Oral AML Treatment That Demonstrated Over 2 years median overall survival1*
There’s something you don’t see every dayONUREG® is indicated for continued treatment of adult patients with acute myeloid leukemia who achieved first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) following intensive induction chemotherapy and are not able to complete intensive curative therapy.
The efficacy of ONUREG® was evaluated in QUAZAR® AML-001, a multicenter, randomized, double-blind, placebo-controlled, phase III study. Eligible patients were ages 55 years or older, had AML, and were within 4 months of achieving first complete remission (CR) or complete remission with incomplete blood count recovery (CRi) with intensive induction chemotherapy. A total of 472 patients who completed induction with or without consolidation therapy were randomized 1:1 to receive ONUREG® 300 mg (n=238) or placebo (n=234) orally on Days 1 to 14 of each 28-day treatment cycle. Efficacy was established on the basis of overall survival (OS). The trial demonstrated a statistically significant improvement in OS for patients randomized to ONUREG® compared with placebo. In the trial, ONUREG® showed a median OS of 24.7 months (95% CI: 18.7, 30.5) vs 14.8 months (95% CI: 11.7, 17.6) for patients receiving placebo (HR 0.69 [95% CI: 0.55, 0.86; P=0.0009]).
AML, acute myeloid leukemia; CBF, core binding factor; CI, confidence interval; GI, gastrointestinal; HCT, hematopoietic cell transplantation; HR, hazard ratio; MRD, minimal residual disease.
†≥60 with no specifications and <60 years old with intermediate-risk cytogenetics and/or molecular abnormalities, including MRD-positive (category 2B) or treatment-related disease other than CBF and/or unfavorable cytogenetics and/or molecular abnormalities.
‡This is not intended to replace consolidation chemotherapy, which can be curative in some cases. In addition, fit patients with intermediate- and/or adverse-risk cytogenetics may benefit from HCT in first CR, and there are no data to suggest that post-remission therapy with oral azacitidine can replace HCT. The panel also notes that the trial did not include younger patients or those with CBF-AML; it was restricted to patients ≥55 years of age with intermediate or adverse cytogenetics who were not felt to be candidates for HCT. Most patients received at least 1 cycle of consolidation prior to starting oral azacitidine.