Lexicon Pharmaceuticals, Inc. has been granted Orphan Drug Designation for telotristat etiprate (LX1032) by the U.S. Food and Drug Administration (FDA). LX1032 is used to treat carcinoid syndrome, a chronic condition caused by neuroendocrine tumors, characterized by severe diarrhea and flushing. One of the long-term consequences of carcinoid syndrome is carcinoid heart disease. For patients with carcinoid syndrome, the concentration of blood serotonin correlates with the severity of the symptoms of the syndrome — flushing, diarrhea, and wheezing – and with the potential damage to the heart. When carcinoid has metastasized to the liver, the tumors there may produce a high concentration of serotonin which flows directly into the right side of the heart. Telotristat etiprate is an orally-delivered serotonin synthesis inhibitor (SSI) drug that acts to reduce serotonin levels.
According to the FDA, the Orphan Drug designation program “provides orphan status to drugs and biologics which are defined as those intended for the safe and effective treatment, diagnosis or prevention of rare diseases/disorders that affect fewer than 200,000 people in the U.S., or that affect more than 200,000 persons but not are not expected to recover the costs of developing and marketing a treatment drug.” The program also provides various incentives and preferences, such as seven years’ marketing exclusivity from the date of approval and certain tax advantages.
Dr. Pablo Lapuerta, senior vice president of clinical development and chief medical officer at Lexicon, commented in a press release issued by Lexicon, “The Orphan Drug designation of telotristat etiprate by the FDA provides important benefits for this drug’s clinical development for carcinoid syndrome. We reported positive Phase 2 results in August 2011 in patients with carcinoid syndrome and are proceeding with preparations for a global Phase 3 development plan in this indication.”
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. In clinical studies, LX1032 reduced bowel movement frequency in patients with carcinoid syndrome. Telotristat etiprate is one of a new class of oral drugs invented by Lexicon, the serotonin synthesis inhibitors, which are being developed for various gastrointestinal conditions.
Lexicon, a biopharmaceutical company founded in 1995, is dedicated to discovering and developing breakthrough treatments for human disease based on understanding how genes function.