Azacytidine 300 mg tablets, CC-486 is earmarked for AML patients who achieved their first complete remission following intensive induction chemotherapy.
The US Food and Drug Administration (FDA) has approved azacytidine (Onureg) 300 mg tablets, CC-486 for the continued treatment of adult patients with acute myeloid leukemia (AML).
The approval, awarded to Bristol Myers Squibb, is specifically for AML patients who achieved their first complete remission or complete remission with incomplete blood count recovery following intensive induction chemotherapy and who are not able to complete intensive curative therapy.
The FDA’s decision is based on the results from the phase 3 QUAZAR AML-001 study, where the study drug resulted in a statistically significant and clinically meaningful improvement in the overall survival of AML patients, meeting the 10 month study’s primary endpoint.
“The FDA approval of Onureg is the culmination of over a decade of research and 13 pre-clinical and clinical trials,” Giovanni Caforio, MD, chairman and chief executive officer, Bristol Myers Squibb, said in a statement. “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.”
The median overall survival from time of randomization was 24.7 months (95% CI, 18.7-30.5) among patients receiving azacytidine compared to 14.8 months (95% CI, 11.7-17.6) in the placebo group (HR, 0.69; 95% CI, 0.55-0.86; P = 0.0009).
The study drug was continued until disease progression or unacceptable toxicity.
The FDA approval comes with warnings and precautions for risks of substitution with other azacytidine products, myelosuppression, increased early mortality in patients with myelodysplastic syndromes and embryo-fetal toxicity.
Because of substantial differences in the pharmacokinetic parameters, the treatment should not be substituted for intravenous or subcutaneous azacytidine because it could cause a fatal adverse reaction.
New or worsening grade 3 or 4 neutropenia and thrombocytopenia occurred in 49% and 22% of patients who received the AML drug, respectively, while febrile neutropenia occurred in 12% of patients.
While considered a rare disease, AML is 1 of the more common acute leukemias in adults.