Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) announced the U.S.
Food & Drug Administration (FDA) has extended the approval of
Praluent® (alirocumab) as an adjunct to diet and other low-density
lipoprotein cholesterol (LDL-C) lowering therapies to include
pediatric patients aged 8 and older with heterozygous familial
hypercholesterolemia (HeFH).
“Many children with heterozygous familial hypercholesterolemia
(HeFH) are able to substantially improve their LDL-C (bad
cholesterol) with currently available therapies. But for those
children whose LDL-C remains dangerously high, this approval is an
important milestone as it gives these children and their families
an additional option to help reduce and manage their LDL-C levels
much earlier in their lives,” said Mary P. McGowan, M.D.,
Chief Medical Officer of the Family Heart Foundation.
Familial hypercholesterolemia (FH) is an inherited condition
caused by mutations in one of several genes that control how the
body processes cholesterol, which can lead to very high levels of
LDL-C (bad cholesterol). FH can come in two forms: HeFH, which
develops when one mutated gene is inherited from one parent; and
homozygous familial hypercholesterolemia (HoFH), which develops
when a mutated gene is inherited from both parents. Praluent is
approved to treat both children and adults with HeFH and adults
with HoFH.
The approval is based on a Phase 3, randomized multicenter trial
evaluating pediatric patients aged 8 to 17 with HeFH, who had LDL-C
levels of 130mg/dL or greater and were already being treated with
lipid-lowering medications. Patients were randomized to receive
Praluent (N=101) or placebo (N=52) every two or four weeks in two
consecutive cohorts. Patients who received Praluent every four
weeks had 31% lower LDL-C than placebo at 24 weeks (97.5%
Confidence Interval: -45.0% to -17.9%; p<0.0001). Improvements
in additional key lipid parameters were also observed. Results from
the trial were recently published in the Journal of the American
Medical Association Pediatrics.
No new adverse reactions were identified in this trial, and the
safety profile was consistent with the safety profile observed in
adults with HeFH. Across Praluent trials in patients with primary
hyperlipidemia (N=2,476), the most common adverse reactions (≥5%)
more frequently observed with Praluent than placebo have been
injection site reactions (7%), and influenza (6%) and diarrhea
(5%).
“The approval of Praluent for the treatment of high cholesterol
was a historic landmark achievement, as it was the first approved
therapy targeting the genetically-validated PCSK9 target for heart
disease,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair,
President and Chief Scientific Officer at Regeneron, and a
principal inventor of Praluent. “Praluent has made a meaningful
impact in the treatment of adults with familial
hypercholesterolemia, and we are proud that our innovation will now
be able to help appropriate children with the heterozygous form of
this disease manage their dangerously high levels of LDL-C.”
About the Praluent HeFH Pediatric TrialThe
randomized multicenter Phase 3 trial consisted of a 24-week
double-blind, placebo-controlled evaluating the efficacy and safety
of Praluent in pediatric patients aged 8 to 17 years with HeFH
(N=79). The primary endpoint was the percent change in LDL-C from
baseline to week 24 in the Praluent and placebo treated patients.
At baseline, patients were on a low-fat diet and being treated with
background lipid-lowering therapy. In the trial, patients were
randomized 2:1 to receive Praluent or placebo every 2 or 4 weeks.
The Praluent dose was based on body weight.
About PraluentPraluent inhibits the binding of
PCSK9 to the LDL receptor and thereby increases the number of
available LDL receptors on the surface of liver cells to clear LDL,
which lowers LDL-C levels in the blood. Praluent was developed by
Regeneron and Sanofi under a global collaboration agreement and
invented by Regeneron using the company's proprietary VelocImmune®
technology that yields optimized fully-human monoclonal
antibodies.
In the U.S., Praluent is currently indicated:
- to reduce the risk of myocardial infarction, stroke, and
unstable angina requiring hospitalization in adults with
established cardiovascular disease
- as an adjunct to diet, alone or in combination with other
low-density lipoprotein cholesterol (LDL-C) lowering therapies in
adults with primary hyperlipidemia including HeFH to reduce
LDL-C
- as an adjunct to other LDL-C-lowering therapies in adults with
HoFH to reduce LDL-C
- along with diet and other LDL-C lowering treatments in children
aged 8 years and older with HeFH to reduce LDL-C
In addition to the U.S., Praluent is approved in 60 countries,
including the European Union, Japan, Canada, Switzerland and
Brazil.
About
Regeneron’s VelocImmune TechnologyRegeneron’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 a substantial
proportion 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,
Kevzara® (sarilumab), Inmazeb® (atoltivimab, maftivimab
and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg).
