FDA Updates Tasigna Label to Reflect Treatment-Free Remission in Leukemia

Recently, the FDA updated the label for nilotinib (Tasigna) to include information for health care providers regarding how to discontinue treatment in certain patients who have achieved remission, according to an agency press release.

Nilotinib was first approved in 2007 for the treatment of patients with Philadelphia chromosome positive (Ph+) chronic myeloid leukemia (CML).

According to the newly-approved labeling, patients with early chronic phase CML who have been treated with nilotinib for at least 3 years and who have responded to treatment may be eligible to discontinue therapy.

“Patients diagnosed with CML generally face a lifetime of treatment to keep their leukemia from growing or recurring,” said Richard Pazdur, MD, director of the FDA Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA Center for Drug Evaluation and Research. “Today’s approval shows that some patients may be able to stop treatment with Tasigna altogether if they are showing a strong response to therapy. 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.”

Nearly all patients with CML have Ph+ disease, which produces the BCR-ABL protein, according to the FDA. Nilotinib—a kinase inhibitor—blocks the protein and inhibits abnormal cell growth.

The new approval adds information to the label that discusses patients who are eligible for discontinuation and the subsequent required monitoring for disease recurrence.

The FDA evaluated 2 trials that measured treatment-free remission (TRF) among patients with Ph+ chronic phase CML treated with nilotinib.

The first study included 190 newly-diagnosed patients who stopped nilotinib therapy after at least 3 years and who met additional criteria. Investigators found that 51.6% of patients remained in TRF at 48 weeks and 48.9% remained in TRF at 96 weeks, according to the release.

The second study included 126 patients who discontinued nilotinib after at least 3 years of therapy and after switching from imatinib. Approximately 57.9% of patients were still in TRF at 48 weeks and 53.2% were in TFR after 96 weeks, according to the release.

The FDA noted that a crucial aspect of the studies was monitoring the BCR-ABL protein in the blood, which can detect the first signs of relapse.

Common adverse events for patients who discontinued therapy included musculoskeletal symptoms such as aches, bone pain, and pain in the extremities, according to the release. Other adverse events include nausea, rash, headache, fatigue, pruritus, vomiting, diarrhea, cough, constipation, arthralgia, nasopharyngitis, pyrexia, night sweats, thrombocytopenia, myelosuppression, thrombocytopenia, neutropenia, and anemia.

Current nilotinib labeling includes a boxed warning for the risk of QT prolongation and sudden death, according to the FDA.

The updated labeling was granted under priority review and nilotinib previously received orphan drug designation.

"It has long been our ambition at Novartis to make it possible for some people with CML to discontinue therapy," said Bruno Strigini, CEO, Novartis Oncology. "We are proud that Tasigna is now the first and only [tyrosine kinase inhibitor] with TFR data in its labeling in the US and several countries around the globe. This achievement would not have been possible without the partnership of patients around the world who participated in our groundbreaking TFR trials, helping Novartis to once again reimagine what is possible for people living with CML."