The FDA on Feb. 28 approved telotristat ethyl (Xermelo) tablets, in combination with somatostatin analog (SSA) therapy, to treat adults with carcinoid syndrome diarrhea that is inadequately controlled with SSA therapy alone. The drug, which reduces the frequency of diarrhea by inhibiting the production of serotonin by carcinoid tumors, is approved to be taken orally three times daily with food, according to a press announcement. The drug received designation as an orphan drug and was granted fast-track designation and priority review.
In a 12-week randomized controlled trial of 90 adults with well-differentiated metastatic neuroendocrine tumors and carcinoid syndrome diarrhea, 33% of those randomized to add the drug to their SSA regimen had an average reduction of two bowel movements per day, compared to 4% of those randomized to placebo. The most common side effects include nausea, headache, increased levels of gamma-glutamyl transferase, depression, peripheral edema, flatulence, decreased appetite, and fever. Risk of constipation may be increased in patients who have less than four bowel movements per day, and clinicians should monitor patients for severe constipation.