Efficacy

Lumoxiti (moxetumomab pasudotox) for Hairy Cell Leukemia

Lumoxiti, generically known as moxetumomab pasudotox, is a CD22-directed cytotoxin approved by the FDA for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. HCL is a rare type of leukemia, and Lumoxiti functions by binding to the CD22 antigen on B cells and then internalizing into the cell where it releases a toxin that inhibits protein synthesis, leading to cell death. Clinical trials have demonstrated that Lumoxiti can induce a high rate of durable complete responses in patients with relapsed or refractory HCL.

In a pivotal phase III clinical trial, Lumoxiti showed a 30% complete response rate with patients achieving hematologic remission. Furthermore, 41% of patients achieved an objective response rate (ORR), which includes partial responses. The median duration of response has not been reached, suggesting durability of response in a significant number of patients. These results indicate that Lumoxiti is an effective treatment option for patients with HCL who have limited options due to the relapsed or refractory nature of their disease.

Vanflyta (quizartinib) for Acute Myeloid Leukemia

Vanflyta, known generically as quizartinib, is a kinase inhibitor that has shown efficacy in treating acute myeloid leukemia (AML), specifically in patients with FLT3-ITD (FMS-like tyrosine kinase 3-internal tandem duplication) mutations. This mutation is one of the most common genetic abnormalities in AML and is associated with a poor prognosis. Vanflyta inhibits the FLT3 signaling pathway, which is thought to be critical in the proliferation and survival of leukemic cells in AML patients with the FLT3-ITD mutation.

The efficacy of Vanflyta was evaluated in a multicenter, randomized, controlled trial that included patients with relapsed or refractory AML with the FLT3-ITD mutation. The trial demonstrated a significant improvement in overall survival for patients treated with quizartinib compared to chemotherapy. The median overall survival was markedly higher in the quizartinib group than in the chemotherapy group. Additionally, the response rates were higher with quizartinib, with a greater percentage of patients achieving complete remission with or without full hematologic recovery. These outcomes suggest that Vanflyta is a promising therapeutic option for patients with FLT3-ITD positive AML, particularly for those who are relapsed or refractory to prior therapies.