WASHINGTON - The FDA approved telotristat ethyl (Xermelo) on Tuesday for treatment of carcinoid syndrome diarrhea, the agency announced.
The drug is a tryptophan hydroxylase inhibitor and is to be taken in combination with a somatostatin analogue. The approved indication is carcinoid syndrome diarrhea that is not adequately controlled by a somatostatin analogue alone.
Carcinoid syndrome is a group of symptoms seen in some patients (less than 10%) with carcinoid tumors. These tumors overproduce serotonin. By inhibiting an enzyme that degrades tryptophan, the drug helps decrease serotonin release from tumors.
Telotristat ethyl is an oral drug to be taken three times a day with food.
Efficacy and safety data came mainly from a 90-patient trial that compared telotristat with placebo. Participants were experiencing 4-10 diarrhea episodes daily despite use of somatostatin analogues. One-third of patients taking telotristat showed reductions of at least 2 bowel movements per day, an endpoint reached by only 4% of the placebo group.
The drug was associated with a range of side effects during clinical studies, including nausea, liver enzyme elevations, depression, peripheral edema, flatulence, decreased appetite, and fever. Patients taking too much of the drug developed severe constipation. One patient was hospitalized and two others experienced complications of intestinal obstruction or perforation.
As a result, the FDA recommends monitoring patients for severe constipation or significant abdominal pain.
Telotristat is manufactured by Lexicon Pharmaceuticals.
This article was published first by MedPageToday Xermelo OK'd for Cancer-Associated Diarrhea, a trusted and reliable source for clinical and policy coverage, and free Continuing Medical Education (CME), that directly affects the lives and practices of health care professionals.