FDA clears oral leukemia drug

FDA’s approval of a novel medication to treat acute myeloid leukemia (AML) was a long time coming.

Oral azacitidine (Onureg, Bristol Myers Squibb) took over a decade of research, including 13 pre-clinical and clinical trials, said Giovanni Caforio, MD, chairman and CEO Of Bristol Myers Squibb, in a press release.

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After granting Onureg’s New Drug Application Priority Review designation, FDA okayed the drug treat adult patients with acute myeloid leukemia (AML) who achieved first complete remission (CR) or CR with incomplete blood count recovery (CRi) following intensive induction chemotherapy and who are not able to complete intensive curative therapy.

The approval is based on results from a phase study in which treatment with Onureg resulted in a statistically significant and clinically meaningful improvement in overall survival (OS) of nearly 10 months compared to placebo.

Median OS from time of randomization was greater than two years (24.7 months) among patients who received Onureg compared to 14.8 months among patients receiving placebo, Bristol Myers Squibb said.

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“This approval should help establish continued treatment with Onureg as a standard component of AML therapy for adults who achieved first complete remission following chemotherapy and who cannot proceed to intensive curative therapy, like hematopoietic stem cell transplant,” said Andrew Wei, PhD, the study’s lead investigator and a professor and hematologist at the Alfred Hospital and adjunct professor at Monash University in Melbourne, Australia.

Wei noted that Onureg demonstrated an OS benefit in adults with AML who had achieved first complete remission and that “it has the potential to do this in a convenient manner, given its once daily oral formulation.”

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