Exelixis files NDA with FDA for cabozantinib to treat MTC

Exelixis, Inc. (NASDAQ: EXEL) today announced that it has completed the filing of its rolling New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for cabozantinib as a treatment for patients with progressive, unresectable, locally advanced, or metastatic medullary thyroid cancer (MTC).

The NDA was submitted under the FDA's Fast Track designation, which is designed to potentially accelerate the review of an investigational therapy for an unmet medical need. As part of the NDA filing, Exelixis has requested Priority Review designation from the FDA. If granted, the FDA's goal for completing the review would be six months from the date of receipt of the final submission.

The NDA filing is based on data from the EXAM trial, a phase 3 pivotal trial in patients with advanced MTC. The trial was conducted under a Special Protocol Assessment with the FDA, with progression-free survival (PFS) as the primary endpoint. In October 2011, Exelixis announced top-line results from EXAM demonstrating that the trial had met its primary endpoint of improving PFS: compared with placebo, cabozantinib improved median PFS by 7.2 months. The median PFS on the cabozantinib arm was 11.2 months versus 4.0 months on the placebo arm, with a hazard ratio (HR) of 0.28, (95% CI 0.19, 0.40), p < 0.0001.