The US Food and Drug Administration (FDA) today approved the oral therapy Xermelo (telotristat ethyl) to be used in combination with somatostatin analog (SSA) therapy for the treatment of adults with carcinoid syndrome diarrhea.
Carcinoid syndrome is a cluster of symptoms that occurs in patients with advanced neuroendocrine tumors (NET) and the related diarrhea results from the overproduction of serotonin by these tumors. In addition to discomfort and inconvenience, uncontrolled diarrhea may lead to unwanted weight loss, malnutrition, dehydration, and electrolyte imbalance.
The syndrome is also characterized by facial flushing, abdominal pain, and fatigue. Xermelo is the first and only approved oral therapy for carcinoid syndrome diarrhea that targets the overproduction of serotonin by NET cells, according to a company statement.
The effectiveness of Xermelo was established in a comparative clinical trial in 90 adult participants with well-differentiated NET and carcinoid syndrome diarrhea. These patients experienced between four and 12 daily bowel movements despite the use of a SSA. In the trial, all patients remained on their SSA treatment, and half received Xermelo. Overall 33% of patients in the Xermelo -plus-SSA group had an average reduction of two bowel movements per day compared with only 4% of the non- Xermelo treated patients.
“Today’s approval will provide patients whose carcinoid syndrome diarrhea is not adequately controlled with another treatment option,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research.