FDA Grants Breakthrough Designation to Renal Cell Carcinoma Drug

Cabozantinib is a potential treatment for patients with advanced renal cell carcinoma who received one prior therapy.

Patients with renal cell carcinoma may soon have a significant new tool in their fight against cancer.

The FDA on Monday announced it granted Breakthrough Therapy Designation to cabozantinib (Cometriq) by Exelixis, Inc. The drug offers a potential therapy for patients with advanced renal cell carcinoma (RCC) who received 1 prior therapy.

“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. 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.”

Cabozantinib was found to inhibit tyrosine kinase activity, including MET, VEGF receptors, AXL, and RET. These kinases are involved in normal cellular function and pathologic processes, including oncogenesis, metastasis, tumor angiogenesis, and tumor microenvironment maintenance.

The Breakthrough Therapy Designation for cabozantinib follows the findings of the phase 3 METEOR trial, which compared cabozantinib to everolimus in patients with RCC whose disease progressed after therapy with a VEGF receptor tyrosine kinase inhibitor.

In July, Exelixis announced the trial achieved its primary endpoint in showing a statistically significant increase in progression-free survival from cabozantinib compared with everolimus in the first 375 patients randomized by an independent radiology review committee. Cabozantinib was found to decrease the disease progression rate or death by 42% compared with everolimus.

The drug is currently marketed in capsule form for the treatment of progressive, metastatic medullary thyroid cancer (MTC) and in Europe for adult patients with progressive, unresectable locally advanced or metastatic MTC.

The FDA is also currently reviewing a distinct tablet formulation of cabozantinib for advanced renal cell carcinoma and other types of cancer.