FDA Approves Nilotinib for Pediatric Patients With CML

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Patients one year or older are now eligible to receive the drug.
Patients one year or older are now eligible to receive the drug.

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.

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. https://www.novartis.com/news/media-releases/novartis-drug-tasignar-approved-fda-treat-children-rare-form-leukemia. Accessed March 23, 2018.

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