- Approval based on NETTER-P trial in which Lutathera
demonstrated a consistent safety profile and comparable drug
exposure between pediatric (ages 12-17) and adult patients
- Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are
a rare cancer that is often unresectable and commonly diagnosed in
the late stages of disease
- Novartis, a leader in radioligand therapy (RLT), is
investigating a portfolio of RLTs to treat a broad range of
cancers, including GEP-NETs, lung, prostate, breast, colon, brain
and pancreatic cancers
EAST
HANOVER, N.J., April 23,
2024 /PRNewswire/ -- Novartis today announced that
the U.S. Food and Drug Administration (FDA) approved
Lutathera® (lutetium Lu 177 dotatate) for the treatment
of pediatric patients 12 years and older with somatostatin
receptor-positive (SSTR+) gastroenteropancreatic neuroendocrine
tumors (GEP-NETs), including foregut, midgut, and hindgut NETs.
This approval makes Lutathera the first therapy specifically
reviewed and approved for use in pediatric patients with
GEP-NETs.
"Lutathera is now the very first therapy approved specifically
for children with GEP-NETs, offering new hope to young patients
living with this rare cancer," said Tina
Deignan, Therapeutic Area Head, Oncology US. "Radioligand
therapies have extraordinary potential to shape the future of
cancer care. With this approval, we have taken another vital step
toward fulfilling that vision, strengthening our commitment to
researching and developing the RLT platform across multiple cancer
types and treatment settings."
NETs are a type of cancer that originates in neuroendocrine
cells throughout the body and are commonly considered slow-growing
malignancies1. The diagnosis of NETs is often delayed
due to the inactive nature of the disease, and approximately 10% to
20% of pediatric patients are diagnosed with metastatic
disease2,3. Even though NETs are an orphan disease,
their incidence has increased over the past several
decades1,4-6.
"While GEP-NETs in children and adolescents are rare, the impact
can be devastating. Today's approval addresses a critical need for
new treatment options for these vulnerable patients," said Dr.
Theodore Laetsch, trial investigator
and Director, Developmental Therapeutics Program, Children's
Hospital of Philadelphia (CHOP), a
NETTER-P clinical trial site. "The introduction of radioligand
therapy significantly advanced how we treat GEP-NETs, and I'm
encouraged that younger patients now have the potential to benefit
from this innovation."
The approval was based on the NETTER-P trial, which evaluated
Lutathera in patients aged 12 to <18 years old with SSTR+
GEP-NETs7. The study reported a safety profile
consistent with the adult population studied in NETTER-1, the
pivotal trial for approval of Lutathera in adults. In addition, the
estimated radiation absorbed dose in pediatric patients was within
established organ thresholds for external beam radiation and
comparable to that in adults for the approved dose.
About Lutathera®
Lutathera® (lutetium Lu 177 dotatate) is approved
in the US for the treatment of adults and children 12 years and
older with SSTR-positive GEP-NETs, including those in the foregut,
midgut and hindgut, an indication which includes the populations
studied in the randomized, controlled Phase III trials NETTER-1 and
NETTER-2. Lutathera is also approved in Europe for unresectable or metastatic,
progressive, well-differentiated (G1 and G2), SSTR-positive
GEP-NETs in adults, and in Japan
for SSTR-positive NETs8,9.
Novartis and Radioligand Therapy (RLT)
Novartis is reimagining cancer care with RLT for patients with
advanced cancers. By harnessing the power of radioactive atoms and
applying it to advanced cancers, RLT is theoretically able to
deliver radiation to target cells anywhere in the
body10,11.
Novartis is investigating a broad portfolio of RLTs, exploring
new isotopes, ligands and combination therapies to look beyond
gastroenteropancreatic neuroendocrine tumors (GEP-NETs) and
prostate cancer and into breast, colon, lung and pancreatic
cancer.
With established global expertise, and specialized supply chain
and manufacturing capabilities across the Novartis network, we are
supporting growing demand for our RLT medicines. Our production
capabilities continue to expand and now include sites in
Millburn, US, Zaragoza,
Spain, Ivrea, Italy and our new state-of-the-art facility in
Indianapolis, US. We recently
announced plans to expand our manufacturing capabilities and build
additional points of supply in Sasayama, Japan, and Haiyan, Zhejiang, China, to produce RLTs for patients
in Japan and China. We are continually evaluating
additional opportunities to increase capacity around the
world.
