The Food and Drug Administration has approved the latest in a string of new therapies for acute myeloid leukemia that have hit the market in recent years.
New York-based Bristol-Myers Squibb said Tuesday that the FDA had approved Onureg (azacitidine) for the continuation setting of acute myeloid leukemia, or AML, in patients who have achieved a complete remission with or without full blood count recovery after intensive induction chemotherapy, but who are not able to complete intensive curative therapy. The drug is an oral hypomethylating agent that uses the same molecule as Vidaza, which is given intravenously or subcutaneously in the first-line setting. Vidaza was developed by Celgene, which BMS acquired last year.
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Onureg’s approval is based on results from the Phase III QUAZAR AML-001 study, which showed a median overall survival of 24.7 months among patients receiving the drug, compared with 14.8 months among those receiving placebo. Severe or life-threatening neutropenia and thrombocytopenia occurred in 49% and 22% of patients receiving the drug, respectively, while 12% of patients encountered febrile neutropenia.
“The differentiation here is that we have created a formulation that allows this drug to be given orally, and as an oral drug we can give it in the continuation setting,” said Noah Berkowitz, head of BMS’ hematology development unit, in a phone interview. Onureg’s label cautions that the drug should not be substituted for Vidaza as it may cause fatal side effects.
AML is one of the most common leukemias and mostly affects older adults, with more than 20,000 cases occurring in the U.S. each year. For many years, its few treatment options were mostly limited to the chemotherapy combination 7+3 – consisting of seven days of standard-dose cytarabine and three days of an anthracycline chemotherapy drug like daunorubicin – and IV or subcutaneous hypomethylating agents. More recently, several drugs have received FDA approval for various settings of AML, such as Novartis’ Rydapt (midostaurin), Pfizer’s Mylotarg (gemtuzumab ozogamicin) and AbbVie’s Venclexta (venetoclax).
Berkowitz noted that there have been previous attempts to test drugs in the continuation setting of AML, but none had been able to definitively demonstrate an advantage in overall survival. “And it goes back decades, where there have been attempts and failures,” he said.
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