– Alnylam Act™ sponsors free third-party
genetic counseling and testing services for people at risk for
hereditary ATTR amyloidosis (hATTR amyloidosis) –
Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi
therapeutics company, today marked the 10th annual Rare Disease Day
by underscoring its commitment to enabling diagnosis for people and
caregivers impacted by specific rare diseases, such as hereditary
ATTR amyloidosis (hATTR amyloidosis). As part of this effort,
Alnylam sponsors free third-party genetic counseling and testing
through Alnylam Act™ (previously known as Alnylam Assist), a
program created to empower patients with the knowledge and tools to
make informed decisions about their health and facilitate an early,
accurate diagnosis, potentially leading to improved care. The
services, currently available in the United States, are provided by
independent third parties.
hATTR amyloidosis is an inherited, rapidly progressive
life-threatening disease impacting 50,0001 people worldwide. It is
caused by a mutation in the transthyretin (TTR) gene that results
in misfolded TTR proteins accumulating as amyloid fibrils in
multiple tissues including the nerves, heart and gastrointestinal
tract. The degree and severity of symptoms vary from person to
person but can lead to morbidity, disability and mortality within
two to 15 years of symptom onset.1,2
“hATTR amyloidosis is significantly under-diagnosed and often
misdiagnosed because of its constellation of symptoms that may
overlap with other diseases, leading many patients to experience
inappropriate medical intervention, such as unnecessary medicine
and surgery,” said Sami L. Khella, M.D., Chief, Department of
Neurology, Penn Presbyterian Medical Center and Professor of
Clinical Neurology, University of Pennsylvania School of Medicine.
“For my patients who have symptoms consistent with hATTR
amyloidosis, Alnylam Act helps to make an accurate diagnosis.”
“As of January 2017 approximately one thousand people have been
tested via Alnylam Act, and nearly 16 percent of these tests were
positive for a pathogenic mutation in the TTR gene, demonstrating
the need to increase awareness and improve diagnosis rates,” said
Pritesh Gandhi, Vice President, Medical Affairs at Alnylam.
“Alnylam Act is a reflection of our commitment to the hATTR
amyloidosis community, and we are proud to make these complimentary
third-party services available to the people at risk for, or
impacted by, this progressive rare disease.”
In addition to genetic testing that can be ordered by a
healthcare professional, Alnylam Act allows patients and their
families to connect with genetic counselors who provide education
and support, serving as an advocate to help guide them through the
diagnostic journey.
“It can be beneficial to meet with a genetic counselor prior to
undergoing testing to understand the benefits and risks involved,
including life and health insurance implications and how to work
through a diagnosis,” said Shawna Feely, MS, CGC, Genetic
Counselor, University of Iowa. “It is our goal to help enable those
at risk for hATTR amyloidosis to obtain the answers and support
they need to make more informed decisions about their health and
the health of their family.”
For physicians interested in ordering free hATTR amyloidosis
genetic testing for their patients, or for people interested in
scheduling a genetic counseling session to discuss the benefits,
risks and limitations of genetic testing, visit Alnylam Act.
About hATTR AmyloidosisHereditary ATTR amyloidosis (hATTR
amyloidosis) is an inherited, rapidly progressive, life-threatening
disease. hATTR amyloidosis is a multisystemic disease with a
heterogeneous clinical presentation that includes sensory and
motor, autonomic (e.g., diarrhea, erectile dysfunction,
hypotension) and cardiac symptoms. hATTR amyloidosis can lead to
significant morbidity, disability and mortality within two to 15
years. The disease continuum of hATTR amyloidosis includes patients
who present with predominantly polyneuropathy symptoms,
historically known as familial amyloidotic polyneuropathy (FAP), as
well as patients who present with predominantly cardiomyopathy
symptoms, historically known as familial amyloidotic cardiomyopathy
(FAC). However, many patients suffer from both polyneuropathy and
cardiomyopathy symptoms. hATTR amyloidosis represents a major unmet
medical need, affecting approximately 50,000 people worldwide. The
only approved treatment options for early stage disease are liver
transplantation and tafamidis (approved in Europe, certain
countries in Latin America and Japan, where it is approved for all
stages of the disease). There is a significant need for novel
therapeutics to treat patients with ATTR amyloidosis.
About RNAiRNAi (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 RNA (siRNA), 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 PharmaceuticalsAlnylam 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. Alnylam's pipeline
of investigational RNAi therapeutics is focused in 3 Strategic
Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline
of RNAi therapeutics for the treatment of rare diseases;
Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics
toward genetically validated, liver-expressed disease targets for
unmet needs in cardiovascular and metabolic diseases; and Hepatic
Infectious Disease, with a pipeline of RNAi therapeutics that
address the major global health challenges of hepatic infectious
diseases. In early 2015, Alnylam launched its "Alnylam 2020"
guidance for the advancement and commercialization of RNAi
therapeutics as a whole new class of innovative medicines.
Specifically, by the end of 2020, Alnylam expects to achieve a
company profile with 3 marketed products, 10 RNAi therapeutic
clinical programs - including 4 in late stages of development -
across its 3 STArs. The company's demonstrated commitment to RNAi
therapeutics has enabled it to form major alliances with leading
companies including Ionis, Novartis, Roche, Takeda, Merck,
Monsanto, The Medicines Company, and Sanofi Genzyme. In addition,
Alnylam holds an equity position in Regulus Therapeutics Inc., a
company focused on discovery, development, and commercialization of
microRNA therapeutics. Alnylam scientists and collaborators have
published their research on RNAi therapeutics in over 200
peer-reviewed papers, including many in the world's top scientific
journals such as Nature, Nature Medicine, Nature Biotechnology,
Cell, New England Journal of Medicine, and The Lancet. Founded in
2002, Alnylam maintains headquarters in Cambridge, Massachusetts.
For more information about Alnylam's pipeline of investigational
RNAi therapeutics, please visit www.alnylam.com.
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 potential for RNAi therapeutics, its expectations
regarding its STAr pipeline growth strategy, and 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 our
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
Annual Report on Form 10-K 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.
1 Suanprasert N, et al. J Neurol Sci. 2014.2 Ruberg and Berk,
Circulation; 126:1286-300 (2012).
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Alnylam Pharmaceuticals, Inc.Christine Regan Lindenboom,
617-682-4340(Investors and Media)orJosh Brodsky,
617-551-8276(Investors)
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