− European Commission Decision Expected in
March −
− Positive Opinion is Based on Data from the
Pivotal ENVISION Phase 3 Study −
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, today announced that the Committee for
Medicinal Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has adopted a positive opinion recommending approval
of givosiran, an RNAi therapeutic targeting aminolevulinic acid
synthase 1 (ALAS1) for the treatment of acute hepatic porphyria
(AHP) in adults and adolescents aged 12 years and older. If
approved by the European Commission, givosiran will be
commercialized under the brand name GIVLAARI™.
“Today’s CHMP positive opinion is very encouraging news for
patients living with acute hepatic porphyria and recognizes the
potential of givosiran to address the urgent unmet need that exists
for those living with this ultra-rare, inherited disease,” said
Barry Greene, President, Alnylam Pharmaceuticals. “We are committed
to bringing givosiran to patients in Europe as rapidly as
possible.”
“Acute hepatic porphyria is characterized by debilitating,
potentially life-threatening attacks and chronic disease
manifestations that dramatically impact the lives of patients and
their families,” said Bernhard Kaumanns, Head of Medical Affairs,
Europe, Middle East, Africa and Canada, at Alnylam. “We have seen
in clinical trials that givosiran has the potential to reduce the
frequency of attacks, reduce pain and improve patients’ quality of
life. Today’s recommendation takes us a significant step closer to
making an important new treatment option a reality for AHP patients
in Europe.”
The positive opinion is based on efficacy and safety findings
from the pivotal ENVISION Phase 3 study, including data on the
reduction in the annualized rate of composite porphyria attacks
compared with placebo. Findings were presented in April 2019 at the
54th Annual International Liver Congress of the European
Association for the Study of the Liver (EASL).
Givosiran was granted Priority Medicines (PRIME) Designation by
the EMA as well as Orphan Designation in the European Union.
Givosiran was also granted an accelerated assessment by the EMA,
which is awarded to medicines deemed to be of major public health
interest and therapeutic innovation, and the award is designed to
bring new treatments to patients more quickly. This positive CHMP
opinion follows the recent approval of GIVLAARI™ (givosiran) by the
Food and Drug Administration in November 2019.
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 subcutaneous
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), toward 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.
About ENVISION Phase 3 Study The ENVISION Phase 3 trial
was a randomized, double-blind, placebo-controlled, global,
multicenter study to evaluate the efficacy and safety of givosiran
in patients with a documented diagnosis of acute hepatic porphyria
(AHP). The primary endpoint was reduction relative to placebo in
the annualized rate of composite porphyria attacks, defined as
those requiring hospitalization, urgent healthcare visit, or hemin
administration at home, in patients with acute intermittent
porphyria (AIP, the most common subtype of AHP) over six months.
Key secondary and exploratory endpoints evaluated reductions in
neurotoxic heme intermediates, aminolevulinic acid (ALA) and
porphobilinogen (PBG), usage of hemin, symptoms of AHP, such as
pain, nausea, and fatigue, as well as impact on quality of life.
The trial enrolled 94 patients with AHP, at 36 study sites in 18
countries around the world and is the largest-ever interventional
study conducted in AHP. Patients were randomized 1:1 to givosiran
or placebo, with givosiran administered subcutaneously at 2.5 mg/kg
monthly. Upon completion of dosing in the double-blind period, all
eligible patients (99 percent) enrolled in the ENVISION open-label
extension (OLE) to receive givosiran on an ongoing basis.
About Acute Hepatic Porphyria Acute hepatic porphyria
(AHP) refers to a family of ultra-rare, genetic diseases
characterized by debilitating, potentially life-threatening attacks
and, for some patients, chronic manifestations that negatively
impact daily functioning and quality of life. AHP is comprised of
four subtypes: acute intermittent porphyria (AIP), hereditary
coproporphyria (HCP), variegate porphyria (VP), and ALA
dehydratase-deficiency porphyria (ADP). Each type of AHP results
from a genetic defect leading to deficiency in one of the enzymes
of the heme biosynthesis pathway in the liver. AHP
disproportionately impacts women of working and childbearing age;
symptoms of the disease vary widely. Severe, unexplained abdominal
pain is the most common symptom, which can be accompanied by limb,
back or chest pain, nausea, vomiting, confusion, anxiety, seizures,
weak limbs, constipation, diarrhea, or dark or reddish urine. The
nonspecific nature of AHP signs and symptoms can often lead to
misdiagnoses of other more common conditions, such as viral
gastroenteritis, irritable bowel syndrome (IBS), addiction
withdrawal, and appendicitis. Consequently, patients with AHP can
wait up to 15 years for a confirmed diagnosis. In addition,
long-term complications and comorbidities of AHP can include
hypertension, chronic kidney disease, or liver disease, including
hepatocellular carcinoma. Currently, there are no treatments
approved in Europe 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 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, and Switzerland, and
GIVLAARI™ (givosiran), approved in the U.S. Alnylam has a deep
pipeline of investigational medicines, including five 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 employs
over 1,200 people worldwide and 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 views with
respect to the approval of GIVLAARI™ (givosiran) injection for
subcutaneous use, and the implications of such approval for
patients and their caregivers, the results of the ENVISION Phase 3
clinical trial for givosiran, expectations regarding the review of
GIVLAARI’s marketing authorization application under accelerated
assessment by the EMA for the treatment of patients with AHP, 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, including GIVLAARI,
progress in establishing a commercial and ex-United States
infrastructure, successfully launching, marketing and selling its
approved products globally, including GIVLAARI, 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 and achieve a
self-sustainable financial profile in the future, 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, including Regeneron, for
development, manufacture and distribution of products, and
Ironwood, for assistance with the education about and promotion of
GIVLAARI, 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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Alnylam Pharmaceuticals, Inc.
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
Fiona McMillan (Media, Europe) +44 1628 244960
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