TARRYTOWN, N.Y., Aug. 12, 2020 /PRNewswire/ --
Homozygous familial hypercholesterolemia affects
approximately 1,300 patients in the U.S., the vast majority of whom
are unable to reach target LDL-C levels with currently available
therapies
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced
that the U.S. Food and Drug Administration (FDA) has accepted for
Priority Review a Biologics License Application (BLA) for
evinacumab as an adjunct to other lipid-lowering therapies in
patients with homozygous familial hypercholesterolemia (HoFH).
Evinacumab is the first investigational medicine of its kind to
show efficacy in patients with HoFH – including patients with
little to no low-density lipoprotein (LDL) receptor function – by
binding to and blocking the function of angiopoietin-like 3
(ANGPTL3).
The target action date for the FDA decision is February 11, 2021. The FDA granted evinacumab
Breakthrough Therapy designation in 2017 for the treatment of
hypercholesterolemia in patients with HoFH.
HoFH is an ultra-rare inherited disease that affects
approximately 1,300 patients in the U.S. Patients with HoFH have
severely elevated levels of bad cholesterol (low-density
lipoprotein cholesterol, or LDL-C), which increases their risk for
premature atherosclerotic disease and cardiac events as early as
their teenage years. Treatment guidelines recommend early and
intensive LDL-C lowering, but patients with HoFH are less
responsive (or unresponsive) to standard lipid-lowering therapies,
including statins and PCSK9 (proprotein convertase subtilisin/kexin
type 9) inhibitors.
The BLA was supported by a pivotal Phase 3 trial evaluating the
efficacy and safety of evinacumab in patients with HoFH. Results
from this trial were presented as a late-breaking presentation at
the American College of Cardiology's Annual Scientific Session
together with World Congress of Cardiology (ACC.20) in March 2020.
Previous research published in NEJM in 2017 by the Regeneron
Genetics Center found that patients with loss-of-function mutations
in their ANGPTL3 gene have significantly lower levels of key blood
lipids, including LDL-C. By blocking the ANGPTL3 protein,
evinacumab was designed to replicate this loss-of-function mutation
effect to lower LDL-C in patients with HoFH.
Regulatory submissions for evinacumab are also progressing in
the European Union. In June 2020, the
European Medicines Agency's Committee for Medicinal Products for
Human Use (CHMP) recommended an accelerated assessment for
evinacumab based on the high unmet medical need and therapeutic
innovation demonstrated by the product. The safety and efficacy of
evinacumab have not been fully evaluated by any regulatory
authority.
About evinacumab and the ELIPSE HoFH Trial
Regeneron invented evinacumab using the company's
VelocImmune® technology, a proprietary
genetically-engineered mouse platform endowed with a
genetically-humanized immune system to produce optimized
fully-human monoclonal antibodies. VelocImmune
technology has been used to create multiple FDA-approved
antibodies including Praluent® (alirocumab),
Dupixent® (dupilumab), Libtayo®
(cemiplimab-rwlc) and Kevzara® (sarilumab). Regeneron
previously used these technologies to rapidly develop a treatment
for Ebola virus infection, which is currently under review by the
FDA, and is now being used in efforts to create prophylactic and
treatment medicines for COVID-19.
Evinacumab is an investigational fully-human monoclonal antibody
that binds to and blocks the function of ANGPTL3 and is currently
being studied in patients with HoFH (ongoing Phase 3 extension
trial), refractory hypercholesterolemia (Phase 2) and severe
hypertriglyceridemia (Phase 2).
ELIPSE (Evinacumab LIPid StudiEs)
HoFH was a multi-national Phase 3 randomized, double-blind,
placebo-controlled, parallel-group trial evaluating the efficacy
and safety of evinacumab 15 mg/kg administered intravenously every
four weeks in 65 patients aged 12 years or older with HoFH (43
evinacumab, 22 placebo). The primary endpoint was reduction of
LDL-C from baseline with evinacumab compared to placebo at 24
weeks.
About Regeneron
Regeneron (NASDAQ: REGN)
is a leading biotechnology company that invents life-transforming
medicines for people with serious diseases. Founded and led for
over 30 years by physician-scientists, our unique ability to
repeatedly and consistently translate science into medicine has led
to seven FDA-approved treatments and numerous product candidates in
development, all of which were homegrown in our laboratories. Our
medicines and pipeline are designed to help patients with eye
diseases, allergic and inflammatory diseases, cancer,
cardiovascular and metabolic diseases, pain, infectious diseases
and rare diseases. Regeneron is accelerating and improving the
traditional drug development process through our proprietary
VelociSuite® technologies, such as
VelocImmune, which uses unique genetically-humanized mice to
produce optimized fully-human antibodies and bispecific antibodies,
and through ambitious research initiatives such as the Regeneron
Genetics Center, which is conducting one of the largest genetics
sequencing efforts in the world. For additional information about
the company, please visit www.regeneron.com or follow @Regeneron on
Twitter.
