Celgene's CC-486 set to become standard maintenance therapy in AML, says analyst

US biotech firm Celgene, now a subsidiary of Bristol-Myers Squibb (NYSE: BMY), plans to pursue a New Drug Application (NDA) in first-quarter 2020 for its formulation of oral azacitidine (CC-486) after data from the Phase III QUAZAR AML-001 trial showed that maintenance therapy can prolong the overall survival (OS) of acute myeloid leukemia (AML) patients compared with placebo. As such, CC-486 is set to address a clear unmet need, says analytics firm GlobalData.

Sakis Paliouras, GlobalData oncology and hematology analyst, commented: “In AML, despite several trials in the maintenance setting, no agent has demonstrated a clinical benefit that is significant enough for widespread adoption, leaving the use of maintenance therapy at the physicians’ discretion. The closest competitor in this setting, decitabine, was associated with an OS benefit but showed no statistical significance.”

He continued: “Upon regulatory confirmation, GlobalData expects CC-486 to become the new standard maintenance therapy for patients over 55 years old. Even though its use will be confined to one line of therapy, the lack of competition has the potential to result in widespread adoption by US-based hematologists, leading to significant revenue for Celgene.