FDA Updates Product Label for Nilotinib

The U.S. Food and Drug Administration (FDA) updated the product label for nilotinib to include information for providers about how to discontinue the drug in certain patients. Nilotinib, first approved by the FDA in 2007, is indicated for the treatment of patients with Philadelphia chromosome–positive (Ph+) chronic myeloid leukemia (CML). With the updated dosing recommendations, patients with early chronic-phase CML who have been taking nilotinib for three years or longer, and whose leukemia has responded to treatment, may be eligible to stop taking the medication.

“While we welcome this progress in patient care, it’s important to note that any discontinuation of treatment still means patients must be regularly monitored for disease recurrence,” Richard Pazdur, MD, director of the FDA’s Oncology Center of Excellence, said in a statement.

The information about discontinuing nilotinib was based on two single-arm trials of patients with Ph+ chronic-phase CML. In the first trial, among the 190 newly diagnosed patients with CML who stopped nilotinib after taking it for three or more years, 51.6 percent were still in treatment-free remission (TFR) after 48 weeks, and 48.9 percent were still in TFR after 96 weeks. In the second trial, among the 126 patients who had stopped nilotinib after taking it for three or more years after switching from the cancer drug imatinib, 57.9 percent were still in TFR after 48 weeks, and 53.2 percent were still in TFR after 96 weeks.

An important part of both trials was regular and frequent monitoring of RNA information that specifies the BCR-ABL protein level in the blood, which provides the first signs of relapse.

Common side effects in patients who discontinued nilotinib included musculoskeletal symptoms such as body aches, bone pain, and pain in extremities, which were prolonged in some patients. Severe side effects typically associated with nilotinib (such as myelosuppression, cardiac events, inflammation of the pancreas, liver damage, and severe bleeding) occurred less frequently in patients who discontinued nilotinib. However, the long-term outcomes of discontinuing treatment remain unknown.

Source: FDA news release, December 22, 2017.