– Health Canada Grants Priority Review to
promising medicines intended for the treatment of serious,
debilitating or life-threatening conditions –
TORONTO, Sept. 24, 2018 /CNW/ - Alnylam
Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNA interference
(RNAi) therapeutics company, announced today that Health Canada has
granted Priority Review status to patisiran. Priority review
is granted to regulatory filings in Canada intended for new potentially
life-saving treatments where there is no existing medicine on the
Canadian market or where the new medicine represents a significant
improvement in the benefit/risk profile over existing products.
Currently there are no approved treatments for patients with hATTR
amyloidosis in Canada.
Many serious, chronic and life-threatening diseases, such as
hATTR amyloidosis, are caused by a fault or mutation that
interferes with the way the body manufactures proteins. RNAi
is a completely new approach to the treatment of these diseases;
targeting the faulty protein that is causing the disease rather
than treating the symptoms. The discovery of RNAi was awarded the
Nobel Prize in Medicine in 2006. RNAi therapeutics are an
entirely new class of medicines.
"We are delighted to have received Priority Review status for
patisiran and look forward to continuing to work closely with
Health Canada during the review process," said Jeff Miller, Country Manager, Alnylam Canada.
"The Priority Review underscores the urgent need for an approved
treatment for Canadian patients with hATTR amyloidosis, an
aggressive, rapidly progressive, debilitating and fatal
disease."
The regulatory submission in Canada for the treatment of patients with
hATTR amyloidosis will be based on data from the APOLLO Phase 3
study. The randomized, double-blind, placebo-controlled, global
Phase 3 is the largest-ever study conducted in hATTR amyloidosis
patients with polyneuropathy, and enrolled patients in
Canada. The results were published in the July 5, 2018, issue of The New England Journal of
Medicine.
"In Canada, patients diagnosed
with hATTR amyloidosis, and their families, face an uncertain
future and poor prognosis, with no treatments approved to halt or
reverse the progression of the disease. In the APOLLO study,
patisiran-treated patients saw an improvement in their
polyneuropathy symptoms, quality of life and ability to undertake
day-to-day tasks, compared to those treated with placebo. Priority
review designation for patisiran is an excellent step here in
Canada, and I am looking forward
to the outcome of the regulatory process", said Dr. Vera Bril, Professor of Medicine at the
University of Toronto, Director of
Neurology at University Health Network and Mount Sinai Hospital and
the Krembil Family Chair in Neurology.
"We are delighted that Health Canada has granted Priority Review
status to patisiran, which hopefully means Canadians with hATTR
amyloidosis will soon have access to this important therapy. We are
optimistic that this signals the beginning of a brighter future for
patients in Canada living with
this devastating, fatal disease that can affect families over
generations," said Durhane
Wong-Rieger, President and CEO, Canadian Organization for
Rare Disorders (CORD).
About Patisiran
Patisiran, based on Nobel
Prize-winning science, is an intravenously administered RNAi
therapeutic targeting transthyretin (TTR) for the treatment of
hereditary ATTR amyloidosis. It is designed to target and silence
specific messenger RNA, potentially blocking the production of TTR
protein before it is made. Patisiran blocks the production of
transthyretin in the liver, reducing its accumulation in the body's
tissues in order to halt or slow down the progression of the
disease. In August 2018,
patisiran received U.S. Food and Drug Administration (FDA) approval
for the treatment of the polyneuropathy of hATTR amyloidosis in
adults, as well as European Medicines Agency marketing
authorization for the treatment of hATTR amyloidosis in adults with
Stage 1 or Stage 2 polyneuropathy. In Canada, the safety and
efficacy of patisiran are still under review and market
authorization has not yet been granted.
About hATTR amyloidosis
Hereditary transthyretin
(TTR)-mediated amyloidosis (hATTR amyloidosis) is an inherited,
progressively debilitating, and often fatal disease caused by
mutations in the TTR gene. TTR protein is primarily produced in the
liver and is normally a carrier of vitamin A. Mutations in the TTR
gene 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, as well as other disease
manifestations. hATTR amyloidosis represents a major unmet
medical need with significant morbidity and mortality, affecting
approximately 50,000 people worldwide. The median survival is 4.7
years following diagnosis, with a reduced survival (3.4 years) for
patients presenting with cardiomyopathy. In Canada, there are no approved treatment
options that can modify the course of the disease and there remains
a pressing need for novel medicines to help treat these
patients.
About RNAi
RNAi (RNA interference) is a natural
cellular process of gene silencing that represents one of the most
promising and rapidly advancing frontier 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 continue to
transform the care of patients with genetic and other diseases.
About Alnylam
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 800 people worldwide and is headquartered in Cambridge, MA.
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 data supporting the Canadian regulatory submission and
the potential implications of such data for patients, plans for
regulatory filings in other markets and expectations regarding the
company's "Alnylam 2020" guidance for the advancement and
commercialization of RNAi therapeutics, constitute forward-looking
statements for the purposes of the safe harbour 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, successfully
launching, marketing and selling its approved products globally,
Alnylam's ability to successfully expand the indication for
patisiran in the future, 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.
SOURCE Alnylam Pharmaceuticals, Inc.