Novartis submits application to FDA for KymriahTM (tisagenlecleucel) in adult patients with r/r DLBCL, seeking second indication for first-ever FDA approved CAR-T therapy
(Thomson Reuters ONE) -
Novartis International AG /
Novartis submits application to FDA for KymriahTM (tisagenlecleucel) in adult
patients with r/r DLBCL, seeking second indication for first-ever FDA approved
CAR-T therapy
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The issuer is solely responsible for the content of this announcement.
* Submission based on updated analyses from global, multi-center Phase II
JULIET study, which met primary endpoint, including 6-month data to be
presented at ASH 2017
* Kymriah has demonstrated durable response rates in r/r DLBCL, a highly
aggressive and difficult-to-treat non-Hodgkin lymphoma that is fatal in
nearly 40% of patients due to relapsed or refractory disease
* Novartis plans to submit an application for marketing authorization with the
European Medicines Agency (EMA) in both DLBCL and pediatric ALL later this
year
Basel, October 31, 2017 - Novartis today announced that the company has
submitted a supplemental Biologics License Application (sBLA) to the US Food and
Drug Administration (FDA) for Kymriah(TM) (tisagenlecleucel) suspension for
intravenous infusion, formerly CTL019, for the treatment of adult patients with
relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) who are
ineligible for autologous stem cell transplant (ASCT). In April 2017, Novartis
received Breakthrough Therapy designation for r/r DLBCL which, if approved,
would be the second indication for Kymriah. In August 2017, Kymriah became the
first available chimeric antigen receptor T cell (CAR-T) therapy when it
received FDA approval five weeks prior to its PDUFA date and was launched for
patients up to 25 years of age with B-cell precursor acute lymphoblastic
leukemia (ALL) that is refractory or has relapsed at least twice. Kymriah is a
novel immunocellular therapy and a one-time treatment that uses a patient's own
T cells to fight cancer.
"Kymriah represents a historic breakthrough in the evolution of individualized
immunotherapy and we are committed to bringing this innovation to as many
patients who may benefit as possible," said Vas Narasimhan, Global Head of Drug
Development and Chief Medical Officer, Novartis. "The response rates we've seen
in the JULIET trial show that Kymriah has the potential to transform treatment
for these patients and we look forward to collaborating with the FDA to make it
available to patients for this second indication."
DLBCL is the most common form of non-Hodgkin lymphoma (NHL), a cancer of the
lymphatic system, accounting for up to 40% of all NHL cases globally[1]. Roughly
50-60% of patients with DLBCL achieve and maintain complete remission after
first-line therapy; however, roughly one-third of patients relapse after
receiving first-line treatment[2],[3]. Only about 25% of patients with r/r DLBCL
are eligible for ASCT, the mainstay of secondary treatment. If left untreated,
r/r DLBCL has a life expectancy of three to four months[2].
"The approval of tisagenlecleucel in the treatment of children and young adults
with second relapse or refractory B-cell ALL was a watershed moment in the
journey for researchers to develop immunocellular therapies," said Stephen
Schuster, MD, director of the Lymphoma Program and Lymphoma Translational
Research, University of Pennsylvania Perelman School of Medicine. "The data show
this therapy could change the treatment paradigm for patients with r/r DLBCL as
we've seen durable complete responses in patients who previously relapsed or
were refractory to prior therapies, and this second filing is a significant step
toward realizing its potential for even more patients who are currently battling
fatal blood cancers."
Kymriah is an innovative immunocellular therapy that is a one-time treatment.
Kymriah uses the 4-1BB costimulatory domain in its chimeric antigen receptor to
enhance cellular expansion and persistence. In 2012, Novartis and the University
of Pennsylvania (Penn) entered into a global collaboration to further research,
develop and commercialize CAR-T cell therapies, including Kymriah, for the
investigational treatment of cancers.
The submission is based on results from the Novartis-sponsored, global, multi-
center Phase II JULIET study (NCT02445248), the first global, multi-center
registration study for Kymriah in adult patients with r/r DLBCL. JULIET was
conducted in collaboration with Penn and is the largest study examining a CAR-T
therapy exclusively in DLBCL, enrolling patients from 27 sites in 10 countries
across the US, Canada, Europe, Australia and Japan. Data from the six-month
primary analysis of JULIET will be presented at the annual meeting of the
American Society of Hematology (ASH) in December 2017.
Novartis plans to submit an additional application for marketing authorization
for Kymriah with the European Medicines Agency (EMA) in both DLBCL and pediatric
ALL later this year. Novartis plans additional regulatory filings for Kymriah
outside the US and EU in 2018.
