- 68% of patients receiving Kymriah in the ELARA trial
experienced complete response, with an 86% overall response rate,
along with a remarkable safety profile1
- Sustained clinical benefit from Kymriah treatment
demonstrated – of patients who achieved a complete response, 85%
were still in response at 12 months1
- Kymriah can be administered in the outpatient setting,
offering increased flexibility and potentially reducing the burden
of therapy for patients and their care teams1,2
- Kymriah is now FDA approved in three indications and remains
the only CAR-T cell therapy approved in both adult and pediatric
settings1
EAST
HANOVER, N.J., May 27, 2022
/PRNewswire/ -- Novartis today announced the US Food and Drug
Administration (FDA) has granted accelerated approval for
Kymriah® (tisagenlecleucel) for the treatment of adult
patients with relapsed or refractory (r/r) follicular lymphoma (FL)
after two or more lines of systemic therapy. In accordance with the
Accelerated Approval Program, continued approval for this
indication may be contingent upon verification and description of
clinical benefit in confirmatory trial(s). Kymriah is now FDA
approved in three indications and remains the only CAR-T cell
therapy approved in both adult and pediatric
settings1.
"We are proud of today's FDA approval of a third indication for
Kymriah. We hope this treatment option that has the potential for
long-lasting results may help break the unrelenting cycle of
treatment for patients with follicular lymphoma," said Victor Bulto, President, Novartis Innovative
Medicines US. "We are on a mission to build on our pioneering work
in cell therapy and continue to innovate for patient impact."
The approval is based on data from the Phase II ELARA trial, a
single-arm, open-label trial, in which 90 patients were evaluated
for efficacy with a median follow-up of approximately 17 months.
Eighty-six percent of patients treated with Kymriah achieved a
response including 68% who experienced a complete
response1.
Prolonged durable response to treatment was demonstrated with an
estimated 85% of patients who achieved a complete response still in
response 12 months after initial response1. Kymriah was
shown to be effective in high-risk patients including those who
were heavily pretreated or had refractory disease, POD24, bulky
disease or those with high Follicular Lymphoma International
Prognostic Index (FLIPI) scores1.
For the 97 patients evaluable for safety at 21 months of median
follow-up, the safety profile of Kymriah was
remarkable1. Fifty-three percent of patients experienced
any-grade cytokine release syndrome (CRS), as defined by the Lee
scale, and there were no reported cases of high-grade (grade 3 or
higher) CRS1. Forty-three percent of patients
experienced any-grade neurologic events; grade 3 or higher
neurologic events were seen in only 6% of patients1.
Eighteen percent of patients (17 of 97 patients) were infused in an
outpatient setting3.
"Patients with follicular lymphoma who relapse or don't respond
to treatment have a poor prognosis and may face a series of
treatment options without a meaningful, lasting response," said
Stephen J. Schuster, MD, the Robert
and Margarita Louis-Dreyfus Professor in Chronic Lymphocytic
Leukemia and Lymphoma in the Division of Hematology Oncology and
Director, Lymphoma Program and Translational Research at the
University of Pennsylvania's Abramson
Cancer Center, institutional Principal Investigator on the trial.
"This new, effective option for patients with follicular lymphoma
may offer long-term benefit."
While follicular lymphoma is typically an indolent type of
cancer, patients with FL may be exposed to a median of four lines
of treatment, with an upper range of 13 lines4,5.
Although there are multiple systemic therapies available, the
efficacy of these regimens drops off rapidly in later
lines6.
"The approval of Kymriah offers patients with relapsed or
refractory follicular lymphoma a new treatment option and new hope
for improving patient outcomes," said Meghan Gutierrez, Chief Executive Officer at the
Lymphoma Research Foundation. "Having this single infusion
treatment option helps to transform the way healthcare providers
approach this type of blood cancer and we commend those who have
contributed to the acceleration of scientific research for the
benefit of patients."
In early May 2022, the European
Commission approved Kymriah for the treatment of adult patients
with r/r FL after two or more lines of systemic therapy, the third
indication for which Kymriah is available to patients in the
European Union.
Additional efficacy and safety details for Kymriah and full
Prescribing Information can be found at
https://www.novartis.us/sites/www.novartis.us/files/kymriah.pdf.
About Novartis commitment to Oncology Cell Therapy
As
part of the unique Novartis Oncology strategy to pursue four cancer
treatment platforms – radioligand therapy, targeted therapy,
immunotherapy and cell and gene therapy – we strive for cures
through cell therapies in order to enable more patients to live
cancer-free. We will continue to pioneer the science and invest in
our manufacturing and supply chain process to further advance
transformative innovation.
Novartis was the first pharmaceutical company to significantly
invest in pioneering CAR-T research and initiate global CAR-T
trials. Kymriah, the first approved CAR-T cell therapy, developed
in collaboration with the Perelman School of Medicine at the
University of Pennsylvania, is the
foundation of the Novartis commitment to CAR-T cell therapy.
We have made strong progress in broadening our delivery of
Kymriah, which is currently available for use in at least one
indication in 30 countries and at more than 370 certified treatment
centers, with clinical and real-world experience from
administration to more than 6,900 patients. We continue to pioneer
in cell therapy, leveraging our vast experience to develop
next-generation CAR-T cell therapies. These therapies will utilize
our new T-Charge™ platform being evaluated to expand across
hematological malignancies and bring the hope for a cure to
patients with other cancer types.
Novartis has a comprehensive, integrated global CAR-T
manufacturing footprint that strengthens the flexibility,
resilience and sustainability of the Novartis manufacturing and
supply chain.
