PARIS AND TARRYTOWN, N.Y., Sept.
28, 2018 /PRNewswire/ -- The U.S. Food and Drug
Administration (FDA) has approved Libtayo®
(cemiplimab-rwlc) for the treatment of patients with metastatic
cutaneous squamous cell carcinoma (CSCC) or locally advanced CSCC
who are not candidates for curative surgery or curative radiation.
Libtayo is a fully-human monoclonal antibody targeting the immune
checkpoint receptor PD-1 (programmed cell death protein-1) and is
the first and only treatment specifically approved and available
for advanced CSCC in the U.S.
"Today's FDA decision is great
news for patients with advanced CSCC, who previously had no
approved treatment options. This is especially true because these
patients are no longer candidates for curative surgery or
radiation," said Michael R.
Migden, M.D., a lead investigator in the pivotal CSCC
clinical program and Professor in the Departments of Dermatology
and Head and Neck Surgery at The University of
Texas MD Anderson Cancer Center. "Libtayo is an important
new immunotherapy option for U.S. physicians to help address a
significant unmet need in this patient group."
CSCC is the second most common form of skin cancer and is
responsible for an estimated 7,000 deaths each year in the U.S. It
currently accounts for approximately 20 % of all skin cancers in
the U.S., with the number of newly diagnosed cases expected to rise
annually. When CSCC invades deeper layers of the
skin or adjacent tissues, it is categorized as locally advanced.
Once it spreads to other distant parts of the body, it is
considered metastatic.
"By following the science, we
identified early on that advanced CSCC was a promising target for
investigation with Libtayo," said Israel Lowy, M.D.,
Ph.D., Vice President of Global Clinical Development and Head of
Translational Science and Clinical Oncology, Regeneron. "We are
proud to offer patients in the U.S. this first and only treatment
for advanced CSCC and remain focused on advancing our clinical
research investigating Libtayo as a potential monotherapy and
combination therapy in other cancer types."
Libtayo was evaluated by the FDA under Priority Review, which is
reserved for medicines that represent significant improvements in
safety or efficacy in treating serious conditions, and in 2017 was
granted Breakthrough Therapy Designation status for advanced CSCC.
Breakthrough Therapy Designation was created to expedite the
development and review of drugs that have the potential for
substantial improvement in the treatment of serious or
life-threatening conditions.
"In the U.S., CSCC accounts for
one in five skin cancers, and the number of new diagnoses is
increasing," said Olivier Brandicourt, M.D., Chief
Executive Officer, Sanofi. "We believe Libtayo has the potential
to make a difference for U.S. patients with advanced CSCC, as it
helps to fill a critical gap in treatment options. We are committed
to bringing this important medicine to patients in other countries
around the world as quickly as possible."
The recommended dosage of Libtayo is 350 mg administered as an
intravenous infusion over 30 minutes every three weeks, until
disease progression or unacceptable toxicity. Libtayo is
available as a single-dose 350 mg vial.
Libtayo is expected to provide significant value for patients
with advanced CSCC and those who care for them. The U.S. list
price, or wholesale acquisition cost, is $9,100 per three-week treatment cycle. Actual
costs to patients are generally anticipated to be lower as the list
price does not reflect insurance coverage, copay support, or
financial assistance from patient support programs.
Sanofi and Regeneron are committed to helping U.S. patients who
have been prescribed Libtayo access their medication. The companies
have launched Libtayo Surround™ to help patients understand how
Libtayo may be covered by their health insurance plans.
Additionally, Libtayo Surround is designed to help eligible
patients who need financial assistance with their prescription. For
more information, please call 1-877-LIBTAYO (1-877-542-8296),
Option 1, or visit www.Libtayo.com.
Sanofi Genzyme, the specialty care global business unit of
Sanofi, and Regeneron will market Libtayo jointly in the U.S.
Pivotal advanced CSCC clinical program and results
The FDA approval of Libtayo was based on a combined analysis of
data from an open-label, multi-center, non-randomized Phase 2 trial
known as EMPOWER-CSCC-1 (Study 1540) and two advanced CSCC
expansion cohorts from a multi-center, open-label, non-randomized
Phase 1 trial (Study 1423). Together, the trials represent the
largest prospective data set in advanced CSCC.
The major efficacy outcome measures for the integrated analysis
of EMPOWER-CSCC-1 and the two CSCC expansion cohorts were confirmed
objective response rate (ORR), as assessed by independent central
review (ICR), and ICR-assessed duration of response (DOR). The
efficacy analysis was conducted when all patients had the
opportunity for at least six months of
follow-up.
