Exelixis, Inc. (NASDAQ: EXEL) today announced that the U.S. Food and Drug Administration (FDA) has approved COMETRIQ™ (cabozantinib) for the treatment of progressive, metastatic medullary thyroid cancer (MTC). COMETRIQ is an inhibitor of multiple receptor tyrosine kinases involved in both normal cellular function and pathologic processes such as oncogenesis, metastasis, tumor angiogenesis, and maintenance of the tumor microenvironment. The COMETRIQ label has boxed warnings concerning risk of perforations and fistulas, and hemorrhage.
Exelixis completed its rolling New Drug Application (NDA) in May 2012, and the FDA granted Priority Review Designation to the filing, assigning a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2012. The COMETRIQ approval is based on the results of EXAM, a randomized phase 3 clinical trial conducted in 330 patients with progressive, metastatic MTC, which met its primary efficacy endpoint of improving progression-free survival (PFS).
"The approval of COMETRIQ is an important milestone for patients with progressive, metastatic medullary thyroid cancer, their families, and their physicians, as well as for Exelixis," said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. "We are grateful to the many patients who participated in the clinical development of COMETRIQ in MTC, and we are committed to making this important new therapy available as quickly as possible."
The recommended dose of COMETRIQ is 140 mg orally, once daily (one 80 mg capsule and three 20 mg capsules). COMETRIQ should not be taken with food, and patients are advised to not eat for at least 2 hours before and at least 1 hour after taking COMETRIQ. Full prescribing information, including Boxed Warning, is available at www.exelixis.com or www.COMETRIQ.com.
"There has been little clinical progress in treating advanced MTC until the introduction of targeted therapies, and it is gratifying to give these patients a new treatment option that has been shown in clinical trials to improve progression-free survival remarkably by nearly three-fold," said Steven I. Sherman, M.D., Naguib Samaan Distinguished Professor in Endocrinology at M.D. Anderson Cancer Center and a senior investigator in the phase 3 study. "The availability of a new therapeutic approach that has the potential to improve patient care and outcomes changes the MTC treatment landscape and provides patients and physicians with a new way to manage the disease."