Efficacy

Lumoxiti (moxetumomab pasudotox) for Hairy Cell Leukemia

Lumoxiti, also known by its generic name moxetumomab pasudotox, is a CD22-directed cytotoxin approved by the Food and Drug Administration (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. Hairy cell leukemia is a rare, slow-growing cancer of the blood where the bone marrow produces too many B cells, which are a type of white blood cell that fights infection. These excess B cells are abnormal and look "hairy" under a microscope.

The efficacy of Lumoxiti was evaluated in a single-arm, open-label clinical trial which included 80 patients with relapsed or refractory HCL. The major efficacy outcome measures were the overall response rate (ORR) and the duration of response (DOR). In this study, Lumoxiti demonstrated an ORR of 75%, with 30% of patients achieving a complete response. Furthermore, among the responders, 80% maintained their response for six months or longer, indicating a significant duration of response for those affected by this rare leukemia.

Daurismo (glasdegib) for Acute Myeloid Leukemia

Daurismo, with the generic name glasdegib, is a medication used in combination with low-dose cytarabine 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. AML is a rapidly progressing cancer that forms in the bone marrow and results in an increased number of abnormal white blood cells in the bloodstream.

The approval of Daurismo was based on a multicenter, randomized, open-label clinical trial in which the efficacy of glasdegib in combination with low-dose cytarabine was compared with low-dose cytarabine alone in patients with newly diagnosed AML or high-risk myelodysplastic syndrome. The trial demonstrated that patients treated with the combination of Daurismo and low-dose cytarabine had a median overall survival of 8.3 months compared to 4.3 months for patients treated with low-dose cytarabine alone. This indicates that the addition of Daurismo to standard therapy can provide a survival benefit to older patients with AML who are not candidates for intensive chemotherapy.