− Company Plans to Complete Submission by
Year-End −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced the company has initiated
submission of a rolling New Drug Application (NDA) to the U.S. Food
and Drug Administration (FDA) for patisiran, an investigational
RNAi therapeutic targeting transthyretin (TTR), for the treatment
of hereditary ATTR (hATTR) amyloidosis. The rolling submission
allows completed portions of an NDA to be reviewed by the FDA on an
ongoing basis. Alnylam has submitted the non-clinical and
chemistry, manufacturing and controls components and expects to
submit the final clinical data component by the end of the year.
Alnylam has also requested a priority review of the application
which, if granted, could result in a six-month review process.
Patisiran previously received Fast Track Designation from the
FDA.
“The initiation of the NDA filing marks an exciting moment in
Alnylam’s 15-year journey – the company’s first NDA, and the
first-ever application for regulatory approval of an RNAi
therapeutic,” said Eric Green, Vice President and General Manager
of the TTR program. “With the recent APOLLO Phase 3 study results,
we believe that patisiran is poised to potentially become an
important option for the treatment of hATTR amyloidosis, a rapidly
progressive, debilitating and often fatal disease.”
“Commencing the NDA submission for patisiran is wonderful news
for the hereditary amyloidosis community. We are deeply encouraged
by the potential impact patisiran can make on the lives of people
living with hATTR amyloidosis,” said Muriel Finkel, president of
the Amyloidosis Support Group. “With patisiran moving one step
closer to potentially being available to patients in the U.S., we
can now have hope for a promising treatment option for this
disease.”
Alnylam, in alliance with Sanofi Genzyme, plans to submit a
Marketing Authorisation Application to the European Medicines
Agency around year end. Sanofi Genzyme is currently preparing for
regulatory filings for patisiran in Japan, Brazil and other
countries, 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.
About PatisiranPatisiran is an investigational
intravenously administered RNAi therapeutic targeting transthyretin
(TTR) in development for the treatment of hereditary ATTR
amyloidosis with polyneuropathy. 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 StudyThe 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.
About hATTR amyloidosisHereditary 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 TechnologyAlnylam has licenses to Arbutus
Biopharma LNP intellectual property for use in RNAi therapeutic
products using LNP technology.
Alnylam - Sanofi Genzyme AllianceIn 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 RNAiRNAi (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 PharmaceuticalsAlnylam (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.
Alnylam Forward Looking StatementsVarious 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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version on businesswire.com: http://www.businesswire.com/news/home/20171116005157/en/
Alnylam Pharmaceuticals, Inc.Investors and MediaChristine
Regan Lindenboom, 617-682-4340orInvestorsJosh Brodsky,
617-551-8276
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