Efficacy

Onureg (Azacitidine) for Leukemia

Onureg (azacitidine) is a nucleoside metabolic inhibitor that is FDA-approved for the continued treatment of adult patients with acute myeloid leukemia (AML) 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 a randomized, double-blind, placebo-controlled trial (QURE). Patients treated with Onureg demonstrated a statistically significant improvement in overall survival compared to those receiving placebo. The median overall survival was 24.7 months for patients on Onureg compared to 14.8 months for those on placebo.

The use of Onureg is also associated with a delay in relapse as part of its efficacy profile. This is particularly important in the management of AML, where relapse after initial treatment is common and associated with a poor prognosis. By extending the duration of remission, Onureg provides a valuable treatment option for patients who are in remission but are not candidates for intensive curative therapies such as stem cell transplant.

Daurismo (Glasdegib) for Leukemia

Daurismo (glasdegib) is a hedgehog pathway inhibitor approved by the FDA for use in combination with low-dose cytarabine (LDAC) for the treatment of newly-diagnosed acute myeloid leukemia (AML) in adult patients who are 75 years or older or who have comorbidities that preclude the use of intensive induction chemotherapy. The efficacy of Daurismo was established in a multicenter, randomized, double-blind, placebo-controlled study. Patients who received Daurismo in combination with LDAC showed a significant improvement in overall survival compared to those treated with LDAC alone. The median overall survival was 8.3 months for patients treated with Daurismo plus LDAC, compared to 4.3 months for those treated with LDAC alone.

Additionally, the combination of Daurismo with LDAC was shown to provide a higher rate of complete remission (CR) and complete remission with partial hematologic recovery (CRh) than LDAC alone. This highlights the potential of Daurismo as part of a combination therapy to induce remission in a patient population with limited treatment options due to age or comorbidities. It is important to note that the use of Daurismo is not without risk, and potential side effects should be carefully considered in the context of its efficacy.