− First and Only FDA-approved Treatment
Available in the United States for this Indication –
− ONPATTRO Shown to Improve Polyneuropathy
Relative to Placebo, with Reversal of Neuropathy Impairment
Compared to Baseline in Majority of Patients –
− Improvement in Specified Measures of Quality
of Life and Disease Burden Demonstrated Across Diverse, Global
Patient Population –
− Alnylam to Host Conference Call Today at 3:00
p.m. ET. −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading
RNAi therapeutics company, announced today that the United States
Food and Drug Administration (FDA) approved 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 is the first and only FDA-approved treatment for this
indication. hATTR amyloidosis is a rare, inherited, rapidly
progressive and life-threatening disease with a constellation of
manifestations. In addition to polyneuropathy, hATTR amyloidosis
can lead to other significant disabilities including decreased
ambulation with the loss of the ability to walk unaided, a reduced
quality of life, and a decline in cardiac functioning. In the
largest controlled study of hATTR amyloidosis, ONPATTRO was shown
to improve polyneuropathy – with reversal of neuropathy impairment
in a majority of patients – and to improve a composite quality of
life measure, reduce autonomic symptoms, and improve activities of
daily living.
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ONPATTRO™ (patisiran) packaging and
product vial (Photo: Business Wire)
“Alnylam was founded on the vision of harnessing the potential
of RNAi therapeutics to treat human disease, and this approval
heralds the arrival of an entirely new class of medicines. We
believe today draws us ever-closer to achieving our Alnylam 2020
goals of becoming a fully integrated, multi-product
biopharmaceutical company with a sustainable pipeline,” said John
Maraganore, Ph.D., Chief Executive Officer of Alnylam. “With the
potential for the sequential launches of several new medicines in
the coming years, we believe we have the opportunity to
meaningfully impact the lives of people around the world in need of
new approaches to address serious diseases with significant unmet
medical needs.”
“Today’s historic approval marks the arrival of a first-of-its
kind treatment option for a rare and devastating condition with
limited treatment options,” said Akshay Vaishnaw, M.D., Ph.D.,
President of R&D at Alnylam. “We extend our deepest gratitude
to the patients who participated in the ONPATTRO clinical trials
and their families and caregivers who supported them. We are also
grateful for the tireless efforts of the investigators and study
staff, without whom this important milestone would not have been
possible. We also look forward to working with the FDA to
potentially expand the ONPATTRO indication in the future.”
The FDA approval of ONPATTRO was based on positive results from
the randomized, double-blind, placebo-controlled, global Phase 3
APOLLO study, the largest-ever study in hATTR amyloidosis patients
with polyneuropathy. Results from the APOLLO study were published
in the July 5, 2018, issue of The New England Journal of
Medicine.
In APOLLO, the safety and efficacy of ONPATTRO were evaluated in
a diverse, global population of hATTR amyloidosis patients in 19
countries, with a total of 39 TTR mutations. Patients were
randomized in a 2:1 ratio to receive intravenous ONPATTRO (0.3 mg
per kg of body weight) or placebo once every 3 weeks for 18 months.
The study showed that ONPATTRO improved measures of polyneuropathy,
quality of life, activities of daily living, ambulation,
nutritional status and autonomic symptoms relative to placebo in
adult patients with hATTR amyloidosis with polyneuropathy. The
primary endpoint of the APOLLO study was the modified Neuropathy
Impairment Score +7 (mNIS+7), which assesses motor strength,
reflexes, sensation, nerve conduction and postural blood
pressure.
- Patients treated with ONPATTRO had a
mean 6.0-point decrease (improvement) in mNIS+7 score from baseline
compared to a mean 28.0-point increase (worsening) for patients in
the placebo group, resulting in a mean 34.0-point difference
relative to placebo, after 18 months of treatment.
- While nearly all ONPATTRO-treated
patients experienced a treatment benefit relative to placebo, 56
percent of ONPATTRO-treated patients at 18 months of treatment
experienced reversal of neuropathy impairment (as assessed by
mNIS+7 score) relative to their own baseline, compared to four
percent of patients who received placebo.
