FDA grants breakthrough drug designation to cabozantinib for advanced renal cell carcinoma

Issue: October 10, 2015

The FDA granted breakthrough drug designation to cabozantinib for the treatment of patients with renal cell carcinoma who received one prior therapy, according to a press release from the drug’s manufacturer.

The FDA based its decision in part on results of the phase 3 pivotal METEOR trial, which compared cabozantinib (Cometriq, Exelixis) to everolimus (Afinitor, Novartis) in patients with renal cell carcinoma who experienced disease progression after treatment with a VEGF receptor tyrosine kinase inhibitor.

Exelixis announced top-line results for METEOR in July. Cabozantinib significantly prolonged PFS compared with everolimus in the first 375 randomly assigned patients, thus meeting the trial’s primary endpoint.

Cabozantinib demonstrated a 42% reduction in the rate of disease progression or death compared with everolimus (HR = 0.58, 95% CI, 0.45-0.75).

Cabozantinib is currently approved in capsule form in the U.S. for the treatment of patients with progressive, metastatic medullary thyroid cancer. A distinct tablet formulation of cabozantinib is being evaluated for the treatment of patients with advanced renal cell carcinoma and other types of cancer.

“Receiving Breakthrough Therapy Designation is an important regulatory achievement for cabozantinib in renal cell carcinoma,” Michael M. Morrissey, PhD, president and CEO of Exelixis, said in a press release. “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 [renal cell carcinoma] 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.”