The US Food and Drug Administration (FDA) has approved AstraZeneca’s Lumoxiti (moxetumomab pasudotox-tdfk) for the treatment of adult patients with relapsed or refractory hairy cell leukaemia (HCL).
Lumoxiti, which is a CD22-directed cytotoxin, has been approved for the patients who had two or prior systemic therapies, including treatment with a purine nucleoside analog.
AstraZeneca executive vice-president and oncology business unit global head Dave Fredrickson said: “Today’s FDA approval of Lumoxiti represents a significant milestone for people living with hairy cell leukaemia, a rare blood cancer that can result in serious and life-threatening conditions. For patients, this approval provides the first FDA-approved medicine for this condition in more than 20 years.”
The FDA approval of the drug was based on the data of a phase 3 single-arm trial which assessed the leukaemia drug as monotherapy in 80 patients with hairy cell leukaemia.
Dubbed as 1053, the late-stage trial showed that 30% of the patients treated with Lumoxiti had durable complete response (CR). On the other hand, 75% of patients treated with the CD22-directed cytotoxin showed partial or complete response.
Durable CR was the primary endpoint of the phase 3 trial which has been defined as CR with blood count normalization for more than 180 days.
According to AstraZeneca, the early discovery of moxetumomab pasudotox was headed by the National Cancer Institute (NCI) which collaborated with MedImmune, AstraZeneca’s biologics research and development arm, to develop the drug.
Lumoxiti is made up of the CD22 binding portion of an antibody fused to a truncated bacterial toxin. The toxin in turn prevents protein synthesis and eventually triggers apoptotic cell death.
FDA center for drug evaluation and research office of hematology and oncology products acting director Richard Pazdur said: “Lumoxiti fills an unmet need for patients with hairy cell leukemia whose disease has progressed after trying other FDA-approved therapies.
“This therapy is the result of important research conducted by the National Cancer Institute that led to the development and clinical trials of this new type of treatment for patients with this rare blood cancer.”