Exelixis announced that the FDA will discuss its supplemental New Drug Application for cabozantinib, intended to treat advanced pancreatic and extra-pancreatic neuroendocrine tumors (pNET and epNET), at an Oncologic Drugs Advisory Committee meeting in March 2025. This sNDA is based on the positive results of the Phase 3 CABINET trial, which showed significant improvement in progression-free survival for patients treated with cabozantinib compared to placebo. The FDA granted orphan drug designation to cabozantinib for pNET in August 2024 and set a target action date of April 3, 2025.
This news is significant for Exelixis, as a positive ODAC recommendation and subsequent FDA approval would expand the addressable market for cabozantinib, potentially driving revenue growth. It’s also important for patients with advanced pNET and epNET, who often face limited treatment options after disease progression. Current treatments may not effectively control the disease, and the five-year survival rates for advanced GI and lung NET tumors are 68% and 55% respectively, while the five-year survival rate for advanced pancreatic NET is only 23%. A new therapy offering improved progression-free survival would significantly advance the treatment landscape for these challenging cancers.
The CABINET trial demonstrated clinically meaningful improvements in progression-free survival with cabozantinib across all clinical subgroups, including primary tumor site, grade, and prior systemic anticancer therapy. The FDA’s decision to convene an ODAC meeting indicates that they are carefully considering the data, and the meeting outcome will be a critical step in the regulatory process. The planned ODAC meeting does not pertain to the currently approved indications for CABOMETYX in the U.S. The trial included 298 patients randomized to receive either cabozantinib or placebo. The primary endpoint was progression-free survival, and secondary endpoints included overall survival, radiographic response rate, and safety.
The upcoming ODAC meeting represents a pivotal moment for Exelixis and for the future of pNET and epNET treatment. A positive outcome could lead to FDA approval and make cabozantinib available to patients with these difficult-to-treat cancers, offering a new option with demonstrated efficacy in improving progression-free survival. While current treatments such as somatostatin analogs, chemotherapy, targeted therapy, and peptide-receptor radionuclide therapy exist, there remains a need for more effective options. The potential approval of cabozantinib could significantly alter the treatment paradigm and offer new hope for patients battling these rare and often aggressive tumors.
Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.
