The US Food and Drug Administration (FDA) approved ceritinib (Zykadia; Novartis) for the treatment of patients with metastatic, anaplastic lymphoma kinase (ALK)-positive non–small-cell lung cancer (NSCLC). Ceritinib is an ALK tyrosine kinase inhibitor that blocks proteins that promote cancer cell growth. The drug is approved for the treatment of patients with late-stage NSCLC who were previously treated with crizotinib, the first and only other ALK tyrosine kinase inhibitor approved by the FDA.
The FDA approved ceritinib under the FDA’s accelerated approval process, 4 months ahead of its scheduled final review date, to expedite access to this drug for a patient population with a life-threatening condition and very few treatment options available.
“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. “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.”
The safety and efficacy of ceritinib were established in a clinical trial with 163 patients with metastatic, ALK-positive NSCLC. All patients received ceritinib: approximately 50% of the patients had their tumor shrink with this therapy, and this result lasted an average of approximately 7 months.
Reported side effects were mainly gastrointestinal, such as diarrhea, nausea, vomiting, and abdominal pain. Laboratory abnormalities included increased liver enzymes and pancreatic enzymes, as well as increased glucose levels. April 29, 2014
