Vidaza Received New Indication for Patients with Newly Diagnosed Juvenile Myelomonocytic Leukemia

JHOP - June 2022 Vol 12, No 3 - FDA Oncology Update, Leukemia, Pediatric Cancer
Download PDF

On May 20, 2022, the FDA accelerated the approval of a new indication for azacitidine injection (Vidaza; Celgene) for the treatment of pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML).

The FDA granted this indication a breakthrough therapy designation. Azacitidine injection was previously approved for the treatment of patients with myelodysplastic syndromes.

This approval was based on the results of the AZA-JMML-001 study, an international, multicenter, open-label clinical trial that evaluated the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine before hematopoietic stem-cell transplant (HSCT) in 18 pediatric patients with JMML. The patients received intravenous (IV) azacitidine on days 1 to 7 of a 28-day cycle, for a minimum of 3 cycles and a maximum of 6 cycles. Patients were included in the study if they did not have disease progression or were not ready for HSCT between cycles 4 and 6.

The main efficacy outcome measures were clinical complete remission or partial remission according to the International Juvenile Myelomonocytic Leukemia Working Group response criteria at 3 months (cycle 3, day 28). The responses must have been sustained for at least 4 weeks in the 4-week period preceding or succeeding cycle 3, or for day 28.

Of the 18 patients, 9 (50%) patients had confirmed clinical responses. Of these 9 patients, 3 patients had a complete response and 6 patients had a partial response to IV azacitidine therapy. 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).

The most common (>30%) adverse reactions reported with IV azacitidine in pediatric patients with JMML were pyrexia, rash, upper respiratory tract infection, and anemia.

The recommended dose of IV azacitidine for patients aged 1 month to <1 year or those weighing <10 kg is 2.5 mg/kg; the recommended dose for patients aged ≥1 years and weighing ≥10 kg is 75 mg/m2.

Related Items
FDA-Approved Nirogacestat Demonstrates Improved Patient Outcomes in Desmoid Tumor Management
Web Exclusives published on February 12, 2024 in FDA Oncology Update
Iwilfin FDA Approved for Adults and Pediatric Patients with High-Risk Neuroblastoma
Web Exclusives published on January 17, 2024 in FDA Oncology Update
Welireg Now FDA Approved for Patients with Advanced Renal Cell Carcinoma
Web Exclusives published on January 17, 2024 in FDA Oncology Update
Keytruda Plus Chemotherapy Receives New FDA Approvals for Advanced Biliary Tract Cancer and 2 Forms of Advanced Gastroesophageal Junction Adenocarcinoma
Web Exclusives published on December 19, 2023 in FDA Oncology Update
FDA Approved Loqtorzi, a PD-1 Inhibitor, for the Treatment of Adults With Metastatic or Recurrent Nasopharyngeal Carcinoma
Web Exclusives published on December 18, 2023 in FDA Oncology Update
Tibsovo FDA Approved for Patients With Relapsed Myelodysplastic Syndromes and IDH1 Mutation
Web Exclusives published on December 18, 2023 in FDA Oncology Update
Fruzaqla FDA Approved for Refractory Metastatic Colorectal Cancer
JHOP - December 2023 Vol 13, No 6 published on December 6, 2023 in FDA Oncology Update
Rozlytrek Now Approved for Pediatric Patients Older Than 1 Month With Solid Tumors and NTRK Gene Fusion and in New Oral Pellet Form
JHOP - December 2023 Vol 13, No 6 published on December 6, 2023 in FDA Oncology Update
FDA Authorizes Updated COVID-19 Vaccine Formulations for Better Protection Against Current Variants
JHOP - December 2023 Vol 13, No 6 published on November 17, 2023 in FDA Oncology Update
Bosulif Now FDA Approved for Pediatric Patients With Chronic Myelogenous Leukemia
JHOP - December 2023 Vol 13, No 6 published on November 3, 2023 in FDA Oncology Update
© Amplity Health. All rights reserved.

Subscribe Today!

To sign up for our newsletter or print publications, please enter your contact information below.

I'd like to receive: