FDA OKs Cabozantinib for Pancreatic Neuroendocrine Tumors

The US Food and Drug Administration (FDA) has approved cabozantinib (Cabometyx, Exelixis, Inc.) for certain adults and children aged ≥ 12 years with previously treated, well-differentiated pancreatic neuroendocrine tumors (pNET) and extrapancreatic NET (epNET).

Specifically, the approval is for those with unresectable, locally advanced, or metastatic pNET and epNET, according to the FDA approval notice. 

The approval was based on progression-free survival (PFS) and overall response rates (ORRs) in the pivotal, placebo-controlled CABINET trial, the FDA said. 

Median PFS among 99 patients with pNET who were randomized 2:1 to receive cabozantinib or placebo was 13.8 months vs 3.3 months, respectively (hazard ratio [HR], 0.22). The ORRs were 18% and 0%, respectively.

Median PFS among 199 patients with epNET who were randomized 2:1 to receive cabozantinib or placebo was 8.5 vs 4.2 months, respectively (HR, 0.40), and the ORR was 5% and 0%, respectively.

Overall survival (OS) data were not mature, but 52% and 37% of placebo arm patients in the pNET and epNET arms crossed over to open label cabozantinib, which may impact the evaluation of OS, according to the FDA notice.

Study participants received either 60 mg oral cabozantinib once daily or placebo until disease progression or unacceptable toxicity. 

The safety profile for cabozantinib, which is currently approved for multiple other indications, was consistent with the approved product label. No new safety signals were observed.

According to the full prescribing information, the recommended doses are 60 mg once daily until disease progression or unacceptable toxicity for adults and children aged ≥ 12 years who weigh at least 40 kg and 40 mg once daily until disease progression or unacceptable toxicity for pediatric patients aged ≥ 12 years who weigh less than 40 kg.

“The characteristics of NET vary widely from patient to patient, and very few treatment options have demonstrated the ability to improve outcomes across such a heterogeneous population,” study chair Jennifer Chan, MD, of Dana-Farber Cancer Institute, Boston, stated in a press release from Exelixis. “It was encouraging to see that cabozantinib resulted in significant delays in disease progression…regardless of primary tumor site and grade. 

Sharon Worcester, MA, is an award-winning medical journalist based in Birmingham, Alabama, writing for Medscape Medical News, MDedge, and other affiliate sites. She currently covers oncology, but she has also written on a variety of other medical specialties and healthcare topics. She can be reached at [email protected] or on X @SW_MedReporter.