CAMBRIDGE, Mass. & PARIS--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, and Sanofi Genzyme, the specialty care global business unit of Sanofi, announced today the submission of a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for patisiran, an investigational RNAi therapeutic targeting transthyretin (TTR) for the treatment of adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). Patisiran was previously granted accelerated assessment by the EMA, potentially reducing the Agency’s evaluation time from 210 to 150 days.
“The MAA submission for patisiran represents another important milestone for Alnylam and a critical step toward bringing RNAi therapeutics to people living with hATTR amyloidosis,” said Eric Green, Vice President and General Manager of the TTR program at Alnylam. “Based on the results of the APOLLO study, we believe patisiran has the potential to become the standard of care for the treatment of hATTR amyloidosis. We look forward to working with the EMA and the Committee for Medicinal Products for Human Use (CHMP) during the review process.”
“People with hATTR amyloidosis have limited treatment options,” said Rand Sutherland, M.D., Therapeutic Area Head, Rare Diseases Development at Sanofi. “With this MAA submission, we are one step closer to making patisiran available in Europe and executing on our shared vision to bring this RNAi treatment to patients globally.”
Alnylam announced completion of the submission of a New Drug Application with the U.S. Food and Drug Administration on December 12, 2017. Sanofi Genzyme is currently preparing regulatory filings for patisiran in Japan, Brazil and other countries, with submissions expected to begin in the first half of 2018. Pending regulatory approvals, Alnylam will commercialize patisiran in the U.S., Canada and Western Europe, with Sanofi Genzyme commercializing the product in the rest of the world, including certain Central and Eastern European countries of the European Union.
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 target and 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 APOLLO Phase 3 Study
The APOLLO Phase 3 study (N=225)
was a randomized, double-blind, placebo-controlled, global study
designed to evaluate the efficacy and safety of patisiran in hATTR
amyloidosis patients with polyneuropathy. The study was completed in
August 2017 and detailed study results were presented at the 1st
European ATTR Amyloidosis Meeting for Patients and Doctors on November
2, 2017. All patients completing the APOLLO Phase 3 study are eligible
to screen for the Global OLE study, in which they have the opportunity
to receive patisiran on an ongoing basis.
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
In January 2014, Alnylam
and Sanofi Genzyme, the specialty care global business unit of Sanofi,
formed an alliance to accelerate the advancement of RNAi therapeutics as
a potential new class of innovative medicines for patients around the
world with rare genetic diseases. The alliance enables Sanofi Genzyme to
expand its rare disease pipeline with Alnylam’s novel RNAi technology
and provides access to Alnylam’s R&D engine, while Alnylam benefits from
Sanofi Genzyme’s proven global capabilities to advance late-stage
development and, upon commercialization, accelerate market access for
these promising genetic medicine products. In the case of patisiran,
Alnylam will advance the product in the United States, Canada and
Western Europe, while Sanofi Genzyme will advance the product in the
rest of the world.
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 600 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.
About Sanofi
Sanofi is dedicated to supporting people
through their health challenges. We are a global biopharmaceutical
company focused on human health. We prevent illness with vaccines,
provide innovative treatments to fight pain and ease suffering. We stand
by the few who suffer from rare diseases and the millions with long-term
chronic conditions. With more than 100,000 people in 100 countries,
Sanofi is transforming scientific innovation into healthcare solutions
around the globe.
Sanofi Genzyme focuses on developing specialty treatments for debilitating diseases that are often difficult to diagnose and treat, providing hope to patients and their families. Learn more at www.sanofigenzyme.com.
Sanofi, Empowering Life.
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 complete results from its APOLLO Phase 3 clinical trial for
patisiran and the potential implications of such results for patients,
its plans for and the expected timing of regulatory filings seeking
approval for patisiran from regulatory authorities in the United States,
Europe and ROW countries, its expectations regarding the potential for
patisiran to improve the lives of hATTR amyloidosis patients with
polyneuropathy and their families, its plans for the commercialization
of patisiran if approved by regulatory authorities, 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 the safety or effectiveness of this investigational therapeutic.
Sanofi Forward Looking Statements
This press release
contains forward-looking statements as defined in the Private Securities
Litigation Reform Act of 1995, as amended. Forward-looking statements
are statements that are not historical facts. These statements include
projections and estimates regarding the potential marketing approvals
for the product. Forward-looking statements are generally identified by
the words “expects”, “anticipates”, “believes”, “intends”, “estimates”,
“plans”, “will be” and similar expressions. Although Sanofi’s management
believes that the expectations reflected in such forward-looking
statements are reasonable, investors are cautioned that forward-looking
information and statements are subject to various risks and
uncertainties, many of which are difficult to predict and generally
beyond the control of Sanofi, that could cause actual results and
developments to differ materially from those expressed in, or implied or
projected by, the forward-looking information and statements. These
risks and uncertainties include among other things, the uncertainties
inherent in research and development, including future clinical data
relating to the product ,decisions by regulatory authorities, such as
the FDA or the EMA, regarding whether and when to approve the product as
well as their decisions regarding labeling and other matters that could
affect the availability or commercial potential of the product, the
absence of guarantee that the product if approved will be commercially
successful, , risks associated with intellectual property, future
litigation, the future approval and commercial success of therapeutic
alternatives, and volatile economic conditions, as well as those risks
discussed or identified in the public filings with the SEC and the AMF
made by Sanofi, including those listed under “Risk Factors” and
“Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s
annual report on Form 20-F for the year ended December 31, 2016. Other
than as required by applicable law, Sanofi does not undertake any
obligation to update or revise any forward-looking information or
statements.
