The US Food and Drug Administration (FDA) expanded the approval of nilotinib, a tyrosine kinase inhibitor (TKI), to include pediatric patients with chronic phase–, Philadelphia chromosome–positive chronic myeloid leukemia (CML), according to a press release.1

Nilotinib is now approved by the FDA for this group of patients as a first- or second-line therapy. Patients one year or older are eligible to receive the drug.

The approval, which was granted under the FDA’s Priority Review designation, was based on data from 2 studies showing promising safety and efficacy in both newly diagnosed patients and those intolerant or resistant to a prior TKI, including imatinib.

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According to the press release, 69 pediatric patients with newly diagnosed or resistant/intolerant disease were treated with nilotinib. Among newly diagnosed patients, the major molecular response rate was 60% at 12 cycles; among resistant/intolerant patients, the major molecular response rate was 40.9% at 12 cycles. One patient progressed to blast crisis after 10 months of nilotinib treatment.

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Grade 3 or 4 adverse events that occurred more frequently in pediatric patients vs adults treated with nilotinib included hyperbilirubinemia and elevated liver enzyme levels.


  1. Novartis drug Tasigna® approved by FDA to treat children with rare form of leukemia [news release]. Basel, Switzerland: Novartis; March 22, 2018. Accessed March 23, 2018.