FDA Accepts Supplemental New Drug Application for Cabometyx in Pancreatic NETs

The FDA accepted a supplemental new drug application for Cabometyx to treat select patients with pancreatic neuroendocrine tumors.

A supplemental new drug application has been accepted by the Food and Drug Administration (FDA) for Cabometyx (cabozantinib) for the treatment of patients with pretreated, locally advanced/unresectable or metastatic, well- or moderately differentiated pancreatic neuroendocrine tumors (NETs).

According to a press release from Exelixis, the manufacturer of Cabometyx, the new drug application also pertains to the potential treatment of patients with extra-pancreatic NETs. In particular, extra-pancreatic NETs are tumors that originate outside the pancreas in areas such as the lung, as noted in a news release.

“The FDA’s acceptance of this application marks another important milestone in our commitment to bringing [Cabometyx] to patients living with difficult-to-treat cancers and who have limited treatment options,” Dr. Amy Peterson, executive vice president of product development and medical affairs, and chief medical officer of Exelixis, said in the release. “We appreciate the opportunity to work with the FDA in the coming months as they review our application, with the goal to bring this new, effective treatment option to patients with advanced [NETs] as quickly as possible.”

With a supplemental new drug application, a pharmaceutical company seeks to make changes to drug labels after they have been approved for other indications, for example, according to the FDA’s website. Cabometyx has been previously approved by the FDA for other diseases such as thyroid cancer and renal cell carcinoma, a type of kidney cancer.

This supplemental new drug application is based on findings from the phase 3 CABINET trial, which compared Cabometyx with placebo in patients with advanced pancreatic NETs and those with extra-pancreatic NETs. The release noted that the CABINET trial was stopped early due to a dramatic improvement in progression-free survival (the time during and after treatment when a patient with cancer lives with the disease without worsening) in both patient groups.

In the CABINET trial, 290 patients with pancreatic or extra-pancreatic NETs were enrolled and randomly assigned to receive Cabometyx or placebo. Patients in this trial experienced either disease progression or intolerance after at least one prior therapy other than somatostatin analogs (designed to control hormone levels and shrink tumors). Of note, the group of patients with extra-pancreatic NETs included primary tumor sites such as the lungs and gastrointestinal tract, among others.

Researchers focused on certain outcomes throughout the trial including progression-free survival, overall survival (the time when a patient with cancer is still alive), radiographic response rate (a way to measure how a patient’s disease reacts to therapy) and safety.

The FDA has assigned the supplemental new drug application for pancreatic NETs a target action date of April 3, 2025. With this date, the FDA has to decide whether to approve or request more information by that time.

According to the release, NETs are cancers that typically originate in specific cells of the neuroendocrine system. An estimated 161,000 to 192,000 patients are currently living with unresectable (unable to be surgically removed), locally advanced (cancer that spreads to nearby tissue or lymph nodes) or metastatic (cancer that spreads from the primary site to other areas of the body) NETs.

Although NETs can develop in any part of the body, they most commonly originate in the gastrointestinal tract or the lungs (extra-pancreatic NETs). NETs can also originate in the pancreas, which can often be a more aggressive disease, according to the release. Treatments for advanced NETs often include chemotherapy, somatostatin analogs, peptide-receptor radionuclide therapy and targeted therapy.

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