Efficacy

Efficacy of Onureg (azacitidine) in Leukemia

Onureg (azacitidine) is a hypomethylating agent approved by the FDA 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 demonstrated in the QUAZAR AML-001 study, a randomized, controlled, phase 3 trial. Patients treated with Onureg showed a significant improvement in overall survival compared to those who received a placebo. The median overall survival was 24.7 months for patients receiving Onureg compared to 14.8 months for placebo.

Additionally, Onureg has been shown to maintain hematologic remission, which is a crucial aspect of AML management. By providing an oral formulation that can be taken at home, Onureg offers a convenient option for patients who are in remission but are not candidates for intensive post-remission chemotherapy or bone marrow transplant. This can be particularly beneficial for older patients or those with comorbidities that make traditional post-remission therapies less feasible.

Efficacy of Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) in Leukemia

Rylaze, also known as asparaginase erwinia chrysanthemi (recombinant)-rywn, is an asparagine-specific enzyme indicated as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL) and lymphoblastic lymphoma (LBL) in adult and pediatric patients who have developed hypersensitivity to E. coli-derived asparaginase. Rylaze is designed to provide a consistent supply of asparaginase activity during treatment when other forms of asparaginase are not tolerated due to allergic reactions.

The efficacy of Rylaze was evaluated in a clinical trial that included patients with ALL or LBL who were allergic to E. coli-derived asparaginase. The study focused on achieving and maintaining nadir serum asparaginase activity (NSAA) above the threshold believed to be therapeutic. The results demonstrated that Rylaze, administered intramuscularly, was effective in maintaining NSAA levels above the target in the majority of patients, ensuring that the therapeutic activity of asparaginase was sustained throughout the treatment period. This is vital for the effective management of ALL and LBL, as asparaginase is a key enzyme that deprives leukemia cells of the amino acid asparagine, which is necessary for their growth and survival.