Nilotinib (Tasigna) was previously granted priority review for the treatment of pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia.
Today, the FDA approved an expanded indication for nilotinib (Tasigna) as a first- and second-line treatment for pediatric patients 1 year and older with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase (Ph+ CML-CP), according to a press release.
Nilotinib is currently indicated to treat both adults and pediatric patients with newly-diagnosed Ph+ CML-CP. It is also indicated to treat pediatric patients 1 year and older with Ph+ CML-CP resistant or intolerant to prior tyrosine kinase inhibitor (TKI) treatment.
Nilotinib is further indicated in adults with Ph+ CML in the chronic and accelerated phases who are resistant or intolerant to prior treatments, including imatinib, according to the release.
Novartis reported that nearly all patients with CML have an abnormality called the Philadelphia chromosome, which produces the BCR-ABL protein and facilitates the proliferation of cancerous white blood cells.
The new indication was granted priority review based on findings from 2 studies that evaluated the efficacy and safety of nilotinib in 69 pediatric patients with Ph+ CML-CP. Nilotinib was used as a first-line therapy in newly-diagnosed patients and as a second-line therapy among those who were resistant or intolerant to TKI therapy.
The researchers found that the major molecular response rate (MMR) was 60% at 12 cycles among newly-diagnosed patients. The cumulative MMR rate among newly-diagnosed pediatric patients was 64% by cycle 12, with a median time to first MMR of 5.6 months, according to the release.
In the second-line setting, the MMR rate was 40.9% at 12 cycles. The cumulative MMR rate among patients with resistance or intolerance was 47.7% by cycle 12, with a median time to first MMR of 2.8 months, according to Novartis.
Adverse reactions observed in pediatric patients were consistent with those in adults; however, pediatric patients were more likely to develop laboratory abnormalities of hyperbilirubinemia. Additionally, a resistant or intolerant pediatric patient progressed to advanced phase/blast crisis after 10 months of treatment with nilotinib, according to the release.
"Novartis' commitment to people living with CML is reinforced by today's FDA approval of Tasigna in children," said Liz Barrett, CEO, Novartis Oncology. "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."