— PDUFA Date Set for December 3, 2020 —
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the U.S. Food and Drug
Administration (FDA) has accepted the Company’s New Drug
Application (NDA) for lumasiran, an investigational RNAi
therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene
encoding glycolate oxidase (GO) – in development for the treatment
of primary hyperoxaluria type 1 (PH1). The FDA also granted
Priority Review for the NDA, a designation for medicines that have
the potential to provide significant improvements in the treatment,
prevention or diagnosis of a serious disease, with the goal of FDA
taking action within six months compared to 10 months under
standard review.
“We are pleased to have received Priority Review status for
lumasiran and look forward to continuing to work closely with the
FDA during the review process,” said Pritesh J. Gandhi, PharmD.,
Vice President and General Manager, Lumasiran Program at Alnylam.
“Award of Priority Review status adds to the previous grants of
Breakthrough Therapy and Pediatric Rare Disease Designations.
Together, we believe these underscore the potential of lumasiran to
address the underlying pathophysiology of PH1 and the urgent need
for an FDA-approved treatment for this progressive, devastating
disease where liver transplantation is currently the only treatment
that addresses the root cause of disease.”
The FDA has set an action date of December 3, 2020 under the
Prescription Drug User Fee Act (PDUFA), and the Agency has
indicated that they are not currently planning an advisory
committee meeting as part of the NDA review.
In addition, the Marketing Authorisation Application (MAA) for
lumasiran has been submitted to and validated by the European
Medicines Agency (EMA). Lumasiran was previously granted an
accelerated assessment by the EMA, which is awarded to medicines
deemed to be of major public health interest and therapeutic
innovation, and is designed to bring new treatments to patients
more quickly. Accelerated assessment potentially reduces the
Agency’s evaluation time from 210 to 150 days.
Lumasiran has also previously received Orphan Drug Designations
for the treatment of PH1 in the U.S. and Europe and has received a
Priority Medicines (PRIME) designation from the EMA.
About Lumasiran
Lumasiran is an investigational, subcutaneously administered
RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in
development for the treatment of primary hyperoxaluria type 1
(PH1). HAO1 encodes glycolate oxidase (GO).Thus, by silencing HAO1
and depleting the GO enzyme, lumasiran inhibits production of
oxalate – the metabolite that directly contributes to the
pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced
Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which
enables quarterly subcutaneous maintainence dosing with increased
potency and durability and a wide therapeutic index. Lumasiran has
received both U.S. and EU Orphan Drug Designations, a Breakthrough
Therapy Designation and pediatric rare disease designation from the
U.S. Food and Drug Administration (FDA), and a Priority Medicines
(PRIME) designation from the European Medicines Agency (EMA). The
safety and efficacy of lumasiran are being evaluated by the FDA and
EMA.
About Primary Hyperoxaluria Type 1 (PH1)
PH1 is an ultra-rare disease in which excessive oxalate
production results in the deposition of calcium oxalate crystals in
the kidneys and urinary tract and can lead to the formation of
painful and recurrent kidney stones and nephrocalcinosis. Renal
damage is caused by a combination of tubular toxicity from oxalate,
calcium oxalate deposition in the kidneys, and urinary obstruction
by calcium oxalate stones. Compromised kidney function exacerbates
the disease as the excess oxalate can no longer be effectively
excreted, resulting in subsequent accumulation and crystallization
in bones, eyes, skin, and heart, leading to severe illness and
death. Current treatment options are very limited and include
frequent renal dialysis or combined organ transplantation of liver
and kidney, a procedure with high morbidity that is limited due to
organ availability. Although a minority of patients are fully
responsive to vitamin B6 therapy, there are no approved
pharmaceutical therapies for PH1.
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 Pharmaceuticals
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 six 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.
Alnylam Forward Looking Statements
Various statements in this release concerning Alnylam's future
expectations, plans and prospects, including, without limitation,
Alnylam's expectations with respect to the review timelines for the
lumasiran NDA and MAA by the FDA and EMA, respectively, its views
with respect to the implications of accelerated review as it
relates to the potential of lumasiran to address the underlying
pathophysiology of PH1 and the urgent need for an FDA-approved
treatment for PHI, and 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: the direct or indirect impact of the COVID-19 global
pandemic or a future pandemic, such as the scope and duration of
the outbreak, government actions and restrictive measures
implemented in response, material delays in diagnoses of rare
diseases, initiation or continuation of treatment for diseases
addressed by Alnylam products, or in patient enrollment in clinical
trials, potential supply chain disruptions, and other potential
impacts to Alnylam’s business, the effectiveness or timeliness of
steps taken by Alnylam to mitigate the impact of the pandemic, and
Alnylam’s ability to execute business continuity plans to address
disruptions caused by the COVID-19 or a future pandemic; 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
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, including lumasiran, or its marketed products;
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 maintaining regulatory approval and obtaining
pricing and reimbursement for its products, including ONPATTRO and
GIVLAARI; progress in continuing to establish a commercial and
ex-United States infrastructure; successfully launching, marketing
and selling its approved products globally, including ONPATTRO and
GIVLAARI, and achieving net product revenues for ONPATTRO within
its revised expected range during 2020; 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
guidance provided by Alnylam through the implementation of further
discipline in operations to moderate spend and its ability to
achieve a self-sustainable financial profile in the future without
the need for future equity financing; Alnylam’s ability to
establish and maintain strategic business alliances and new
business initiatives, including completing an agreement for funding
by Blackstone of certain R&D activities for vutrisiran and
ALN-AGT; Alnylam's dependence on third parties, including
Regeneron, for development, manufacture and distribution of certain
products, including eye and CNS products, Ironwood, for assistance
with the education about and promotion of GIVLAARI, and Vir for the
development of ALN-COV and other potential RNAi therapeutics
targeting SARS-CoV-2 and host factors for SARS-CoV-2; 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200526005203/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)
Historical Stock Chart
From Feb 2024 to Mar 2024
Alnylam Pharmaceuticals (NASDAQ:ALNY)
Historical Stock Chart
From Mar 2023 to Mar 2024