Houston—Lexicon Pharmaceuticals announced Tuesday that the FDA has approved its treatment for diarrhea caused by carcinoid syndrome, telotristat ethyl (Xermelo).
Lexicon (NASDAQ: LXRX) says it is the first orally administered therapy to treat carcinoid syndrome diarrhea in combination with somatostatin analog, or SSA, therapies, which target the root cause of the problem: overproduction of hormones.
Carcinoid syndrome is a condition that affects 14,000 people in the U.S. that have metastatic neuroendocrine tumors, or mNETs. When these tumors spread to the liver and other organs in the gastrointestinal tract, it can cause carcinoid syndrome, which is characterized by an overproduction of serotonin, causing symptoms such as diarrhea, facial flushing, abdominal pain, fatigue, and heart valve damage.
Lexicon CEO Lonnel Coats noted that the agency approval comes on “Rare Disease Day,” which is designated as February 28. It is the first drug developed by the company to receive FDA approval.
“There are very few companies that get to take a drug they discovered in their own labs all the way to commercialization,” says Jeff Wade, executive vice president for corporate and administrative affairs, chief financial officer, and a 15-year company veteran.
Xermelo inhibits tryptophan hydroxylase, an amino acid that plays a role in forming serotonin, and can be used to treat carcinoid syndrome diarrhea when symptoms are not adequately controlled by SSA therapy alone. Lexicon says the combination of the two drugs decreases both the production of serotonin within the mNET and the release of serotonin outside the cell, which then reduces the frequency of diarrhea.
The company reported that in a recent Phase 3 clinical trial, 33 percent of patients receiving both Xermelo and somatostatin therapy had at least two fewer bowel movements per day. Only 4 percent of patients who just received the somatostatin therapy had reported the same, Lexicon said.
Xermelo will be available by prescription Monday at a cost of $5,164 for a 28-day supply, Lexicon said.
Lexicon, which was founded in 1995, says its scientists have spent more than a decade analyzing nearly 5,000 genes to find key targets for drug development— especially in diseases for which there have not been improvement to treatment in years, possibly decades. Coats said in a call with investors Tuesday that Xermelo represents the first change to standard of care for carcinoid syndrome patients in three decades.