Exelixis Seeks Approval For CABOMETYX® In Advanced Neuroendocrine Tumors

Exelixis, Inc. has revealed that the U.S. Food and Drug Administration (FDA) will be reviewing the company’s supplemental New Drug Application (sNDA) for cabozantinib (CABOMETYX®) as a potential treatment for adults with previously treated advanced pancreatic neuroendocrine tumors (pNET) and advanced extra-pancreatic neuroendocrine tumors (epNET). This review will take place during an Oncologic Drugs Advisory Committee (ODAC) meeting scheduled for March 2025. 

The sNDA is supported by the final findings from the phase 3 CABINET pivotal trial, which was led by the National Cancer Institute’s National Clinical Trials Network and compared cabozantinib to a placebo in patients with advanced pNET and epNET. In August 2024, Exelixis also announced that the FDA had granted orphan drug designation for cabozantinib for pNET and set a Prescription Drug User Fee Act action date for April 3, 2025.

In August 2023, the independent Data and Safety Monitoring Board of the Alliance for Clinical Trials in Oncology recommended halting enrollment in the CABINET trial, and unblinding the randomized patients. This decision was based on a substantial improvement in progression-free survival (PFS) observed during an interim analysis, which also allowed for patients on placebo to crossover to cabozantinib. 

The final data from the enrolled patient population, presented at the 2024 European Society of Medical Oncology Congress and published simultaneously in the *New England Journal of Medicine*, confirmed that cabozantinib demonstrated statistically significant and clinically meaningful improvements over placebo in the primary endpoint of PFS, as determined by blinded independent central review. Additional analyses further confirmed that the benefits of cabozantinib were consistent across various clinical subgroups, including primary tumor site, grade, and prior systemic anticancer therapy.

The Oncologic Drugs Advisory Committee (ODAC) is set to review and assess data on the safety and efficacy of both marketed and investigational drug products intended for cancer treatment, offering recommendations to the Commissioner of Food and Drugs. For more details on ODAC reviews, additional information is available on the FDA’s website. It’s important to note that this upcoming ODAC meeting will not be related to the current approved uses of CABOMETYX in the U.S.