Vyxeos Now Indicated for Treatment of Pediatric Patients with AML

Web Exclusives - FDA Approvals, Select Drug Profiles

On March 30, 2021, the FDA approved a new indication for daunorubicin and cytarabine (Vyxeos; Jazz Pharmaceuticals) for the treatment of pediatric patients aged ≥1 years with newly diagnosed, therapy-related acute myeloid leukemia (AML) or patients with AML and myelodysplasia-related changes. Daunorubicin and cytarabine is a liposomal combination of an anthracycline topoisomerase inhibitor (daunorubicin) and a nucleoside metabolic inhibitor (cytarabine).

The FDA has previously approved the combination of daunorubicin and cytarabine for the treatment of adults with newly diagnosed, therapy-related AML or AML with myelodysplasia-related changes.

The FDA approved this new indication in pediatric patients based on safety data from 2 single-arm clinical trials: the AAML1421 study and the CPX-MA-1201 study, as well as on efficacy evidence shown in a previous clinical trial in adults.

“The expansion of the Vyxeos label to include children is a welcome and necessary advancement in support of some of our most vulnerable patients,” said Edward Anders Kolb, MD, Director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children, and Chair of the Myeloid Disease Committee of the Children’s Oncology Group.

In all, 38 pediatric patients aged 1 year to 21 years with AML who were in first relapse were enrolled in the phase 1/2 AAML1421 study, and 27 patients aged 1 year to 19 years with relapsed or refractory hematologic malignancies were enrolled in the phase 1 CPX-MA-1201 study. In both studies, no differences were found in the safety profile of daunorubicin and cytarabine based on the patients’ age.

The most common (≥25%) adverse reactions with daunorubicin and cytarabine include bleeding events, fever, rash, swelling, nausea, sores in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain, difficulty breathing, headache, cough, decreased appetite, irregular heartbeat, pneumonia, blood infection, chills, sleep disorders, and vomiting.

Related Items
Breyanzi Now FDA Approved for Second-Line Treatment of Large B-Cell Lymphoma
Web Exclusives published on September 22, 2022 in FDA Approvals
Tafinlar plus Mekinist First Tumor-Agnostic Therapy Approved for Any Unresectable or Metastatic Solid Tumors with BRAF Mutation
Web Exclusives published on September 2, 2022 in FDA Approvals
Xalkori Now FDA Approved for the Treatment of ALK-Positive Inflammatory Myofibroblastic Tumor
Web Exclusives published on August 20, 2022 in FDA Approvals
Yescarta First CAR T-Cell Therapy FDA Approved for Large B-Cell Lymphoma
Web Exclusives published on July 29, 2022 in FDA Approvals
Tibsovo Received a New Indication, in Combination with Azacitidine, for Newly Diagnosed Patients with AML and IDH1 Mutation
Web Exclusives published on July 22, 2022 in FDA Approvals
Last modified: August 30, 2021
Copyright © Engage Healthcare Communications, LLC. All rights reserved.