– Positive Scientific Opinion Under Early
Access to Medicines Scheme (EAMS) Will Make Lumasiran Treatment
Available for UK Primary Hyperoxaluria Type 1 (PH1) Patients Before
Marketing Authorization --
– Lumasiran Indicated Within EAMS for the
Treatment of Patients with PH1 --
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the UK’s Medicines and
Healthcare Products Regulatory Agency (MHRA) has granted lumasiran,
an investigational RNAi therapeutic in development for the
treatment of primary hyperoxaluria type 1 (PH1), a positive
scientific opinion through the Early Access to Medicines Scheme
(EAMS). With this decision, eligible PH1 patients in the UK, many
of whom are children, can gain access to lumasiran before the drug
is granted marketing authorization by the European Commission
(EC).
The aim of EAMS is to provide early availability of innovative,
unlicensed medicines to UK patients who have a high degree of unmet
clinical need. The medicines included in the scheme are those that
are intended to treat, diagnose or prevent seriously debilitating
or life-threatening conditions where there are no adequate
treatment options.
PH1 is an ultra-rare orphan disease characterized by excessive
oxalate production, which can lead to end-stage kidney disease
(ESKD) and other systemic complications. PH1 affects 1-3
individuals per million in Europe and the United States; in the
United Kingdom, it is estimated that there are 100 patients
diagnosed with PH1. Current treatment approaches do not prevent
oxalate overproduction and aim to lessen damage to the kidneys and
delay progression to ESKD. PH1 patients with advanced kidney
disease require dialysis to help filter waste products from their
blood, until they are able and eligible to receive a dual or
sequential liver/kidney transplant. However, long-term dialysis and
post-transplant complications can severely impact patients’ quality
of life.
“This positive scientific opinion to make lumasiran available
through the EAMS is wonderful news for PH1 patients and their
families, who currently have limited treatment options,” said
Brendan Martin, Country Manager, UK & Ireland at Alnylam. “New
medicines that address the underlying cause of this ultra-rare
condition, and have the potential for a favorable impact on disease
manifestations, are urgently needed. This decision will allow
eligible PH1 patients in the UK to have access to lumasiran at the
earliest opportunity.”
The MHRA’s decision is based on the evaluation of the effects of
lumasiran in PH1 patients and its safety profile, including data
from the ILLUMINATE-A Phase 3 study. The results of the
ILLUMINATE-A study were presented at the European Renal
Association-European Dialysis and Transplant Association (ERA-EDTA)
International Congress, held June 6-9, 2020 as a virtual event.
A marketing authorization application (MAA) for lumasiran has
been submitted to the European Medicines Agency (EMA) in April 2020
and was granted Accelerated Assessment. Lumasiran previously
received Priority Medicines (PRIME) designation. The EC decision,
which will apply to the UK, is expected in late 2020. In addition,
Alnylam filed a New Drug Application (NDA) with the U.S. Food and
Drug Administration (FDA). The FDA has granted a Priority Review
for the NDA and has set an action date of December 3, 2020 under
the Prescription Drug User Fee Act (PDUFA).
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 subcutaneous dosing with increased potency and durability
and a wide therapeutic index. Lumasiran has received both EU and
U.S. Orphan Drug Designations, Priority Medicines (PRIME)
designation from the European Medicines Agency (EMA) and
Breakthrough Therapy Designation from the U.S. Food and Drug
Administration (FDA). The safety and efficacy of lumasiran are
under evaluation by the FDA and EMA.
About ILLUMINATE-A Phase 3 Study
ILLUMINATE-A (NCT03681184) is a six-month randomized,
double-blind, placebo-controlled, global, multicenter Phase 3 study
(with a 54-month extension period) to evaluate the efficacy and
safety of lumasiran in 39 patients with a documented diagnosis of
PH1. Patients were randomized 2:1 to receive three monthly doses of
lumasiran or placebo followed by quarterly maintenance doses at 3
mg/kg. The primary endpoint was the percent change in 24-hour
urinary oxalate excretion from baseline to the average of months 3
to 6 in the patients treated with lumasiran as compared to placebo.
Treatment arms were stratified at randomization based upon mean
24-hour urinary oxalate during screening (≤ 1.7 or > 1.7
mmol/24hr/1.73m2). Key secondary and exploratory endpoints were
designed to evaluate additional measures of urinary oxalate, plasma
oxalate, estimated glomerular filtration rate (eGFR),
nephrocalcinosis, renal stone events, safety and tolerability.
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 small minority of patients respond
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 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 (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 views with respect to the safety and efficacy of
lumasiran as demonstrated in the ILLUMINATE-A Phase 3 study and the
potential for lumasiran to have a favorable impact on PH1 disease
manifestations, Alnylam’s expectations regarding the implications
of the positive scientific opinion through the EAMS for eligible
PH1 patients in the UK, many of whom are children, Alnylam's
expectations with respect to the review timelines for the lumasiran
NDA and MAA by the FDA and EMA, respectively, Alnylam’s plans,
assuming favorable regulatory reviews, to bring lumasiran to
patients with PH1 around the world, 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.
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Alnylam Pharmaceuticals, Inc.
Fiona McMillan (EU & Canada Head of Communications) +44 1628
244960
Christine Regan Lindenboom (Investors and Media)
+1-617-682-4340
Josh Brodsky (Investors) +1-617-551-8276
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