Lexicon Pharmaceuticals, Inc.,a fully integrated biopharmaceutical company, announced that the US Food and Drug Administration (FDA) has 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 (SSA) therapy in adults inadequately controlled by SSA therapy.

Carcinoid syndrome is a rare and debilitating condition that affects people with metastatic neuroendocrine tumors (mNETs).  Xermelo targets the overproduction of serotonin inside mNET cells , providing a new treatment option for patients suffering from carcinoid syndrome diarrhea. This new treatment is now available by prescription and will be in select specialty pharmacies beginning March 6, 2017.

“Today’s approval of Xermelo represents a shift in the treatment paradigm of carcinoid syndrome diarrhea for cancer patients who are inadequately controlled by SSA therapy, and until now, have had limited options to manage this debilitating condition,” said Lonnel Coats, Lexicon’s president and chief executive officer. “We are proud to have discovered and developed this ground-breaking orphan drug, and it is an honor to make it available for the thousands of patients currently suffering from this condition who wish to lead a more routine life with fewer incidences of severe diarrhea.”

Carcinoid syndrome is a rare condition that occurs in patients living with mNETs  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 H. Kulke, M.D., TELESTAR primary investigator, director of the Program in Neuroendocrine and Carcinoid Tumors at Dana Farber Cancer Institute and Professor of Medicine, Harvard Medical School.  “Inhibition of tumoral serotonin production represents a novel approach for patients with this condition.  Studies have shown that Xermelo can reduce the debilitating effects of carcinoid syndrome diarrhea and has a favorable efficacy and safety profile in patients who currently have limited treatment options.”

Discovered using Lexicon’s unique 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.

Lexicon has built the in-house capability and infrastructure to launch and market Xermelo in the US, where it retains all commercialization rights.  Lexicon also retains rights to market telotristat ethyl in Japan.  Lexicon has established a license and collaboration agreement with Ipsen to commercialize telotristat ethyl in Europe and other countries outside of US and Japan.  

Lexicon is a fully integrated biopharmaceutical company that is applying a unique approach to gene science based on Nobel Prize-winning technology to discover and develop precise medicines for patients with serious, chronic conditions.