− Structure of Value-Based Arrangements Agreed
in Principle with Harvard Pilgrim Health Care and Other Major
Health Insurers –
− Alnylam Assist™ Program Designed to
Facilitate Access to ONPATTRO and Provide Dedicated Patient Support
–
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, today announced it has agreed on the
structure and key terms of value-based agreements (VBA) with
leading health insurers for ONPATTRO™ (patisiran) lipid complex
injection, a first-of-its-kind RNA interference (RNAi) therapeutic
for the treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults. ONPATTRO,
approved today by the U.S. Food and Drug Administration (FDA), is
the first FDA-approved treatment available in the United States for
this indication. ONPATTRO was shown to improve polyneuropathy, with
reversal of neuropathy impairment in a majority of patients, as
measured by change from baseline in the modified Neuropathy
Impairment Score +7 (mNIS+7) primary endpoint in the APOLLO pivotal
study. In the largest controlled study of hATTR amyloidosis
patients with polyneuropathy, ONPATTRO was also shown to improve
quality of life, as measured by the Norfolk Quality of
Life-Diabetic Neuropathy (QoL-DN) patient-reported assessment,
reduce autonomic symptoms, and improve activities of daily
living.
“While preparing for approval and launch, we knew that our more
than 15-year effort to advance RNAi therapeutics would only be
successful if ONPATTRO gets to the patients who need it,” said
Barry Greene, President of Alnylam. “Our pursuit of value-based
agreements with insurance providers and our Alnylam Assist™ program
demonstrate how we intend to operate as a commercial company,
consistent with our Patient Access Philosophy. We believe these
initiatives are the right things to do for patients and will
deliver value to the healthcare system.”
Alnylam is actively discussing VBAs with leading commercial
insurers, and has reached agreement in principle on the structure
of value-based agreements with Harvard Pilgrim Health Care and
other major health insurers, with many ongoing discussions. Based
on MMIT, a leading medical policy reporting agency, these engaged
payers cover approximately 76 percent of commercial medical lives
in the U.S. The goal of these agreements is to ensure that Alnylam
is paid based on the ability of ONPATTRO to deliver outcomes in the
real world setting comparable to those demonstrated in clinical
trials. Partnering with payers on these agreements is intended to
provide more certainty to them for their investment, and help
accelerate coverage decisions for patients. The agreements are
structured to link ONPATTRO’s performance in real-world use to
financial terms.
“Alnylam’s proactive approach to working closely with Harvard
Pilgrim far ahead of the drug approval date to develop a
value-based agreement highlights its commitment to patients,
patient access, and delivering meaningful outcomes to the
healthcare system,” said Michael Sherman, M.D., M.B.A., Chief
Medical Officer of Harvard Pilgrim Health Care. “By virtue of
linking level of reimbursement to meaningful patient outcomes, this
agreement will help us meet the needs of hATTR amyloidosis patients
by supporting our efforts to balance access and affordability.”
The VBAs build upon Alnylam’s Patient Access Philosophy
announced last year, which focuses the Company’s commercial
objectives on being proactive about patient access while delivering
value to patients, physicians, and insurers. This Philosophy
commits Alnylam to act with urgency for patients, pursue
value-based agreements and to not increase the annualized price of
its medicines above the consumer price index (CPI-U) unless
valuable new innovation has been achieved.
"Express Scripts is enthusiastic about this approval as it
represents a new therapeutic technology that should improve the
lives of patients. We applaud Alnylam for taking a responsible
approach to pricing and patient access in the rare disease space,
an increasingly complicated challenge," said Steve Miller, M.D.,
Chief Medical Officer, Express Scripts. "We want to work together
toward a common goal of ensuring broad, affordable access to
important medicines for rare conditions."
Other insurers have also recognized Alnylam’s approach to
delivering good value through reimbursement design:
“Alnylam’s approach is to start with a clinical package that
provides real benefit to patients and then ensure value is
delivered in the real world over time. It's about reducing
uncertainty and knowing that you're paying for the outcomes you
expect,” said Jim Clement, Executive Director, Value Based Care and
Supply Chain Management, Aetna Pharmacy Management. “I congratulate
Alnylam for establishing the Company as a proactive, innovative and
influential leader in the industry, and am looking forward to being
a part of this Alnylam initiative.”
Alnylam Assist™Today, Alnylam also launched Alnylam
Assist, a comprehensive support services program to help patients.
Alnylam Assist will offer a wide range of personalized services
that include access to in-house Case Managers who will assist with
verification of insurance benefits and financial support for
eligible patients, and field-based Patient Education Liaisons who
will offer patients education on hATTR amyloidosis.
