Exelixis, Inc. announced that it has completed the submission of its rolling New Drug Application (NDA) with the U.S. Food and Drug Administration (FDA) for cabozantinib as a treatment for patients with advanced renal cell carcinoma (RCC) who have received one prior therapy. Exelixis has requested Priority Review as part of the NDA filing.
In August 2015, the FDA granted Breakthrough Therapy designation to cabozantinib for this potential advanced RCC indication. Breakthrough Therapy designation can expedite the development and review of drugs that are intended to treat serious or life-threatening diseases, and for which preliminary clinical evidence indicates the drug may demonstrate substantial improvement over existing therapies on one or more clinically significant endpoints. Drugs that receive Breakthrough Therapy designation may benefit from the involvement of FDA senior leadership in the review process, rolling submission, and other benefits. Prior to receiving Breakthrough Therapy designation, cabozantinib received Fast Track designation for its potential advanced RCC indication in April 2015.
“Completing the submission of our rolling New Drug Application brings us closer to our goal of improving the treatment options for patients with advanced kidney cancer,” said Michael M. Morrissey, Ph.D., president and chief executive officer of Exelixis. “Following the release of positive top-line results from our phase 3 pivotal trial in July, the Exelixis team worked expeditiously to complete the U.S. regulatory filing by year end. We look forward to continuing to work with the FDA team during the review process.”
The NDA submission is based on results of METEOR, a phase 3 pivotal trial comparing cabozantinib to everolimus in patients with advanced RCC who experienced disease progression following treatment with a VEGF receptor tyrosine kinase inhibitor. In July 2015, Exelixis announced top-line results from METEOR demonstrating that the trial had met its primary endpoint of improving progression-free survival; compared with everolimus, cabozantinib was associated with a 42% reduction in the rate of disease progression or death. These data were later presented at the European Cancer Congress in September 2015 and concurrently published in The New England Journal of Medicine.
In the European Union, Exelixis aims to complete its Marketing Authorization Application (MAA) in early 2016. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recently granted accelerated assessment to cabozantinib for advanced RCC. As a result, when filed, the company’s MAA may be eligible for a 150-day review, versus the standard 210 days (excluding clock stops when information is requested from CHMP).
Cabozantinib is currently marketed in capsule form under the brand name COMETRIQ® in the United States for the treatment of progressive, metastatic medullary thyroid cancer (MTC), and in the European Union for the treatment of adult patients with progressive, unresectable locally advanced or metastatic MTC. COMETRIQ is not indicated for patients with RCC. In the METEOR trial, and all other cancer trials currently underway, Exelixis is investigating a tablet formulation of cabozantinib distinct from the COMETRIQ capsule form. The tablet formulation of cabozantinib is the subject of the NDA for advanced RCC.
