FDA Approves Vidaza for Rare Pediatric Blood Cancer

The Food and Drug Administration (FDA) approved Vidaza (azacytidine) for the treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia, a rare blood cancer where certain types of white blood cells do not mature normally.

The approval is based off findings from the open-label AZA-JMML-001 clinical trial, which evaluated Vidaza in 18 children with juvenile myelomonocytic leukemia before undergoing hematopoietic stem cell transplant — which is currently the only known successful treatment for the disease.

The main goal of the study was clinical complete remission (no evidence of cancer after treatment) or clinical partial remission (reduction in cancer after treatment) at three months since the start of treatment. Half of study participants (nine patients) had confirmed clinical responses, which included three clinical complete remissions.

Average time to response was 1.2 months, and 94% of patients went on to receive a stem cell transplant, with the average time to transplant being 4.6 months.

The recommended dose for patients between the age of one month to one year who weigh less than 10 kg is 2.5 mg/kg, and the recommended dose for children older than one year and weighing over 10 kg is 75 mg/m2.

In the AZA-JMML-001 trial, patients received Vidaza intravenously on days one through seven of a 28-day cycle. Patients underwent a minimum of three cycles, and a maximum of six cycles as long as they did not have their disease get worse or were ready for transplant between cycles 4 and 6.

The most common side effects that occurred in 30% or more of patients were fever, rash, upper respiratory tract infection and anemia (a low red blood cell count that may lead to fatigue).

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