Emerging treatments
Surufatinib
Surufatinib, a tyrosine kinase inhibitor, was granted fast track designation by the US Food and Drug Administration (FDA) for the treatment of advanced and progressive pancreatic and extrapancreatic neuroendocrine tumours (NET) based on two phase 3 trials carried out in China, and one phase 1 trial carried out in the US. In one China-based phase 3 trial of 198 adult patients with extrapancreatic NETs, progression-free survival was 9.2 months with surufatinib, compared with 3.8 months with placebo.[63] In 2022, the FDA rejected approval due to lack of multiregional clinical data (among other reasons). The manufacturer has since withdrawn its application for a marketing authorisation in Europe. Surufatinib is approved in China.
Cabozantinib
Cabozantinib, a tyrosine kinase inhibitor, significantly improved progression-free survival in a randomised, placebo-controlled phase 3 trial of patients with previously treated, progressive advanced extra-pancreatic or pancreatic neuroendocrine tumours.[64] The National Comprehensive Cancer Network (NCCN) recommends cabozantinib as a treatment option for locoregional advanced disease or distant metastases in patients with well-differentiated pancreatic neuroendocrine tumours.[3]
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