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European Commission Expands Azacitidine Approval in AML

Jason M. Broderick @jasoncology
Published: Friday, Oct 30, 2015

Hervé Dombret, MD

Hervé Dombret, MD

The European Commission (EC) has expanded the approval of azacitidine (Vidaza) in acute myeloid leukemia (AML) to include patients aged ≥65 years who are ineligible for hematopoietic stem cell transplantation (HSCT) and who have >30% myeloblasts, according to Celgene International Sàrl, a subsidiary of Celgene Corporation.

Previously, the approval of azacitidine in this setting was limited to patients with <30% blasts (per WHO classification). The expanded indication follows a recommendation issued by the Committee for Medicinal Products for Human Use (CHMP) in September 2015.

“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, Hervé Dombret, MD, chief, Blood Disease Department (Leukaemia Unit), University Hospital Saint-Louis, AP-HP, Paris, France, said in a statement. “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.”

The expanded indication was based on data from the international, multicenter, open-label, phase III AML-001 trial which included 488 patients with newly diagnosed or secondary AML with >30% bone marrow blasts. Patients first received investigator’s choice of intensive chemotherapy (standard 7+3 regimen), low-dose Ara-Cytarabine, or best supportive care alone. Subsequently, patients were randomized to azacitidine (75 mg/m2/day subcutaneous for 7 days of each 28-day cycle) plus best supportive care (n = 241) or conventional care (n = 247). The primary endpoint of the study was overall survival (OS).

Treatment with azacitidine was associated with a 15% reduction in the risk of death. Median OS for the azacitidine arm was 10.4 months (95% CI, 8.0-12.7) versus 6.5 months (95% CI, 5.0-8.6) for patients in the control arm (HR, 0.85; 95% CI, 0.69-1.03; P = .1009). The 1-year survival rate with azacitidine was 46.5% compared with 34.2% with conventional therapy (95% CI, 3.5%-21%).

Grade 3/4 adverse events were reported for patients receiving azacitidine versus best supportive care versus low-dose Ara-Cytarabine versus intensive chemotherapy: anemia (16% vs 5% vs 23% vs 14%), neutropenia (26% vs 5% vs 25% vs 33%), febrile neutropenia (28% vs 28% vs 30% vs 31%), and thrombocytopenia rates (24% vs 5% vs 28% vs 21%).

“Celgene is committed to bringing innovative medicines to patients with haematological 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,” Tuomo Pätsi, president of Celgene in Europe, Middle East and Africa, said in a statement.

“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,” added Pätsi.

Azacitidine is not approved for the treatment of AML in the United States, but the FDA has authorized its use for the treatment of patients with all subtypes of myelodysplastic syndromes.

AML is a disease that progresses rapidly and without stem cell transplantation, it is usually fatal. OS in patients aged >65 years has not improved in more than 4 decades, according to a Celgene press release.


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