The US Food and Drug Administration (FDA) has approved Lexicon Pharmaceuticals' Xermelo (telotristat ethyl) tablets in combination with somatostatin (SSA) therapy to treat adults with carcinoid syndrome diarrhea.
Carcinoid syndrome is a cluster of symptoms sometimes seen in people with carcinoid tumors. Carcinoid syndrome occurs in less than 10% of patients with carcinoid tumors, usually after the tumor has spread to the liver.
These tumors are rare, and often slow-growing. Most carcinoid tumors are found in the gastrointestinal tract.
The tumors in patients with carcinoid syndrome diarrhea release more amounts of the hormone serotonin, resulting in diarrhea.
FDA Center for Drug Evaluation and Research Office of Drug Evaluation III director Julie Beitz said: “Today’s approval will provide patients whose carcinoid syndrome diarrhea is not adequately controlled with another treatment option.”
"Today’s approval will provide patients whose carcinoid syndrome diarrhea is not adequately controlled with another treatment option."
Approved in tablet form, Xermelo inhibits the production of serotonin by carcinoid tumors and reduces the frequency of carcinoid syndrome diarrhea.
The tablet's safety and efficacy were established in a double-blind, placebo-controlled trial carried out in 90 adult participants with metastatic neuroendocrine tumors and carcinoid syndrome diarrhea for 12 weeks.
The most common side effects of Xermelo include nausea, headache, high levels of the liver enzyme gamma-glutamyl transferase, depression, accumulation of fluid-causing swelling, flatulence, decreased appetite, and fever.
The tablet may cause constipation, and the risk of developing this may be high in patients whose bowel movement frequency is less than four bowel movements a day.
Xermelo also received orphan drug designation, which provides incentives to assist the development of drugs for rare diseases.