FDA Approves Cabometyx for Advanced Neuroendocrine Tumors

The FDA has approved cabozantinib (Cabometyx) for use in patients aged 12 and older with previously treated, unresectable pancreatic and extra-pancreatic neuroendocrine tumors (pNET and epNET). This decision was based on data from the CABINET trial, which demonstrated significant improvements in progression-free survival (PFS) with cabozantinib compared to placebo, although overall survival data remain immature and complicated by patient crossovers. The pNET cohort showed a median PFS of 13.8 months versus 3.3 months in the placebo group, while the epNET cohort had a median PFS of 8.5 months compared to 4.2 months for placebo. Despite the promising PFS results, the trial noted a higher risk of mortality in patients treated with cabozantinib, raising concerns about its safety profile. The approval is seen as a potential breakthrough for patients facing limited treatment options in this challenging cancer type. Cabozantinib works by targeting multiple pathways related to tumor growth and has a side effect profile similar to other therapies in its class.