CAMBRIDGE, Mass.--(BUSINESS WIRE)--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 Patisiran
Patisiran 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 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.
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.
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 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 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 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.
