Celgene International Sàrl, a wholly owned subsidiary of Celgene Corporation has announced that the European Commission (EC) has approved VIDAZA® (azacitidine for injection) for the treatment of adult patients aged 65 years or older with acute myeloid leukemia (AML) who are not eligible for hematopoietic stem cell transplantation (HSCT).
The VIDAZA Marketing Authorization has been updated to include this new indication in AML, covering 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 anemia and hemorrhages.
For many patients, AML is typically associated with a poor prognosis particularly for those patients who cannot tolerate potentially 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 one year. As an acute leukemia, AML progresses rapidly and is typically fatal within months if stem cell transplantation is not an option. In elderly patients ( >65 years), 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.
“Today’s announcement brings hope to patients with AML, particularly the elderly and more frail patients who cannot undergo intensive therapies such as stem cell transplantation,” said Hervé Dombret, M.D., Chief, Blood Disease Department (Leukemia Unit), University Hospital Saint-Louis, AP-HP, Paris, France. “Azacitidine has demonstrated a median overall survival of 10.4 months in these patients, which is a clinically relevant benefit and gives us a new treatment option in a previously underserved group of patients.”
Adds Tuomo Pätsi, President of Celgene in Europe, Middle East and Africa (EMEA): “Celgene is committed to bringing innovative medicines to patients with hematological diseases including AML. The approval of VIDAZA in this segment of AML patients now gives us a new opportunity to help these patients and underscores our commitment to delivering medicines that can have a significant impact on patients with severe and debilitating diseases. Our next step will be to work with each of the member countries to provide access to VIDAZA in this indication, ensuring that patients who can benefit from its use have the opportunity to do so.”
The EC decision is based on data from AML-001, a global, multi-center, 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 anemia, 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.
The EC decision for the use of VIDAZA in adult patients with AML who are not eligible for HSCT follows the positive opinion issued by the Committee for Medicinal Products for Human Use (CHMP) in September 2015. Additionally, because this new therapeutic indication brings significant clinical benefit in comparison with existing therapies as determined through the Regulatory Review process, VIDAZA will receive extended market protection in all its indications for an additional year throughout the European Economic Area.
Today’s approval marks the fourth new product or extension of the indication approved by the EC in the EU for Celgene in 2015. Celgene received approvals in the first quarter of the year for REVLIMID® in newly diagnosed multiple myeloma in adult patients ineligible for transplantation; OTEZLA®, the first phosphodiesterase-4 (PDE-4) inhibitor in psoriasis and psoriatic arthritis; and ABRAXANE® in non-small cell lung cancer.
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 anemia (RA) or refractory anemia with ringed sideroblasts (RARS) (if accompanied by neutropenia or thrombocytopenia or requiring transfusions), refractory anemia with excess blasts (RAEB), refractory anemia with excess blasts in transformation (RAEB-T), and chronic myelomonocytic leukemia (CMMoL).