The Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to cabozantinib for the treatment of patients with previously treated radioactive iodine-refractory differentiated thyroid cancer.

The designation is supported by data from the randomized, double-blind, placebo-controlled phase 3 COSMIC-311 study (ClinicalTrials.gov: NCT03690388), which compared the efficacy and safety of cabozantinib to placebo in patients with radioiodine-refractory differentiated thyroid cancer who had progressed after up to 2 prior vascular endothelial growth factor receptor-targeted therapies. Patients were randomly assigned 2:1 to receive either cabozantinib 60mg orally once daily or placebo.

Interim analysis showed that treatment with cabozantinib significantly reduced the risk of disease progression or death by 78% compared with placebo (hazard ratio 0.22; 95% CI, 0.13-0.36; P <.0001). The safety profile of cabozantinib was found to be consistent with that seen in previous studies.


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“We look forward to submitting our regulatory application in 2021 and to working closely with the FDA during the review process, with the goal of bringing cabozantinib to this patient population with a high unmet medical need for whom there is currently no available standard of care,” said Gisela Schwab, MD, President, Product Development and Medical Affairs and Chief Medical Officer, Exelixis.

Cabozantinib, a kinase inhibitor, is marketed under the trade name Cabometyx and is currently indicated for advanced renal cell carcinoma and hepatocellular carcinoma.

References

  1. Exelixis announces Breakthrough Therapy designation granted to cabozantinib for the treatment of patients with previously treated radioactive iodine-refractory differentiated thyroid cancer. [press release]. Alameda, CA: Exelixis, Inc.; February 25, 2021. 
  2. Exelixis announces cabozantinib significantly improved progression-free survival in COSMIC-311 phase 3 pivotal trial in patients with previously treated radioiodine-refractory differentiated thyroid cancer. [press release]. Alameda, CA: Exelixis, Inc.; December 21, 2021. 

This article originally appeared on MPR