− PDUFA Date Set for February 4, 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 givosiran, an investigational RNAi
therapeutic targeting aminolevulinic acid synthase 1 (ALAS1) in
development for the treatment of acute hepatic porphyria (AHP), and
granted Priority Review for the NDA. A Priority Review designation
is granted to medicines that the FDA has determined have the
potential to provide significant improvements in the treatment,
prevention or diagnosis of a serious disease, and FDA’s goal is to
take action within six months compared to 10 months under standard
review.
The FDA has set an action date of February 4, 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.
Additionally, the Marketing Authorisation Application (MAA) for
givosiran has been submitted to and validated by the European
Medicines Agency (EMA). Givosiran 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.
Givosiran also previously received Breakthrough Therapy
Designation from the FDA and Orphan Drug Designation in the U.S.,
as well as Priority Medicines (PRIME) Designation from the EMA and
Orphan Drug Designation in the EU.
About Givosiran Givosiran is an investigational,
subcutaneously administered RNAi therapeutic targeting
aminolevulinic acid synthase 1 (ALAS1) in development for the
treatment of acute hepatic porphyria (AHP). Monthly administration
of givosiran has the potential to significantly lower induced liver
ALAS1 levels in a sustained manner and thereby decrease neurotoxic
heme intermediates, aminolevulinic acid (ALA) and porphobilinogen
(PBG), towards normal levels. By reducing accumulation of these
intermediates, givosiran has the potential to prevent or reduce the
occurrence of severe and life-threatening attacks, control chronic
symptoms, and decrease the burden of the disease. Givosiran
utilizes Alnylam’s Enhanced Stabilization Chemistry ESC-GalNAc
conjugate technology, which enables subcutaneous dosing with
increased potency and durability and a wide therapeutic index. The
safety and efficacy of givosiran were evaluated in the ENVISION
Phase 3 trial with positive results; these results have not been
evaluated by the FDA, the EMA or any other health authority.
About Acute Hepatic Porphyria Acute hepatic porphyria
(AHP) refers to a family of rare, genetic diseases characterized by
potentially life-threatening attacks and for some patients chronic
debilitating symptoms that negatively impact daily functioning and
quality of life. AHP is comprised of four subtypes, each resulting
from a genetic defect leading to deficiency in one of the enzymes
of the heme biosynthesis pathway in the liver: acute intermittent
porphyria (AIP), hereditary coproporphyria (HCP), variegate
porphyria (VP), and ALAD-deficiency porphyria (ADP). These defects
cause the accumulation of neurotoxic heme intermediates
aminolevulinic acid (ALA) and porphobilinogen (PBG), with ALA
believed to be the primary neurotoxic intermediate responsible for
causing both attacks and ongoing symptoms between attacks. Common
symptoms of AHP include severe, diffuse abdominal pain, weakness,
nausea, and fatigue. The nonspecific nature of AHP signs and
symptoms can often lead to misdiagnoses of other more common
conditions such as irritable bowel syndrome, appendicitis,
fibromyalgia, and endometriosis, and consequently, patients
afflicted by AHP often remain without a proper diagnosis for up to
15 years. In addition, long-term complications of AHP and its
treatment can include chronic neuropathic pain, hypertension,
chronic kidney disease and liver disease, including iron overload,
fibrosis, cirrhosis and hepatocellular carcinoma. Currently, there
are no treatments approved to prevent debilitating attacks or to
treat the chronic manifestations of the disease.
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 discovery
platform. Alnylam’s first commercial RNAi therapeutic is ONPATTRO®
(patisiran) approved in the U.S., EU, Japan and Canada. Alnylam has
a deep pipeline of investigational medicines, including five
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 1,200 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 Statements Various statements in
this release concerning Alnylam's future expectations, plans and
prospects, including, without limitation, Alnylam's views with
respect to the potential treatment benefits of givosiran and
potential for givosiran to impact the lives of patients, the
acceptance of the givosiran NDA by the FDA and the expected timing
of review based upon the FDA’s Priority Review of the NDA and the
established PDUFA date, expectations regarding the lack of a need
for an advisory committee meeting for givosiran based upon the
FDA’s current plans, the validation of the MAA for givosiran by the
EMA and the expected timing for review based upon the EMA’s grant
of an accelerated assessment, 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/20190805005489/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media) 617-682-4340
Josh Brodsky (Investors) 617-551-8276
Alnylam Pharmaceuticals (NASDAQ:ALNY)
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