Celgene subsidiary Celgene International has received approval from the European Commission (EC) for Vidaza (azacitidine for injection) to treat adults aged 65 years or older with acute myeloid leukaemia (AML) who are not eligible for haematopoietic stem cell transplantation (HSCT).
The company said that the expanded indication brings medicine to greater number of elderly AML patients not eligible for HSCT and have >30% myeloblasts in their bone marrow.
According to the WHO classification, the indication of Vidaza previously covered AML patients with <30% blasts.
In AML, myeloblasts’ 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.
University Hospital Saint-Louis Blood Disease Department (Leukaemia Unit) chief Hervé Dombret said: "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.
"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 approval was based on data from AML-001, a global, multi-centre, randomised, open-label pivotal trial of patients at least 65 years old with newly diagnosed or secondary AML with >30% bone marrow blasts.
In the trial, Vidaza plus best supportive care was compared with conventional care regimens.
The primary endpoint of the trial, median overall survival (OS) was 10.4 months for patients receiving azacitidine compared with 6.5 months for patients receiving conventional treatment regimens.
Celgene Europe , Middle East and Africa (EMEA) president Tuomo Pätsi said: "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.
"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."
For many patients, AML is typically associated with a poor prognosis particularly for those who cannot tolerate potentially curative therapies like stem cell transplantation.
In September, the European Medicines Agency ‘s (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for the use of Vidaza in adults with AML who are not eligible for HSCT.
The EC approval marks the fourth new product or extension of the indication approved by the agency in the EU for the company this year.
