Novartis reaches another regulatory milestone for CTL019 (tisagenlecleucel) with submission of its MAA* to EMA for children, young adults with r/r B-cell ALL and adult patients with r/r DLBCL
(Thomson Reuters ONE) -
Novartis International AG /
Novartis reaches another regulatory milestone for CTL019 (tisagenlecleucel) with
submission of its MAA* to EMA for children, young adults with r/r B-cell ALL and
adult patients with r/r DLBCL
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The issuer is solely responsible for the content of this announcement.
* Application follows sBLA submission to the FDA for r/r DLBCL which marked
second US application for first-ever FDA approved CAR-T therapy
* Building on the US r/r B-cell ALL experience, Novartis is working closely
with EMA and European treatment centers to make CTL019 available in this
region
* Submission of MAA includes data from global, multi-center Phase II ELIANA
and JULIET studies, including 6-month JULIET data to be presented at ASH
2017
Basel, November 6, 2017 - Novartis today announced that the company has
submitted a Marketing Authorization Application (MAA) to the European Medicines
Agency (EMA) for CTL019 (tisagenlecleucel) for two indications. The application
is for the treatment of children and young adults with relapsed or refractory
(r/r) B-cell acute lymphoblastic leukemia (ALL) and for adult patients with r/r
diffuse large B-cell lymphoma (DLBCL) who are ineligible for autologous stem
cell transplant (ASCT). CTL019 is a novel immunocellular therapy and a one-time
treatment that uses a patient's own T cells to fight cancer.
"Since the historic FDA approval of Kymriah, formerly CTL019, we have launched,
manufactured and supplied this highly individualized immunocelluar therapy in a
commercial setting and the submission to the EMA is a major step toward our goal
of delivering it to more critically ill cancer patients around the world," said
Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer,
Novartis. "We look forward to working with the EMA to make CTL019 available to
the children and adults who may benefit from this novel therapy."
There has been a dire need for innovative therapies to treat pediatric and young
adult patients with r/r B-cell ALL and adult patients with r/r DLBCL, who have
few options and historically poor outcomes. DLBCL is the most common subtype of
non-Hodgkin lymphoma (NHL), accounting for 40% of all NHL cases globally[1]. If
left untreated, r/r DLBCL has a life expectancy of three to four months[2]. In
Europe, ALL accounts for approximately 80% of leukemia cases among children[3].
Less than 10% of patients with relapsed or refractory ALL survive five years[4].
"When tisagenlecleucel became a reality for certain patients and their families
in the US after approval by the FDA for patients with relapsed or refractory ALL
this year, I believe it forever changed the face of cancer treatment," said the
study's principal investigator Stephen J. Schuster, MD, the Robert and Margarita
Louis-Dreyfus Professor in Chronic Lymphocytic Leukemia and Lymphoma Clinical
Care and Research in Penn's Perelman School of Medicine and director of the
Lymphoma Program at the Abramson Cancer Center. "The data show this is a
groundbreaking immunocellular therapy that has the potential to alter outcomes
in patients who have limited options. This submission brings us closer to
realizing that potential for more patients with fatal blood cancers."
CTL019 is an innovative immunocellular therapy that is a one-time treatment.
CTL019 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 CTL019, for the
investigational treatment of cancers.
"For patients in the EU living with these aggressive forms of blood cancer, we
have very limited options to improve their chances of sustained remission after
their disease has relapsed or become refractory to initial treatment," said
Professor Gilles Salles, MD, PhD, Head of Hematology Department, Hospices Civils
de Lyon, Lyon, France. "The data for tisagenlecleucel has provided an optimistic
look at the potential to achieve durable responses in two distinct and
difficult-to-treat patient populations, helping to address a dire unmet need for
patients."
The MAA submission is based on the Novartis-sponsored global, multicenter, phase
II ELIANA and JULIET trials, which were conducted in collaboration with Penn.
ELIANA is the first pediatric global CAR-T cell therapy registration trial,
examining patients in 25 centers in the US, Canada, Australia, Japan and the EU,
including: Austria, Belgium, France, Germany, Italy Norway, and Spain.
JULIET is the first multi-center global registration study for CTL019 in adult
patients with r/r DLBCL. JULIET is the largest study examining a CAR-T therapy
exclusively in DLBCL, enrolling patients from 27 sites in 10 countries across
the US, Canada, Australia, Japan and Europe, including: Austria, France,
Germany, Italy, Norway and the Netherlands. 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 additional regulatory submissions for CTL019 in pediatric and
young adult patients with r/r B-cell ALL and adult patients with r/r DLBCL
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 recognize and
fight the patient's cancer cells and other B cells expressing a particular
antigen.
About CTL019 Manufacturing
CTL019 is manufactured for each individual patient using their own cells at the
Novartis Morris Plains, New Jersey facility. Novartis has designed a reliable
and integrated manufacturing and supply chain platform that allows for an
individualized treatment approach on a global scale. This process includes
cryopreservation of a patient's harvested (or leukapheresed) cells, giving
treating physicians and centers the flexibility to initiate therapy with CTL019
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
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 has also successfully established the CTL019 manufacturing process at
the Fraunhofer-Institut for cell therapy and immunology (Fraunhofer-Institut für
Zelltherapie and Immunologie) facility in Leipzig, Germany, which currently
supports the manufacturing of CTL019 for global clinical trials.
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.
Kymriah(TM), the first approved CAR-T cell therapy, is the cornerstone of this
strategy. 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) US 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/kymriah.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
/DiffuseLargeBCellLymphoma.pdf. Accessed November 2017
[2] Raut, L., Chakrabarti, P. "Management of relapsed-refractory diffuse large B
cell lymphoma." South Asian J Can, 2014 Jan-Mar; 3(1): 66-70.
[3] World Health Organization and European Environment and Health Information
System, "Incidence of Childhood Leukaemia." December 2009. Available at:
http://www.euro.who.int/__data/assets/pdf_file/0005/97016/4.1.-Incidence-of-
childhood-leukaemia-EDITED_layouted.pdf. Accessed November 2017.
[4] Ronson, A., Tvito, A., Rowe, JM. "Treatment of Relapsed/Refractory Acute
Lymphoblastic Leukemia in Adults." Current Oncology Reports, 2016 Jun;18(6):39.
https://www.ncbi.nlm.nih.gov/pubmed/27207612. Accessed November 2017.
# # #
Novartis Media Relations
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Source: Novartis International AG via GlobeNewswire
Unternehmensinformation / Kurzprofil:
Datum: 06.11.2017 - 07:10 Uhr
Sprache: Deutsch
News-ID 566662
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contact information:
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