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The U.S. Food & Drug Administration has granted approval for a new drug to treat pancreatic neuroendocrine tumors, also called PNET or PanNET.
“I am delighted that patients with pancreatic neuroendocrine tumors now have another treatment option,” said PanCAN Chief Scientific and Medical Officer Anna Berkenblit, MD, MMSc. “While PNETs tend to grow more slowly than the most common type of pancreatic cancer, called pancreatic ductal adenocarcinoma, the five-year relative survival rate for PNETs that have spread is 23%, highlighting the need for new treatment options.”
Here, PanCAN answers questions related to this new treatment option.
What is CABOMETYX® (cabozantinib)?
CABOMETYX® is a targeted therapy. It has been granted approval by the FDA for a specific subset of patients with pancreatic cancer who are diagnosed with a PNET. PNETs originate from the endocrine (hormone-producing) cells of the pancreas. PNETs are less common than pancreatic ductal adenocarcinoma (PDAC) and are very different biologically and clinically and require specialized care.
This newest FDA approval also makes CABOMETYX available to patients diagnosed with an extra-pancreatic neuroendocrine tumor, also known as epNETs. This type of tumor originates in cells outside of the pancreas, most often in the gastrointestinal tract or in the lungs.
CABOMETYX was previously approved to treat several other types of cancer, including advanced kidney cancer, liver cancer and differentiated thyroid cancer.
What does this FDA approval mean?
CABOMETYX is approved for adults with a PNET who have received previous treatment, who are ineligible for surgery and who have “well-differentiated pancreatic neuroendocrine tumors.” This means that the tumor cells look more like healthy cells and are growing more slowly than poorly differentiated tumor cells.
What is the benefit for patients diagnosed with a PNET?
A Phase III clinical trial tested the benefit of CABOMETYX. Ninety-nine patients diagnosed with PNET whose cancer progressed following prior therapy were included in the study. In the 66 patients with PNET who received CABOMETYX, the median progression-free survival, or the time it took for their cancer to get worse, was 13.8 months. In the 33 patients with PNET in the control group who did not receive CABOMETYX, their median progression-free survival was 3.3 months. The overall response rate, or the percentage of PNET patients in the study whose tumors responded to CABOMETYX, was 18%. These findings led to the study stopping early. Those in the control group were given the option to begin taking CABOMETYX.
What are the side effects of CABOMETYX?
The most common side effects reported by the oncology company Exelixis include the following: tiredness, decreased appetite, nausea and vomiting, weight loss and constipation. Over the course of the study, 49% of patients had to reduce their dose due to side effects and 19% of patients stopped taking CABOMETYX also because of side effects. High blood pressure is reported as common and sometimes severe, as is diarrhea and a skin problem called hand-foot skin reaction. For a full list of side effects, see the CABOMETYX website.
I am a patient interested in CABOMETYX. What should I do?
People with a PNET diagnosis should talk to their healthcare team about this treatment option. Contact PanCAN Patient Services for additional information. Our expert Case Managers can provide PNET patients and their caregivers with accurate, up-to-date information about neuroendocrine tumors of the pancreas. Resources available to PNET patients include personalized clinical trials searches, a list of PNET specialists across the country and a free booklet with in-depth information specific to PNET diagnosis and treatment.
Does insurance cover this treatment?
FDA approval means this drug combination is safe and effective, and although the FDA does not decide what is covered by insurance, when a drug gets FDA approval, Medicare and Medicaid will usually cover it. Coverage for cancer drugs will vary based on the specific plan and insurance company a person uses.
Contact PanCAN Patient Services for more information on financial assistance programs for those experiencing or anticipating cost-related barriers to care.
Why is this news important?
PNET is a rare form of cancer with limited treatment options. Less than 10% of the 67,440 pancreatic cancer diagnoses expected in 2025 will be PNETs. Surgery may be an option for cases diagnosed early enough. In addition to targeted therapy, other options include radiation therapy, liver directed therapy, hormone therapy, chemotherapy and clinical trials. They may be used alone or together.
This is the first new treatment approved for PNETs since 2018. Every new drug approval means additional options for patients. We need continued investment in research and advocacy for federal funding to continue this progress.
