On May 20, 2022, the Food and Drug Administration approved azacitidine (Vidaza, Celgene Corp.) for pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).
Efficacy was evaluated in AZA-JMML-001 (NCT02447666), an international, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to hematopoietic stem cell transplantation (HSCT) in 18 pediatric patients with JMML. Patients were treated with intravenous azacitidine daily on Days 1-7 of a 28-day cycle for a minimum of 3 cycles and a maximum of 6 cycles, provided patients did not have disease progression or were ready for HSCT between Cycles 4 and 6.
The main efficacy outcome measures were clinical complete remission (cCR) or clinical partial remission (cPR) according to the International JMML response criteria at 3 months (Cycle 3, Day 28). Responses must have been sustained for at least 4 weeks either in the 4-week period preceding or succeeding Cycle 3, Day 28. A total of 9 patients (50%, 95% CI: 26, 74) had confirmed clinical responses. Of these 9 patients, there were 3 cCR and 6 cPR. The median time to response was 1.2 months (range 0.95-1.87 months). The proportion of patients undergoing HSCT was 94% and the median time to HSCT was 4.6 months (range 2.8-19 months).
Most common adverse reactions (>30%) occurring in pediatric patients with JMML were pyrexia, rash, upper respiratory tract infection, and anemia.
The recommended dose for patients 1 month to less than 1 year of age or weighing less than 10 kg is 2.5 mg/kg. The recommended dose for patients ≥ age 1 and weighing ≥ 10 kg is 75 mg/m2.
This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application 1 month ahead of the FDA goal date.
This application was granted priority review, breakthrough designation. A description of FDA expedited programs is in the Guidance for Industry: Expedited Programs for Serious Conditions-Drugs and Biologics.
Healthcare professionals should report all serious adverse events suspected to be associated with the use of any medicine and device to FDA’s MedWatch Reporting System or by calling 1-800-FDA-1088.
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