Lexicon Pharmaceuticals has won FDA approval for its carcinoid syndrome diarrhea treatment Xermelo™ (telotristat ethyl) in combination with somatostatin analog (SSA) therapy in adults inadequately controlled by SSA therapy alone.

Xermelo is the first oral therapy to be approved for diarrhea tied to carcinoid syndrome, a rare and debilitating condition affecting people with metastatic neuroendocrine tumors (mNETs).

Xermelo, formerly LX1032, is designed to stop the overproduction of serotonin inside mNET cells by targeting tryptophan hydroxylase, an enzyme that mediates excess serotonin production within those cells. The drug will be offered as 250-mg tablets to be taken three times daily with food.

Lexicon said yesterday Xermelo will be in select specialty pharmacies beginning Monday in the U.S., where the company holds all commercial rights. Ipsen holds commercialization rights to the drug in Europe and other countries outside the U.S. and Japan through a license and collaboration agreement with Lexicon, which developed the drug and steered its development through clinical trials and regulatory review.

Xermelo’s safety and efficacy were established in a Phase III trial in which 33% of participants randomized to add Xermelo with SSA saw an average reduction of two bowel movements per day, compared to 4% of patients randomized to a combination of placebo and SSA.

The 12-week double-blind trial included 90 adult participants with well-differentiated mNETs and carcinoid syndrome diarrhea, all having between four to 12 daily bowel movements despite using SSA at a stable dose for at least 3 months.

“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,” Lexicon president and CEO Lonnel Coats said in a statement.

In addition to Xermelo, Lexicon has a pipeline of clinical and preclinical drug candidates in diabetes and metabolism and neuropathic pain.

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