Physicians and patients can learn more about Alnylam’s
comprehensive patient services by visiting AlnylamAssist.com or
calling 1-833-256-2748.
Speeding Availability & Accurate DiagnosisGiven the
rapid and debilitating progression of hATTR amyloidosis, Alnylam is
working to expedite the availability of ONPATTRO to patients whose
physicians have prescribed it so that they can initiate treatment
as quickly as possible. Alnylam is partnering closely with a
network of specialty pharmacies and distributors to make ONPATTRO
commercially available within 48 hours of FDA approval. Alnylam
continues to offer its third-party genetic testing service in the
U.S. and Canada, called Alnylam Act™, which is provided at no
charge to patients and their physicians and aims to reduce the time
to accurate diagnoses.
Visit ONPATTRO.com for more information, including full
prescribing information.
IMPORTANT SAFETY INFORMATION
Infusion-Related ReactionsInfusion-related reactions
(IRRs) have been observed in patients treated with ONPATTRO. In a
controlled clinical study, 19% of ONPATTRO-treated patients
experienced IRRs, compared to 9% 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, acetaminophen, 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 upper respiratory
tract infections (29%) and infusion related reactions (19%).For
additional information about ONPATTRO, please see the full
Prescribing Information.
About ONPATTRO™ (patisiran) lipid complex
injectionONPATTRO was approved by the U.S. Food and Drug
Administration (FDA) for the treatment of the polyneuropathy of
hereditary transthyretin-mediated (hATTR) amyloidosis in adults.
ONPATTRO is the first and only RNA interference (RNAi) therapeutic
approved by the FDA for this indication. ONPATTRO utilizes a novel
approach to target and reduce production of the TTR protein in the
liver via the RNAi pathway. Reducing the TTR protein leads to a
reduction in the amyloid deposits that accumulate in tissues.
ONPATTRO is administered through intravenous (IV) infusion once
every 3 weeks following required premedication and the dose is
based on actual body weight. Home infusion may be an option for
some patients after an evaluation and recommendation by the
treating physician, and may not be covered by all insurance plans.
Regardless of the setting, ONPATTRO infusions should be performed
by a healthcare professional. For more information about ONPATTRO,
visit ONPATTRO.com.
About hATTR AmyloidosisHereditary transthyretin
(TTR)-mediated amyloidosis (hATTR) 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. The median survival is 4.7
years following diagnosis. Until now, people living with hATTR
amyloidosis in the U.S. had no FDA-approved treatment options.
Alnylam Assist™As part of Alnylam’s commitment to making
therapies available to those who may benefit from them, Alnylam
Assist will offer a wide range of services to guide patients
through treatment with ONPATTRO, including financial assistance
options for eligible patients, benefit verification and claims
support, and ordering assistance and facilitation of delivery via
specialty distributor or specialty pharmacy. Patients will have
access to dedicated Case Managers who can provide personalized
support throughout the treatment process and Patient Education
Liaisons to help patients gain a better understanding of the
disease. Visit AlnylamAssist.com for more information.
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. RNAi therapeutics are a new class of medicines that
harness the natural biological process of RNAi. 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 in developing
medicines to improve the care of patients with genetic and other
diseases.
About AlnylamAlnylam (Nasdaq: ALNY) is leading the
translation of RNA interference (RNAi) into a whole new class of
innovative medicines with the potential to improve 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. ONPATTRO, available in the U.S. for the
treatment of the polyneuropathy of hereditary
transthyretin-mediated (hATTR) amyloidosis in adults, is Alnylam’s
first U.S. FDA-approved RNAi therapeutic. Alnylam has a deep
pipeline of investigational medicines, including three 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. For more information about our
people, science and pipeline, 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 approval of ONPATTRO™ (patisiran) lipid complex
injection, including the approved indication, and the implications
of such approval for patients, the results from its APOLLO Phase 3
clinical trial for patisiran, its expectations concerning when
ONPATTRO will be available for shipment to healthcare providers in
the U.S., its plan to offer comprehensive support services for
people prescribed ONPATTRO through Alnylam Assist™, the expected
timing for additional regulatory filings for approval in global
markets, its expectations regarding the potential for patisiran to
improve the lives of hATTR amyloidosis patients with polyneuropathy
and their families, its plans to work with the FDA to expand the
indication for ONPATTRO in the future, 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 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20180810005400/en/
Alnylam Pharmaceuticals, Inc.Christine Regan Lindenboom,
617-682-4340(Investors and Media)orJosh Brodsky,
617-551-8276(Investors)
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