FDA expands approval of Tasigna for children with leukemia subtype

Issue: May 10, 2018

The FDA expanded the approval of nilotinib to include the first- and second-line treatment of pediatric patients with Philadelphia chromosome-positive chronic myeloid leukemia in the chronic phase, according to the agent’s manufacturer.

Nilotinib (Tasigna, Novartis) is indicated for adults with Ph+CML in chronic phase, pediatric patients with Ph+CML in chronic phase with resistance or intolerance to tyrosine kinase inhibitors, and adults with Ph+CML in chronic phase and accelerated phase with resistance or intolerance to imatinib.

The FDA based this expanded approval on two studies of 69 pediatric patients aged 2 years to younger than 18 years with Ph+CML in chronic phase who received nilotinib. Patients were either newly diagnosed or had resistance or intolerance to prior TKI therapy.

In the frontline setting, researchers observed a major molecular response (MMR) rate of 60% (95% CI; 38.7-78.9) at 12 cycles. Median time to first MMR was 5.6 months (range, 2.7-16.6).

In the second-line setting, the MMR rate was 40.9% (95% CI, 26.3-56.8) at 12 cycles. Median time to first MMR was 2.8 months (range, 0-11.3).

The observed adverse events were generally consistent with those observed in adults, except for hyperbilirubinemia, which occurred at grade 3 or grade 4 among 13% of patients. One patient treated in the second-line setting progressed to advanced phase/blast crisis about 10 months after treatment.

“Novartis’ commitment to people living with CML is reinforced by today’s FDA approval of Tasigna in children,” Liz Barrett, CEO of Novartis Oncology, said in a company-issued press release. “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.”

The FDA approved this indication under priority review.