Crizotinib: A novel, targeted gene therapy for the treatment of non-small-cell lung cancer

Crizotinib is a new kinase inhibitor recently approved by FDA for the treatment of patients with locally advanced or metastatic non-small-cell lung cancer who express the anaplastic lymphoma kinase (ALK) gene. FDA approval was based on objective response rate in 2 multicenter, single-arm studies. Crizotinib demonstrated response rates of 50% to 60% in patients enrolled in these studies significantly better results than any previously reported in this setting. Crizotinib was well tolerated, with less than 10% of patients discontinuing due to treatment-related adverse events. Ongoing clinical trials are evaluating crizotinib's impact on survival, its role in the first-line setting, and its safety and efficacy in other malignancies. The development and accelerated approval of targeted therapies such as crizotinib represent another significant milestone in the treatment of cancer. (Formulary. 2011;46:460-472.)