If approved, Evkeeza would be the first and only treatment of
its kind to help children as young as 5 years old control
dangerously high levels of LDL cholesterol caused by homozygous
familial hypercholesterolemia
TARRYTOWN, N.Y., Nov. 30,
2022 /PRNewswire/
-- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN)
today announced the U.S. Food and Drug Administration (FDA) has
accepted for Priority Review the supplemental Biologics License
Application (sBLA) for Evkeeza® (evinacumab-dgnb) as an
adjunct to other lipid-lowering therapies to treat children
aged 5 to 11 years with homozygous familial hypercholesterolemia
(HoFH). The FDA target action date is March
30, 2023.
HoFH is an ultra-rare inherited condition that affects
approximately 1,300 patients in the U.S. and is the most severe
form of familial hypercholesterolemia (FH). The disease occurs when
two copies of the FH-causing genes are inherited, one from each
parent, resulting in dangerously high levels (usually >400
mg/dL) of low-density lipoprotein-cholesterol (LDL-C or bad
cholesterol). Those living with HoFH are at risk for premature
atherosclerotic disease and cardiac events even in their teenage
years.
The sBLA is supported by data from a three-part trial evaluating
Evkeeza in children aged 5 to 11 years with HoFH. Efficacy was
assessed in 14 children enrolled in the Part B portion of the
trial. Despite treatment with other lipid-lowering therapies, these
children entered the trial with an average LDL-C level of 264
mg/dL, more than twice the target (<110 mg/dL) for pediatric
patients with HoFH. The trial met its primary endpoint, showing
children who added Evkeeza to other lipid-lowering therapies
reduced their LDL-C by 48% at week 24 on average. Furthermore, 79%
(n=11) saw their LDL-C reduced by at least half at 24 weeks
following Evkeeza treatment, with an average absolute reduction in
LDL-C from baseline of 132 mg/dL.
Among 20 children evaluated for long-term safety across Parts A,
B and C of the trial (mean exposure: 52 weeks, range: 42-64 weeks),
the most common adverse events (AEs) occurring in ≥15% of patients
included COVID-19 (n=15), pyrexia (n=5), headache (n=4), throat
pain (oropharyngeal pain, n=4) as well as upper abdominal pain,
diarrhea, vomiting, fatigue, nasopharyngitis, rhinitis and cough
(all n=3). Most reported AEs were mild or moderate, and none led to
study discontinuation. The safety profile of Evkeeza observed in
these patients was generally consistent to those seen in adults and
pediatric patients aged 12 years and older.
Evkeeza is the first angiopoietin-like 3 (ANGPTL3) targeted
therapy approved by the FDA, European Commission and the
United Kingdom's Medicines and
Healthcare products Regulatory Agency as an adjunct therapy for
patients aged 12 years and older with HoFH. The potential use of
Evkeeza in HoFH patients aged 5 to 11 years has not been fully
evaluated by any regulatory authority.
About the Trial
The three-part, single-arm,
open-label trial evaluated Evkeeza in pediatric
patients with HoFH aged 5 to 11 years. Part A
(n=6) was a Phase 1b
trial designed to assess the pharmacokinetics
(PK), safety and tolerability of
Evkeeza.
Efficacy was evaluated in the 24-week treatment period in the
Phase 3 Part B portion, which enrolled 14 patients with an average
age of 9 years. Among them, 86% were on statins, 93% were on
ezetimibe, 50% were on LDL apheresis and 14% were on lomitapide.
Patients received Evkeeza 15 mg/kg every four weeks delivered
intravenously alongside their lipid-lowering treatment regimen. The
primary endpoint was change in LDL-C at week 24. Secondary
endpoints included the effect of Evkeeza on other lipid parameters
(i.e., apolipoprotein B, non-high-density lipoprotein cholesterol,
lipoprotein[a] and total cholesterol), efficacy by mutation status,
safety and tolerability, immunogenicity and PK.
Patients who completed Part A or B were allowed to continue
treatment in Part C (n=20), an ongoing Phase 3 extension trial.
Parts A, B and C were not designed to evaluate the effect of
Evkeeza on cardiovascular events.
About Evkeeza® (evinacumab)
Evkeeza
was invented using Regeneron's
VelocImmune® technology and is a fully
human monoclonal antibody that binds to and blocks the function of
ANGPTL3, a protein that inhibits lipoprotein lipase
(LPL) and endothelial lipase (EL) and regulates
circulating lipids, including LDL-C.
Regeneron scientists discovered the angiopoietin gene family
more than two decades ago. Human genetics research published in
New England Journal of Medicine in 2017 by scientists from
the Regeneron Genetics Center® found that patients whose
ANGPTL3 gene did not function properly (called a "loss-of function
mutation") have significantly lower levels of key blood lipids,
including LDL-C, and that this is associated with a significantly
lower risk of coronary artery disease.
The generic name for Evkeeza in its approved U.S. indications is
evinacumab-dgnb, with dgnb the suffix designated in accordance with
Nonproprietary Naming of Biological Products Guidance for Industry
issued by the FDA. The safety and effectiveness of Evkeeza
have not been established in patients with other causes of
hypercholesterolemia, including those with heterozygous familial
hypercholesterolemia (HeFH). The effect of Evkeeza on
cardiovascular morbidity and mortality has not been determined.
Regeneron is responsible for the development and distribution of
Evkeeza in the U.S. and is collaborating with Ultragenyx to
clinically develop, commercialize and distribute Evkeeza outside of
the U.S.
