FDA Approves Ceritinib (Zykadia) for Metastatic Lung Cancer

Ceritinib is the second FDA-approved drug for ALK-positive lung cancer.

Earlier today the US Food and Drug Administration granted accelerated approval to ceritinib (Zykadia) for the treatment of patients with metastatic ALK-positive non–small-cell lung cancer (NSCLC). About 2% to 7% percent of NSCLC patients have ALK-positive disease.

The new drug, a tyrosine kinase inhibitor, was approved 4 months early under the FDA's accelerated approval program and is intended for the treatment of patients who previously received the ALK-inhibitor crizotinib.

“Today’s approval illustrates how a greater understanding of the underlying molecular pathways of a disease can lead to the development of specific therapies aimed at these pathways,” said Richard Pazdur, MD, director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research, in a press release. “It also demonstrates the FDA’s commitment to working cooperatively with companies to expedite a drug’s development, review, and approval, reflecting the promise of the breakthrough therapy designation program.”

Ceritinib was granted breakthrough therapy designation as the trial results suggest the drug may be a substantial improvement over current therapies.

The trial that led to the approval of ceritinib included 163 NSCLC patients with metastatic ALK-positive disease, all of whom received the study drug. Tumor shrinkage occurred in about half of the trial participants, lasting about 7 months on average.

In the trial, common adverse events included diarrhea, abdominal pain, nausea, and vomiting. Also observed were laboratory abnormalities such as increased glucose levels, and increased liver and pancreatic enzymes.

In 2014, it is estimated that 224,210 people will be diagnosed with lung cancer in the United States, according to the National Cancer Institute. The leading cause of cancer-related deaths, it is estimated that 159,260 men and women will die from lung cancer this year. NSCLC is the most common type of lung cancer, comprising about 85% of cases.