Combined efficacy results (n=108) from EMPOWER-CSCC-1 and the
two advanced CSCC expansion cohorts from the Phase 1 trial were as
follows:
Efficacy
Endpoints*
|
Metastatic
CSCC
(n =
75)
|
Locally
Advanced
CSCC
(n=
33)
|
Combined
CSCC
(n =
108)
|
Confirmed
ORR
|
ORR
(95% confidence
interval [CI])
|
47%
(35, 59)
|
49%
(31, 67)
|
47%
(38, 57)
|
Complete response
rate†
|
5%
|
0%
|
4%
|
Partial response
rate
|
41%
|
49%
|
44%
|
DOR
|
Range in
months
|
3-15+
|
1-13+
|
1-15+
|
Patients with DOR ≥ 6
months, n (%)
|
21 (60%)
|
10 (63%)
|
31 (61%)
|
+Denotes ongoing at last assessment
*Median duration of follow-up: metastatic CSCC: 8.1 months; locally
advanced CSCC: 10.2 months; combined CSCC: 8.9 months
†Only includes patients with complete healing of prior
cutaneous involvement; locally advanced CSCC patients in
EMPOWER-CSCC-1 required biopsy to confirm complete response.
For the combined safety analysis (n=163) of EMPOWER-CSCC-1 and
the two advanced CSCC expansion cohorts, the most common adverse
reactions reported were fatigue (29%), rash (25%) and diarrhea
(22%). Libtayo was permanently discontinued due to adverse
reactions in 5% of patients; adverse reactions resulting in
permanent discontinuation were pneumonitis, autoimmune myocarditis,
hepatitis, aseptic meningitis, complex regional pain syndrome,
cough and muscular weakness. Serious adverse reactions (SAEs)
occurred in 28% of patients. SAEs that occurred in at least 2% of
patients were cellulitis, sepsis, pneumonia, pneumonitis and
urinary tract infection.
Cemiplimab-rwlc development program overview
Cemiplimab-rwlc is being jointly developed by Regeneron and
Sanofi under a global collaboration agreement.
In April 2018, the European
Medicines Agency (EMA) accepted for review the Marketing
Authorization Application for Libtayo for the treatment of patients
with metastatic CSCC or with locally advanced CSCC who are not
candidates for surgery. The EMA review process is anticipated to be
complete in the first half of 2019. There are currently no
EMA-approved treatments for advanced CSCC. Regulatory applications
in additional countries are also being considered for submission
later in 2018.
In addition to advanced CSCC, cemiplimab-rwlc is being
investigated in trials in non-small cell lung cancer, basal cell
carcinoma, and cervical cancer along with trials in squamous cell
carcinoma of the head and neck, melanoma, colorectal cancer,
prostate cancer, multiple myeloma, Hodgkin lymphoma and non-Hodgkin
lymphoma. These potential uses are investigational, and their
safety and efficacy have not been evaluated by any regulatory
authority.
IMPORTANT SAFETY INFORMATION AND INDICATION FOR U.S.
PATIENTS
What is the most important information I should know about
Libtayo?
Libtayo is a medicine that may treat a type of skin
cancer by working with your immune system. Libtayo can cause your
immune system to attack normal organs and tissues in any area of
your body and can affect the way they work. These problems can
sometimes become severe or life-threatening and can lead to death.
These problems may happen anytime during treatment or even after
your treatment has ended.
Call or see your healthcare provider right away if you
develop any symptoms of the following problems or these symptoms
get worse:
- Lung problems (pneumonitis). Signs and symptoms
of pneumonitis may include new or worsening cough, shortness of
breath, and chest pain.
- Intestinal problems (colitis) that can lead to tears or
holes in your intestine. Signs and symptoms of colitis
may include diarrhea (loose stools) or more frequent bowel
movements than usual; stools that are black, tarry, or sticky or
that have blood or mucus; and severe stomach-area (abdomen) pain or
tenderness.
- Liver problems (hepatitis). Signs and symptoms of
hepatitis may include yellowing of your skin or the whites of your
eyes, severe nausea or vomiting, pain on the right side of your
stomach area (abdomen), drowsiness, dark urine (tea colored),
bleeding or bruising more easily than normal, and feeling less
hungry than usual.
