Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today the completion of the rolling submission of a 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 adults with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis). The rolling submission began on November 15, 2017 with the nonclinical and chemistry, manufacturing and controls components submitted, and now submission of the clinical data completes the filing. Alnylam has requested priority review for the application which, if granted, could result in a six-month review process. Patisiran previously received Fast Track and Breakthrough Therapy designations from the FDA, and recently received an expanded Orphan Drug Designation for ATTR amyloidosis.
“In November, we reported promising results from the APOLLO Phase 3 study of patisiran which showed amelioration of neurological impairment, improved quality of life, and reduced disease symptoms and disability in hATTR amyloidosis patients with polyneuropathy,” said Eric Green, Vice President and General Manager of the TTR program. “In less than 90 days from first reporting the top-line data, the completion of Alnylam’s first NDA submission is an historic event, bringing patisiran one step closer to patients living with hATTR amyloidosis. We look forward to the exciting prospect of introducing the first FDA-approved RNAi therapeutic, marking the arrival of a new class of medicines.”
“People living with hATTR amyloidosis currently have no FDA-approved options to treat this devastating, often fatal disease,” said Isabelle Lousada, President and Chief Executive Officer of the Amyloidosis Research Consortium (ARC). “The ARC applauds this significant milestone for patisiran and we are hopeful this therapy will be approved for patients in the U.S. who are desperately awaiting treatment options.”
Patisiran also has been granted accelerated assessment by the European Medicines Agency. Alnylam, in collaboration with Sanofi Genzyme, intends to file a Marketing Authorization Application in the European Union 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, 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 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 FDA recently granted
a request to amend the Orphan Drug Designation for patisiran to the
treatment of transthyretin-mediated amyloidosis (ATTR amyloidosis). 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 symptomatic 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 open-label extension 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.
Alnylam Forward Looking Statements
Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's plans for and the
expected timing of a regulatory filing seeking approval for patisiran
from regulatory authorities in the United States, Europe and ROW
countries, 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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