TARRYTOWN, N.Y., Feb. 11, 2021 /PRNewswire/ --
Homozygous familial hypercholesterolemia (HoFH) is an
ultra-rare inherited condition that affects approximately 1,300
patients in the U.S. and is characterized by extremely high
low-density lipoprotein cholesterol (LDL-C)
In pivotal Phase 3 HoFH trial, adding Evkeeza to standard
lipid-lowering therapies reduced LDL-C by nearly half at 24 weeks,
compared to placebo
Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced
that the U.S. Food and Drug Administration (FDA) approved
EvkeezaTM (evinacumab-dgnb) as an adjunct to other
low-density lipoprotein cholesterol (LDL-C) lowering therapies to
treat adult and pediatric patients aged 12 years and older with
homozygous familial hypercholesterolemia (HoFH). Evkeeza is the
first FDA-approved treatment that binds to and blocks the function
of angiopoietin-like 3 (ANGPTL3), a protein that plays a key role
in lipid metabolism.
"The FDA's approval of Evkeeza is a watershed moment for
individuals born with HoFH, a severe form of familial
hypercholesterolemia," said Katherine A.
Wilemon, Founder and CEO of the FH Foundation. "Those living
with HoFH have faced devastatingly high LDL-C levels and an
uncertain future. Evkeeza significantly lowered LDL-C levels in
clinical trials and this new treatment offers an important new
option for people living with HoFH."
HoFH, also known as homozygous FH, is an ultra-rare inherited
condition that affects approximately 1,300 patients in the U.S.
HoFH occurs when two copies of the familial hypercholesterolemia
(FH)-causing genes are inherited, one from each parent, resulting
in dangerously high levels (>400 mg/dL) of LDL-C (bad
cholesterol). Patients with HoFH are at risk for premature
atherosclerotic disease and cardiac events as early as their
teenage years.
"Evkeeza is a potentially transformational new treatment for
people with HoFH," said Daniel J.
Rader, M.D., Professor and Chair of the Department of
Genetics in the Perelman School of Medicine of the University of Pennsylvania, a leading HoFH expert
who was involved with Evkeeza clinical trials. "Existing therapies
for HoFH are insufficient for the majority of patients. Evkeeza,
through its unique mechanism of action, was shown to reduce LDL-C
levels in patients with all forms of HoFH, even those with nearly
no LDL receptor activity, and represents a highly meaningful
improvement in our ability to control LDL-C levels in patients with
HoFH."
"Evkeeza is the first FDA-approved ANGPTL3 inhibitor and the
latest example of the promise of Regeneron's development approach
that harnesses genetic insights and pioneering technology to
deliver new treatment options for patients who need them," said
George D. Yancopoulos, M.D., Ph.D.,
President and Chief Scientific
Officer at Regeneron. "We are proud to bring Evkeeza to patients
with HoFH, and Regeneron is grateful to the patients and doctors
who participated in our trials to make this a reality."
The FDA approval is based on results from the Phase 3 ELIPSE
HoFH trial, published in the New England Journal of Medicine
(NEJM) in August 2020. In the
trial, 65 patients were randomized to receive either Evkeeza 15
mg/kg intravenously every four weeks (n=43) plus other
lipid-lowering therapies, compared to lipid-lowering therapies
alone (placebo, n=22). The mean baseline LDL-C level of patients in
both groups was 255 mg/dL.
The trial met its primary endpoint, with Evkeeza-treated
patients reducing their LDL-C from baseline by 49% on average
compared to placebo at week 24 (47% reduction Evkeeza, 2% increase
placebo, p<0.0001). At the same time point, compared to
baseline, Evkeeza-treated patients also experienced:
- 132 mg/dL average reduction in LDL-C compared to placebo (135
mg/dL reduction Evkeeza, 3 mg/dL reduction placebo,
p<0.0001).
- Significant reductions were also observed in other key
secondary endpoints including levels of apolipoprotein B (ApoB),
non-high-density lipoprotein cholesterol (non-HDL-C) and total
cholesterol, compared to placebo (p<0.0001 for all).
- Similar levels of LDL-C lowering were also observed in the most
difficult-to-treat patients who often don't respond to certain
other therapies because of limited LDL receptor function, described
as "null/null" (<15% LDL receptor function by in vitro assays)
or "negative/negative" (genetic variants likely to result in
minimal to no LDL receptor function by mutation analysis)
patients.
Reductions in LDL-C seen with Evkeeza were observed as early as
week 2 and maintained throughout the double-blind treatment period
(week 24) and open label trial period (through week 48).
The most common adverse reactions (>3% of patients) reported
from the combined safety analysis of placebo-controlled trials
after 24 weeks that occurred more frequently in Evkeeza patients
(n=81) than placebo (n=54) were nasopharyngitis (16% Evkeeza, 13%
placebo), influenza-like illness (7% Evkeeza, 6% placebo),
dizziness (6% Evkeeza, 0% placebo), rhinorrhea (5% Evkeeza, 0%
placebo), nausea (5% Evkeeza, 2% placebo), pain in extremity (4%
Evkeeza, 0% placebo) and asthenia (4% Evkeeza, 0% placebo). In
clinical trials, adverse reactions led to discontinuation of
treatment in 2% of patients treated with Evkeeza, including 1 case
of anaphylaxis that resolved with treatment, and 2% of patients who
received placebo.
