− In the United States, Company Completes Tenth
Value-Based Agreement with Commercial Payers, and Achieves
Confirmed Access to ONPATTRO for Greater than 90 Percent of Covered
Lives –
− In Europe, Company Achieves Favorable Health
Technology Assessment Ratings for ONPATTRO in Multiple Countries
–
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, today announced that it has made
substantial progress in the global commercialization of ONPATTRO
for the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults. The Company
has now completed definitive value-based agreements (VBAs) with 10
commercial payers in the United States. Furthermore, greater than
90 percent of U.S. lives across commercial, Medicare, Medicaid, and
other government payer categories are now confirmed to have access
to ONPATTRO if prescribed.
“We’re encouraged by the positive reception for global access to
ONPATTRO from many private and public payers. With 10 VBAs
completed with commercial payers in the U.S., we’re fulfilling our
commitment that ONPATTRO is reimbursed based on its ability to
deliver outcomes in the real-world setting comparable to those
demonstrated in clinical trials,” said Barry Greene, President of
Alnylam. “We have also made great progress advancing pricing and
reimbursement procedures with authorities across Europe. In
particular, we’re encouraged by the favorable outcomes achieved in
health technology assessment proceedings in major EU markets
including Germany and France, among others. Overall, our market
access progress in the U.S. and EU is aligned with our commitment
to bring ONPATTRO to patients around the world.”
Alnylam has made significant progress securing access to
ONPATTRO for multiple countries in the EU. The Company remains on
track with pricing and reimbursement procedures in nearly all EU
markets, with encouraging assessment outcomes and ongoing
discussions with authorities. Key updates include:
- Germany: Launched ONPATTRO one month
following EC authorization (October 2018). Since then, ONPATTRO has
become the only product for the treatment of hATTR amyloidosis to
receive a “considerable benefit” rating from the Joint Federal
Committee (G-BA).
- France: Recognized ONPATTRO as being
the only product for the treatment of hATTR with a significant
public health impact as noted by its ASMR III and Intêret Santé
Publique (ISP) ratings. ONPATTRO is currently reimbursed through
Temporary Authorization for Use.
- Austria: Achieved access as of October
2018.
- Luxembourg: Achieved access as of
October 2018.
- Netherlands: Reached a nationwide
agreement to reimburse ONPATTRO for all eligible patients through
joint negotiations coordinated by Zorgverzekeraars Nederland (ZN),
the Dutch Association of Health Insurers.
- Sweden: Published HTA assessment from
the Dental and Pharmaceutical Benefits Agency in Sweden (TLV)
allowing a subsequent ongoing negotiation with the New Therapy
Council (NT- Council) to allow usage of ONPATTRO within the County
Councils.
Pricing and reimbursement procedures are progressing in several
additional countries, including the United Kingdom, Italy, Spain,
Portugal, Ireland and key markets in Central and Eastern Europe
where several hATTR endemic regions exist. In addition, authorities
have approved access under specific named patient procedures in
several countries while reimbursement proceedings move forward.
Alnylam remains committed to making ONPATTRO available and
reimbursed worldwide in alignment with its Patient Access
Philosophy, which outlines the Company’s commitment to access while
delivering value to patients, physicians, and payers.
Physicians and patients in the United States can learn more
about Alnylam’s comprehensive patient services by visiting
AlnylamAssist.com or calling 1-833-256-2748.
Visit ONPATTRO.com for more information, including full
prescribing information.
About ONPATTRO® (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
TTR messenger RNA, thereby blocking the production of TTR protein
before it is made. Patisiran 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 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.
Important Safety Information
Infusion-Related ReactionsInfusion-related reactions
(IRRs) have been observed in patients treated with ONPATTRO. In a
controlled clinical study, 19 percent of ONPATTRO-treated patients
experienced IRRs, compared to 9 percent of placebo-treated
patients. The most common symptoms of IRRs with ONPATTRO were
flushing, back pain, nausea, abdominal pain, dyspnea, and
headache.
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
ONPATTRO 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
SupplementationONPATTRO treatment leads to a decrease in serum
vitamin A levels. Supplementation at the recommended daily
allowance (RDA) of vitamin A is advised for patients taking
ONPATTRO. Higher doses than the RDA should not be given to try to
achieve normal serum vitamin A levels during treatment with
ONPATTRO, 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.
night blindness).
Adverse ReactionsThe most common adverse reactions that
occurred in patients treated with ONPATTRO were respiratory-tract
infection (29 percent) and infusion-related reactions (19
percent).
About LNP TechnologyAlnylam has licenses to Arbutus
Biopharma LNP intellectual property for use in RNAi therapeutic
products using LNP technology.
About RNAiRNAi (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 PharmaceuticalsAlnylam (Nasdaq: ALNY) is
leading the translation of RNA interference (RNAi) into a 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/ocular diseases. Based on
Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach for the treatment of
diseases with high unmet need. ONPATTRO® (patisiran) is the
first-ever RNAi therapeutic approved by the U.S. FDA for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults and by the EMA
for the treatment of hATTR amyloidosis in adults with stage 1 or
stage 2 polyneuropathy. Alnylam has a deep pipeline of
investigational medicines, including six product candidates in
Phase 3 studies. 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. Headquartered in
Cambridge, MA, Alnylam employs over 1,000 people worldwide. For
more information, please visit www.alnylam.com and engage with
us on Twitter at @Alnylam or on LinkedIn.
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 availability and reimbursement for ONPATTRO in
multiple geographies around the world, 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, successfully launching, marketing and
selling its approved products globally, 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, 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20190430006066/en/
Alnylam Pharmaceuticals, IncChristine Regan
Lindenboom(Investors and Media)617-682-4340
Josh Brodsky(Investors)617-551-8276
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