FDA approves late-stage lung cancer drug

FDA approved ceritinib (Zykadia, Novartis) for patients with metastatic non-small cell lung cancer (NSCLC).

Ceritinib, the fourth drug with breakthrough therapy designation to be approved, received approval 4 months ahead of the product’s prescription drug user fee goal date of August 24, 2014.

Ceritinib is an anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor that blocks proteins that promote the development of cancerous cells. It is indicated for individuals with metastatic ALK-positive NSCLC who had been treated with crizotinib.

Disease progression is often inevitable for NSCLC patients whose tumor is driven by a rearrangement in the ALK gene. Most patients with ALK-positive NSCLC are relatively young on average, and have not smoked. These patients may relapse less than 1 year after beginning initial therapy with crizotinib and the approval of ceritinib addresses an unmet medical need for these patients, according to Novartis.

NSCLC is the most common type of lung cancer, accounting for 85% to 90% of all cases. However, only 2% to 7% of patients with NSCLC have the ALK gene rearrangement. Lung cancer is the leading cause of cancer-related deaths among men and women. According to the National Cancer Institute, almost 225,000 Americans will be diagnosed with lung cancer, and nearly 160,000 will die from it this year.

The approval of ceritinib is based on a pivotal trial that included 163 patients with metastatic ALK-positive NSCLC whose disease progressed or were intolerant to treatment with crizotinib. The most common sites of metastases in the patient population studied were brain (60%), liver (42%), and bone (42%).

Among previously treated patients, ceritinib achieved an overall response rate of 54.6% [95% CI, 47-62%] and a median duration of response of 7.4 months [95% CI, 5.4-10.1 months].   

Gastrointestinal symptoms including diarrhea, nausea, vomiting, and abdominal pain are common side effects of ceritinib. Laboratory abnormalities such as increased liver enzymes, pancreatic enzymes, and increased glucose levels also occurred.