FDA Approves Azacitidine for Newly Diagnosed Juvenile Myelomonocytic Leukemia

The FDA has granted approval to azacitidine for the treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia following positive phase 2 research.

The FDA has granted approval to azacitidine (Vidaza) for the treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML), according to a press release by the FDA.1

Azacitidine was granted approval in the JMML patient population based on findings from the phase 2 multicenter, open-label AZA-JMML-001 clinical trial (NCT02447666), which evaluated the agent’s use in 28 patients between the ages of 1 month and 18 years.

The confirmed clinical response rate observed with azacitidine in the study was 50% (95% CI, 26-74), which consisted of these 3 confirmed complete responses (CRs) and 6 confirmed partial responses (PRs).

In the JMML cohort, patients received azacitidine 75 mg/m2 via Intravenous (IV) or subcutaneous (SC) administration once daily (QD) on days 1 to 7 of a 28-day cycle. Treatment lasted for a minimum of 3 cycles and a maximum of 6 cycles and continued in all patients until transplantation or disease progression.2

The primary end point of the AZA-JMML-001study was response rate at 3 months. The secondary end points used to determine efficacy included cytogenic response, molecular response, duration of response, time to response (TTR), TTR of clinical complete or partial response, and time to progression. The survival-related secondary end points were leukemia-free survival and overall survival. The study also assessed deoxyribonucleic acid methylation status in, time to hematopoietic stem cell transplant, adverse events, and pharmacokinetics.

In terms of the secondary end points, the study results showed that the median time to response was 1.2 months (range, 0.95-1.87 months). Ninety-four percent of patients underwent HSCT with the median time to HSCT being 4.6 months (range 2.8-19 months).1,3

The most common AEs observed with azacitidine in the JMML population were pyrexia, rash, upper respiratory tract infection, and anemia. These AEs collectively occurred in > 30% of patients in the study.1

Based on multiple phase 1 and 2 studies that utilized azacitidine to treat 220 patients total, the frequently observed adverse reactions categorized as blood and lymphatic system disorders included anemia (70%), thrombocytopenia (66%), leukopenia (48%), neutropenia (38%), febrile neutropenia (16%), and anemia aggravated (6%). Frequently observed gastrointestinal (GI) adverse reactions include nausea (70%), vomiting (54%), diarrhea (36%), constipation (12%), and other GI conditions occurred in 10% of patients or less. There are also general disorders, infections, and psychiatric conditions that have previously been observed in patients treated with azacitidine.4

For the treatment of JMML, the FDA recommends 2.5 mg/kg of azacitidine for patients 1 month to less than 1 year of age or children who weigh less than 10 kg. For children ≥ age 1 and weighing ≥ 10 kg, the FDA recommends a dose 75 mg/m2.1


1. FDA approves azacitidine for newly diagnosed juvenile myelomonocytic leukemia. News release. FDA. May 20, 2022. Accessed May 20, 2022. https://bit.ly/39LSZRa

2. Niemeyer CM, Flotho C, Lipka DB, et al. Response to upfront azacitidine in juvenile myelomonocytic leukemia in the AZA-JMML-001 trial. Blood Adv. 2021;5(14):2901-2908. doi:10.1182/bloodadvances.2020004144

3. Study with azacitidine in pediatric subjects with newly diagnosed advanced myelodysplastic syndrome (mds) and juvenile myelomonocytic leukemia (JMML). Clinicaltrials.gov. Updated July 11, 2019. Accessed May 20, 2022.

4. VIDAZA (azacitidine for injection), for subcutaneous or intravenous useInitial U.S. Approval: 2004. FDA.gov. Accessed May 20, 2022.