- Hormone gland problems (especially the adrenal glands,
pituitary, thyroid and pancreas). Signs and symptoms that your
hormone glands are not working properly may include headaches that
will not go away or unusual headaches, rapid heartbeat, increased
sweating, extreme tiredness, weight gain or weight loss, dizziness
or fainting, feeling more hungry or thirsty than usual, hair loss,
feeling cold, constipation, deeper voice, very low blood pressure,
urinating more often than usual, nausea or vomiting, stomach-area
(abdomen) pain, and changes in mood or behavior, such as decreased
sex drive, irritability, or forgetfulness.
- Kidney problems, including nephritis and kidney failure.
Signs of these problems may include decrease in your amount of
urine, blood in your urine, swelling in your ankles, and loss of
appetite.
- Skin problems. Signs of these problems may include rash,
itching, skin blistering, and painful sores or ulcers in the mouth,
nose, throat, or genital area.
- Problems in other organs. Signs of these problems may
include headache, tiredness or weakness, sleepiness, changes in
heartbeat (such as beating fast, seeming to skip a beat, or a
pounding sensation), confusion, fever, muscle weakness, balance
problems, nausea, vomiting, stiff neck, memory problems, seizures
(encephalitis), swollen lymph nodes, rash or tender lumps on skin,
cough, shortness of breath, vision changes, or eye pain
(sarcoidosis), seeing or hearing things that are not there
(hallucinations), severe muscle weakness, low red blood cells
(anemia), bruises on the skin or bleeding, and changes in
eyesight.
- Rejection of a transplanted organ. Your doctor should
tell you what signs and symptoms you should report and monitor you,
depending on the type of organ transplant that you have had.
- Infusion (IV) reactions that can sometimes be severe
and life-threatening. Signs of these problems may include
chills or shaking, itching or rash, flushing, shortness of breath
or wheezing, dizziness, fever, feeling of passing out, back or neck
pain, and facial swelling.
Getting medical treatment right away may help keep these
problems from becoming more serious.
Your healthcare provider will check you for these problems
during your treatment with Libtayo. Your healthcare provider may
treat you with corticosteroid or hormone replacement medicines.
Your healthcare provider may delay or completely stop treatment if
you have severe side effects.
Before you receive Libtayo, tell your healthcare
provider about all your medical conditions, including if
you:
- have immune system problems such as Crohn's disease, ulcerative
colitis, or lupus;
- have had an organ transplant;
- have lung or breathing problems;
- have liver or kidney problems;
- have diabetes;
- are pregnant or plan to become pregnant; Libtayo can harm your
unborn baby
Females who are able to become pregnant:
-
- Your healthcare provider will give you a pregnancy test before
you start treatment.
- You should use an effective method of birth control during your
treatment and for at least 4 months after your last dose of
Libtayo. Talk with your healthcare provider about birth control
methods that you can use during this time.
- Tell your healthcare provider right away if you become pregnant
or think you may be pregnant during treatment with Libtayo.
- are breastfeeding or plan to breastfeed. It is not known if
Libtayo passes into your breast milk. Do not breastfeed during
treatment and for at least 4 months after the last dose of
Libtayo.
Tell your healthcare provider about all the medicines you
take, including prescription and
over-the-counter medicines, vitamins, and herbal supplements.
The most common side effects of Libtayo include tiredness, rash,
and diarrhea. These are not all the possible side effects of
Libtayo. Call your doctor for medical advice about side effects.
You may report side effects to FDA at 1-800-FDA-1088. You may
also report side effects to Regeneron Pharmaceuticals and Sanofi at
1-877-542-8296.
Please see accompanying full
Prescribing Information, including Medication
Guide.
What is Libtayo?
Libtayo is a prescription medicine used to treat people with a
type of skin cancer called cutaneous squamous cell carcinoma (CSCC)
that has spread or cannot be cured by surgery or radiation.
It is not known if Libtayo is safe and effective in
children.
About Regeneron Pharmaceuticals, Inc
Regeneron (NASDAQ: REGN) is a leading biotechnology company that
invents life-transforming medicines for people with serious
diseases. Founded and led for 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, neuromuscular
diseases,infectious diseases and rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our proprietary
VelociSuite® technologies, such
asVelocImmune® which produces optimized
fully-human antibodies, and 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.
About Sanofi
Sanofi is dedicated to supporting people through their health
challenges. We are a global biopharmaceutical company focused on
human health. We prevent illness with vaccines, provide innovative
treatments to fight pain and ease suffering. We stand by the few
who suffer from rare diseases and the millions with long-term
chronic conditions.
With more than 100,000 people in 100 countries, Sanofi is
transforming scientific innovation into healthcare solutions around
the globe.