About Regeneron's VelocImmune
Technology
Regeneron's VelocImmune technology
utilizes a proprietary genetically engineered mouse platform
endowed with a genetically humanized immune system to produce
optimized fully human antibodies. When Regeneron's
President and Chief Scientific Officer George D.
Yancopoulos was a graduate student with his
mentor Frederick W. Alt in 1985, they were the
first to envision making such a genetically
humanized mouse, and Regeneron has spent decades
inventing and developing VelocImmune and
related VelociSuite®
technologies. Dr. Yancopoulos and his team have
used VelocImmune technology to create
approximately one in five of all original, FDA-approved fully human
monoclonal antibodies currently available. This includes
Evkeeza® (evinacumab-dgnb),
REGEN-COV® (casirivimab and
imdevimab), Dupixent®
(dupilumab), Libtayo®
(cemiplimab-rwlc), Praluent®
(alirocumab), Kevzara®
(sarilumab) and Inmazeb®
(atoltivimab, maftivimab and
odesivimab-ebgn).
IMPORTANT SAFETY INFORMATION FOR EVKEEZA®
(evinacumab-dgnb) INJECTION
Who should not use EVKEEZA?
Do not use EVKEEZA if you are allergic to
evinacumab-dgnb or to any of the ingredients in EVKEEZA.
Before receiving EVKEEZA, tell your healthcare provider about
all of your medical conditions, including if you:
- Are pregnant or plan to become pregnant. EVKEEZA may harm your
unborn baby. Tell your healthcare provider if you become pregnant
while using EVKEEZA. People who are able to become
pregnant:
-
- Your healthcare provider may do a pregnancy test before you
start treatment with EVKEEZA
- You should use an effective method of birth control during
treatment and for at least 5 months after the last
dose of EVKEEZA. Talk with your healthcare provider about birth
control methods that you can use during this time.
- Are breastfeeding or plan to breastfeed. It is not known if
EVKEEZA passes into your breast milk. You and your healthcare
provider should decide if you will receive EVKEEZA or
breastfeed.
Tell your healthcare provider about all the medicines you
take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements.
What are the possible side effects of EVKEEZA?
EVKEEZA can cause serious side effects, including:
Allergic reactions (hypersensitivity), including a severe reaction
known as anaphylaxis. Tell your healthcare provider right away
if you get any of the following symptoms: swelling (mainly of the
lips, tongue or throat which makes it difficult to swallow or
breathe), breathing problems or wheezing, feeling dizzy or
fainting, rash, hives, and itching.
The most common side effects of EVKEEZA include symptoms
of the common cold, flu-like symptoms, dizziness, pain in legs or
arms, nausea, and decreased energy.
Tell your healthcare provider if you have any side effect that
bothers you or does not go away. These are not all the possible
side effects of EVKEEZA. Call your doctor for medical advice about
side effects. You may report side effects to FDA at
1-800-FDA-1088.
Please see full Prescribing Information,
including Patient Information.
About Regeneron
Regeneron (NASDAQ: REGN) is a leading
biotechnology company that invents, develops and commercializes
life-transforming medicines for people with serious diseases.
Founded and led for nearly 35 years by physician-scientists, our
unique ability to repeatedly and consistently translate science
into medicine has led to nine FDA-approved treatments and numerous
product candidates in development, almost 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, hematologic conditions, 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 more information, please visit www.Regeneron.com or follow
@Regeneron on Twitter.
Forward-Looking Statements and Use of Digital
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
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These statements concern, and these risks and 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 programs, Regeneron's
ability to manage its supply chain, net product sales of products
marketed or otherwise commercialized by Regeneron and/or its
collaborators or licensees (collectively, "Regeneron's Products"),
and the global economy; the nature, timing, and possible success
and therapeutic applications of Regeneron's Products and product
candidates being developed by Regeneron and/or its collaborators or
licensees (collectively, "Regeneron's Product Candidates") and
research and clinical programs now underway or planned, including
without limitation Evkeeza® (evinacumab-dgnb); 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 Evkeeza for the
treatment of children aged 5 to 11 with homozygous familial
hypercholesterolemia (including potential regulatory approval by
the U.S. Food and Drug Administration based on the supplemental
Biologics License Application discussed in this press release);
uncertainty of the utilization, market acceptance, and commercial
success of Regeneron's Products (such as Evkeeza) and Regeneron's
Product Candidates and the impact of studies (whether conducted by
Regeneron or others and whether mandated or voluntary), including
the study discussed in this press release, on any of the foregoing
or any potential regulatory approval of Regeneron's Products and
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applicable) to perform manufacturing, filling, finishing,
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ability of Regeneron to manage supply chains for multiple products
and product candidates; safety issues resulting from the
administration of Regeneron's Products and Regeneron's Product
Candidates in patients, including serious complications or side
effects in connection with the use of Regeneron's Products and
Regeneron's 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 Regeneron's Product
Candidates, including without limitation Evkeeza; ongoing
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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,
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to which the results from the research and development programs
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replicated in other studies and/or lead to advancement of product
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(or their respective affiliated companies, as applicable), as well
as Regeneron's collaboration with Ultragenyx referenced in this
press release, to be cancelled or terminated; 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, Praluent®
(alirocumab), and REGEN-COV® (casirivimab and
imdevimab)), 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, 2021 and its Form 10-Q for the
quarterly period ended September 30,
2022. 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 or otherwise) any forward-looking statement, including
without limitation any financial projection or guidance, whether as
a result of new information, future events, or otherwise.
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Relations
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Relations
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SOURCE Regeneron Pharmaceuticals, Inc.