On May 20, 2022, the FDA accelerated the approval of a new indication for azacitidine injection (Vidaza; Celgene) for the treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).
The FDA granted this indication a breakthrough therapy designation. Azacitidine injection was previously approved for the treatment of adult patients with myelodysplastic syndromes.
This new indication was approved based on a multicenter, open-label clinical trial that included 18 pediatric patients with JMML. Researchers assessed the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to hematopoietic stem-cell transplantation (HSCT).
The patients received intravenous (IV) azacitidine once daily on days 1 to 7 of a 28-day cycle, for a minimum of 3 cycles and a maximum of 6 cycles. Patients were included in the study if they did not have disease progression or were not ready for HSCT between cycles 4 and 6.
The main efficacy outcome measures were clinical complete remission or clinical partial remission according to the International Juvenile Myelomonocytic Leukemia Working Group response criteria at 3 months (cycle 3, day 28). The responses must have been sustained for at least 4 weeks in the 4-week period preceding or succeeding cycle 3.
Nine of the 18 patients (50%) had confirmed clinical responses, with 3 having a clinical complete response and 6 having a clinical partial response. The median time to response was 1.2 months (range, 0.95-1.87 months).
Most (94%) patients underwent HSCT, with a median time to HSCT of 4.6 months (range, 2.8-19 months).
The most common (>30%) adverse reactions reported with IV azacitidine in pediatric patients with JMML were pyrexia, rash, upper respiratory tract infection, and anemia.
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