Exelixis Advances CABOMETYX® for Neuroendocrine Tumors Ahead of FDA Review

Exelixis Advances Cabozantinib for Neuroendocrine Tumors with Upcoming FDA Review

Exelixis, Inc. is set to make significant strides in oncology with its supplemental New Drug Application (sNDA) for cabozantinib, marketed as CABOMETYX®, which is scheduled for discussion by the U.S. Food and Drug Administration’s Oncologic Drugs Advisory Committee (ODAC) in March 2025. This application focuses on the treatment of adults with previously treated advanced pancreatic neuroendocrine tumors (pNET) and advanced extra-pancreatic neuroendocrine tumors (epNET). The sNDA is supported by the final results of the phase 3 CABINET trial, a pivotal study conducted by the National Cancer Institute's National Clinical Trials Network. The upcoming ODAC meeting underscores the growing importance of cabozantinib in managing these complex tumor types, which have historically posed treatment challenges.

The CABINET trial, a multicenter, randomized, double-blinded, placebo-controlled study, aims to evaluate the effectiveness of cabozantinib in patients with advanced neuroendocrine tumors after prior therapies. Notably, the trial's independent Data and Safety Monitoring Board recommended halting patient enrollment last August due to significant improvements in progression-free survival (PFS) associated with cabozantinib compared to placebo. This recommendation led to the unblinding of patients, allowing those in the placebo group to switch to cabozantinib. The final results of the trial, which were presented at the 2024 European Society of Medical Oncology Congress and subsequently published in the New England Journal of Medicine, affirm the drug's efficacy across a range of clinical subgroups, further strengthening Exelixis' position in the oncology landscape.

In addition to the sNDA submission, cabozantinib has also received orphan drug designation from the FDA for pNET as of August 2024, with a Prescription Drug User Fee Act target action date of April 3, 2025. This designation highlights the unmet medical need in treating rare diseases, potentially expediting the development and review processes. Exelixis' collaboration with the National Cancer Institute exemplifies its commitment to advancing cancer research, reinforcing the company’s role as a key player in developing therapies for challenging tumor types.

As Exelixis prepares for the upcoming ODAC review, the emphasis on cabozantinib reflects a broader trend in oncology where targeted therapies are becoming increasingly vital in the treatment of rare and complex cancers. The results from the CABINET trial not only provide hope for patients with advanced neuroendocrine tumors but also signal a promising avenue for further innovation in the field of oncology.