Nilotinib is now approved for use by children aged 1 year and older with Philadelphia chromosome–positive chronic myeloid leukemia (Ph+ CML) in the chronic phase.

The Food and Drug Administration expanded the drug’s indication to include use as first- and second-line treatment in children.

The approval, which was announced March 22, was made under the agency’s Priority Review designation. It is based on results from two recent studies showing efficacy in children. In total, the studies included 69 patients aged 2-18 years with Ph+ CML in the chronic phase. Among newly diagnosed patients, the major molecular response (MMR) rate was 60% at 12 cycles with 15 patients achieving MMR. Among patients with resistance or intolerance to prior tyrosine kinase inhibitor therapy, the MMR rate was 40.9% at 12 cycles with 18 patients achieving MMR, according to Novartis , which markets the drug.

Adverse events in the pediatric studies were similar to those observed in adults. However, children experienced hyperbilirubinemia (grade 3/4: 13%) and transaminase elevation (AST grade 3/4: 1%; ALT grade 3/4: 9%). Additionally, one previously treated pediatric patient progressed with advance phase/blast crisis after about 10 months of treatment.

Nilotinib (Tasigna) was already approved in adults with newly diagnosed Ph+ CML in the chronic phase and adults with chronic phase and accelerated phase Ph+ CML resistant or intolerant to prior therapy.


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