BMS Updates Vidaza Label After Pediatric Indication Approval

The label now includes a new section about the risk of substituting an oral azacitidine product, Onureg, for the injection therapy.

Bristol Myers Squibb has updated the prescribing information of Vidaza (azacitidine) to include a new section about the risks of substitution with other azacitidine products, especially oral versions of the medication.

This move was made after the FDA approval of Vidaza in May 2022 to treat pediatric patients with newly diagnosed juvenile myelomonocytic leukemia (JMML), a rare blood cancer where the white blood cells don’t mature normally. Vidaza is an intravenous therapy that had previously been approved to treat adults with myelodysplastic syndrome, a group of blood cancers.

The product also is approved as a tablet and marketed by Bristol Myers Squibb under the brand name Onureg to treat adult patients with acute myeloid leukemia who achieved first complete remission.

The label now includes a warning not to substitute the oral for the injection therapy. The new subsection reads: “Due to substantial differences in the pharmacokinetic parameters, the recommended dose and schedule for Vidaza are different from those of oral azacitidine products. Treatment of patients using Vidaza at the recommended dosage of oral azacitidine may result in a fatal adverse reaction. Treatment of patients using oral azacitidine at the doses recommended for Vidaza may not be effective.”

The new pediatric indication was approved based on data from an open-label study to evaluate the pharmacokinetics, pharmacodynamics and safety of azacitidine prior to hematopoietic stem cell transplantation (HSCT) in 18 pediatric patients with JMML.

The main efficacy outcome measures were clinical complete remission or clinical partial remission. Half of the patients had confirmed clinical responses. Of these 9 patients, there were three complete remissions and six partial remissions. The median time to response was 1.2 months. The proportion of patients undergoing HSCT was 94% and the median time to HSCT was 4.6 months.

Most common adverse reactions (>30%) occurring in pediatric patients with JMML were fever, rash, upper respiratory tract infection, and anemia.

This application was granted priority review and breakthrough designation.