About CAR-T
CAR-T is different from typical small molecule or biologic therapies because it
is manufactured for each individual patient using their own cells. During the
treatment process, T cells are drawn from a patient's blood and reprogrammed in
the laboratory to create T cells that are genetically coded to hunt the
patient's cancer cells and other B cells expressing a particular antigen.
About Kymriah Manufacturing
Kymriah is manufactured for each individual patient using their own cells at the
Novartis Morris Plains, New Jersey facility. Novartis has successfully
demonstrated a 22-day turnaround time for manufacturing Kymriah in the
commercial setting, and this will continue to be the target. The reliable and
integrated manufacturing and supply chain platform for Kymriah allows for an
individualized treatment approach on a global scale. The process includes
cryopreservation of a patient's harvested (or leukapheresed) cells, giving
treating physicians and centers the flexibility to initiate therapy with Kymriah
based on the individual patient's condition.
Building on the company's experience, having manufactured CAR-T cells for over
250 patients from 11 countries across various indications in clinical trials, it
has demonstrated a high-quality and reproducible product. Novartis continues to
advance its CAR-T manufacturing expertise and make investments to support the
anticipated demand to meet the needs of patients.
Novartis Leadership in Immuno-Oncology
Novartis is at the forefront of investigational immunocellular therapy as the
first pharmaceutical company to initiate global CAR-T trials, and has
significantly invested in CAR-T research and worked with pioneers in the field.
Active research programs are underway targeting other hematologic malignancies
and solid tumors, and include efforts focused on next generation CAR-Ts that
involve simplified manufacturing schemes and gene edited cells.
Kymriah(TM) (tisagenlecleucel) Important Safety information (for pediatric and
young adult patients with B-cell precursor acute lymphoblastic leukemia)
The full prescribing information, including Boxed WARNING, for Kymriah can be
found at:
https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/kymria
h.pdf
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 high fever, difficulty breathing,
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) in the US called Kymriah REMS.
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 (cytopenia), where one
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 of 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, 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
virus vaccine.
After treatment with Kymriah, patients will be monitored life-long by their
health care provider, as they may develop secondary cancers or recurrence of
their leukemia.
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, and
dizziness/lightheadedness. However, these are not all of 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. There is no information available for Kymriah
use in pregnant or breast-feeding women. Therefore, Kymriah is not recommended
for women who are pregnant or breast feeding. If either sex partner has received
Kymriah, 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, or tissues and cells for transplantation after
receiving Kymriah.
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," "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, regarding our ability to implement, scale and sustain commercial
manufacturing for the investigational or approved products described in this
press release, regarding our ability to build and sustain a network of treatment
centers to offer 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. Neither can
there be any guarantee that Novartis will successfully implement, scale and
sustain commercial manufacturing for the investigational or approved products
described in this press release, or successfully build and sustain a network of
treatment centers to offer the investigational or approved products described in
this press release. 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, our ability to
successfully implement, scale and sustain commercial manufacturing and build and
sustain a network of treatment centers; 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; our ability to obtain or maintain proprietary intellectual property
protection; the particular prescribing preferences of physicians and patients;
global trends toward health care cost containment, including government, payor
and general public pricing and reimbursement pressures; general economic and
industry conditions, including the effects of the persistently weak economic and
financial environment in many countries; safety, quality or manufacturing
issues, 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 provides innovative healthcare solutions that address the evolving
needs of patients and societies. Headquartered in Basel, Switzerland, Novartis
offers a diversified portfolio to best meet these needs: innovative medicines,
cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has
leading positions globally in each of these areas. In 2016, the Group achieved
net sales of USD 48.5 billion, while R&D throughout the Group amounted to
approximately USD 9.0 billion. Novartis Group companies employ approximately
121,000 full-time-equivalent associates. Novartis products are sold in
approximately 155 countries around the world. For more information, please visit
http://www.novartis.com.
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References
[1] World Health Organization. Diffuse large B-cell lymphoma. Review of cancer
medicines on the WHO list of essential medicines. Available at:
http://www.who.int/selection_medicines/committees/expert/20/applications/Diffuse
LargeBCellLymphoma.pdf. Accessed October 2017
[2] Friedberg JW (2011) Relapsed/refractory diffuse large B-cell lymphoma.
Hematology Am Soc Hematol Educ Program 2011 498-505. Accessed October 2017.
[3] Raut, L., Chakrabarti, P. "Management of relapsed-refractory diffuse large B
cell lymphoma." South Asian J Can, 2014 Jan-Mar; 3(1): 66-70.
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Datum: 31.10.2017 - 07:15 Uhr
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