AstraZeneca has achieved a major regulatory milestone with the US Food and Drug Administration (FDA) approval of Lumoxiti (moxetumomab pasudotox-tdfk), a CD22-directed cytotoxin for the treatment of relapsed or refractory hairy cell leukemia (HCL). This approval marks a significant advancement in the treatment of this rare and chronic form of leukemia, offering new hope to patients who have exhausted other therapeutic options.

Lumoxiti is specifically approved for adult patients with hairy cell leukemia who have previously undergone at least two systemic therapies, including treatment with a purine nucleoside analog. This patient group often faces limited treatment options, making this FDA approval particularly impactful.

Hairy cell leukemia is characterized by the bone marrow’s overproduction of abnormal B cell lymphocytes. This slow-growing leukemia can lead to severe life-threatening conditions, including bleeding, infections, and anemia, highlighting the critical need for effective treatment options.

The recommended dosage of Lumoxiti for treating hairy cell leukemia is 0.04 mg/kg, administered as an intravenous infusion over 30 minutes on days 1, 3, and 5 of each 28-day cycle, for up to six cycles. Treatment continues until disease progression or unacceptable toxicity levels are reached. However, Lumoxiti is not recommended for patients with severe renal impairment.

The FDA’s approval of Lumoxiti was supported by the results of the phase 3 single-arm 1053 trial, which evaluated the drug as a monotherapy in 80 patients who had previously received at least two prior therapies. The trial’s primary endpoint was durable complete response (CR), defined as the maintenance of hematologic remission for over 180 days after achieving CR. Impressively, 30% of participants recorded durable CR, while the overall response rate was 75%.

Dave Fredrickson, Executive Vice-President and Global Head of the Oncology Business Unit at AstraZeneca, commented on the approval: “Today’s FDA approval of Lumoxiti represents a significant milestone for people living with hairy cell leukemia, 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.”

Richard Pazdur, director of the FDA’s Oncology Center of Excellence, also remarked, “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.”

With the FDA’s priority review and approval of Lumoxiti, AstraZeneca continues to lead in the oncology sector, providing critical solutions for challenging and rare conditions. The success of Lumoxiti in clinical trials and its subsequent approval is a testament to the ongoing innovation in cancer treatment and patient care.

  AstraZeneca, Cancer treatment, Dave Fredrickson, FDA approval, Hairy cell leukaemia, hairy cell leukemia, Lumoxiti, Moxetumomab pasudotox-tdfk, Oncology, oncology research, Richard Pazdur, US FDA, USA