Lexicon receives FDA Orphan Drug designation for telotristat etiprate to treat carcinoid syndrome

Mar 21 2012

Lexicon Pharmaceuticals, Inc. (Nasdaq: LXRX), announced today that the U.S. Food and Drug Administration (FDA) has granted its request for Orphan Drug designation for telotristat etiprate (LX1032) for the treatment of carcinoid syndrome.  Carcinoid syndrome is a chronic condition caused by neuroendocrine tumors that usually originate from the gastrointestinal tract and is characterized by severe diarrhea and flushing episodes with long-term consequences that may include cardiac valve disease.  Symptoms of carcinoid syndrome have been linked to excess production of serotonin by metastatic tumor cells.  Telotristat etiprate is an orally-delivered serotonin synthesis inhibitor (SSI) drug that acts to reduce serotonin levels.

"The Orphan Drug designation of telotristat etiprate by the FDA provides important benefits for this drug's clinical development for carcinoid syndrome," commented Dr. Pablo Lapuerta, senior vice president of clinical development and chief medical officer at Lexicon. "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."

In the U.S., Orphan Drug designation is generally granted for drugs intended for the treatment of diseases that affect fewer than 200,000 people and provides various incentives and preferences, such as seven years marketing exclusivity from the date of approval and certain tax advantages.  The marketing of drugs which have received Orphan Drug designation remains subject to the receipt of marketing approval from the FDA.  Lexicon has previously received Orphan Drug designation from the European Medicines Agency and Fast Track status from the FDA for telotristat etiprate. 

SOURCE Lexicon Pharmaceuticals, Inc.