Sanofi, Empowering Life
Sanofi Media
Relations Contact Ashleigh Koss
Tel.: +1 (908) 981-8745
Ashleigh.Koss@sanofi.com
Regeneron Media
Relations Contact Sarah
Cornhill Tel.: +1 (914)
847-5018
sarah.cornhill@regeneron.com
|
Investor Relations
Contact George Grofik
Tel.: +33 (0)1 53 77 45 45
ir@sanofi.com
Regeneron Investor
Relations Contact Manisha
Narasimhan, Ph.D. Tel.: +1
(914) 847-5126 Manisha.Narasimhan@regeneron.com
|
Sanofi
Forward-Looking Statements
This press release
contains forward-looking statements as defined in the Private
Securities Litigation Reform Act of 1995, as amended.
Forward-looking statements are statements that are not historical
facts. These statements include projections and estimates regarding
the marketing and other potential of the product, or regarding
potential future revenues from the product. Forward-looking
statements are generally identified by the words "expects",
"anticipates", "believes", "intends", "estimates", "plans" and
similar expressions. Although Sanofi's management believes that the
expectations reflected in such forward-looking statements are
reasonable, investors are cautioned that forward-looking
information and statements are subject to various risks and
uncertainties, many of which are difficult to predict and generally
beyond the control of Sanofi, that could cause actual results and
developments to differ materially from those expressed in, or
implied or projected by, the forward-looking information and
statements. These risks and uncertainties include among other
things, unexpected regulatory actions or delays, or government
regulation generally, that could affect the availability or
commercial potential of the product, the absence of guarantee that
the product will be commercially successful, the uncertainties
inherent in research and development, including future clinical
data and analysis of existing clinical data relating to the
product, including post marketing, unexpected safety, quality or
manufacturing issues, competition in general, risks associated with
intellectual property and any related future litigation and the
ultimate outcome of such litigation, and volatile economic
conditions, as well as those risks discussed or identified in the
public filings with the SEC and the AMF made by Sanofi, including
those listed under "Risk Factors" and "Cautionary Statement
Regarding Forward-Looking Statements" in Sanofi's annual report on
Form 20-F for the year ended December 31, 2017. Other than as
required by applicable law, Sanofi does not undertake any
obligation to update or revise any forward-looking information or
statements.
Regeneron
Forward-Looking Statements and Use of Digital
Media This
news 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 Regeneron's products, product
candidates, and research and clinical programs now underway or
planned, including without limitation Libtayo®
(cemiplimab-rwlc) for the treatment of patients with metastatic
cutaneous squamous cell carcinoma ("CSCC") or patients with locally
advanced CSCC who are not candidates for curative surgery or
curative radiation and other potential indications; the
likelihood and timing of achieving any of Regeneron's anticipated
clinical development milestones; unforeseen safety issues resulting
from the administration of products and product candidates in
patients, including serious complications or side effects in
connection with the use of Regeneron's product candidates (such as
Libtayo) in clinical trials; the likelihood and timing of possible
regulatory approval and commercial launch of Regeneron's late-stage
product candidates and new indications for marketed products,
including without limitation Libtayo for the treatment of
non-small cell lung cancer, basal cell carcinoma, cervical cancer,
squamous cell carcinoma of the head and neck, melanoma, colorectal
cancer, prostate cancer, multiple myeloma, Hodgkin lymphoma,
non-Hodgkin lymphoma, and other potential indications; the
extent to which the results from the research and development
programs conducted by Regeneron or its collaborators may be
replicated in other studies and lead to therapeutic applications;
ongoing regulatory obligations and oversight impacting Regeneron's
marketed products (such as Libtayo), research and clinical
programs, and business, including those relating to patient
privacy; 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; competing drugs and product
candidates that may be superior to Regeneron's products and product
candidates; uncertainty of market acceptance and commercial success
of Regeneron's products and product candidates; 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 to perform
filling, finishing, packaging, labeling, distribution, and other
steps related to Regeneron's products and product candidates;
coverage and reimbursement determinations by third-party payers,
including Medicare and Medicaid; 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
proceedings relating to EYLEA® (aflibercept) Injection, Dupixent®
(dupilumab) Injection, Praluent®
(alirocumab) Injection, the ultimate outcome of any such
litigation proceedings, 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
fiscal year ended December 31, 2017 and its Form 10-Q for the
quarterly period ended June 30, 2018. 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, 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).
|
View original content to download
multimedia:http://www.prnewswire.com/news-releases/fda-approves-libtayo-cemiplimab-rwlc-as-first-and-only-treatment-for-advanced-cutaneous-squamous-cell-carcinoma-300721192.html
SOURCE Sanofi