First-in-Class Treatment Approved for Hairy Cell Leukemia

The Food and Drug Administration (FDA) has approved Lumoxiti (moxetumomab pasudotox-tdfk; AstraZeneca), a CD22-directed cytotoxin, for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who received ≥2 prior systemic therapies, including treatment with a purine nucleoside analog.

The approval of Lumoxiti was based on data from a Phase 3 single-arm, open-label trial conducted in patients with histologically confirmed HCL or HCL variant with a need for therapy based on presence of cytopenias or splenomegaly and who had received prior treatment with at least 2 systemic therapies, including a purine nucleoside analog (N=80). Patients received Lumoxiti 0.04mg/kg as an intravenous infusion over 30 minutes on Days 1, 3, and 5 of each 28-day cycle for a maximum of 6 cycles or until documentation of complete response, disease progression, or unacceptable toxicity. 

The primary endpoint of the study was durable complete response, defined as patients who achieved

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