FDA Approves Expanded Label of Vyxeos to Include Pediatric Patients with Secondary AML

March 31, 2021
Kristie L. Kahl
Kristie L. Kahl

The agency granted approval to the revised label of Vyxeos to now include treatment for newly diagnosed therapy-related acute myeloid leukemia or disease with myelodysplasia-related changes in pediatric patients aged 1 and older.

The Food and Drug Administration (FDA) approved a revised label for Vyxeos (daunorubicin and cytarabine) that now includes the indication to treat newly diagnosed therapy-related acute myeloid leukemia (AML) or AML with myelodysplasia-related changes (AML-MRC) in pediatric patients aged 1 and older, according to Jazz Pharmaceuticals, the agent’s manufacturer.

The agency granted approval to the revised label of Vyxeos — a liposomal combination of daunorubicin (an anthracycline topoisomerase inhibitor) and cytarabine (a nucleoside metabolic inhibitor) — based on safety data from two single-arm trials — AAML1421 and CPX-MA-1201 — as well as efficacy data from the CPX351-301 study 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," Dr. Edward Anders Kolb, director of the Center for Cancer and Blood Disorders at Nemours/Alfred I. DuPont Hospital for Children and chair of the Myeloid Disease Committee at Children's Oncology Group (COG) — who conducted the AAML1421 trial — said in a press release.

"Jazz has been a wonderful partner in pediatric drug development and we are grateful for the continued work being done to provide safe and effective therapies for children,” he added.

In the two clinical trials supporting the agent’s safety pharmacokinetics, Vyxeos was evaluated in children and young adults with AML or relapsed/refractory blood cancers. In total, 38 pediatric patients aged 1 to 21 years with second-line AML were enrolled in the phase 1/2 AAML1421 study and 27 patients aged 1 to 19 years with relapsed/refractory blood cancers were enrolled in the phase 1 CPX-MA-1201 study.

There was no difference observed in the safety profile from either study based on age.

In the phase 3 CPX351-301 study, the most common side effects included 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.

The agent was previously indicated for adult patients with newly diagnosed therapy-related AML or AML-MRC.

"At Jazz Pharmaceuticals, we believe all patients living with complex conditions deserve solutions and work diligently to expand the science behind our therapies to ensure the greatest number of patients can benefit from our medicines," Dr. Robert Iannone, executive vice president of research and development and chief medical officer of Jazz Pharmaceuticals, said in the release. "While pediatric patients represent a relatively small percentage of total AML patients, there is a critical need for more effective therapies in this setting. With the expansion of the Vyxeos label to include the pediatric population, Jazz demonstrates our continued commitment to broadening our cancer research and focusing on the people for whom we can have the greatest impact."

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