- Anticipated approval could provide new
option for population with limited treatments
- Expanded indication brings medicine to
elderly AML patients who are not eligible for haematopoietic stem
cell transplantationand who have >30% myeloblasts in their bone
marrow
Celgene International Sàrl, a wholly owned subsidiary of Celgene
Corporation (NASDAQ: CELG) today announced that that the European
Medicines Agency's (EMA) Committee for Medicinal Products for Human
Use (CHMP) has adopted a positive opinion for an expanded
indication of VIDAZA® (azacitidine for injection) for the treatment
of adult patients aged 65 years or older with acute myeloid
leukaemia (AML) who are not eligible for haematopoietic stem cell
transplantation (HSCT). The expanded indication now covers patients
who have >30% myeloblasts according to the WHO classification;
previously, the indication covered AML patients with <30%
blasts.
Myeloblasts are white cells in the bone marrow; in AML, their
functioning is disrupted and results in numerous non-functioning
white cells, which can potentially interfere with the body’s
ability to control infections and can lead to anaemia and
haemorrhages.
For many patients, AML is typically associated with a poor
prognosis and deteriorating quality of life, particularly for those
patients who cannot tolerate curative therapies like stem cell
transplantation. In Europe, more than 14,000 people suffer from
AML, and most of these patients will die within less than 1 year.
As an acute leukaemia, AML progresses rapidly and is typically
fatal within months if stem cell transplant is not an option.
Specific to elderly patients, overall survival with AML has not
improved in more than 40 years1, and there is a clear need for
treatments that can support this patient population.
“While progress has been made in treating younger, fitter AML
patients who can undergo intensive and potentially curative
therapies such as stem cell transplant, there is still a clear need
for treatments for elderly and more frail patients,” said Hervé
Dombret, M.D., Chief, Blood Disease Department (Leukaemia Unit),
University Hospital Saint-Louis, AP-HP, Paris, France. “Azacitidine
has demonstrated a median overall survival of 10.4 months, and
these results suggest that, if approved, azacitidine could provide
a valuable treatment option for patients who have limited options
today.”
Adds Tuomo Pätsi, President of Celgene in Europe, Middle East
and Africa (EMEA): “Celgene is committed to bringing innovative
medicines to patients with haematological diseases including AML.
With the positive CHMP opinion for VIDAZA in AML, Celgene has an
opportunity to advance the treatment options available to patients
with AML. And, we will continue to focus on meeting the unmet needs
of patients with myeloid disease, as we have several partnerships
and development programmes that will build on what we are learning
about treating these diseases.”
The CHMP decision was based on data from AML-001, a global,
multi-centre, randomized, open-label pivotal study of patients at
least 65 years old with newly diagnosed or secondary AML with
>30% bone marrow blasts. VIDAZA plus best supportive care
(n=241) was compared with conventional care regimens (n=247).
Median overall survival (OS), the primary endpoint of the study,
was 10.4 months (95% CI 8.0-12.7 months) for patients receiving
azacitidine compared with 6.5 months (95% CI: 5.0-8.6) for patients
receiving conventional treatment regimens (HR=0.85 [95% CI 0.69,
1.03], stratified log-rank p=0.1009). One-year survival rates with
azacitidine and conventional treatment regimens were 46.5% and
34.2%, respectively (difference 12.3% [95% CI: 3.5% - 21%]).
In the study, grade 3-4 anaemia, neutropenia, febrile
neutropenia, and thrombocytopenia rates, respectively, were 16%,
26%, 28%, and 24% with azacitidine; 5%, 5%, 28%, 5% with best
supportive care; 23%, 25%, 30%, 28% with low-dose Ara-Cytarabine;
and 14%, 33%, 31%, 21% with intensive chemotherapy.
In addition to recommending the marketing authorisation for the
new indication to the European Commission, the CHMP also noted
that this new therapeutic indication brings significant clinical
benefit in comparison with existing therapies; if the European
Commission adopts the CHMP decision in full, VIDAZA will
receive extended market protection in all its indications for an
additional year throughout the European Economic Area.
The CHMP reviews applications for all 28 member states in the
European Union (EU), as well as Norway, Liechtenstein and Iceland.
The European Commission, which generally follows the recommendation
of the CHMP, is expected to make its final decision within two
months. If approval is granted, detailed conditions for the use of
this product will be described in the Summary of Product
Characteristics (SmPC), which will be published in the revised
European Public Assessment Report (EPAR).
The anticipated European Commission decision would add to the
portfolio of indications VIDAZA is authorised for across high-risk
myeloid diseases, including myelodysplastic syndromes (MDS) and
AML. VIDAZA has been approved in the EU since 2008 for the
treatment of adult patients ineligible for transplantation
diagnosed with intermediate 2 and high-risk MDS; chronic
myelomonocytic leukaemia (CMML) with 10-29 % marrow blasts without
myeloproliferative disorder; or acute myeloid leukaemia (AML) with
20-30 % blasts and multi-lineage dysplasia.
