Novartis has received approval from the US Food and Drug Administration (FDA) for the use of its Tasigna (nilotinib) drug to treat Philadelphia chromosome-positive chronic myeloid leukaemia in the chronic phase (Ph+ CML-CP) in children.
Ph+CML is caused by the abnormal production of protein from a gene called BCR-ABL1. A signal sent by the BCR-ABL1 protein is responsible for the production of leukemic cells. Tasigna is designed to block this signal to allow the growth of healthy blood cells.
Tasigna has been approved for use in first and second-line paediatric patients aged one year or above.
Previously, the drug was indicated for newly diagnosed Ph+CML-CP in adults and paediatric patients with Ph+CML-CP resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy. It is also indicated for adult with Ph-CML in chronic and accelerated phase, or resistant to prior therapy.
Novartis Oncology CEO Liz Barrett said: “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 decision is based on findings from two clinical trials conducted to assess the safety and efficacy of nilotinib in 69 paediatric patients aged 2-18 with newly diagnosed Ph+ CML-CP or patients resistant to previous TKI therapy.
In newly diagnosed subjects, the cumulative major molecular response (MMR) was 64% at 12 cycles, with 15 patients experiencing the response.
The MMR rate was 40.9% at 12 cycles in subjects resistant or intolerant to prior TKI therapy. The median time to the first response was 2.8 months and 18 patients achieved the MMR.