Exelixis has completed the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) for CABOMETYX (cabozantinib) tablets as a treatment for patients with previously treated advanced hepatocellular carcinoma (HCC). The sNDA submission is based on results from the CELESTIAL randomized pivotal phase 3 trial of CABOMETYX in patients with advanced HCC who received prior sorafenib.
“We look forward to working closely with regulatory authorities through the review process in anticipation of bringing CABOMETYX to people diagnosed with advanced hepatocellular carcinoma, an underserved patient community that urgently needs new treatment options,” said Gisela Schwab, M.D., President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis. "We would sincerely like to thank the study patients and clinicians who participated in the CELESTIAL trial as well as our dedicated clinical development, medical and regulatory teams for bringing us another step closer to our goal of fully exploring the potential of CABOMETYX and making it accessible to every patient who may benefit from its use.”
On October 16, 2017, Exelixis announced that the independent data monitoring committee for the study recommended that the CELESTIAL trial be stopped for efficacy following review at the second planned interim analysis, with cabozantinib providing a statistically significant and clinically meaningful improvement in OS compared with placebo in patients with previously treated advanced HCC (pre-specified critical p-value ≤ 0.021). In March 2017, the FDA granted orphan drug designation to cabozantinib for the treatment of advanced HCC.
An sNDA is an application to the FDA that, if approved, will allow a drug sponsor to make changes to a previously approved product label, including modifications to the indication.