The Food and Drug Admin-istration has granted accelerated approval to Zykadia (ceritinib) for patients with late-stage (metastatic) non-small cell lung cancer (NSCLC). The drug had previously been granted Breakthrough Drug status, and the approval was given four months ahead of the review completion drug date.
Zykadia is an anaplastic lymphoma kinase (ALK) tyrosine kinase inhibitor (TKI) that blocks proteins that promote the development of cancerous cells. It is intended for patients with metastatic ALK-positive NSCLC who were previously treated with crizotinib, the only other approved ALK TKI.
ALK-positive NSCLCs are rare: Of the approximately 85 percent of lung cancers that are NSCLC, only two to seven percent are ALK-positive.
"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," Richard Pazdur, MD, the Director of the FDA's Ofice of Hematology and Oncology Products, said in a news 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."
Zykadia is the fourth drug with breakthrough therapy designation to receive FDA approval. The agency said the drug was granted breakthrough therapy designation, priority review, and orphan product designation because, respectively:
* The sponsor (Novartis) demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies;
* The drug had the potential, at the time the application was submitted, to be a significant improvement in safety or effectiveness in the treatment of a serious condition; and
* The drug is intended to treat a rare disease.
Zykadia's safety and effectiveness were established in a clinical trial of 163 participants with metastatic ALK-positive NSCLC, all of whom received the drug. Results showed that about half of the participants had their tumors shrink, and this effect lasted an average of about seven months.
"Zykadia represents an important treatment option for ALK+ NSCLC patients who relapse after starting initial therapy with crizotinib," the lead investigator for the researcher, Alice T. Shaw, MD, PhD, of Massachusetts General Hospital Cancer Center, said in a Novartis news release.
"This approval will affect the way we manage and monitor patients with this type of lung cancer, as we will now be able to offer them the opportunity for continued treatment response with a new ALK inhibitor."
Common side effects of Zykadia include gastrointestinal symptoms such as diarrhea, nausea, vomiting, and abdominal pain. Laboratory abnormalities such as increased liver enzymes, pancreatic enzymes, and increased glucose levels were also observed.