What is Lutathera?
LUTATHERA is a prescription medicine used to treat adult and
pediatric patients 12 years and older with a type of cancer known
as gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that are
positive for the hormone receptor somatostatin, including GEP-NETs
in the foregut, midgut, and hindgut.
Lutathera Important Safety Information
What are some important things to know about the safety of
LUTATHERA?
LUTATHERA is associated with some serious safety considerations
and, in some cases, these may require your health care provider to
adjust or stop your treatment. You should always follow your health
care provider's instructions. Safety considerations
include:
- Radiation exposure: Treatment with LUTATHERA will expose
you to radiation, which can contribute to your long-term radiation
exposure. Overall radiation exposure is associated with an
increased risk for cancer. The radiation will be detectable in your
urine for up to 30 days following administration of the drug. It is
important to minimize radiation exposure to household contacts
consistent with good radiation safety practices as advised by your
health care provider.
- Bone marrow problems: Treatment with LUTATHERA increases
the risk of myelosuppression, a condition in which bone marrow
activity is decreased, resulting in a drop in blood cell counts.
You may experience blood-related side effects such as low red blood
cells (anemia), low numbers of cells that are responsible for blood
clotting (thrombocytopenia), and low numbers of white blood cells
(neutropenia). Speak with your health care provider if you
experience any signs or symptoms of infection, fever, chills,
dizziness, shortness of breath, or increased bleeding or bruising.
Your health care provider may need to adjust or stop your treatment
accordingly.
- Secondary bone marrow and blood cancers: Other serious
conditions that you may develop as a direct result of treatment
with LUTATHERA include blood and bone marrow disorders known as
secondary myelodysplastic syndrome and cancer known as acute
leukemia. Your health care provider will routinely check your blood
cell counts and tell you if they are too low or too high.
- Kidney problems: Treatment with LUTATHERA will expose
your kidneys to radiation and may impair their ability to work as
normal. You may be at an increased risk for kidney problems after
LUTATHERA treatment if you already have kidney impairment before
treatment. In some cases, patients have experienced kidney failure
after treatment with LUTATHERA. Your health care provider will
provide you with an amino acid solution before, during, and after
LUTATHERA to help protect your kidneys. You should stay well
hydrated before, on the day of, and on the day after your
treatment. You should urinate frequently before, on the day of, and
on the day after administration of LUTATHERA. Your doctor will
monitor your kidney function and may withhold, reduce, or stop your
LUTATHERA treatment accordingly.
- Liver problems: In clinical studies of LUTATHERA, less
than 1% of patients were reported to have tumor bleeding
(hemorrhage), swelling (edema), or tissue damage (necrosis) to the
liver. If you have tumors in your liver, you may be more likely to
experience these side effects. Tell your health care provider right
away if you have any of these signs and symptoms of liver problems:
yellowing of the skin or the whites of the eyes (jaundice), unusual
darkening of the urine, unusual tiredness, right upper stomach area
(abdomen) pain, confusion, and/or swelling of the stomach area
(abdomen). Your health care provider will monitor your liver using
blood tests and may need to withhold, reduce, or stop your
LUTATHERA treatment accordingly.
- Allergic reactions: Allergic reactions have occurred in
people who were treated with LUTATHERA. Notify your health care
provider if you develop symptoms of an allergic reaction. Seek
emergency help right away for any serious allergic reactions.
Symptoms may include trouble breathing or swallowing; raised bumps
(hives); rash or itching; and swelling of the face, lips, tongue,
throat, or arms.
- Hormonal gland problems (carcinoid crisis): During your
treatment you may experience certain symptoms that are related to
hormones released from your cancer. These symptoms may include
flushing, diarrhea, difficulty breathing (bronchospasm), and low
blood pressure (hypotension), and may occur during or within the 24
hours after your first LUTATHERA treatment. Your health care
provider will monitor you closely. Speak with your health care
provider if you experience any of these signs or
symptoms.
