FDA Approves Azacitidine for Newly Diagnosed Juvenile Myelomonocytic Leukaemia

On 20 May 2022, the US Food and Drug Administration (FDA) approved azacitidine (Vidaza, Celgene Corp.) for paediatric patients with newly diagnosed juvenile myelomonocytic leukaemia (JMML).

Efficacy was evaluated in AZA-JMML-001 (NCT02447666), an international, multicentre, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to haematopoietic stem cell transplantation (HSCT) in 18 paediatric 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% confidence interval 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 paediatric patients with JMML were pyrexia, rash, upper respiratory tract infection, and anaemia.

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.

Full prescribing information for Vidaza is available here.

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.

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.

For assistance with single-patient INDs for investigational oncology products, healthcare professionals may contact FDA’s Oncology Center of Excellence Project Facilitate.