– Cardiovascular and Renal Drugs Advisory
Committee Voted 9:3 That the Benefits of Patisiran Outweigh its
Risks for the Treatment of the Cardiomyopathy of ATTR Amyloidosis
–
– Prescription Drug User Fee Act Target Action
Date is October 8, 2023 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced the positive outcome of the
U.S. Food and Drug Administration’s (FDA) Cardiovascular and Renal
Drugs Advisory Committee (CRDAC) meeting to discuss the
supplemental New Drug Application (sNDA) for patisiran, an
investigational RNAi therapeutic in development for the treatment
of the cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis.
The CRDAC voted 9:3 that the benefits of patisiran outweigh its
risks for the treatment of the cardiomyopathy of ATTR
amyloidosis.
“We are grateful for the CRDAC’s thoughtful review and
discussion, and thank the patients, physicians and advocacy
community who shared their valuable insights today,” said Pushkal
Garg, M.D., Chief Medical Officer at Alnylam. “The positive outcome
of today’s meeting is supported by the efficacy and safety data
observed in the APOLLO-B Phase 3 study, and is another step toward
bringing patients with the cardiomyopathy of ATTR amyloidosis a
novel treatment option that addresses the underlying cause of
disease and has the potential to meaningfully benefit patients’
functional capacity and quality of life. We look forward to
continuing to work with the FDA as they complete their review of
our sNDA.”
ATTR amyloidosis is an underdiagnosed, rapidly progressive,
debilitating and fatal disease caused by misfolded transthyretin
(TTR) proteins, which accumulate as amyloid deposits in various
parts of the body, including the heart, resulting in cardiomyopathy
and heart failure. The cardiac manifestations associated with ATTR
amyloidosis can have a devastating impact on patients’ lives and
treatment options today are limited, with many patients continuing
to progress on, or unable to access, current standard-of-care.
The positive decision by the FDA’s CRDAC panel of independent
experts was based on a discussion of the data supporting the sNDA
for patisiran, which include positive results from the APOLLO-B
Phase 3 study that demonstrate favorable effects on functional
capacity and health status and quality of life in patients with
ATTR amyloidosis with cardiomyopathy relative to placebo, as
measured by the 6-Minute Walk Test (6-MWT) and the Kansas City
Cardiomyopathy Questionnaire Overall Summary (KCCQ-OS) score,
respectively.
The CRDAC provides the FDA with independent, expert advice and
recommendations on marketed and investigational medicines for use
in the treatment of cardiovascular and renal disorders. The CRDAC’s
vote, while not binding, will be considered by the FDA when making
its decision regarding the potential expanded indication for
patisiran. The FDA has set an action date of October 8, 2023, under
the Prescription Drug User Fee Act.
Patisiran is the established name for ONPATTRO®, which is
approved by the FDA for the treatment of the polyneuropathy of
hereditary ATTR amyloidosis in adults.
ONPATTRO Indication and Important Safety Information
Indication
ONPATTRO is indicated for the treatment of the polyneuropathy of
hereditary transthyretin-mediated amyloidosis in adults.
Important Safety Information
Infusion-Related Reactions
Infusion-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
Supplementation
ONPATTRO 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 Reactions
The 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
U.S. Prescribing Information.
About ONPATTRO® (patisiran)
ONPATTRO (patisiran) is an RNAi therapeutic that is approved in
the United States and Canada for the treatment of the
polyneuropathy of hereditary ATTR (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. ONPATTRO is an intravenously
administered RNAi therapeutic targeting transthyretin (TTR). It is
designed to target and silence TTR messenger RNA, thereby reducing
the production of TTR protein before it is made. Reducing the
pathogenic protein leads to a reduction in amyloid deposits in
tissues. Patisiran is also being evaluated for the treatment of the
cardiomyopathy of transthyretin-mediated (ATTR) amyloidosis; the
safety and efficacy of patisiran in this indication have not been
established or evaluated by the FDA, EMA or any other health
authority.
About ATTR Amyloidosis
Transthyretin-mediated (ATTR) amyloidosis is an underdiagnosed,
rapidly progressive, debilitating and fatal disease caused by
misfolded transthyretin (TTR) proteins, which accumulate as amyloid
deposits in various parts of the body, including the nerves, heart
and gastrointestinal tract. Patients may present with
polyneuropathy, cardiomyopathy, or both manifestations of disease.
There are two different forms of ATTR amyloidosis – hereditary ATTR
(hATTR) amyloidosis, which is caused by a TTR gene variant and
affects approximately 50,000 people worldwide, and wild-type ATTR
(wtATTR) amyloidosis, which occurs without a TTR gene variant and
impacts an estimated 200,000 – 300,000 people worldwide.
