The European Commission has approved Novartis’ Tasigna (nilotinib) for the treatment of children with a specific type of chronic myeloid leukemia (CML). The treatment is now approved for both newly diagnosed patients and those who failed earlier treatment that included Glivec (imatinib).
“Treatment options for children with CML have historically been limited, and with this new indication an unmet need has been addressed,” Bruno Strigini, chief executive officer at Novartis Oncology, said in a press release.
The drug’s approval is specifically aimed at patients in the chronic phase of CML who have a chromosomal abnormality called a Philadelphia chromosome, by far the most common type of CML.
In September 2017, the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended the approval of Tasigna for these children after reviewing evidence from two clinical trials — a Phase 1 study (NCT01077544) and a Phase 2 study (NCT01844765).
The studies, developed with the advice of the EMA, included 69 children and adolescents ages 2-18. Study findings showed that among those with newly diagnosed CML, 60% had a molecular response to the drug, meaning that researchers could detect normal gene activity in the disease-related gene.
Among patients who had previously been treated, the molecular response rate was 40.9%.
Tasigna had a similar safety profile in these children as seen earlier in adults, with some exceptions. Children showed increased levels of bilirubin in their blood analyses and signs of liver toxicity more often than adults.
“Data from two prospective studies demonstrated Tasigna is safe and effective in patients as young as two years old, which is consistent with the established safety profile of Tasigna in adults,” Strigini said.
Tasigna is a drug that blocks an enzyme called tyrosine kinase, which boosts cancer growth. CML cells from patients with the Philadelphia chromosome produce this enzyme.
Tasigna already had been approved for adults with various types of CML in both Europe and the U.S. In June, the European Commission also allowed Novartis to include information on stopping the treatment, as data demonstrated that patients with chronic phase CML may have the ability to maintain a treatment response after stopping the treatment.
The drug is currently not approved for the treatment of CML in children in the U.S.