Lexicon Pharmaceuticals Inc. said FDA approved Xermelo (telotristat ethyl) 250 mg as a first and only orally administered therapy for the treatment of carcinoid syndrome diarrhea in combination with somatostatin analog therapy in adults inadequately controlled by SSA therapyi.
Carcinoid syndrome is a rare and debilitating condition that affects people with metastatic neuroendocrine tumors (mNETs)ii.
Xermelo targets the overproduction of serotonin inside mNET cellsiii, providing a new treatment option for patients suffering from carcinoid syndrome diarrhea.
Carcinoid syndrome is a rare condition that occurs in patients living with mNETsiv and is characterized by frequent and debilitating diarrhea that often prevents patients from leading active, predictable lives, as well as by facial flushing, abdominal pain, fatigue and, over time, heart valve damage.
“The approval of XERMELO establishes a new treatment option for patients with carcinoid syndrome diarrhea that is inadequately controlled by SSA therapy,” said Matthew Kulke, primary investigator of the company trial and director of the Program in Neuroendocrine and Carcinoid Tumors at Dana Farber Cancer Institute. “Inhibition of tumoral serotonin production represents a novel approach for patients with this condition.”
Discovered using Lexicon’s approach to gene science, Xermelo is the first and only approved oral therapy for carcinoid syndrome diarrhea. Xermelo targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within mNET cells.
According to the label, a 12-week double-blind, placebo-controlled, randomized, multicenter trial of Xermelo was conducted in adult patients with a well-differentiated metastatic neuroendocrine tumor and carcinoid syndrome diarrhea who were having between four to 12 daily bowel movements despite the use of SSA therapy at a stable dose for at least three months.
Patients were randomized to placebo or treatment with Xermelo 250 mg three times daily. A total of 90 patients were evaluated for efficacy. The primary efficacy endpoint was the change from baseline in the number of daily bowel movements averaged over the 12-week treatment period.
In the 12-week study, a difference in average weekly reductions in bowel movement frequency between Xermelo and placebo was observed as early as one to three weeks, and persisted for the remaining nine weeks of the study.