- Pregnancy warning: Tell your health care provider if you
are pregnant. LUTATHERA can harm your unborn baby. Females should
use an effective method of birth control during treatment and for 7
months after the last dose of LUTATHERA. Males with female partners
should use an effective method of birth control during treatment
with LUTATHERA and for 4 months after the last dose.
- Breastfeeding warning: You should not breastfeed during
treatment with LUTATHERA and for 2.5 months after your last dose of
LUTATHERA.
- Fertility problems: Treatment with LUTATHERA may cause
infertility. This is because radiation absorbed by your testes or
ovaries over the treatment period falls within the range of
exposure in which temporary or permanent infertility may
occur.
What are the most common side effects of
LUTATHERA?
The most common and most serious side effects of LUTATHERA include
decreased blood cell counts, increased liver enzymes, vomiting,
nausea, increased blood glucose, and decreased blood potassium
levels.
Talk to your doctor if you experience any of these side effects.
There are other possible side effects of LUTATHERA. For more
information and to learn more about LUTATHERA, talk to your doctor
or health care provider.
Adverse reactions observed in pediatric patients were similar to
those observed in adults treated with LUTATHERA.
What other medicines may interact with
LUTATHERA?
Tell your health care provider if you are taking any other
medications. Somatostatin analogs and glucocorticoids may affect
how your LUTATHERA treatment works. You should stop taking your
long-acting somatostatin analog at least 4 weeks before LUTATHERA
treatment. You may continue taking short-acting somatostatin
analogs up to 24 hours before your LUTATHERA treatment. Avoid
repeated high doses of glucocorticoids during treatment with
LUTATHERA.
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 see full Prescribing Information for LUTATHERA
at
https://www.novartis.com/us-en/sites/novartis_us/files/lutathera.pdf.
Disclaimer
This press release contains
forward-looking statements within the meaning of the United States
Private Securities Litigation Reform Act of 1995. Forward-looking
statements can generally be identified by words such as
"potential," "can," "will," "plan," "may," "could,"
"would," "expect," "anticipate," "look forward," "believe,"
"committed," "investigational," "pipeline," "launch," or similar
terms, or by express or implied discussions regarding potential
marketing approvals, new indications or labeling for the
investigational or approved products described in this press
release, or regarding potential future revenues from such products.
You should not place undue reliance on these statements. Such
forward-looking statements are based on our current beliefs and
expectations regarding future events, and are subject to
significant known and unknown risks and uncertainties. Should one
or more of these risks or uncertainties materialize, or should
underlying assumptions prove incorrect, actual results may vary
materially from those set forth in the forward-looking statements.
There can be no guarantee that the investigational or approved
products described in this press release will be submitted or
approved for sale or for any additional indications or labeling in
any market, or at any particular time. Nor can there be any
guarantee that such products will be commercially successful in the
future. In particular, our expectations regarding such products
could be affected by, among other things, the uncertainties
inherent in research and development, including clinical trial
results and additional analysis of existing clinical data;
regulatory actions or delays or government regulation generally;
global trends toward health care cost containment, including
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ability to obtain or maintain proprietary intellectual property
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and patients; general political, economic and business conditions,
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of our information technology systems, and other risks and factors
referred to in Novartis AG's current Form 20-F on file with the US
Securities and Exchange Commission. Novartis is providing the
information in this press release as of this date and does not
undertake any obligation to update any forward-looking statements
contained in this press release as a result of new information,
future events or otherwise.
About Novartis
Novartis is an innovative medicines
company. Every day, we work to reimagine medicine to improve and
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and societies are empowered in the face of serious disease. Our
medicines reach more than 250 million people worldwide.
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Safety and Dosimetry of Lutathera in Adolescent Patients With
GEP-NETs and PPGLs. Accessed March
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- Lutathera. Full Prescribing Information. 2018. Revised March
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Revised February 2023.
https://www.ema.europa.eu/en/documents/product-information/lutathera-epar-product-information_en.pdf.
- Jadvar H. Targeted radionuclide therapy: an evolution toward
precision cancer treatment. AJR Am J Roentagenol.
2017;209(2);277-288.
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SOURCE Novartis US