– EMA Will Evaluate MAA Under Accelerated Assessment –
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Jan. 25, 2018--
Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics
company, announced today that the European Medicines Agency (EMA) has
accepted the Marketing Authorisation Application (MAA) and initiated its
review for patisiran, an investigational RNAi therapeutic targeting
transthyretin (TTR) for the treatment of hereditary ATTR (hATTR)
amyloidosis. The filing of the MAA was previously announced on December
18, 2017, and patisiran was previously granted accelerated assessment by
the EMA, potentially reducing the Agency’s evaluation time from 210 to
150 days.
“By evaluating the patisiran application under accelerated assessment,
the EMA acknowledges the urgent need for a new treatment approach to
address the debilitating and devastating effects of hATTR amyloidosis,”
said Eric Green, Vice President and General Manager of the TTR program
at Alnylam. “We plan to work closely with the EMA and Committee for
Medicinal Products for Human Use (CHMP) toward the goal of bringing
patisiran to patients with hATTR amyloidosis in the EU as quickly as
possible.”
In addition, Alnylam announced today that the UK Medicines and
Healthcare Products Regulatory Agency (MHRA) has granted designation of
patisiran as a Promising Innovative Medicine (PIM), supportive of its
consideration for entry into the UK’s Early Access to Medicines Scheme
(EAMS). EAMS aims to give patients with life-threatening or seriously
debilitating conditions access to medicines that do not yet have a
marketing authorization when there is a clear unmet medical need.
Alnylam announced completion of the submission of a New Drug Application
with the U.S. Food and Drug Administration (FDA) on December 12, 2017.
In the U.S., patisiran has Fast Track Designation, Breakthrough Therapy
Designation, and an expanded Orphan Drug Designation for ATTR
amyloidosis from the FDA.
About Patisiran
Patisiran is an investigational intravenously administered RNAi
therapeutic targeting transthyretin (TTR) in development for the
treatment of hereditary ATTR amyloidosis. It is designed to silence
specific messenger RNA, potentially blocking the production of TTR
protein before it is made. This may help to enable the clearance of TTR
amyloid deposits in peripheral tissues and potentially restore function
to these tissues. The safety and efficacy of patisiran have not been
evaluated by the U.S. Food and Drug Administration or any other health
authority.
About hATTR Amyloidosis
Hereditary transthyretin (TTR)-mediated (hATTR) amyloidosis is an
inherited, progressively debilitating, and often fatal disease caused by
mutations in the TTR gene. TTR protein is produced primarily in the
liver and is normally a carrier of vitamin A. Mutations in TTR cause
abnormal amyloid proteins to accumulate and damage body organs and
tissue, such as the peripheral nerves and heart, resulting in
intractable peripheral sensory neuropathy, autonomic neuropathy, and/or
cardiomyopathy. hATTR amyloidosis represents a major unmet medical need
with significant morbidity and mortality, affecting approximately 50,000
people worldwide. hATTR amyloidosis patients have a life expectancy of
2.5 to 15 years from symptom onset, and the only approved treatment
options are liver transplantation for early stage disease and tafamidis
(approved in Europe, Japan and certain countries in Latin America,
specific indication varies by region). There is a significant need for
novel therapeutics to help treat patients with hATTR amyloidosis.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual property for
use in RNAi therapeutic products using LNP technology.
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.
Alnylam – Sanofi Genzyme Alliance
On January 7, 2018, Alnylam and Sanofi announced a restructuring of
their RNAi therapeutics alliance. As part of this restructuring, Alnylam
will obtain global development and commercialization rights to patisiran
and Sanofi Genzyme will be eligible to receive royalties on sales of
patisiran in territories outside the United States, Canada and Western
Europe. Sanofi intends to substantially complete the transition of its
patisiran activities in regions outside the United States, Canada, and
Western Europe, consistent with the original scope of its license rights
to patisiran, by mid-2018. The transaction is subject to customary
closing conditions and clearances, including clearance under the
Hart-Scott Rodino Antitrust Improvements Act.
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 700 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,
Alnylam's views with respect to the potential for RNAi therapeutics,
including patisiran, its expectations regarding the review, potential
regulatory approval and commercial launch of patisiran in the United
States and 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, 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.
Patisiran 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 its safety
or effectiveness.

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Source: Alnylam Pharmaceuticals, Inc.
Alnylam Pharmaceuticals, Inc.
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