Evkeeza is administered based on weight (15 mg/kg) once a month
via intravenous infusion. The average Wholesale Acquisition Cost
(WAC) per patient in the U.S. will vary based on weight, and is
expected to be approximately $450,000
per year on average. Regeneron is committed to helping
patients who have been prescribed Evkeeza access their medication.
Regeneron's myRARE™ patient support program offers financial
assistance to eligible patients who need help with the
out-of-pocket cost of Evkeeza. Under the program, eligible patients
with commercial insurance may pay as little as $0 in out-of-pocket costs for Evkeeza. In
addition, myRARE™ offers resources to help patients and healthcare
providers get started with Evkeeza including product information,
insurance benefit verification, community resources and appointment
reminders. For more information, call 1-833-EVKEEZA (833-385-3392)
or visit www.EVKEEZA.com.
The FDA evaluated Evkeeza under Priority Review, following the
decision in 2017 to grant Evkeeza Breakthrough Therapy designation
for the treatment of hypercholesterolemia in patients with HoFH.
The FDA reserves its Priority Review for medicines that represent
significant improvements in safety or efficacy in treating serious
conditions, and its Breakthrough Therapy designation is designed to
expedite the development and U.S. review of drugs that target
serious or life-threatening conditions.
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.
About EvkeezaTM (evinacumab-dgnb)
Evkeeza
is a fully-human monoclonal antibody that binds to and blocks the
function of ANGPTL3. Regeneron scientists discovered the
angiopoietin gene family more than two decades ago. Human genetics
research published in NEJM 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 this is associated with a significantly lower risk of
coronary artery disease.
Evkeeza was invented using Regeneron's
VelocImmune® technology that utilizes a
proprietary genetically-engineered mouse platform endowed with a
genetically-humanized immune system to produce optimized
fully-human antibodies. VelocImmune technology has also been
used to create multiple antibodies including Dupixent®
(dupilumab), Libtayo® (cemiplimab-rwlc),
Praluent® (alirocumab), Kevzara® (sarilumab),
Inmazeb™ (atoltivimab, maftivimab, and odesivimab-ebgn) and
Regeneron's antibody cocktail for COVID-19, which was recently
granted Emergency Use Authorization (EUA) in the U.S.
Regulatory review for Evkeeza is ongoing 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 Evkeeza based on the high
unmet medical need and therapeutic innovation demonstrated by the
product.
Important disclosures:
- The FH Foundation is a 501(c)-3 public charity and
receives funding support from federal grants, pharmaceutical
companies including Regeneron, laboratory and medical device
companies, as well as donations from individuals and families
impacted by familial hypercholesterolemia
and lipoprotein(a).
- Dr. Rader has received research support and consulting fees
from Regeneron, including for service on an evinacumab advisory
board, and Regeneron has provided in-kind support for work
conducted in the Penn Medicine Biobank.
IMPORTANT SAFETY INFORMATION FOR
EVKEEZATM (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
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 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, 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.
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 "anticipate," "expect," "intend," "plan," "believe,"
"seek," "estimate," variations of such words, and similar
expressions are intended to identify such forward-looking
statements, although not all forward-looking statements contain
these identifying words. 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 (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 (collectively, "Regeneron's Product Candidates")
and research and clinical programs now underway or planned,
including without limitation EvkeezaTM (evinacumab-dgnb)
as an adjunct to other low-density lipoprotein cholesterol (LDL C)
lowering therapies to treat adult and pediatric patients aged 12
years and older with homozygous familial hypercholesterolemia
(HoFH); uncertainty of 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) on the commercial success of Regeneron's Products
and Regeneron's Product Candidates; the likelihood, timing,
and scope of possible regulatory approval and commercial launch of
Regeneron's Product Candidates (including possible regulatory
approval of Evkeeza in the European Union) and new indications for
Regeneron's Products; safety issues resulting from the
administration of Regeneron's Products (such as Evkeeza) 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; ongoing regulatory obligations and oversight impacting
Regeneron's Products, research and clinical programs, and business,
including those relating to patient privacy; the availability and
extent of reimbursement of Regeneron's Products (such as Evkeeza)
from third-party payers, 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 Regeneron's
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
Regeneron's 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, collaboration, or supply
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; 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), Praluent® (alirocumab), and
REGEN-COVTM (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, 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 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.
Regeneron uses its media and investor relations website and
social media outlets to publish important information about the
Company, including information that may be deemed material to
investors. Financial and other information about Regeneron is
routinely posted and is accessible on Regeneron's media and
investor relations website (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
View original
content:http://www.prnewswire.com/news-releases/fda-approves-first-in-class-evkeeza-evinacumab-dgnb-for-patients-with-ultra-rare-inherited-form-of-high-cholesterol-301227183.html
SOURCE Regeneron Pharmaceuticals, Inc.