FDA approves orphan drug Xermelo for frequent, severe diarrhea

The U.S. Food and Drug Administration (FDA) approved Lexicon Pharmaceuticals’ Xermelo tablets in combination with somatostatin analog (SSA) therapy for the treatment of adults with carcinoid syndrome diarrhea that SSA therapy alone couldn’t control properly.

The FDA approved Xermelo, in a regimen with SSA therapy, in tablet form to be taken orally three times daily with food. Xermelo inhibits the production of serotonin by carcinoid tumors and reduces the frequency of carcinoid syndrome diarrhea, said the FDA.

“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.” 

The FDA granted this application fast track designation and priority review. The drug also received orphan drug designation, which provides incentives to assist and encourage the development of drugs for rare diseases.

“Today’s approval will provide patients whose carcinoid syndrome diarrhea is not adequately controlled with another treatment option,” said Julie Beitz, M.D., director of the Office of Drug Evaluation III in the FDA’s Center for Drug Evaluation and Research.

About Xermelo

Lexicon points out that Xermelo is the first and only approved oral therapy for carcinoid syndrome diarrhea. It further explains that Xermelo targets tryptophan hydroxylase, an enzyme that mediates the excess serotonin production within mNET cells.

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