FDA Grants Orphan Drug Designation for Treatment of Pancreatic Neuroendocrine Tumors
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FDA Grants Orphan Drug Designation for Treatment of Pancreatic Neuroendocrine Tumors


Surufatinib was recently granted Orphan Drug designation by the FDA. This drug was made for the treatment of pancreatic neuroendocrine tumors (PanNETs). It was created by Hutchison China MediTech Limited (Chi-Med), and the CEO of the company has released statements that stress the importance of this treatment. He says that it addresses a “serious unmet medical need,” as there are few treatments currently available for PanNETs. The company is excited about the new designation and hopes to further develop surufatinib so that it is available for more people.

About Pancreatic Neuroendocrine Tumors (PanNETs)

Tumors are masses of abnormal cells that have grown out of control and can be malignant or benign. When genes meant to regulate normal cell growth mutate or are damaged, the result is a tumor, as the cells can grow at an unregulated or accelerated pace. Pancreatic neuroendocrine tumors (PanNETs) are tumors that appear in the pancreas due to cells releasing hormones into the bloodstream in response to signals from the nervous system. The pancreas is responsible for hormones and enzymes that aid in digestion, so tumors in this organ affect these functions.

Pancreatic neuroendocrine tumors are also referred to as islet cell tumors. Islet cells make hormones in the pancreas, and the tumors composed of them are different than those of pancreatic cancer, which are usually adenocarcinomas. Most islet cell tumors are benign, but others are malignant and cause symptoms. These symptoms include back and abdominal pain, lumps in the abdomen, indigestion, diarrhea, and yellowing of the skin and eyes. There are two types of these islet cell tumors, functional and nonfunctional. Functional tumors are in endocrine tissue that produce hormones while nonfunctional tumors are in endocrine tissue that does not produce hormones. The majority of islet cell tumors are functional and create hormones like gastrin, insulin, glucagon, vasoactive intestinal peptides, and somatostatin. Depending on the type of hormone that the tumor makes, there can be additional symptoms. They include recurring stomach ulcers, low blood sugar, fast heartbeat, rashes, sore mouth, high blood sugar, blood clots, altered bowel habits, unintended weight loss, dehydration, and gallstones. Risk factors for islet cell tumors are multiple endocrine neoplasia type 1 and neurofibromatosis type 1.

There is a large variety of methods for the diagnosis of pancreatic NETs. Some of these methods are CT scans, MRIs, tumor marker tests, colonoscopies, or biopsies. For islet cell tumors that produce specific hormones, there are tests used to diagnosis the type of islet cell tumor. Doctors use the information obtained during diagnosis to assign a stage to the cancer, which then helps them develop a treatment plan. Treatment depends on the location, size, how far the tumor has spread, patient age and health, and whether the cancer is newly diagnosed or is a recurrence. Treatments include chemotherapy, radiation, surgery, hormone therapy, targeted therapy, and supportive care. Treatments that are specific to islet cell tumors are hepatic arterial occlusion and chemoembolization.

About Surufatinib

Surufatinib is the second novel drug developed by Chi-Med. It inhibits tyrosine kinase activity, which is associated the vascular endothelial growth factor receptors and fibroblast growth factor receptors. In June of 2019 Chi-Med’s study of Surufatinib met the requirements for progression-free survival and they then started the process for the pre-New Drug Application.

About the Study

Clinical Cancer Research posted the results of the study in March of 2019. The study was randomized, with participants taking either 300 mg of the drug or a placebo, both on a 28 day cycle. In order to enroll in the study, participants were required to have radiological documentation of disease progression along with certain counts of platelets, neutrophil, hemoglobin, and serum total bilirubin. The study revealed that surufatinib led to antitumor activity in participants.The overall response rate of patients with pancreatic NETs was 15%, and the disease control rate was 91%. The median progression-free survival was 21.2 months. Along with these statistics, surufatinib was found to have manageable toxicity.

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