The FDA has approved cabozantinib (Cabometyx) for adults and adolescents (aged 12+) with advanced pancreatic (pNET) or extra-pancreatic neuroendocrine tumors (epNET) that have progressed after prior treatments. This oral targeted therapy slows cancer growth and is now available for patients with unresectable or metastatic disease.
Key Benefits for Patients
- Delays Cancer Progression:
- For pNET patients, cabozantinib tripled progression-free survival (PFS) to 13.8 months vs. 3.3 months on placebo.
- For epNET patients, PFS doubled to 8.5 months vs. 4.2 months.
- Response Rates: 18% of pNET patients saw tumor shrinkage with cabozantinib (vs. 0% on placebo).
Who Can Benefit?
- Patients with well-differentiated, advanced pNET or epNET that worsened after prior therapies (e.g., chemotherapy, PRRT).
- Adolescents (≥12 years) are eligible, with dosing adjusted for body weight.
Safety and Side Effects
- Common Side Effects: Fatigue, diarrhea, hypertension, and rash—mostly mild to moderate.
- No New Safety Risks: The safety profile aligns with cabozantinib’s use in other cancers (e.g., kidney, liver).
Why This Matters
Neuroendocrine tumors are rare and often resistant to standard treatments. “This approval fills a critical gap for patients who’ve run out of options,” said Dr. Jennifer Chan of Dana-Farber Cancer Institute. While overall survival data are still pending, delaying progression is a meaningful win for quality of life.
Next Steps for Patients
- Discuss cabozantinib with your oncologist if your NET has progressed.
- Pediatric dosing (40 mg/day) is available for adolescents under 40 kg.
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