FDA D.I.S.C.O. Burst Edition: FDA approval of Vidaza (azacitidine) for pediatric patients with newly diagnosed juvenile myelomonocytic leukemia
Welcome back to the D.I.S.C.O., FDA’s Drug Information Soundcast in Clinical Oncology, Burst Edition, brought to you by FDA’s Division of Drug Information in partnership with FDA’s Oncology Center of Excellence. Today we’ll provide another quick update on a recent FDA cancer drug approval.
On May 20, 2022, the FDA approved azacitidine (brand name Vidaza) for pediatric patients with newly diagnosed juvenile myelomonocytic leukemia.
Efficacy was evaluated in AZA-JMML-001, an international, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to hematopoietic stem cell transplantation in 18 pediatric patients with juvenile myelomonocytic leukemia. 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 hematopoietic stem cell transplantation 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 response criteria at 3 months. 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 had confirmed clinical responses. Of these 9 patients, there were 3 clinical complete remission and 6 clinical partial remission. The median time to response was 1.2 months. The proportion of patients undergoing hematopoietic stem cell transplantation was 94% and the median time to hematopoietic stem cell transplantation was 4.6 months.
The most common adverse reactions occurring in more than 30% in pediatric patients with juvenile myelomonocytic leukemia were pyrexia, rash, upper respiratory tract infection, and anemia.
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.
Full prescribing information for these approvals can be found on the web at www.fda.gov/drugsatFDA.
Health care professionals should report serious adverse events to FDA’s MedWatch Reporting Program at www.fda.gov/medwatch.
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