FDA OKs New Treatment for Hairy Cell Leukemia

Officials with the FDA have approved AstraZeneca’s moxetumomab pasudotox-tdfk (Lumoxiti) injection for intravenous use for the treatment of adult patients with relapsed or refractory hairy cell leukemia (HCL) who have received at least 2 prior systemic therapies, including treatment with a purine nucleoside analog.

Lumoxiti is a CD22-directed cytotoxin and is the first of this type of treatment for patients with HCL.

“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 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, in a prepared statement.

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“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.”

According to AstraZeneca’s Dave Fredrickson, Executive Vice-President, Global Head Oncology Business Unit, Lumoxiti represents the first FDA-approved medicine for HCL in more than 20 years.

The FDA granted approval for this drug under 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 approval is based on data from the Phase III single-arm, open-label ‘1053’ trial of Lumoxiti monotherapy in 80 patients who have received at least 2 prior therapies, including a purine nucleoside analog. The primary endpoint of the trial was durable complete response (CR), defined as maintenance of hematologic remission for more than 180 days after achievement of CR. Thirty percent of patients in the trial achieved durable CR, and the overall response rate was 75%.

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