- Patients treated with ONPATTRO had a
mean 6.7-point decrease (improvement) in Norfolk Quality of Life
Diabetic Neuropathy (QoL-DN) score from baseline compared to a mean
14.4-point increase (worsening) for patients in the placebo group,
resulting in a mean 21.1-point difference relative to placebo,
after 18 months of treatment.
- As measured by Norfolk QoL-DN, 51
percent of patients treated with ONPATTRO experienced improvement
in quality of life at 18 months relative to their own baseline,
compared to 10 percent of the placebo-treated patients.
- Over 18 months of treatment, patients
treated with ONPATTRO experienced significant benefit vs. placebo
for all other secondary efficacy endpoints, including measures of
activities of daily living, walking ability, nutritional status,
and autonomic symptoms.
- The most common adverse events that
occurred more frequently with ONPATTRO than with placebo were upper
respiratory tract infections and infusion-related reactions. To
reduce the risk of infusion-related reactions, patients received
premedications prior to infusion.
“FDA approval of ONPATTRO represents an entirely new approach to
treating patients with polyneuropathy in hATTR amyloidosis and
shows promise as a new era in patient care,” said John Berk, M.D.,
Associate Professor of Medicine at Boston University School of
Medicine and assistant director of the Amyloidosis Center at Boston
University School of Medicine. “Given the strength of the APOLLO
data, including data showing the possibility of halting or
improving disease progression in many patients, ONPATTRO holds
tremendous promise for people living with this disease.”
“For years I have witnessed the tragic impact of hATTR
amyloidosis on generations of families. Today, we celebrate the FDA
approval of ONPATTRO,” said Muriel Finkel, President of Amyloidosis
Support Groups. “It’s extremely gratifying to see promising science
translate into a treatment option that will allow patients to
potentially experience an improvement in their disease and an
improvement in their overall quality of life.”
“Today’s approval is significant in so many respects. It means
the hATTR amyloidosis community of patients, families, caregivers
and healthcare professionals in the United States now has a
treatment option that offers renewed hope,” said Isabelle Lousada,
Founder and Chief Executive Officer of the Amyloidosis Research
Consortium. “With an FDA-approved treatment now available, I am
more optimistic than ever that we can increase awareness of this
rare disease and encourage more people to get tested and receive
the proper diagnosis.”
ONPATTRO is expected to be available for shipment to healthcare
providers in the U.S. within 48 hours.
Alnylam is committed to helping people access the medicines they
are prescribed and will be offering comprehensive support services
for people prescribed ONPATTRO through Alnylam Assist™. Visit
AlnylamAssist.com for more information or call 1-833-256-2748.
ONPATTRO was reviewed by the FDA under Priority Review and had
previously been granted Breakthrough Therapy and Orphan Drug
Designations. On July 27, patisiran received a positive opinion
from the Committee for Medicinal Products for Human Use (CHMP) for
the treatment of hereditary transthyretin-mediated amyloidosis in
adults with stage 1 or stage 2 polyneuropathy under accelerated
assessment by the European Medicines Agency. The recommended
Summary of Product Characteristics (SmPC) for the European Union
(EU) includes data on secondary and exploratory endpoints. Expected
in September, the European Commission will review the CHMP
recommendation to make a final decision on marketing authorization,
applicable to all 28 EU member states, plus Iceland, Liechtenstein
and Norway. Regulatory filings in other markets, including Japan,
are planned beginning in mid-2018.
Visit ONPATTRO.com for more information, including full
Prescribing Information.
Conference Call Details
Alnylam management will discuss the FDA approval via conference
call today, August 10, 2018, at 3:00 p.m. ET. A webcast
presentation will also be available on the Investors page of the
Company's website, www.alnylam.com. To access the call, please dial
(800) 682-0995 (domestic) or (334) 323-0509 (international) five
minutes prior to the start time and refer to conference ID 7371935.
A replay of the call will be available beginning at 6:00 p.m. ET on
August 10, 2018. To access the replay, please dial (888) 203-1112
(domestic) or (719) 457-0820 (international) and refer to
conference ID 7371935.
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/20180810005398/en/
Alnylam Pharmaceuticals, Inc.Christine Regan Lindenboom,
617-682-4340(Investors and Media)orJosh Brodsky,
617-551-8276(Investors)
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