Efficacy

Lumoxiti (moxetumomab pasudotox) for Hairy Cell Leukemia

Lumoxiti, also known by its generic name moxetumomab pasudotox, is a medication specifically indicated for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL). Hairy cell leukemia is a rare type of chronic leukemia characterized by an excess of abnormal B cells. Lumoxiti is a CD22-directed cytotoxin and works by binding to the CD22 antigen on B cells, then internalizing into the cell where it releases a toxin that inhibits protein synthesis, leading to cell death.

The efficacy of Lumoxiti was evaluated in a pivotal single-arm, open-label clinical trial which included 80 patients with relapsed or refractory HCL. The results of this trial demonstrated a high rate of durable complete response (CR), which is defined as the disappearance of all signs of cancer in response to treatment. In the trial, 30% of patients achieved CR with Lumoxiti, and 41% of patients achieved an objective response rate (ORR), which includes partial responses in addition to complete responses.

Rylaze (asparaginase erwinia chrysanthemi (recombinant)-rywn) for Acute Lymphoblastic Leukemia

Rylaze, also known as asparaginase erwinia chrysanthemi (recombinant)-rywn, is used as a component of a multi-agent chemotherapeutic regimen for the treatment of acute lymphoblastic leukemia (ALL). ALL is a type of cancer of the blood and bone marrow that affects white blood cells. Rylaze is an asparagine specific enzyme that depletes the levels of asparagine, an amino acid that is necessary for the survival of leukemia cells, thus inhibiting their growth.

The efficacy of Rylaze was primarily based on achieving and maintaining nadir serum asparaginase activity (NSAA) above the level of 0.1 U/mL, a threshold associated with good clinical outcomes in patients with ALL. In a clinical study involving patients with hypersensitivity to E. coli-derived asparaginase, Rylaze was shown to maintain the NSAA above the desired threshold with no evidence of asparagine synthesis. This pharmacodynamic effect is associated with a positive clinical response in patients with ALL when used as part of a multi-agent regimen.