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| Alnylam Receives Orphan Drug Designation from U.S. Food & Drug Administration for ALN-TTR02, an RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis (ATTR) |
“We are very pleased to have received Orphan Drug Designation from the
About Transthyretin-Mediated Amyloidosis
Transthyretin (TTR)-mediated amyloidosis (ATTR) is a hereditary,
systemic disease caused by mutations in the TTR gene. TTR protein is
produced primarily in the liver and is normally a carrier for thyroid
hormones and retinol binding proteins. 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. In its
severest form, ATTR represents a major unmet medical need with
significant morbidity and mortality as an orphan disease; FAP (familial
amyloidotic polyneuropathy) affects approximately 10,000 people
worldwide and FAC (familial amyloidotic cardiomyopathy) affects at least
40,000 people worldwide. ATTR patients with FAP have a mean life
expectancy of five to 15 years from symptom onset and the only treatment
options for early stage disease are liver transplantation and tafamidis
(approved in About ALN-TTR Program
ALN-TTR02 is a systemically delivered RNAi therapeutic being developed
for the treatment of ATTR. Alnylam has completed enrollment in a Phase I
trial with ALN-TTR02 and expects to present data early in the third
quarter of 2012. The company recently initiated a Phase II trial in
About RNA Interference (RNAi) RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNAs (siRNAs), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way.
About
Alnylam is a biopharmaceutical company developing novel therapeutics
based on RNA interference, or RNAi. The company is leading the
translation of RNAi as a new class of innovative medicines with a core
focus on RNAi therapeutics for the treatment of genetically defined
diseases, including ALN-TTR for the treatment of transthyretin-mediated
amyloidosis (ATTR), ALN-PCS for the treatment of severe
hypercholesterolemia, ALN-HPN for the treatment of refractory anemia,
ALN-APC for the treatment of hemophilia, and ALN-TMP for the treatment
of hemoglobinopathies. As part of its “Alnylam 5x15TM”
strategy, the company expects to have five RNAi therapeutic products for
genetically defined diseases in clinical development, including programs
in advanced stages, on its own or with a partner by the end of 2015.
Alnylam has additional partner-based programs in clinical or development
stages, including ALN-RSV01 for the treatment of respiratory syncytial
virus (RSV) infection, ALN-VSP for the treatment of liver cancers, and
ALN-HTT for the treatment of Huntington’s disease. The company’s
leadership position on RNAi therapeutics and intellectual property have
enabled it to form major alliances with leading companies including
Merck, About “Alnylam 5x15™”
The “Alnylam 5x15” strategy, launched in Alnylam Forward-Looking Statements
Various statements in this release concerning Alnylam’s future
expectations, plans and prospects, including without limitation,
statements regarding Alnylam’s views with respect to the potential for
RNAi therapeutics, including ALN-TTR02 and ALN-TTRsc, its expectations
with respect to the timing and success of its clinical trials for
ALN-TTR02, its expectations regarding the reporting of data from its
ALN-TTR02 clinical trials, and Alnylam’s expectations regarding its
“Alnylam 5x15” product strategy, constitute forward-looking statements
for the purposes of the safe harbor provisions under The Private
Securities Litigation Reform Act of 1995. Actual results may differ
materially from those indicated by these forward-looking statements as a
result of various important factors, including, without limitation,
Alnylam’s ability to discover and develop novel drug candidates,
successfully demonstrate the efficacy and safety of its drug candidates,
including ALN-TTR02 and ALN-TTRsc, the pre-clinical and clinical results
for these product candidates, which may not support further development
of such product candidates, actions of regulatory agencies, which may
affect the initiation, timing and progress of clinical trials for such
product candidates, obtaining, maintaining and protecting intellectual
property, obtaining regulatory approval for products, competition from
others using technology similar to Alnylam’s and others developing
products for similar uses, and Alnylam’s ability to establish and
maintain strategic business alliances and new business initiatives, as
well as those risks more fully discussed in the “Risk Factors” section
of its most recent quarterly report on Form 10-Q on file with the
Source:
Alnylam Pharmaceuticals, Inc.
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