Exelixis scored a double victory yesterday when FDA approved the company’s first product, the thyroid cancer drug Cometriq (cabozantinib), while the European Medicines Agency accepted for review the drug’s Marketing Authorization Application (MAA) allowing for its sale in the 27-nation European Union.
FDA approved Cometriq for treatment of progressive, metastatic medullary thyroid cancer (MTC). The drug inhibits 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.
FDA based its approval of Cometriq on the results of the Phase III EXAM clinical trial, conducted in 330 patients with progressive, metastatic MTC. EXAM met its primary efficacy endpoint of improving progression-free survival (PFS), with 219 Cometriq patients experience a median PFS time of 11.2 months, compared with 4 months for the 111 patients receiving placebo.
“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,” Steven I. Sherman, M.D., a senior investigator in the Phase III study. Dr. Sherman is the Naguib Samaan distinguished professor in endocrinology at M.D. Anderson Cancer Center.
Cometriq was recommended at a once-daily dose of 140 mg orally—one 80 mg capsule, and three 20 mg capsules. The drug 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. The drug carries boxed warnings concerning risk of perforations and fistulas, and hemorrhage.
FDA granted Priority Review Designation to Exelixis’ rolling NDA, completed in May. The agency met its Prescription Drug User Fee Act (PDUFA) action date for a decision on the NDA, set for Nov. 29. Cometriq is the 31st new drug to win FDA approval this year, compared with 30 all of 2011.
In Europe, Cometriq submitted a slightly different indication of progressive, unresectable, locally advanced, or metastatic MTC. Earlier, Cometriq received the European Union’s orphan drug designation for the treatment of MTC from the Committee for Orphan Medicinal Products.