Efficacy

Lumoxiti (moxetumomab pasudotox) for Hairy Cell Leukemia

Lumoxiti, also known by its generic name moxetumomab pasudotox, is a novel medication approved for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL). Hairy cell leukemia is a rare, slow-growing cancer of the blood in which the bone marrow produces too many B cells, a type of white blood cell that fights infection. These excess B cells have fine, hair-like projections when viewed under a microscope. Lumoxiti is a recombinant immunotoxin that targets CD22, a protein found on the surface of B cells, and is used when patients have received at least two prior systemic therapies, including treatment with a purine nucleoside analog.

The efficacy of Lumoxiti was established in a pivotal clinical trial that included 80 participants with relapsed or refractory HCL. The trial's results demonstrated that 30% of patients achieved a durable complete response, meaning the absence of disease symptoms and evidence for more than 180 days. Additionally, 41% of participants achieved an overall response rate, which includes both complete and partial responses. These results indicate that Lumoxiti can induce a significant response in a substantial proportion of patients with limited treatment options.

Xospata (gilteritinib) for Acute Myeloid Leukemia

Xospata, with the generic name gilteritinib, is an oral medication approved for the treatment of adult patients who have relapsed or refractory acute myeloid leukemia (AML) with a specific genetic mutation known as FLT3. AML is a rapidly progressing cancer that starts in the bone marrow and often moves into the blood. It can lead to the production of abnormal white blood cells that interfere with normal hematopoiesis. Gilteritinib is a type of drug known as a kinase inhibitor, which works by blocking the action of the abnormal protein that signals cancer cells to multiply.

The approval of Xospata was supported by clinical trial data from patients with relapsed or refractory AML who had the FLT3 mutation as detected by an FDA-approved test. In a clinical trial involving 138 patients with FLT3 mutation-positive relapsed or refractory AML, gilteritinib demonstrated a combined complete remission (CR) and complete remission with partial hematologic recovery (CRh) rate of 21%. The median duration of these remissions was 4.6 months. The trial provided evidence that Xospata is effective in inducing remission in a subset of patients with a particularly challenging form of AML.