FDA Approves Onureg for Continued Treatment of Adults with AML Who Achieved First Complete Remission


The approval for patients with acute myeloid leukemia was based on data from the phase 3 QUAZAR AML-001 trial, which demonstrated that patients who received Onureg achieved a median overall survival of 24.7 months compared to 14.8 months for those who received placebo.

The Food and Drug Administration (FDA) approved the oral therapy Onureg (azacitidine 300 milligram (mg) tablets, CC-486) for the continued treatment of adults with acute myeloid leukemia (AML) who have achieved an initial complete remission following intensive induction chemotherapy and are not able to complete intensive curative therapy, according to Bristol Myers Squibb.

The approval was based on data from the randomized, double-blind phase 3 QUAZAR AML-001 trial, which demonstrated that Onureg showed a significant and meaningful improvement in overall survival, which was the main goal of the study.

“Continued treatment with Onureg demonstrated an overall survival benefit in adults with AML who had achieved first complete remission in the QUAZAR® AML-001 study and, notably, it has the potential to do this in a convenient manner, given its once daily oral formulation,” said QUAZAR AML-001 lead investigator Dr. Andrew Wei in a press release. “This approval should help establish continued treatment with Onureg as a standard component of AML therapy for adults who achieved (their) first complete remission following chemotherapy and who cannot proceed to intensive curative therapy, like hematopoietic stem cell transplant.”

The median overall survival was 24.7 months in patients who received Onureg 300 mg (238 patients) compared to 14.8 months in those who received placebo (234 patients). Patients continued to receive Onureg until disease progression or when they reached unacceptable toxicity.

Fifteen percent of patients in the Onureg arm experienced serious side effects. Pneumonia (8%) and febrile neutropenia (7%) were the most common serious side effects. One patient who received Onureg developed sepsis and died.

The most common side effects, regardless of severity, for patients who received Onureg included, but were not limited to, nausea (65%), vomiting (60%) and diarrhea (50%). Patients within the Onureg arm were also more likely to experience any side effect compared to those in placebo arm.

Eight percent of patients in the Onureg arm discontinued treatment as a result of a side effect.

“This milestone is representative of our commitment to helping patients with hard-to-treat cancers live longer, and the approval of Onureg as an oral therapy option for patients is more relevant now than ever as the world continues to navigate the COVID-19 pandemic,” said Bristol Myers Squibb’s chairman and CEO Dr. Giovanni Caforio in the release.

For more on this approval check back later.

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