Efficacy

Elzonris (tagraxofusp-erzs) Efficacy in Treating Leukemia

Elzonris (tagraxofusp-erzs) is an innovative targeted therapy approved by the U.S. Food and Drug Administration (FDA) for the treatment of blastic plasmacytoid dendritic cell neoplasm (BPDCN) in adults and in pediatric patients, 2 years of age and older. BPDCN is a rare and aggressive form of leukemia. The efficacy of Elzonris was evaluated in clinical trials, primarily in a pivotal trial that included 13 patients with treatment-naive BPDCN. The results were promising, with a reported overall response rate (ORR) of 90%, indicating a significant proportion of patients experienced a reduction or disappearance of their leukemia-related symptoms after treatment with Elzonris.

The duration of response (DOR) to Elzonris also underscored its efficacy. Among responders, the median DOR was not reached, suggesting that the majority of responses were sustained for a considerable period. Furthermore, the treatment demonstrated a clinically meaningful benefit in terms of survival outcomes, with a median overall survival (OS) that was not reached after 20.7 months of follow-up, indicating that many patients were still alive at the end of the study period.

Daurismo (glasdegib) Efficacy in Treating Leukemia

Daurismo (glasdegib) is another medication that has been approved by the FDA 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, in combination with low-dose cytarabine (LDAC), was demonstrated in a randomized clinical trial that compared it to LDAC alone. The trial showed that patients treated with the combination had a median overall survival of 8.3 months compared to 4.3 months for patients treated with LDAC alone, indicating a significant improvement in survival for the Daurismo combination group.

In addition to the improved OS, the combination of Daurismo and LDAC resulted in a higher rate of complete remission (CR) compared to LDAC alone. The CR rates were 17% for the combination therapy versus 2.3% for LDAC alone. This improvement in CR rates further supports the efficacy of Daurismo as a treatment option for older patients with AML who are not candidates for intensive chemotherapy.