IMPORTANT SAFETY INFORMATION AND
INDICATIONS
INDICATIONSPRALUENT is an injectable
prescription medicine used:
- in adults with cardiovascular disease to reduce the risk of
heart attack, stroke, and certain types of chest pain conditions
(unstable angina) requiring hospitalization.
- along with diet, alone or together with other
cholesterol-lowering medicines in adults with high blood
cholesterol levels called primary hyperlipidemia (including a type
of high cholesterol called heterozygous familial
hypercholesterolemia [HeFH]), to reduce low-density lipoprotein
cholesterol (LDL-C) or bad cholesterol.
- along with other LDL-lowering treatments in adults with a type
of high cholesterol called homozygous familial
hypercholesterolemia, who need additional lowering of LDL-C.
- along with diet and other LDL-C lowering treatments in children
aged 8 years and older with HeFH to reduce LDL-C.
It is not known if PRALUENT is safe and effective in children
who are younger than 8 years of age or in children with other types
of high cholesterol (hyperlipemias).
IMPORTANT SAFETY INFORMATION
Do not use PRALUENT if you are allergic to alirocumab or to any
of the ingredients in PRALUENT.
Before you start using PRALUENT, tell your healthcare provider
about all of your medical conditions, including allergies, and if
you are pregnant or plan to become pregnant or if you are
breastfeeding or plan to breastfeed.
Tell your healthcare provider or pharmacist about any medicines
you take, including prescription and over-the-counter medicines,
vitamins, or herbal supplements.
PRALUENT can cause serious side effects, including allergic
reactions that can be severe and require treatment in a hospital.
Stop using PRALUENT and call your healthcare provider or go to the
nearest hospital emergency room right away if you have any symptoms
of an allergic reaction including a severe rash, redness, hives,
severe itching, trouble breathing, or swelling of the face, lips,
throat, or tongue.
The common side effects of PRALUENT include: redness, itching,
swelling, or pain/tenderness at the injection site, flu or flu-like
symptoms, diarrhea, muscle pain, muscle spasms and bruising. Tell
your healthcare provider if you have any side effect that bothers
you or that does not go away.
Talk to your doctor about the right way to prepare and give
yourself a PRALUENT injection and follow the “Instructions For Use”
that comes with PRALUENT. In children aged 12 to 17 years, it is
recommended that PRALUENT be given by or under the supervision of
an adult. In children aged 8 to 11 years, PRALUENT should be given
by a caregiver.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit
www.fda.gov/medwatch or call
1-800-FDA-1088.
Please click here for
full Prescribing Information.
About RegeneronRegeneron (NASDAQ: REGN) is a
leading biotechnology company that invents, develops and
commercializes life-transforming medicines for people with serious
diseases. Founded and led for over 35 years by
physician-scientists, our unique ability to repeatedly and
consistently translate science into medicine has led to numerous
FDA-approved treatments and 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, 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 LinkedIn.
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 nature, timing, and possible success and
therapeutic applications of products marketed or otherwise
commercialized by Regeneron and/or its collaborators or licensees
(collectively, “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 Praluent® (alirocumab) for the treatment of pediatric
patients aged 8 to 17 with heterozygous familial
hypercholesterolemia; uncertainty of the utilization, market
acceptance, and commercial success of Regeneron’s Products and
Regeneron’s Product Candidates and the impact of studies (whether
conducted by Regeneron or others and whether mandated or
voluntary), including the studies discussed or referenced in this
press release, on any of the foregoing; the likelihood, timing, and
scope of possible regulatory approval and commercial launch of
Regeneron’s Product Candidates and new indications for Regeneron’s
Products; the ability of Regeneron’s collaborators, licensees,
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; the ability of Regeneron to manage
supply chains for multiple products and product candidates; safety
issues resulting from the administration of Regeneron’s Products
(such as Praluent) 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 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 or licensees may be
replicated in other studies and/or lead to advancement of product
candidates to clinical trials, therapeutic applications, or
regulatory approval; 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 and Bayer
(or their respective affiliated companies, as applicable) to be
cancelled or terminated; the impact of public health outbreaks,
epidemics, or pandemics (such as the COVID-19 pandemic) on
Regeneron's business; 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), 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,
2023. 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
(https://investor.regeneron.com) and its LinkedIn page
(https://www.linkedin.com/company/regeneron-pharmaceuticals).
Contacts:Media RelationsMary
HeatherTel: +1 914-847-8650mary.heather@regeneron.com |
Investor RelationsMark HudsonTel: +1
914-847-3482mark.hudson@regeneron.com |
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