FDA approves azacitidine for pediatric leukemia indication
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The FDA approved azacitidine for treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia.
Azacitidine (Vidaza, Celgene) is a nucleoside metabolic inhibitor indicated for treatment of certain patients with myelodysplastic syndrome.
The FDA based the new indication on results of the multicenter, open-label AZA-JMML-001 study, which included 18 pediatric patients with juvenile myelomonocytic leukemia. Researchers assessed the pharmacokinetics, pharmacodynamics, safety and activity of azacitidine prior to hematopoietic stem cell transplantation.
Patients received IV azacitidine daily on days 1-7 of 28-day cycles. Treatment continued for a minimum three cycles and maximum six cycles, provided patients did not experience disease progression or were ready to undergo HSCT between the fourth and sixth cycles.
Main efficacy outcomes included clinical complete remission or clinical partial remission at 3 months. Responses must have been sustained for at least 4 weeks either in the 4-week period prior to or following the end of the third cycle.
Nine patients (50%) achieved confirmed clinical responses, with three achieving clinical complete remission and six achieving clinical partial remission. Median time to response was 1.2 months (range, 0.95-1.87).
Most (94%) study participants underwent HSCT, with median time to HSCT of 4.6 months (range, 2.8-19).
Adverse events that occurred among more than 30% of study participants included pyrexia, rash, upper respiratory tract infection and anemia.
The FDA previously granted priority review and breakthrough therapy designation to azacitidine for this indication.
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