FDA Approves AstraZeneca’s Lumoxiti for Hairy Cell Leukemia

Rare Daily Staff

The U.S. Food and Drug Administration approved AstraZeneca’s Lumoxiti, a first-in-class medicine for hairy cell leukemia (HCL), and the first new treatment option for patients with the rare cancer in more than 20 years.

HCL is a chronic, and slow-growing leukemia in which the bone marrow overproduces abnormal B cell lymphocytes. HCL can result in serious and life-threatening conditions, including infections, bleeding and anemia. Approximately 1,000 people are diagnosed with HCL in the United States each year.

While many patients initially respond to treatment, 30 percent to 40 percent will relapse five to ten years after their first treatment. With no established standard of care and very few treatments available, there remains significant unmet medical need for people with relapsed or refractory HCL.

Lumoxiti is a CD22-directed cytotoxin for adult patients with relapsed or refractory HCL who have received at least two prior systemic therapies, including treatment with a purine nucleoside analog. It consists of the CD22 binding portion of an antibody fused to a truncated bacterial toxin. The toxin inhibits protein synthesis and ultimately triggers apoptotic cell death.

“Lumoxiti fills an unmet need for patients with hairy cell leukemia whose disease has progressed after trying other FDA-approved therapies,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the 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.”

The FDA granted Lumoxiti Fast Track and Priority Review designations. Lumoxiti also received Orphan Drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

The efficacy of Lumoxiti was studied in a single-arm, open-label clinical trial of 80 patients who had received prior treatment for HCL with at least two systemic therapies, including a purine nucleoside analog. The trial measured durable complete response, defined as maintenance of hematologic remission for more than 180 days after achievement of complete response. Thirty percent of patients in the trial achieved durable complete response, and 75 percent of patients had a partial or complete response to therapy.

Common side effects of Lumoxiti include infusion-related reactions, edema, nausea, fatigue, headache, fever, constipation, anemia, and diarrhea.

The prescribing information for Lumoxiti includes a Boxed Warning to advise healthcare professionals and patients about the risk of developing capillary leak syndrome, a condition in which fluid and proteins leak out of tiny blood vessels into surrounding tissues. The Boxed Warning also notes the risk of hemolytic uremic syndrome, a condition caused by the abnormal destruction of red blood cells.

September 14, 2018
Photo: AstraZeneca scientist at work