Nilotinib Gets Priority Review for Newly Diagnosed Early CML

Nilotinib Gets Priority Review for Newly Diagnosed Early CML

Novartis recently announced that nilotinib (Tasigna) has been granted priority review by the US Food and Drug Administration (FDA) for the treatment of adult patients with newly diagnosed Philadelphia chromosome–positive chronic myeloid leukemia (Ph+ CML) in chronic phase.

FDA priority review status is granted to therapies that offer major advances in treatment or provide a treatment where no adequate therapy exists. This status accelerates the standard review time from 10 to 6 months.

In addition to the US filing, regulatory submissions have been filed in the EU and Japan. All filings are based on data showing superior efficacy for nilotinib in the first head-to-head comparison of the drug against the standard of care imatinib in newly diagnosed Ph+ CML patients. If approved for the first-line indication, nilotinib will be the first drug for newly diagnosed patients to become available since the approval of imatinib in 2002.

ENESTnd Trial

The regulatory submissions are based on data from the ENESTnd (Evaluating Nilotinib Efficacy and Safety in Clinical Trials of Newly Diagnosed Ph+ CML Patients) phase III clinical trial. This randomized, open-label, multicenter trial compared the efficacy and safety of nilotinib vs imatinib in adult patients with newly diagnosed Ph+ CML in chronic phase. It is the largest global randomized comparison of two oral therapies ever conducted in newly diagnosed Ph+ CML patients.

In the ENESTnd trial, significantly fewer patients at 12 months progressed to accelerated or blastic phase on nilotinib, 300 mg twice daily, than on imatinib, 400 mg once daily (2 vs 11 patients), demonstrating a significant improvement in disease control. Fewer patients discontinued due to adverse events from the nilotinib 300 mg twice daily arm of the study compared to the imatinib 400 mg once daily arm.

Nilotinib is a potent and selective inhibitor of the Bcr-Abl protein that causes production of cancer cells in Ph+ CML. The first clinical trials of nilotinib began 21 months after its discovery, with the drug receiving its first regulatory approval in the second-line indication in 2007.