FDA approves new kind of treatment for hairy cell leukemia

FDA approved on September 13 moxetumomab pasudotox-tdfk (Lumoxiti—AstraZeneca) injection for the treatment of hairy cell leukemia (HCL).

FDA approved on September 13 moxetumomab pasudotox-tdfk (Lumoxiti—AstraZeneca) injection for the treatment of hairy cell leukemia (HCL). The drug, a CD22-directed cytotoxin, is approved to treat adults with relapsed or refractory HCL who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. "Lumoxiti fills an unmet need for patients with hairy cell leukemia whose disease has progressed after trying other FDA-approved therapies," said Richard Pazdur, MD, director of FDA's Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in FDA's Center for Drug Evaluation and Research. "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." To determine the efficacy of moxetumomab pasudotox-tdfk, researchers conducted a single-arm clinical trial involving 80 patients. They found that 30% of the patients achieved a durable complete response (CR), defined as maintenance of hematologic remission for more than 180 days after achievement of CR, while the overall response rate (the number of patients with a partial or complete response to therapy) was 75%. Common adverse effects of the drug include infusion-related reactions, swelling caused by excess fluid in body tissue, nausea, fatigue, headache, fever, constipation, anemia, and diarrhea. The drug's prescribing information includes a boxed warning advising health care professionals and patients about the risk of developing capillary leak syndrome and notes the risk of hemolytic uremic syndrome. Additional serious warnings include decreased renal function, infusion-related reactions, and electrolyte abnormalities.