Important Safety Information
KYMRIAH may cause side
effects that are severe or life-threatening, such as cytokine
release syndrome (CRS) or neurological toxicities. Patients
with CRS may experience symptoms including difficulty breathing,
fever (100.4◦F/38◦C or higher),
chills/shaking chills, severe nausea, vomiting and diarrhea, severe
muscle or joint pain, very low blood pressure, or
dizziness/lightheadedness. Patients may be admitted to the hospital
for CRS and treated with other medications.
Patients with neurological toxicities may experience symptoms
such as altered or decreased consciousness, headaches, delirium,
confusion, agitation, anxiety, seizures, difficulty speaking and
understanding, or loss of balance. Patients should be advised to
call their health care provider or get emergency help right away if
they experience any of these signs and symptoms of CRS or
neurological toxicities.
Because of the risk of CRS and neurological toxicities, KYMRIAH
is only available through a restricted program under a Risk
Evaluation and Mitigation Strategy (REMS) called the KYMRIAH
REMS.
Patients may experience hemophagocytic
lymphohistiocytosis/macrophagocytic activation syndrome. Patients
should discuss the possibility of developing this life-threatening
condition with their health care provider.
Serious allergic reactions, including anaphylaxis, may occur
after KYMRIAH infusion. KYMRIAH can increase the risk of
life-threatening infections that may lead to death. Patients should
be advised to tell their health care provider right away if they
develop fever, chills, or any signs or symptoms of an
infection.
Patients may experience prolonged low blood cell counts
(cytopenias), where 1 or more types of blood cells (red blood
cells, white blood cells, or platelets) are decreased. The
patient's health care provider will do blood tests to check all
their blood cell counts after treatment with KYMRIAH. Patients
should be advised to tell their health care provider right away if
they get a fever, are feeling tired, weak, or short of breath, or
have bruising or bleeding.
Patients may experience hypogammaglobulinemia, a condition in
which the level of immunoglobulins (antibodies) in the blood is low
and the risk of infection is increased. It is expected that
patients may develop hypogammaglobulinemia with KYMRIAH and may
need to receive immunoglobulin replacement for an indefinite amount
of time following treatment with KYMRIAH. Patients should tell
their health care provider about their treatment with KYMRIAH
before receiving a live vaccine.
After treatment with KYMRIAH, patients will be monitored
lifelong by their health care provider, as they may develop
secondary cancers or recurrence of their cancer.
Patients should not drive, operate heavy machinery, or do other
dangerous activities for 8 weeks after receiving KYMRIAH because
the treatment can cause temporary memory and coordination problems,
including sleepiness, confusion, weakness, dizziness, and
seizures.
Some of the most common side effects of KYMRIAH are difficulty
breathing, fever (100.4°F/38°C or higher), chills/shaking chills,
confusion, severe nausea, vomiting and diarrhea, severe muscle or
joint pain, very low blood pressure, dizziness/lightheadedness, and
headache. However, these are not all the possible side effects of
KYMRIAH. Patients should talk to their health care provider for
medical advice about side effects.
Prior to a female patient starting treatment with KYMRIAH, their
health care provider may do a pregnancy test. No information is
available for KYMRIAH use in pregnant or breastfeeding women.
Therefore, KYMRIAH is not recommended for women who are pregnant or
breastfeeding. Patients should talk to their health care provider
about birth control and pregnancy.
Patients should tell their health care provider about all the
medicines they take, including prescription and over-the-counter
medicines, vitamins, and herbal supplements.
After receiving KYMRIAH, patients should be advised that some
commercial HIV tests may cause a false-positive test result.
Patients should also be advised not to donate blood, organs,
tissues, sperm, oocytes, and other cells after receiving
KYMRIAH.
Please see the full Prescribing Information for KYMRIAH,
including Boxed WARNING, and Medication Guide at
www.KYMRIAH.com
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 government, payor and general public
pricing and reimbursement pressures and requirements for increased
pricing transparency; our ability to obtain or maintain proprietary
intellectual property protection; the particular prescribing
preferences of physicians and patients; general political, economic
and business conditions, including the effects of and efforts to
mitigate pandemic diseases such as COVID-19; safety, quality, data
integrity or manufacturing issues; potential or actual data
security and data privacy breaches, or disruptions 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
Located in East Hanover, NJ Novartis Pharmaceuticals
Corporation – an affiliate of Novartis – is reimagining medicine to
improve and extend people's lives. As a leading global medicines
company, we use innovative science and digital technologies to
create transformative treatments in areas of great medical need. In
our quest to find new medicines, we consistently rank among the
world's top companies investing in research and development.
Novartis employs nearly 15,000 people in the United States. For more information,
please visit https://www.novartis.us.
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References
- Kymriah [prescribing information]. East Hanover, NJ: Novartis Pharmaceuticals
Corp; 2022.
- Fowler, N.H., et.al. Assessment of Healthcare Resource
Utilization and Costs in Patients with Relapsed or Refractory
Follicular Lymphoma Undergoing CAR-T Cell Therapy with
Tisagenlecleucel: Results from the Elara Study. Abstract #3533.
2021 American Society of Hematology (ASH) Annual Meeting,
Dec 11-14, Atlanta, GA and Virtual.
- Fowler, N.H., et al.Tisagenlecleucel in adult relapsed or
refractory follicular lymphoma: the phase 2 ELARA trial. Nature
Medicine. 2021;10.1038/s41591-021-01622-0.
- Data on File, Novartis, 2020.
- Schuster, S., et al. Chimeric antigen receptor T cells in
refractory B-cell lymphomas. NEJM. 2017;377(26):2545–2554.
- Sutamtewagul, G. & Link, B.K. Novel treatment approaches
and future perspectives in follicular lymphoma. Ther Adv
Hematol. 2019; 10:1–20.
Novartis Media Relations
E-mail: media.relations@novartis.com
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SOURCE Novartis US