Nilotinib Approval Expanded to Include Pediatric CML Type

The U.S. Food and Drug Administration (FDA) has expanded the approved indication for nilotinib (Tasigna) to include the treatment of first- and second-line pediatric patients ages one year and older with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP).

Nilotinib is now indicated in the United States for the treatment of adult and pediatric patients with newly diagnosed Ph+CML-CP; pediatric patients ages one year and older with Ph+CML-CP resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy; and adult patients with Ph+ CM in chronic phase and accelerated phase, resistant or intolerant to prior therapy that included imatinib.

"This expanded use, along with the other recent global regulatory Tasigna milestones, underscores our dedication to reimagining medicine and addressing the needs for people with CML, including children with this cancer,” said Liz Barrett, CEO, Novartis Oncology in the company’s official press release.

The approval is based on 2 studies that evaluated the efficacy and safety of nilotinib in pediatric patients.

A total of 69 first- or second-line pediatric patients aged 2 to 18 years with Ph+CML-CP received nilotinib, and in the former, the major molecular response (MMR) rate was 60.0% at 12 cycles, with 15 patients achieving MMR. The MMR rate at 12 cycles for newly diagnosed pediatric patients was 64.0%, and the median time to first MMR was 5.6 months. In the latter, the MMR rate was 40.9% at 12 cycles, with 18 patients being in MMR. The cumulative MMR rate among pediatric patients were 64.0% and 47.7% among the first- and second-line patients, respectively.

In these pediatric studies, adverse reactions were generally consistent with what has historically been observed in adults, however, one notable exception included laboratory abnormalities of hyperbilirubinemia, which was reported at a higher frequency than in adult patients. Neither study reported any deaths during treatment or following discontinuation.

Other reported adverse effects (AEs) of nilotinib are typical of anti-cancer drugs, and include headache, fatigue, nausea, vomiting, diarrhea, constipation, muscle and joint pain, rash, flu-like symptoms, and reduced blood cell count.

This is the second recent FDA approval for this indication, as dasatinib (Sprycel) for the treatment of pediatric patients with Ph+CML-CP was given the go-ahead in November 2017.

CML originates in the blood-generating cells of the bone marrow and eventually invades the blood. According to the National Cancer Institute (NCI), it accounts for approximately 10% of all leukemias. There were an estimated 8,950 new cases in 2017, and 1,080 estimated deaths.

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