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Media
This press release includes forward-looking statements that
involve risks and uncertainties relating to future events and the
future performance of Regeneron Pharmaceuticals, Inc. ("Regeneron"
or the "Company"), and actual events or results may differ
materially from these forward-looking statements. Words such
as "anticipate," "expect," "intend," "plan," "believe," "seek,"
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uncertainties include, among others, the impact of SARS-CoV-2 (the
virus that has caused the COVID-19 pandemic) on Regeneron's
business and its employees, collaborators, and suppliers and other
third parties on which Regeneron relies, Regeneron's and its
collaborators' ability to continue to conduct research and clinical
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product sales of products marketed by Regeneron and/or its
collaborators (collectively, "Regeneron's Products"), and the
global economy; the nature, timing, and possible success and
therapeutic applications of Regeneron's Products and Regeneron's
product candidates and research and clinical programs now underway
or planned, including without limitation evinacumab; the
likelihood, timing, and scope of possible regulatory approval and
commercial launch of Regeneron's product candidates and new
indications for Regeneron's Products, such as
evinacumab for the treatment of patients with homozygous familial
hypercholesterolemia, refractory hypercholesterolemia, or
severe hypertriglyceridemia; uncertainty of market acceptance and
commercial success of Regeneron's Products and product candidates
and the impact of studies (whether conducted by Regeneron or others
and whether mandated or voluntary) on the commercial success of
Regeneron's Products and product candidates; safety issues
resulting from the administration of Regeneron's Products and
product candidates (such as evinacumab) in patients, including
serious complications or side effects in connection with the use of
Regeneron's Products and product candidates in clinical trials;
determinations by regulatory and administrative governmental
authorities which may delay or restrict Regeneron's ability to
continue to develop or commercialize Regeneron's Products and
product candidates, including without limitation evinacumab;
ongoing regulatory obligations and oversight impacting Regeneron's
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those relating to patient privacy; the availability and extent of
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including private payer healthcare and insurance programs, health
maintenance organizations, pharmacy benefit management companies,
and government programs such as Medicare and Medicaid; coverage and
reimbursement determinations by such payers and new policies and
procedures adopted by such payers; competing drugs and product
candidates that may be superior to, or more cost effective than,
Regeneron's Products and product candidates; the extent to which
the results from the research and development programs conducted by
Regeneron and/or its collaborators may be replicated in other
studies and/or lead to advancement of product candidates to
clinical trials, therapeutic applications, or regulatory approval;
the ability of Regeneron to manufacture and manage supply chains
for multiple products and product candidates; the ability of
Regeneron's collaborators, suppliers, or other third parties (as
applicable) to perform manufacturing, filling, finishing,
packaging, labeling, distribution, and other steps related to
Regeneron's Products and product candidates; unanticipated
expenses; the costs of developing, producing, and selling products;
the ability of Regeneron to meet any of its financial projections
or guidance and changes to the assumptions underlying those
projections or guidance; the potential for any license or
collaboration agreement, including Regeneron's agreements with
Sanofi, Bayer, and Teva Pharmaceutical Industries Ltd. (or their
respective affiliated companies, as applicable), to be cancelled or
terminated without any further product success; and risks
associated with intellectual property of other parties and pending
or future litigation relating thereto (including without limitation
the patent litigation and other related proceedings relating to
EYLEA® (aflibercept) Injection, Dupixent®
(dupilumab), and Praluent® (alirocumab)), other
litigation and other proceedings and government investigations
relating to the Company and/or its operations, the ultimate outcome
of any such proceedings and investigations, and the impact any of
the foregoing may have on Regeneron's business, prospects,
operating results, and financial condition. A more complete
description of these and other material risks can be found in
Regeneron's filings with the U.S. Securities and Exchange
Commission, including its Form 10-K for the year ended December 31, 2019 and its Form 10-Q for the
quarterly period ended June 30, 2020.
Any forward-looking statements are made based on management's
current beliefs and judgment, and the reader is cautioned not to
rely on any forward-looking statements made by Regeneron. Regeneron
does not undertake any obligation to update publicly any
forward-looking statement, including without limitation any
financial projection or guidance, whether as a result of new
information, future events, or otherwise.
Regeneron uses its media and investor relations website and
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Company, including information that may be deemed material to
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(http://newsroom.regeneron.com) and its
Twitter feed (http://twitter.com/regeneron).
Contacts:
Media Relations
Joseph Ricculli
Tel: +1 (914) 418-0405
Joseph.Ricculli@regeneron.com
Investor Relations
Mark Hudson
Tel: +1 (914) 355-0213
Mark.Hudson@regeneron.com
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