About APOLLO-B Phase 3 Study
APOLLO-B is a Phase 3, randomized, double-blind,
placebo-controlled multicenter global study designed and powered to
evaluate the effects of patisiran on functional capacity and
quality of life in patients with ATTR amyloidosis with
cardiomyopathy. The study enrolled 360 adult patients with ATTR
amyloidosis (hereditary or wild-type) with cardiomyopathy at 69
sites in 21 countries. Patients were randomized 1:1 to receive 0.3
mg/kg of patisiran or placebo intravenously administered every
three weeks over a 12-month treatment period. After 12 months, all
patients received patisiran in an open-label extension period.
About LNP Technology
Alnylam has licenses to Arbutus Biopharma LNP intellectual
property for use in RNAi therapeutic products using LNP
technology.
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
or disease pathway 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 Pharmaceuticals
Alnylam Pharmaceuticals (Nasdaq: ALNY) has led 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 and prevalent diseases with unmet need. Based
on Nobel Prize-winning science, RNAi therapeutics represent a
powerful, clinically validated approach yielding transformative
medicines. Since its founding in 2002, Alnylam has led the RNAi
Revolution and continues to deliver on a bold vision to turn
scientific possibility into reality. Alnylam’s commercial RNAi
therapeutic products are ONPATTRO® (patisiran), AMVUTTRA®
(vutrisiran), GIVLAARI® (givosiran), OXLUMO® (lumasiran), and
Leqvio® (inclisiran), which is being developed and commercialized
by Alnylam’s partner, Novartis. Alnylam has a deep pipeline of
investigational medicines, including multiple product candidates
that are in late-stage development. Alnylam is executing on its
“Alnylam P5x25” strategy to deliver transformative medicines in
both rare and common diseases benefiting patients around the world
through sustainable innovation and exceptional financial
performance, resulting in a leading biotech profile. Alnylam is
headquartered in Cambridge, MA. For more information about our
people, science and pipeline, please visit www.alnylam.com and
engage with us on X (formerly Twitter) at @Alnylam, or on LinkedIn,
Facebook, or Instagram.
Alnylam Forward Looking Statements
This press release contains forward-looking statements within
the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. All statements
other than historical statements of fact regarding Alnylam’s
expectations, beliefs, goals, plans or prospects including, without
limitation, statements, express or implied, relating to the FDA
Advisory Committee’s statements and recommendations regarding the
potential benefits of patisiran for the treatment of the
cardiomyopathy of ATTR amyloidosis in adults, Alnylam’s plans to
continue working with the FDA as they complete their review of the
patisiran sNDA; that the FDA is not bound by the advisory committee
recommendation but takes its advice in consideration when reviewing
applications; and expectations regarding Alnylam’s aspiration to
become a leading biotech company and the planned achievement of its
“Alnylam P5x25” strategy, should be considered forward-looking
statements. 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: the FDA may not approve the sNDA for
patisiran by the application PDUFA date or at all, we may not be
able to comply with all FDA requests, including with respect to our
patisiran sNDA, in a timely manner or at all; Alnylam’s ability to
successfully execute on its “Alnylam P5x25” strategy; Alnylam's
ability to discover and develop novel drug candidates and delivery
approaches and successfully demonstrate the efficacy and safety of
its product candidates; the pre-clinical and clinical results for
Alnylam’s product candidates, including patisiran and vutrisiran;
actions or advice of regulatory agencies and Alnylam’s ability to
obtain and maintain regulatory approval for its product candidates,
including patisiran and vutrisiran, as well as favorable pricing
and reimbursement; successfully launching, marketing and selling
Alnylam’s approved products globally; delays, interruptions or
failures in the manufacture and supply of Alnylam’s product
candidates or its marketed products; delays or interruptions in the
supply of resources needed to advance Alnylam’s research and
development programs, including as may arise from recent
disruptions in the supply of non-human primates; obtaining,
maintaining and protecting intellectual property; Alnylam’s ability
to successfully expand the indication for ONPATTRO or AMVUTTRA in
the future; Alnylam's ability to manage its growth and operating
expenses through disciplined investment in operations and its
ability to achieve a self-sustainable financial profile in the
future without the need for future equity financing; Alnylam’s
ability to maintain strategic business collaborations; Alnylam's
dependence on third parties for the development and
commercialization of certain products, including Roche, Novartis,
Sanofi, Regeneron and Vir; the outcome of litigation; the potential
risks of future government investigations; and unexpected
expenditures; as well as those risks more fully discussed in the
“Risk Factors” filed with Alnylam's 2022 Annual Report on Form 10-K
filed with the Securities and Exchange Commission (SEC), as may be
updated from time to time in Alnylam’s subsequent Quarterly Reports
on Form 10-Q and in its other SEC filings. 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.
This release discusses investigational RNAi therapeutics and
uses of previously approved RNAi therapeutics in development and is
not intended to convey conclusions about efficacy or safety as to
those investigational therapeutics or uses. Patisiran has not been
approved by any regulatory agency for the treatment of ATTR
amyloidosis with cardiomyopathy. No conclusions can or should be
drawn regarding its safety or effectiveness in treating
cardiomyopathy in this population. There is no guarantee that any
investigational therapeutics or expanded uses of commercial
products will successfully complete clinical development or gain
health authority approval.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230913147234/en/
Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024