brodsky,
617-551-8276(Investors)
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