September 17, 2021--Alameda, CA--(BUSINESS WIRE)
Exelixis, Inc. (NASDAQ: EXEL) announced that the U.S. Food and Drug Administration (FDA) has approved cabozantinib for the treatment of adult and pediatric patients aged 12 years and older with locally advanced or metastatic differentiated thyroid cancer (DTC) whose disease has progressed after prior vascular endothelial growth factor receptor (VEGFR) targeted therapy and who are radioiodine-refractory or unsuitable for radioiodine therapy. The FDA granted cabozantinib Breakthrough Therapy Designation and Priority Review designation, more than two months ahead of the Prescription Drug User Fee Act (PDUFA) target action date of December 4, 2021. Differentiated thyroid cancer is the most common type of thyroid cancer in the United States, and patients resistant to radioactive iodine therapy have a poor prognosis.
“Prior to today, patients with radioiodine-refractory differentiated thyroid cancer who had previously received VEGFR-targeted therapy and experienced disease progression faced aggressive disease with no standard treatment options,” said Marcia S. Brose, MD, PhD, Chief Operating Officer of the Sidney Kimmel Cancer Center at Jefferson Torresdale Hospital in Philadelphia, Co-Director of Community Clinical Trials at the Sidney Kimmel Cancer Center at Thomas Jefferson University, and Principal Investigator of the COSMIC-311 trial. “In the pivotal Phase 3 COSMIC-311 trial, cabozantinib prolonged progression-free survival in patients. FDA approval of cabozantinib is an important step forward for these patients who urgently need new treatment options.”
This approval is based on the results of the COSMIC-311 trial, a pivotal Phase 3 trial evaluating the efficacy of cabozantinib versus placebo in patients with radioiodine-refractory DTC who had experienced disease progression after receiving up to two prior VEGFR-targeted therapies. In the planned interim analysis, cabozantinib significantly reduced the risk of disease progression or death in the intention-to-treat population compared to placebo (p<0.0001). In a subsequent analysis with a median follow-up of 10.1 months, assessed by a blinded independent imaging committee, the median progression-free survival (PFS) was 11.0 months for patients treated with cabozantinib (n=170) and 1.9 months for those treated with placebo (n=88); hazard ratio (HR): 0.22; 95% confidence interval (CI): 0.15–0.31. These results will be presented at the 2021 European Society for Medical Oncology (ESMO) Congress this month.
“This approval of cabozantinib builds on our existing achievements in providing transformative medicines for patients with difficult-to-treat cancers,” said Dr. Michael M. Morrissey, President and CEO of Exelixis. “We thank the patients who participated in our clinical trials, the physicians and staff involved in the COSMIC-311 trial, and the FDA team for their cooperation during the expedited review of our application.”
About Cabozantinib
In the United States, cabozantinib tablets are approved for the treatment of patients with advanced renal cell carcinoma (RCC); for the treatment of patients with hepatocellular carcinoma previously treated with sorafenib; as first-line treatment for patients with advanced RCC in combination with nivolumab; and for the treatment of adult and pediatric patients aged 12 years and older with locally advanced or metastatic differentiated thyroid cancer whose disease has progressed after prior VEGFR-targeted therapy and who are radioiodine refractory or unsuitable for radioiodine therapy.
Cabozantinib tablets have also received regulatory approval in the EU and other countries and regions.
