Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, and Gen, a GMP-certified pharmaceutical
company specializing in rare diseases, today announced an exclusive
Distribution Agreement for ONPATTRO®, a first-in-class RNAi
therapeutic for the treatment of hATTR amyloidosis in adults with
Stage 1 or Stage 2 polyneuropathy.
“Our partnership with Gen enables us to extend access to
ONPATTRO to patients suffering from hereditary ATTR (hATTR)
amyloidosis with polyneuropathy in Turkey where we currently don’t
have a presence,” said Brendan Martin, Vice President and Acting
Head of Canada, Europe, Middle East and Africa, Alnylam
Pharmaceuticals. “There are a significant number of patients in
Turkey who urgently need new treatment options and we are delighted
to partner with Gen to bring ONPATTRO to those in need.”
Abidin Gülmüs, CEO of Gen stated: “We are proud of our
reputation as one of Turkey's leading specialty pharmaceutical
companies and are excited to have partnered with Alnylam. Through
collaborations with leading international companies, we aim to
bring innovative medicines to patients in Turkey in the fastest and
most reliable way possible.”
Patients in Turkey were among those who participated in the
randomized, double-blind, placebo-controlled, global Phase 3 APOLLO
study, the largest-ever study in hATTR amyloidosis patients with
polyneuropathy, which led to the approval of ONPATTRO in the U.S.
and EU in 2018.
About ONPATTRO® (patisiran)
ONPATTRO is an RNAi therapeutic that was approved in the United
States and Canada for the treatment of the polyneuropathy of hATTR
amyloidosis in adults. ONPATTRO is also approved in the European
Union, Switzerland and Brazil for the treatment of hATTR
amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and
in Japan for the treatment of hATTR amyloidosis with
polyneuropathy. Based on Nobel Prize-winning science, ONPATTRO is
an intravenously administered RNAi therapeutic targeting
transthyretin (TTR). It is designed to target and silence TTR
messenger RNA, thereby blocking the production of TTR protein
before it is made. ONPATTRO blocks the production of TTR in the
liver, reducing its accumulation in the body’s tissues in order to
halt or slow down the progression of the polyneuropathy associated
with the disease. For more information about ONPATTRO, visit
ONPATTRO.com.
Important Safety Information (ISI) for ONPATTRO
Infusion-Related Reactions
Infusion-related reactions (IRRs) have been observed in patients
treated with patisiran. In a controlled clinical study, 19% of
patisiran-treated patients experienced IRRs, compared to 9% of
placebo-treated patients. The most common symptoms of IRRs with
patisiran were flushing, back pain, nausea, abdominal pain,
dyspnoea, and headache. Hypotension, which may include syncope, has
also been reported as a symptom of IRRs.
To reduce the risk of IRRs, patients should receive
premedication with a corticosteroid, paracetamol, and
antihistamines (H1 and H2 blockers) at least 60 minutes prior to
patisiran infusion. Monitor patients during the infusion for signs
and symptoms of IRRs. If an IRR occurs, consider slowing or
interrupting the infusion and instituting medical management as
clinically indicated. If the infusion is interrupted, consider
resuming at a slower infusion rate only if symptoms have resolved.
In the case of a serious or life-threatening IRR, the infusion
should be discontinued and not resumed.
Reduced Serum Vitamin A Levels and Recommended
Supplementation
Patisiran treatment leads to a decrease in serum vitamin A
levels. Patients receiving patisiran should take oral
supplementation of approximately 2500 IU vitamin A per day to
reduce the potential risk of ocular toxicity due to vitamin A
deficiency. Doses higher than 2500 IU vitamin A per day should not
be given to try to achieve normal serum vitamin A levels during
treatment with patisiran, as serum levels do not reflect the total
vitamin A in the body. Patients should be referred to an
ophthalmologist if they develop ocular symptoms suggestive of
vitamin A deficiency (e.g. including reduced night vision or night
blindness, persistent dry eyes, eye inflammation, corneal
inflammation or ulceration, corneal thickening or corneal
perforation).
Adverse Reactions
The most common adverse reactions that occurred in patients
treated with patisiran were peripheral oedema (30%) and
infusion-related reactions (19%).
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 new class of medicines, known as RNAi therapeutics, is now
a reality. 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
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, hepatic infectious, and central
nervous system (CNS)/ocular 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 RNAi therapeutics platform. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), approved in the
U.S., EU, Canada, Japan, Brazil and Switzerland, and GIVLAARI®
(givosiran), approved in the U.S and the EU. Alnylam has a deep
pipeline of investigational medicines, including five product
candidates that are in late-stage development. Alnylam is executing
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 is
headquartered in Cambridge, MA.
About Gen
Gen is the fastest growing pharmaceutical company in Turkey.
Teamed up with its leading international partners and compliant
with ethical and scientific principles, Gen supplies products used
in treatment of rare diseases and disorders in different branches
and aims to bring these products to patients in the easiest,
fastest and most reliable way possible while striving to find and
bring new treatments to patients with unmet medical needs. With its
GMP certificated production facility based in Ankara, Gen exports
its products to different countries and has offices in Ankara (HQ),
İstanbul, İzmir, Trabzon, Azerbaijan, Kazakhstan and Russia with
400+ employees. For more information please visit the Gen
website.
Alnylam Forward Looking Statements
Various statements in this release concerning future
expectations, plans and prospects, including, without limitation,
Alnylam's views and plans with respect to the ability to extend
patient access to ONPATTRO in Turkey through the announced
Distribution Agreement with Gen, and the number of patients in
Turkey within the approved indication for ONPATTRO who are in need
of new treatment options, Gen’s views and plans with respect to the
speed and reliability with which it is able to bring innovative
medicines to patients in Turkey, and Alnylam’s expectations
regarding the continued execution on 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; 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 or its marketed products, including ONPATTRO in
Turkey; obtaining, maintaining and protecting intellectual
property; intellectual property matters including potential patent
litigation relating to its platform, products or product
candidates; obtaining regulatory approval for its product
candidates, including lumasiran and product candidates developed in
collaboration with others, including inclisiran, and maintaining
regulatory approval and obtaining pricing, reimbursement and access
for its products, including ONPATTRO and GIVLAARI; progress in
continuing to establish a commercial and ex-United States
infrastructure, including in Europe; successfully launching,
marketing and selling its approved products globally, including
ONPATTRO and GIVLAARI, and achieve net product revenues for
ONPATTRO within its expected range during 2020; potential risks to
Alnylam’s business, activities and prospects as a result of the
COVID-19 pandemic, or delays or interruptions resulting therefrom,
including without limitation, any risks affecting access to
ONPATTRO in Turkey, Alnylam’s ability to successfully expand the
indication for ONPATTRO 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 within the ranges of its expected guidance
and achieve a self-sustainable financial profile in the future,
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,
including Regeneron, for development, manufacture and distribution
of certain products, including eye and CNS products, and Ironwood,
for assistance with the education about and promotion of GIVLAARI
in the U.S.; 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20200325005133/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) +1-617-682-4340
Fiona McMillan (Media, Europe) +44 1628 244960
Gen Ayhan Yener, MD (Medical Director) +90 554 566 57
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