Exelixis, Inc. has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation to cabozantinib as a potential treatment for patients with advanced renal cell carcinoma (RCC) who have received one prior treatment.

“Receiving Breakthrough Therapy Designation is an important regulatory achievement for cabozantinib in renal cell carcinoma,” said Michael M. Morrissey, PhD, president and chief executive officer of Exelixis.

“Following the positive top-line results announced in July and a productive dialogue with the FDA, Exelixis believes we can expedite our regulatory timelines and complete the cabozantinib NDA submission in advanced RCC prior to the end of 2015.”

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Breakthrough Therapy Designation is based on findings from the phase 3 METEOR trial, which compared cabozantinib to everolimus in patients with RCC who experienced disease progression after treatment with a VEGF receptor kinase inhibitor. Results showed that cabozantinib significantly reduced the rate of disease progression or death by 42% compared with everolimus.

“We look forward to working closely with the FDA during the submission and review process, keeping in mind our ultimate goal of bringing a new therapeutic option to the renal cell carcinoma community as soon as possible,” Dr. Morrissey said.

RELATED: Second-line Everolimus Safe, Effective for VEGF-refractory Renal Cell Carcinoma

Cabozantinib, a tyrosine kinase inhibitor, is currently approved under the brand name Cometriq for the treatment of patients with progressive, metastatic medullary thyroid cancer.

Cabozantinib labeling carries a warning for gastrointestinal perforations and fistulas as well as severe and sometimes fatal hemorrhages. Common adverse events include diarrhea, stomatitis, palmar-plantar erythrodysesthesia syndrome, weight loss, loss of appetite, nausea, fatigue, oral pain, change in hair color, dysgeusia, hypertension, abdominal pain, and constipation.


  1. FDA grants Breakthrough Therapy Designation to Exelixis’ cabozantinib for the treatment of renal cell carcinoma in patients who received one prior therapy [news release]. South San Francisco, CA: Exelixis, Inc; August 24, 2015. http://www.exelixis.com/investors-media/press-releases. Accessed August 24, 2015.