FDA Clears All-Oral AML Regimen for Older Patients Who Can't Take Intensive Chemo

The U.S. Food and Drug Administration has approved a fully oral regimen for people newly diagnosed with acute myeloid leukemia who are older or aren't candidates for intensive chemotherapy, according to News-Medical. The decision rests on results from the ASCERTAIN-V study, published in the New England Journal of Medicine. The regimen stacks decitabine-cedazuridine tablets with venetoclax.

Here's how it works: decitabine is a chemotherapy drug, and cedazuridine is what makes it swallowable instead of injectable. Venetoclax goes after a protein cancer cells rely on to stay alive. Until now, these patients typically came into a clinic every month for injections lasting five to seven days, on top of a daily tablet. The oral version could mean treatment at home instead.

The phase I-II trial enrolled 189 patients who were 75 or older or had health conditions that ruled out intensive chemotherapy. Once researchers found no signs of cancer in patients' bone marrow, they cut back how long medication was used in later cycles, factoring in how blood counts were recovering. A complete response showed up in 47% of participants, and 63% had a meaningful clinical response. Among those who regularly needed blood or platelet transfusions, 60% stopped needing them during treatment. Median overall survival came in at 15.5 months.

Amer Zeidan, a hematologist at Yale School of Medicine and a co-author of the study, said an indirect comparison pointed to survival, treatment response and safety rates in line with the standard injectable regimen. He also said trimming the medication period after a response helped blood counts bounce back faster and lowered the risk of infections or bleeding.