In the United States, VIDAZA is not indicated for treatment of
patients with AML. VIDAZA is indicated for treatment of patients
with the following French-American-British (FAB) myelodysplastic
syndrome subtypes: refractory anaemia (RA) or refractory anaemia
with ringed sideroblasts (RARS) (if accompanied by neutropenia or
thrombocytopenia or requiring transfusions), refractory anaemia
with excess blasts (RAEB), refractory anaemia with excess blasts in
transformation (RAEB-T), and chronic myelomonocytic leukemia
(CMMoL).
IMPORTANT SAFETY INFORMATION
CONTRAINDICATIONS:
VIDAZA is contraindicated in patients with a known
hypersensitivity to azacitidine or mannitol and in patients with
advanced malignant hepatic tumors
WARNINGS AND PRECAUTIONS:
Anemia, Neutropenia and Thrombocytopenia:
Because treatment with VIDAZA causes anemia, neutropenia, and
thrombocytopenia, monitor complete blood counts frequently for
response and/or toxicity, at a minimum, prior to each dosing
cycle
VIDAZA Toxicity in Patients with Severe Pre-existing Hepatic
Impairment:
Because azacitidine is potentially hepatotoxic in patients with
severe preexisting hepatic impairment, caution is needed in
patients with liver disease.
Renal Toxicity:
Azacitidine and its metabolites are primarily excreted by the
kidneys and the risk of toxic reactions to this drug may be greater
in patients with impaired renal function. These patients, including
the elderly should be closely monitored for toxicity
Use in Pregnancy:
VIDAZA may cause fetal harm when administered to a pregnant
woman. Women of childbearing potential should be apprised of the
potential hazard to the fetus. Men should be advised not to father
a child while receiving VIDAZA
USE IN SPECIFIC POPULATIONS:
Nursing Mothers:
Nursing mothers should be advised to discontinue nursing or the
drug, taking into consideration the importance of the drug to the
mother
ADVERSE REACTIONS:
In Studies 1 and 2, the most commonly occurring adverse
reactions by SC route were nausea (70.5%), anemia (69.5%),
thrombocytopenia (65.5%), vomiting (54.1%), pyrexia (51.8%),
leukopenia (48.2%), diarrhea (36.4%), injection site erythema
(35.0%), constipation (33.6%), neutropenia (32.3%), and ecchymosis
(30.5%). Other adverse reactions included dizziness (18.6%), chest
pain (16.4%), febrile neutropenia (16.4%), myalgia (15.9%),
injection site reaction (13.6%), and malaise (10.9%). In Study 3,
the most common adverse reactions by IV route also included
petechiae (45.8%), weakness (35.4%), rigors (35.4%), and
hypokalemia (31.3%)
In Study 4, the most commonly occurring adverse reactions were
thrombocytopenia (69.7%), neutropenia (65.7%), anemia (51.4%),
constipation (50.3%), nausea (48.0%), injection site erythema
(42.9%), and pyrexia (30.3%). The most commonly occurring Grade 3/4
adverse reactions were neutropenia (61.1%), thrombocytopenia
(58.3%), leukopenia (14.9%), anemia (13.7%), and febrile
neutropenia (12.6%)
About Acute Myeloid Leukaemia
For many patients, AML is a disease that is associated with a
poor prognosis and deteriorating quality of life. AML patients tend
to be older with poor-risk features; as such, a large proportion
are ineligible for intensive but potentially curative therapies,
and while there have been some advances recently, treatment options
remain limited. Celgene is committed to providing breakthrough
treatments and innovative technologies for patients with AML,
including those with a poor prognosis. Multiple Celgene products
are under investigation and are at various stages of development in
AML.
About Celgene
Celgene International Sàrl, located in Boudry, in the Canton of
Neuchâtel, Switzerland, is a wholly-owned subsidiary and
international headquarters of Celgene Corporation. Celgene
Corporation, headquartered in Summit, New Jersey, is an integrated
global pharmaceutical company engaged primarily in the discovery,
development and commercialization of innovative therapies for the
treatment of cancer and inflammatory diseases through gene and
protein regulation. For more information, please visit
www.celgene.com. Follow Celgene on Social Media:
@Celgene, Pinterest, LinkedIn and YouTube.
Forward-Looking Statements
This press release contains forward-looking statements, which
are generally statements that are not historical facts.
Forward-looking statements can be identified by the words
"expects," "anticipates," "believes," "intends," "estimates,"
"plans," "will," “outlook” and similar expressions. Forward-looking
statements are based on management’s current plans, estimates,
assumptions and projections, and speak only as of the date they are
made. We undertake no obligation to update any forward-looking
statement in light of new information or future events, except as
otherwise required by law. Forward-looking statements involve
inherent risks and uncertainties, most of which are difficult to
predict and are generally beyond our control. Actual results or
outcomes may differ materially from those implied by the
forward-looking statements as a result of the impact of a number of
factors, many of which are discussed in more detail in our Annual
Report on Form 10-K and our other reports filed with the Securities
and Exchange Commission.
# # #
1 Alan K. Burnett ASH 2012 Ham-Wasserman lecture; Hematology
2012:1–6
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