Efficacy

Onureg (Azacitidine) Efficacy in Leukemia

Onureg, known generically as azacitidine, is a hypomethylating agent approved by the U.S. Food and Drug Administration (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 in this indication was demonstrated in a randomized, controlled trial that showed a significant improvement in overall survival among patients treated with azacitidine compared to those who received a placebo. This marked a significant advancement in the management of AML, particularly for patients who are unable to undergo further intensive chemotherapy or bone marrow transplant.

The clinical trial, known as QUAZAR AML-001, showed that patients receiving Onureg had a median overall survival of 24.7 months compared to 14.8 months for those on placebo. Furthermore, the drug was associated with a delay in relapse, which is a critical aspect of AML management, as relapse after initial treatment is common and associated with a poor prognosis. The improvement in survival and relapse rates underscores the efficacy of Onureg as a maintenance therapy in AML patients who are in remission but cannot pursue more aggressive treatment options.

Elzonris (Tagraxofusp-erzs) Efficacy in Leukemia

Elzonris, with the generic name tagraxofusp-erzs, is the first FDA-approved therapy for blastic plasmacytoid dendritic cell neoplasm (BPDCN), a rare and aggressive form of leukemia. The efficacy of Elzonris in treating BPDCN was evaluated in a multicenter, open-label, single-arm clinical trial, which included patients with untreated and relapsed or refractory BPDCN. The results demonstrated a clinically meaningful and durable response to treatment, with a significant proportion of patients achieving complete remission or complete remission with a skin normalization.

The trial reported that 45% of patients achieved complete remission (CR) or CR with skin normalization (CRc), with a median duration of response not reached for those who achieved CR/CRc. This indicates that Elzonris can induce a high rate of response in patients with BPDCN, which is particularly noteworthy given the historically poor outcomes associated with this disease. The approval of Elzonris provides a targeted treatment option for patients with BPDCN, addressing a previously unmet need in the therapeutic landscape of leukemia.