Good to Know > FDA Approves Azacitidine for Rare Form of Leukemia in Pediatric Patients

FDA Approves Azacitidine for Rare Form of Leukemia in Pediatric Patients

The US Food and Drug Administration recently approved Azalpho (Azacitidine) to treat pediatric patients with newly diagnosed JMML (Juvenile Myelomonocytic Leukemia).


On May 20, 2022, Azacitidine received the approval of the US FDA for the treatment of patients aged 1 month and older diagnosed with a rare type of blood cancer namely Juvenile Myelomonocytic Leukemia. Earlier, Azacitidine was approved and prescribed for patients suffering from myelodysplastic syndromes (a group of blood/bone marrow disorders).


Efficacy was evaluated in AZA-JMML-001, an international, open-label, multicenter study to assess the pharmacokinetics, pharmacodynamics, safety, and activity of Azacitidine prior to hematopoietic stem cell transplantation (HSCT) in 18 pediatric patients suffering from juvenile myelomonocytic leukemia. Patients were treated with intravenous Azacitidine injection daily on days 1 to 7 of a 28-day cycle. For 3 to 6 cycles provided, patients did not have symptoms of disease progression.

Clinical full or partial remission according to the International JMML response criteria at 3 months was the primary efficacy outcome measure (cycle 3, day 28). Responses sustained for at least 4 weeks. 9 patients had proven clinical responses. There were three clinical full remissions and six clinical partial remissions among these 9 patients. The average response time was 1.2 months (range: 0.95–1.87). The median time to HSCT was 4.6 months (range = 2.8–19 months) and the proportion of patients who had HSCT was 94%.

The most common side effects of Azacitidine injection in pediatric patients included:

upper respiratory tract infection, low red blood cell count, and skin rash.


The recommended dose of Azacitidine for pediatrics aged between 1 to 12 months (having bodyweight of less than 10kg) is 2.5 mg/kg. Whereas, patients aged above 12 months and weighing greater than or equal to 10kg must be administered a 75 mg/m2  dose.


About JMML

JMML (juvenile myelomonocytic leukemia) is an uncommon blood malignancy that affects children. When monocytes and myelocytes, two types of white blood cells, do not mature properly, JMML develops. In certain youngsters, this disease might occur spontaneously or be linked to other genetic illnesses.


About Azacitidine

Azalpho (Azacitidine) is an anti-cancer drug belonging to the class of pyrimidine nucleoside analogs. Experts believe that it exerts antineoplastic effects. The medicine does so by causing hypomethylation (loss of DNA methylation). At high doses, it induces cytotoxicity via incorporation into DNA and RNA.