Azacitidine received U.S. Food and Drug AdminÂistration approval for the treatment of newly diagnosed juvenile myelomonocytic leukemia. Approval was granted based on the results of the AZA-JMML-001 study.
The international, multicenter, open-label trial included 18 pediatric patients. Researchers sought to assess the safety, pharmacodynamics, pharmacokinetics, and activity of the drug before hematopoietic cell transplant.
Half of the patients had confirmed clinical responses at three months; three had a complete clinical remission and six had a partial clinical remission. Rash, upper respiratory tract infection, pyrexia, and anemia occurred in more than 30% of patients and were the most common adverse events.
Source: U.S. Food & Drug Administration, June 3, 2022.