CHMP adopts positive opinion of azacitidine for elderly patients with AML

Celgene International Sàrl has announced that the 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. 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, 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.”

In a study, median overall survival was longer for patients receiving azacitidine compared with conventional treatments

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. Azacitidine plus best supportive care was compared with conventional care regimens. Median overall survival (OS), the primary endpoint of the study, was 10.4 months for patients receiving azacitidine compared with 6.5 months for patients receiving conventional treatment regimens. One-year survival rates with azacitidine and conventional treatment regimens were 46.5% and 34.2%, respectively.

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, azacitidine will receive extended market protection in all its indications for an additional year throughout the European Economic Area.