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Orphan Drug Status Conveyed on Telotristat Etiprate, Treatment for Carcinoid Syndrome
The drug, LX1032 or telotristat etiprate, is designed to treat carcinoid syndrome -- a group of symptoms associated with tumors that usually originate from the gastrointestinal tract.
Telotristat etiprate was discovered and developed at Lexicon to reduce serotonin production by inhibiting tryptophan hydroxylase (TPH), a key enzyme in the synthesis of serotonin. Excessive levels of serotonin have been implicated in symptoms associated with carcinoid syndrome, especially diarrhea and carcinoid heart disease. In preclinical studies, telotristat etiprate reduced peripheral serotonin in several different species without affecting serotonin levels in the brain. In clinical studies, telotristat etiprate reduced serotonin levels consistent with preclinical results and reduced bowel movement frequency in patients with carcinoid syndrome. Telotristat etiprate is being developed under Fast Track and Orphan Drug designation from the U.S. Food and Drug Administration and Orphan Drug designation from the European Medicines Agency. Telotristat etiprate is a member of a new class of oral drugs invented by Lexicon, the serotonin synthesis inhibitors, which are being developed in a spectrum of gastrointestinal indications. Lexicon is also currently planning a Phase 2 trial of telotristat etiprate in mild to moderate ulcerative colitis.
Read the full news story on Reuters.