BOUDRY, Switzerland--(BUSINESS WIRE)--Sep 25, 2015--Celgene International Sarl, 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 R (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 years 1, 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 Herve 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 Patsi, 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
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.
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 should be advised to discontinue nursing or the drug, taking into consideration the importance of the drug to the mother
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.
Celgene International Sarl, located in Boudry, in the Canton of Neuchatel, 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.
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.
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1 Alan K. Burnett ASH 2012 Ham-Wasserman lecture; Hematology 2012:1?V6
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