− Company Plans to Complete Submission in Early
2020 –
– Lumasiran Granted Pediatric Rare Disease
Designation by FDA for the Treatment of PH1 –
Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi
therapeutics company, announced today that the Company has
initiated a rolling submission of its New Drug Application (NDA) to
the U.S. Food and Drug Administration (FDA) for lumasiran, an
investigational RNAi therapeutic targeting glycolate oxidase for
the treatment of primary hyperoxaluria type 1 (PH1). The rolling
submission allows completed sections of an NDA to be reviewed by
the FDA on an ongoing basis. Specifically, Alnylam has submitted
the non-clinical components to the FDA and expects to submit the
remaining components in early 2020. Alnylam also announced that it
has been granted a pediatric rare disease designation from the FDA
for lumasiran for the treatment of PH1.
“Having recently announced positive topline results in the
ILLUMINATE-A Phase 3 study of lumasiran, we’re now pleased to
initiate our NDA filing, a critical milestone in making this
important medicine available to patients as rapidly as possible. We
look forward to working with the FDA to advance this medicine to
patients and anticipate receiving approval in late 2020, assuming
favorable regulatory review,” said Pritesh J. Gandhi, PharmD, Vice
President and General Manager, Lumasiran program at Alnylam. “In
addition, we are pleased to have received a pediatric rare disease
designation for lumasiran from the FDA, recognizing the serious and
life-threatening manifestations of PH1 affecting pediatric
patients.”
Lumasiran has also received both U.S. and EU Orphan Drug
Designations, a Breakthrough Therapy Designation from the FDA and a
Priority Medicines (PRIME) designation from the European Medicines
Agency (EMA). Alnylam intends to file a Marketing Authorisation
Application (MAA) with the EMA in early 2020.
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 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 have not been evaluated by the
FDA, EMA or any other health authority.
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 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 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, 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. 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 implications of the positive topline results from
the ILLUMINATE-A study, as well as the pediatric rare disease
designation for lumasiran from the FDA, the initiation and expected
timing for completion of its rolling NDA submission to the FDA and
its plans and expected timing for filing an MAA for lumasiran with
the EMA, and expectations regarding 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: 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 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; 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 certain products,
including eye and CNS 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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version on businesswire.com: https://www.businesswire.com/news/home/20200110005467/en/
Alnylam Pharmaceuticals, Inc. Christine Regan Lindenboom
(Investors and Media)
617-682-4340
Joshua Brodsky
(Investors) 617-551-8276
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