Sanofi and Alnylam submit Marketing Authorization Application to the European Medicines Agency for patisiran for the
treatment of hereditary ATTR amyloidosis
Patisiran Marketing Authorization Application to be reviewed under accelerated assessment Paris and
Cambridge, Mass. - December 18, 2017 - Sanofi Genzyme, the specialty care global business unit of Sanofi, and Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the submission of a Marketing
Authorization 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 EMA'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."
U.S and other regulatory submissions
Alnylam announced completion of the submission of a New Drug Application with the U.S. Food and Drug Administration (FDA) 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.
Patisiran has not been approved by the FDA, EMA, 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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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
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.
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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.
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.
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